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Agreed.
I didn't know that about the researchers, but I like what I hear. I like this as a 5 year + play and will add when I can if it falls below $4.00.
Happy St. Patrick's day all.
I'm eager to learn more about SGMO's patent coverage. My understanding is that it is extensive enough that some researchers in academia feel constrained by it. That said, I actually think that it is a good thing for early stage companies like Sangamo to have a fairly tight grip on new technological protocols because it incentivizes their development and gives them a decided stake in scientific advance.
sorry, I didn't mean to mislead anyone, March 30, 2012 is the date I meant to display. I hate to talk about other companies on other boards ,but I wanted to show that the ZNF have many applications and can bring in revenue from many places.
Thanks for the clarification.
Long term hold for me.
The Kraig Labs conference call was delayed until March 30, 2012.
In the title the date is wrong, but in the subsequent paragraphs the date is correct.
SGMO will have royalty's from many sources.
Keep an eye on this;
Kraig Biocraft Laboratories to Host Conference Call to Discuss Commercialization and Laboratory Highlights
Date : 02/28/2012 @ 12:45PM
http://ih.advfn.com/p.php?pid=nmona&article=51397165
Because of this;
Kraig Biocraft Laboratories Receives New Custom Zinc Finger Configurations for Advanced Fibers Development
Date : 12/12/2011 @ 6:05AMhttp://ih.advfn.com/p.php?pid=nmona&article=50385206
Results From HIV Gene Therapy Trial Show Long-Term Positive Effects (CROI 2012)
Results from two Phase 1 gene therapy trials sponsored by Sangamo BioSciences indicate that infusion with genetically modified immune cells, called SB-728-T, lead to long-lasting effects on the immune system of HIV-positive individuals, including increased white blood cell counts and possibly suppressed HIV replication.
“The infusion of these genetically-modified [CD4] T-cells is safe and well tolerated,” said Dr. Pablo Tebas, director of the AIDS Clinical Trials Unit at the University of Pennsylvania and lead investigator of the study.
“[The therapy] leads to durable increases in total CD4 cells,” he added. “These cells engraft, expand, and persist for over a year.”
Dr. Tebas noted that researchers in future clinical trials will attempt to optimize the gene therapy procedure in an attempt to make it more efficient and effective.
The results were presented last week at the 19th Conference on Retroviruses and Opportunistic Infections (CROI) in Seattle.
Anybody buying here? I loaded up at 2.78, thinking of adding more soon...
3:23PM Sangamo BioSci presents new clinical data demonstrating persistent positive effects of ZFN Therapeutic for 'Functional Cure' of HIV/AIDS (SGMO) 5.13 +0.17 : Co says "The results suggest that SB-728-T has a positive effect on immune health of HIV-infected patients and this effect is persistent for over a year in some subjects. Most importantly, SB-728-T treatment provides HIV-resistant T-cells that are capable of mounting an immune response to an inflammatory event in lymphoid tissues and has yielded encouraging effects on HIV viral load when antiretroviral drugs are withdrawn during a treatment interruption... These data add to our understanding of the positive viral load changes in SB-728-T-treated subjects, and suggest that an early cytokine burst may initiate the dramatic and prolonged increase in CD4 counts observed following SB-728-T treatment."
Sangamo ist worth billions already
A HIV Cure worth maybe 50-100b in a few years?
The deal with Shire with milestone payments worth 1.5b and double digit royalty payments...
The royalty payments from protein productions worth some factor x billions
The many, many potential partnerships based on Sangamo's ZFN technology
Most of all the tremendous impact these gene therapies will have on the market
As soon as institutions realize that Sangamo's technology leads to success in so many different areas this company will more than pay off
Re: Monogenetic disorders and their potential...
Will big Pharma partner number two wait until the proof of concept announcement that will be made by Shire in the not too distant future???? When the upfront fee increases to $13 Million per target gene or will they sign with Sangamo prior to shires next announcement?
When will these gene targets receive a partner???
Hemoglobinopathies(SCA, B-thalassemia)
Immune System Disorders (X-SCID, ADA-SCID, W-A)
EL indicated that partnerships are the stated path of advancing progress on gene targets beyond the current capabilities of their own staff... Will the above be Sangamo targets or targets of Big Pharma Partnerships... How many different partnerships are available to Sangamo in the treatment of monogenetic diseases.
Many, many perhaps too many to imagine...
I posted a cash flow analysis of Sangamo with six pharma partnerships comparable to Shire thru 2021 and a projected share price of $320...
Will future partnerships be as inexpensive to Big Pharma as Shire's deal... I don't think so... Sangamo will drive research until the financial benefits to Sangamo are at a higher inflection ratio than the Shire deal and these partnerships will be more beneficial not less.
JMHO,
RM
Re: Monogenetic disorders and their potential...
From Slide 11 in the Leerink Presentation...
Hemoglobinopathies(SCA, B-thalassemia)
Immune System Disorders (X-SCID, ADA-SCID, W-A)
Liposomal Storage Diseases(Gaucher, Fabry, Pompe)
Sangamo's 8K disclosed that Liposomal Storage Diseases were part of the Shire Agreement. Several press releases indicate simply that Shire has rights to Factor 7, 8, 9 and 10, as well as three additional gene targets. Shire seems to want to keep confidential the additional gene targets for competitive reasons, however, clues have been slowly leaking thru presentations and announcements and investment seminars featuring EL and Sangamo. I believe the three additional gene targets are included in slide 11 and are: Liposomal Storage Diseases(Gaucher, Fabry, Pompe). Shire is already active in the Liposomal treatment market and most probably have included these disorders in their additional three gene targets.
What will be the first functional cure announcement out of the Shire Partnership? At first I thought it would be Hemophilia B, however due to the confidentiality around the three additional gene targets I now believe that one of these three diseases will be the first functional cure from this partnership. Anyone care to guess which it will be? Gaucher, Fabry, Pompe???
When the announcement is made that Gaucher disease has been cured in laboratory mice and confirms the Hemophilia B proof of concept... what will the share price do on that date in 2012 or 2013.
Will we see a 25% surge in share price from an $8 per share level? Perhaps a move from $8 to $10 in one day...
Or will we see a move from $10 to $20 due to the "proof of concept" and validation of the $1.5 Billion + shire deal?
Great times are ahead of us if and when this day comes...
JMHO,
RM
Monogenetic disorders and their potential...
Monogenic diseases
Monogenic diseases result from modifications in a single gene occurring in all cells of the body. Though relatively rare, they affect millions of people worldwide. Scientists currently estimate that over 10,000 of human diseases are known to be monogenic. Pure genetic diseases are caused by a single error in a single gene in the human DNA. The nature of disease depends on the functions performed by the modified gene. The single-gene or monogenic diseases can be classified into three main categories:
Dominant
Recessive
X-linked
All human beings have two sets or copies of each gene called “allele”; one copy on each side of the chromosome pair. Recessive diseases are monogenic disorders that occur due to damages in both copies or allele. Dominant diseases are monogenic disorders that involve damage to only one gene copy. X linked diseases are monogenic disorders that are linked to defective genes on the X chromosome which is the sex chromosome. The X linked alleles can also be dominant or recessive. These alleles are expressed equally in men and women, more so in men as they carry only one copy of X chromosome (XY) whereas women carry two (XX).
Monogenic diseases are responsible for a heavy loss of life. The global prevalence of all single gene diseases at birth is approximately 10/1000. In Canada, it has been estimated that taken together, monogenic diseases may account for upto 40% of the work of hospital based paediatric practice (Scriver, 1995).
Thalassaemia
Sickle cell anemia
Haemophilia
Cystic Fibrosis
Tay sachs disease
Fragile X syndrome
Huntington's disease
Thalassaemia
Thalassaemia is a blood related genetic disorder which involves the absence of or errors in genes responsible for production of haemoglobin, a protein present in the red blood cells. Each red blood cell can contain between 240 and 300 million molecules of haemoglobin. The severity of the disease depends on the mutations involved in the genes, and their interplay.
A haemoglobin molecule has sub-units commonly referred to as alpha and beta. Both sub-units are necessary to bind oxygen in the lungs properly and deliver it to tissues in other parts of the body. Genes on chromosome 16 are responsible for alpha subunits, while genes on chromosome 11 control the production of beta subunits. A lack of a particular subunit determines the type of thalassaemia (eg. a lack of alpha subunits results in alpha-thalassemia). The lack of subunits thus corresponds to errors in the genes on the appropriate chromosomes.
There can be various gradations of the disease depending on the gene and the type of mutations.
Prevalence:
The alpha and beta thalassaemias are the most common inherited single-gene disorders in the world with the highest prevalence in areas where malaria was or still is endemic. The burden of this disorder in many regions is of such a magnitude that it represents a major public health concern. For example in Iran, it is estimated that about 8,000 pregnancies are at risk each year. In some endemic countries in the Mediterranean region, long-established control programs have achieved 80-100% prevention of newly affected births.
Map of global distribution of haemoglobin disorders*
pdf, 143kb
Diagnosis/ prognosis:
Diagnosis of thalassaemia can be made as early as 10-11 weeks in pregnancy using procedures such as amniocentesis and chorionic villi sampling. Individuals can also be tested for thalassaemia through routine blood counts. Thalassaemic patients may have reduced fertility or even infertility. Early treatment of thalessaemia has proved to be very effective in improving the quality of life of patients. Currently, genetic testing and counselling, and prenatal diagnosis play an increasingly important role in informing individual as well as professional decisions around the prevention, management and treatment of this disease.
Additional resources:
Argentina: La Fundación Argentina de Talasemia
Argentina: Thalassaemia Argentina
Australia: Thalassaemia Society of Victoria
Australia: The Australian Thalassaemia Association
Canada: Thalassemia Foundation of Canada
Cyprus: Thalassaemia International Federation
Hong Kong: Children's Thalassaemia Foundation
Hong Kong: Thalassaemia Society of Hong Kong
India: Mumbai Thalassaemia Society
India: Thalassemics India
Italy: Berloni Foundation Against Thalassaemia
Lebanon: Lebanon Chronic Care Centre
Malaysia: Sarawak Thalassaemia Society
Middle East: Thalassemia, The Middle East Experience
Nigeria: Sickle Cell Foundation Nigeria
Pakistan: Thalassaemia Society of Pakistan
UK: North of England Thalassaemia Association
UK: UK Thalassaemia Society
USA: Cooley’s anemia foundation
USA: Northern California Comprehensive Thalassemia Center
USA: Impact Children Foundation
Sickle cell anemia
Sickle-cell anemia is a blood related disorder that affects the haemoglobin molecule, and causes the entire blood cell to change shape under stressed conditions. In sickle cell anaemia, the haemoglobin molecule is defective. After haemoglobin molecules give up their oxygen, some may cluster together and form long, rod-like structures which become stiff and assume sickle shape.
Unlike healthy red blood cells, which are usually smooth and donut-shaped, sickled red blood cells cannot squeeze through small blood vessels. Instead, they stack up and cause blockages that deprive organs and tissues of oxygen-carrying blood. This process produces periodic episodes of pain and ultimately can damage tissues and vital organs and lead to other serious medical problems. Normal red blood cells live about 120 days in the bloodstream, but sickled red cells die after about 10 to 20 days. Because they cannot be replaced fast enough, the blood is chronically short of red blood cells, leading to a condition commonly referred to as anemia.
Prevalence:
Sickle cell anemia affects millions throughout the world. It is particularly common among people whose ancestors come from Sub-Saharan Africa, South America, Cuba, Central America, Saudi Arabia, India, and Mediterranean countries such as Turkey, Greece, and Italy. In the Unites States, it affects around 72,000 people, most of whose ancestors come from Africa. The disease occurs in about 1 in every 500 African-American births and 1 in every 1000 to 1400 Hispanic-American births. About 2 million Americans, or 1 in 12 African Americans, carry the sickle cell allele.
Map of global distribution of haemoglobin disorders*
pdf, 143kb
Diagnosis/ prognosis:
The sickle cell disease can be diagnosed in a simple blood test. In many cases, sickle-cell anemia is diagnosed when new-borns are screened. Vaccines, antibiotics, and folic acid supplements are administered, in addition to pain killers. Blood transfusions and surgery are used in severe cases. The only known cure at present is a bone marrow transplant.
Additional resources:
American Sickle Cell Anemia Association
Genetics Home Reference: Sickle Cell Anemia
MEDLINEplus: Sickle Cell Anemia
UK : NHS Sickle Cell & Thalassaemia Screening Programme
Haemophilia
Haemophilia is a hereditary bleeding disorder, in which there is a partial or total lack of an essential blood clotting factor. It is a lifelong disorder, that results in excessive bleeding, and many times spontaneous bleeding, which, very often , is internal. Haemophilia A is the most common form, referred to as classical haemophilia. It is the result of a deficiency in clotting factor 8, while haemophilia B (Christmas Disease) is a deficiency in clotting factor 9. This illness is a sex-linked recessive disorder.
Prevalence:
Due to the sex-linkage of the disorder, there is a greater prominence in males than in females. About a third of new diagnoses are where there is no previous family history. It appears world-wide and occurs in all racial groups. About 6,000 people are affected with haemophilia in the UK. There are about 5400 people in the UK with haemophilia A and about 1100 with haemophilia B.
Map of global distribution of haemoglobin disorders*
pdf, 143kb
Diagnosis/ prognosis:
Blood tests can determine the presence of the haemophilia condition, and more specifically whether it is a type A or a type B disease. Usually, infants do not show signs before 9 months of age. Administration of clotting factors help affected individuals to live with the disease. There are various lifestyle changes that one can make as a haemophiliac, and though a serious disease, it can be tolerable with proper precautions and therapy. The prospects for youngster with haemophilia are excellent. Only a few decades ago, children with haemophilia had a significantly reduced life expectancy. They were often crippled with arthritis and joint deformity by their teens and had to attend special schools for disabled people. Many recent studies have documented a greatly increased life-expectancy among people suffering from haemophilia in developed countries over the last few decades. Children with haemophilia now face few limitations. They certainly attend normal schools, most jobs are open to them, and full participation in society through employment, marriage and having children is now the norm. It is anticipated, however, that the number of people with haemophilia in developed countries will increase steadily over the next few decades
Additional resources:
Canadian Hemophilia Society
National Hemophilia Foundation, USA
The Haemophilia Society, UK
World Federation of Hemophilia
Hemophilia Federation (India)
Cystic Fibrosis
Cystic Fibrosis is a genetic disorder that affects the respiratory, digestive and reproductive systems involving the production of abnormally thick mucus linings in the lungs and can lead to fatal lung infections. The disease can also result in various obstructions of the pancreas, hindering digestion. An individual must inherit two defective cystic fibrosis genes, one from each parent, to have the disease. Each time two carriers of the disease conceive, there is a 25 percent chance of passing cystic fibrosis to their children ; a 50 percent chance that the child will be a carrier of the cystic fibrosis gene; and a 25 percent chance that the child will be a non-carrier.
Prevalence:
The incidence of CF varies across the globe. Although it is severely underdiagnosed in Asia, existing evidence indicates that the prevelance of CF is rare. In the European Union 1 in 2000-3000 new borns is found to be affected by CF . In the United States of America the incidence of CF is reported to be 1 in every 3500 births.
The molecular epidemiology of cystic fibrosis **
pdf, 89kb
Diagnosis/ prognosis:
People with CF have a variety of symptoms including: very salty-tasting skin; persistent coughing, at times with phlegm; wheezing or shortness of breath; an excessive appetite but poor weight gain; and greasy, bulky stools. Symptoms vary from person to person, in part, due to the more than 1,000 mutations of the CF gene, several of which have been identified and sequenced by researchers.The sweat test is the standard diagnostic test for CF. This simple and painless procedure measures the amount of salt in the sweat. A high salt level indicates CF. Although the results of this test are valid any time after a baby is 24 hours old, collecting a large enough sweat sample from a baby younger than 3 or 4 weeks old may be difficult. The sweat test can also confirm the diagnosis in older children and adults. If pancreatic enzyme levels are reduced, an analysis of the person's stool may reveal decreased or absent levels of the digestive enzymes (trypsin and chymotrypsin) or high levels of fat. If insulin secretion is reduced, blood sugar levels are high. Pulmonary function tests may show that breathing is compromised. Also, a chest x-ray may suggest the diagnosis. Relatives other than the parents of a child with cystic fibrosis may want to know if they're likely to have children with the disease. Genetic testing on a small blood sample can help determine who has a defective cystic fibrosis gene. Unless both parents have at least one such gene, their children will not have cystic fibrosis. If both parents carry a defective cystic fibrosis gene, each pregnancy has a 25 percent chance of producing a child with cystic fibrosis. During pregnancy, an accurate diagnosis of cystic fibrosis in the fetus is usually possible.
The severity of cystic fibrosis varies greatly from person to person regardless of age; the severity is determined largely by how much the lungs are affected. However, deterioration is inevitable, leading to debility and eventually death. Nonetheless, the outlook has improved steadily over the past 25 years, mainly because treatments can now postpone some of the changes that occur in the lungs. Half of the people with cystic fibrosis live longer than 28 years. Long-term survival is somewhat better in males, people who don't have pancreatic problems, and people whose initial symptoms are restricted to the digestive system. Despite their many problems, people with cystic fibrosis usually attend school or work until shortly before death. Gene therapy holds great promise for treating cystic fibrosis.
More information on CF gene therapy
According to the CF Foundation's National Patient Registry, the median age of survival for a person with CF is currently 33.4 years. Only thirty years ago, a CF patient was not expected to reach adulthood. Many people even live into their fifties and sixties.
As more advances have been made in the treatment of CF, the number of adults with CF has steadily grown. Today, nearly 40 percent of the CF population is age 18 and older. Adults, however, may experience additional health challenges including CF-related diabetes and osteoporosis. CF also can cause reproductive problems - more than 95 percent of men with CF are sterile. But, with new technologies, some are becoming fathers. Although many women with CF are able to conceive, limited lung function and other health factors may make it difficult to carry a child to term.
Additional resources:
Cystic Fibrosis Worldwide
Cystic Fibrosis Foundation, USA
Cystic Fibrosis.com
Canadian Cystic Fibrosis Foundation, Canada
Cystic Fibrosis Trust, UK
Tay sachs disease
Tay-Sachs disease is a fatal genetic disorder in which harmful quantities of a fatty substance called Ganglioside GM2 accumulate in the nerve cells in the brain. This is caused by a decrease in the functioning of the Hexosaminidase A enzyme. Abnormal Hexosaminidase A enzyme activity causes an accumulation of fat in nerve cells, leading to paralysis, dementia, blindness, psychoses, and even death. Though the degradation of the central nervous system begins at the fetal stage, observations such as loss of peripheral vision and motor co-ordination are not seen until about 6 months of age. This disease is autosomal recessive which means that an individual must inherit two defective genes, one from each parent, to inherit this disease. According to the age of onset there are two existing forms of Tay-Sachs disease.
Infantile Tay-Sachs disease
Late onset Tay-Sachs disease ( chronic GM2-gangliosidosis)
Prevalence:
The frequency of the condition is much higher in in Ashkenazi Jews of Eastern European origin than in others.
Approximately one in every 27 Jews in the United States of America is a carrier of the TSD gene. There is also a noticeable incidence of TSD in non-Jewish French Canadians living near the St. Lawrence River and in the Cajun community of Louisiana. By contrast, the carrier rate in the general population as well as in Jews of Sephardic origin is about one in 250.
Among Jews of Sephardic origin and in the general, non-Jewish population, the carrier rate is about 1 in 250. There are certain exceptions. French-Canadian and the Cajun community of Louisiana have the same carrier rate as Ashkenazi Jews, one in 27. Also, individuals with ancestry from Ireland are at increased risk for the Tay-Sachs gene. Current research indicates that among Irish Americans, the carrier rate is about one in 50.
Diagnosis/ prognosis:
The diagnosis for Tay- Sachs disease (TSD) can be made via a blood test in which the Hex A enzyme can be measured in either the serum, the white blood cells, or in the skin fibroblast. Over the past 25 years, carrier screening and genetic counselling within high-risk populations have greatly reduced the number of children born with TSD in these groups. Therefore, a great percentage of the babies born with Tay-Sachs Disease today are born to couples who were not previously thought to be at significant risk.
Prenatal tests that can diagnose Tay-Sachs in the fetus before birth are available. These procedures are referred to as Amniocentesis and Chorionic Villus Sampling. Amniocentesis sampling is performed between the 15th and 16th week of pregnancy. The procedure involves inserting a needle into the mother's abdomen and obtaining a sample of the fluid that surrounds the baby. In Chorionic Villus Sampling a sample of cells from the placenta is retrieved by the doctor during the 10th and 12th week of pregnancy, and tested for the presence of Hex A.
Additional resources:
Genes and Disease: Tay-Sachs Disease
Late Onset Tay-Sachs Foundation, USA
National Institute of Neurological Disorders and Stroke: Tay-Sachs Disease Information page (USA)
National Tay-Sachs ans Allied Diseases Association,Inc. (USA)
Fragile X syndrome
The Fragile X syndrome is caused by a "fragile" site at the end of the long arm of the X-chromosome. It is a genetic disorder that manifests itself through a complex range of behavioural and cognitive phenotypes. It is the result of genetic mutation which varies considerably in severity among patients. Fragile X syndrome is the most common cause of inherited mental retardation. Although it is a X-linked recessive trait with variable expression and incomplete penetrance, 30% of all carrier women are affected.
Prevalence:
According to the Fragile X association of Southern California, Fragile X syndrome is the single most common inherited cause of mental impairment affecting 1 in 3600 males and 1 in 4000 to 6000 females with full mutation worldwide. Some studies also suggest that fragile X affects 1 in every 2000 males and 1 in every 4000 females of all races and ethnic groups. Studies have also revealed that 1 in 259 women of all races carry fragile X and could pass it to their children. The number of men who are carriers is thought to be 1 in 800 of all races and ethnicity. Carrier females have a 30% to 40>% chance of giving birth to a retarded male child and a 15 to 20% chance of having a retarded female.
Diagnosis/ prognosis:
The diagnosis of Fragile-X syndrome is made through the detection of errors in the FMR1 gene. Over 99% of individuals have a full mutant FMR1 gene. Tests used for diagnosis include chromosome analysis and various protein tests. Diagnosis is usually made when young, and there is no current cure for this illness. Early diagnosis of the syndrome call allow for therapeutic interventions like speech therapy, occupational therapy, psychotherapy and special education, that can considerably improve the quality of the patients' life.
Additional resources:
Fragile X Syndrome Profile, USA
FRAXA Research Foundation, USA
National Institute of Child Health and Human Development, USA
Policy Statement: American College of Medical Genetics; Fragile X Syndrome: Diagnostic and Carrier Testing
The National Fragile X Foundation
Huntington's disease
Huntington’s disease is a degenerative brain disorder, in which afflicted individuals lose their ability to walk, talk, think, and reason. They easily become depressed, and lose their short-term memory capacity. They may also experience a lack of concentration and focus. This disease begins between ages 30-45, and every individual with the gene for the disease will eventually develop the disease. Huntington's is an autosomal dominant genetic disorder which means that if one parent carriers the defective Huntington's gene, his/her offspring have a 50/50 chance of inheriting the disease.
Prevalence:
Huntington's disease (HD) affects males and females equally and crosses all ethnic and racial boundaries. It typically begins in mid-life, between the ages of 30 and 45, though onset may occur as early as the age of 2. Children who develop the juvenile form of the disease rarely live to adulthood. There is a 50/50 chance of inheriting the fatal gene from the parents. Everyone who carries the gene will develop the disease. In Western countries, it's estimated that about five to seven people per 100,000 are affected by HD.
Diagnosis/ prognosis:
There is no treatment or cure for Huntington’s Disease, and the patient eventually becomes completely dependent on others for daily functioning. Individuals may also die due to other secondary complications such as choking, infection, or heart failure. Children who are diagnosed with Huntington’s Disease do not usually live to reach adulthood.
what did I miss?
Single-digit Factor IX levels would not be normal; the patient with severe hemophilia would then have mild hemophilia, still a great improvement. These patients (mild) typically only manifest symptoms with trauma or surgery. At a cost of 1/2 to 1 million dollars a year for conventional prophylactic therapy the economic incentives for development would be huge. I assume the next step will be dog testing, as there is a hemophilia model there. Then?? Any knowledge of the pipeline time-line to human trials and ...successful deployment? 5 years? 10? The two big questions in my mind are whether the ZFN technology can eliminate the mutation issue and subsequently immune response issues (inhibitor development).
How does this translate to Cystic fibrosis? I'm not sure what level of corrected CFTR is necessary for significant disease/symptom abatement. Not sure if it is linear, or somewhat like hemophilia in the need for less than full conversion of target genes. Also there is an issue with the larger size of the CF gene involved as opposed to the FIX gene which is about 1/3 the size, I believe. At what point is gene size a detriment to ZFN deployment and function?
Thanks for replying to my post. I also feel positive about the forward movement of this company. It's fun to be in a company with so much potential. I will admit the science is a bit over my head, but I can see this being a good long term investment.
4.78 in After hours! Some (on IV) think it's just a late post but I strongly disagree. A late post would have to have been from the very start of the day and the not posted til 5:00 PM? NOT likely!
IMHO it's a real trade and well justified from items in the SIAL call.
SIAL call today had some very strongly positive comments about SGMO:
"Our research biology business achieved reported and organic growth of 5%, consistent with our full year 2011 organic growth in this area.
This increase was helped by strong demand and growth of our zinc-finger-related products, which grew about 30% in 2011. In 2011, this innovative technology has enabled us to develop novel programs and cell-based assays amongst other products. We have tried to once again to receive the acknowledgment of our peers in Nature Methods' Method of the Year for gene with engineered nucleus.
...
We have a couple of new extensions of our zinc finger platform technologies to share with you. We expanded our capabilities to provide knockout ZFN for rat models that exhibit features key to studying conditions such as obesity, diabetes, atherosclerosis, high cholesterol and hypertension. For many indications, especially metabolic and cardiovascular diseases, the rat is the most suitable model for drug development research. We are the only providers of these rat models, which enable researchers to more accurately predict the efficacy of their drugs and help to strengthen their drug development timelines. This enables our customers to have more certainty about the safety and efficacy of the drugs that move through their pipeline, hopefully making them more successful and profitable.
We also launched our first offering of zinc finger nuclease modified Chinese hamster ovary cell lines. These products are designed for use in the production of biopharmaceuticals and enable customers to reach the market faster and more cost effectively by reducing timelines for early-stage biological drug development. The distinctive features of these cell lines should appeal to organizations to -- looking to build a robust and comprehensive therapeutic protein monoclonal antibody manufacturing platform."
The CHO cell lines will get royalties coming in very quickly. IMHO it won't be as long as most think before SGMO starts turning a profit.
Very positive IMHO.
the factor everyone appears to be overlooking, which is BIG, is protein production.
SIAL is sellling ZF modified cell lines for protein production. Those royalties will start coming in very soon (it only takes weeks to set up production in cell lines) whereas the royalties (as opposed to just upfront payments) for other things like seeds (DAS) and products developed with ZFs will take a lot longer.
Protein production royalties will get revenues coming in in the near term.
Anyone have any thoughts on today's news? SGMO has lost diabetes funding (and trials), but I like the way they added Shire in the report.
thanks a lot!
sounds great!!!!
From the 8K
Item 1.01. Entry into a Material Definitive Agreement.
On January 31, 2012, Sangamo BioSciences, Inc. (“Sangamo”) entered into a Collaboration and License Agreement (the “Agreement”) with Shire AG (“Shire”), pursuant to which Sangamo and Shire will collaborate to research, develop and commercialize human therapeutics and diagnostics for monogenic diseases based on Sangamo’s zinc finger DNA-binding protein (“ZFP”) technology. Under the Agreement, Sangamo and Shire may develop potential human therapeutic or diagnostic products for seven (7) gene targets. The initial four (4) gene targets are blood clotting Factors VII, VIII, IX and X, and products developed for such initial gene targets would be used for treating or diagnosing hemophilia. Shire has the right, subject to certain limitations, to designate three (3) additional gene targets. Sangamo grants Shire an exclusive, world-wide, royalty-bearing license, with the right to grant sublicenses, to use Sangamo’s ZFP technology for the purpose of developing and commercializing human therapeutic and diagnostic products for the gene targets.
Sangamo and Shire agreed to form a joint steering committee, consisting of an equal number of representatives of Sangamo and Shire, to oversee the research collaboration. The initial research term of the Agreement is six (6) years and is subject to extensions upon mutual agreement and under other specified circumstances. Sangamo is responsible for all research activities through the submission of an Investigative New Drug Application (IND) or European Clinical Trial Application (CTA), while Shire is responsible for clinical development and commercialization of products generated from the research program from and after the acceptance of an IND or CTA for the product. Shire will reimburse Sangamo for its internal and external research program-related costs.
Under the Agreement, Sangamo will receive an upfront license fee of $13.0 million. In addition, for each gene target, Sangamo is eligible to receive milestone payments upon the achievement of specified research, regulatory, clinical development, commercialization and sales milestones. The total amount of potential milestone payments for each of the seven gene targets, assuming the achievement of all specified milestones in the Agreement, is $213.5 million. The milestone payments for each gene target through the acceptance of an IND or CTA submission total $8.5 million. Sangamo will also receive royalty payments that are a tiered double-digit percentage of net sales of products developed under the collaboration.
The Agreement may be terminated by (i) Sangamo or Shire, in whole or in part, for the uncured material breach of the other party, (ii) Sangamo or Shire for the bankruptcy or other insolvency proceeding of the other party and (iii) Shire, in its entirety, beginning 24 months after the effective date of the Agreement upon 90 days advance written notice. In addition, Shire may terminate the Agreement with respect to an individual gene target at any time, and under certain circumstances may designate a replacement gene target for a terminated gene target.
The foregoing description is a summary and qualified in its entirety by the Agreement, a copy of which Sangamo intends to file as an exhibit to its Annual Report on Form 10-K for the period ended December 31, 2011.
Hi
$1.5 BILLION in potential milestones from Shire...
Where does this come from...I mean where can I read about the agreement as far as milestone payments are concerned...?
I mean, that is absolutely incredible, but where is it written?
Yours, Andi
All the good news in SGMO and the current short position can keep this trending higher...
Avg Vol (3 month)3: 338,979
Avg Vol (10 day)3: 532,625
Shares Outstanding5: 52.47M
Float: 49.73M
% Held by Insiders1: 5.67%
% Held by Institutions1: 39.70%
Shares Short (as of Jan 13, 2012)3: 4.33M
Short Ratio (as of Jan 13, 2012)3: 13.80
Short % of Float (as of Jan 13, 2012)3: 10.10%
Shares Short (prior month)3: 4.36M
ANYONE on I.V. board is welcome to PLEASE
cross post this post (I'm replying to) there!
That board (which is usually pretty good) is SERIOUSLY messing this one up!
SHPGY would NOT "save" $1.5 BILLION by buying SGMO. THe milestones are to finance development of the drugs. If SHPGY buys SGMO then IT has to do the drug development and spend that $1.5 billion itself.
How could they get this one so very wrong?!
Shire would NOT "save" $1.5 billion by buying out SGMO despite some misconceptions to that effect on the "other" SGMO board. (Which IMHO is good because I'd really HATE to see a buyout anywhere remotely near current levels!)
The milestones are to be used to finance the development of zinc fingers for the chosen conditions. If Shire bought out SGMO then it would have to do that work itself and so it would just be spending the money directly instead of giving it to SGMO to spend.
Which, frankly, to me, is a very good thing because the LAST thing I'd want to see right now is some big pharma KNOWING it could "save" $1.5 BILLION by acquiring SGMO outright. I plan to get a LOT more than a measly $25 or so for my shares, thanks, and am willing to wait a few years to do it.
Zinc fingers work VERY fast!: KBLB is now using them to make a new GM in EXACTLY the location intended, with EXACTLY the insertion desired with EXACTLY the immediate effect desired (the secondary effects of a GM are, of course, inherently problematic for most changes) in only 2 or 3 months! This is a BLISTERING rate of genetic modifications!
Note that many products will be neither drugs nor foods and thus require no lengthy and expensive clinical trials nor any food safety testing. And some even (like KBLB's silkworms) won't even require any approvals for the release of GMOs into the environment: commercial silkworms are incapable of surviving in the wild). So some products could be paying royalties to SGMO (thru the DAS and SIAL agreements) in relatively short order. That, plus SGMO's 2+ years of cash on hand, plus the SHPGY deal give SGMO the staying power to be around long enough for the other, longer development time products to start paying royalties thru SIAL and DAS and then KBLB's drugs to start bring revenues in.
KBLB's silkworms could be some of the first BIG royalties SGMO gets: there is a huge market for spider silk (both as spun fiber and unspun protein) for a very wide array of uses:
unspun spider silk protein can be altered to biodegrade at any desired rate, it has very valuable optical properties, the fiber is tougher than Kevlar and, uniquely, its properties (like for example, strength and elasticity, that are strongly dependent on each other in all other materials, are independent in silk (thanks to its unique structure) and could be highly customizable to combinations of properties that could not be achieved in other materials.
AND MANY OF THOSE PRODUCTS COULD BE ON THE MARKET WITHIIN SEVERAL YEARS and paying royalties to SGMO(i.e.: before SGMO would run out of cash at its current burn rate even if it received no other income!
There is also an extensive range of potential medical uses (which would, of course, be years down the road: medical sutures, implants, scaffold for growing tissues,
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3136811/
$1.5 BILLION in potential milestones from Shire is no small potatoes (plus double digit royalties)!
The monogenic diseases will be VERY safe bets!: correct the gene and you correct the problem. With one important limitation!:
Depending on the tissues that the gene is expressed in and the delivery method used for the zinc fingers, the correction rate may not be 100%. (and with current knowledge is unlikely to be.) But it doesn't have to be for many monogenic diseases: For hemophilia, for example, it is only necessary for the gen expression to reach high single digits of percent for function to be returned to normal (thanks to the efficiency of the blood clotting cascade). So ZFs will have not problem attaining high levels of efficacy (as demonstrated in the mouse model), which is why four of the genes chosen were involved in the blood clotting pathway.
It will be interesting to see what other three monogenetic diseases Shire intends to go after (my hope is for sickle cell to be one of them. But that's very unlikely: 1) there would be no selection factor in favor of the corrected blood cells because the stem cells producing the defective RBCs would be entirely unaffected by their higher loss rate 2)Killing all of the existing blood forming stem cells would be a serious undertaking (however if it's a life long CURE for the condition it might, nevertheless, be warranted. So ultimately it will be done but I suspect they'll go after easier targets first (plus the FDA would want to see more working ZF treatments before allowing something on the level of knocking out all of the blood forming stem cells).
IMHO this puts a new higher and very stable floor for SGMO at current levels (about 4.50 range). What SGMO now has is financing for four drug candidates and up to three more PLUS some MAJOR validation by a very big player.
Not to mention years of expenditures in cash on hand plus the increasing revenue streams from SIAL and DAS which should start picking up in the near future.
Nice move up 22%
Lee HJ, Kweon J, Kim E, Kim S, Kim J-S. Targeted chromosomal duplications and inversions in the human genome using zinc finger nucleases. Genome Research. http://genome.cshlp.org/content/early/2011/12/16/gr.129635.111
Despite the recent discoveries of and interest in numerous structural variations (SVs)—which include duplications and inversions—in the human and other higher eukaryotic genomes, little is known about the etiology and biology of these SVs, partly due to the lack of molecular tools with which to create individual SVs in cultured cells and model organisms. Here, we present a novel method of inducing duplications and inversions in a targeted manner without pre-manipulation of the genome. We found that zinc finger nucleases (ZFNs) designed to target two different sites in a human chromosome could introduce two concurrent double-strand breaks, whose repair via non-homologous end-joining (NHEJ) gives rise to targeted duplications and inversions of the genomic segments of up to a mega base pair (bp) in length between the two sites. Furthermore, we demonstrated that a ZFN pair could induce the inversion of a 140-kbp chromosomal segment that contains a portion of the blood coagulation factor VIII gene to mimic the inversion genotype that is associated with some cases of severe hemophilia A. This same ZFN pair could be used, in theory, to revert the inverted region to restore genomic integrity in these hemophilia A patients. We propose that ZFNs can be employed as molecular tools to study mechanisms of chromosomal rearrangements and to create SVs in a predetermined manner so as to study their biological roles. In addition, our method raises the possibility of correcting genetic defects caused by chromosomal rearrangements and holds new promise in gene and cell therapy.
Sangamo BioSciences Announces Presentation of New Data from ZFP Therapeutic® Program in Hemophilia B at American Society for Hematology Meeting
Studies Expand Potential of ZFN-Mediated Genome Editing as a Therapeutic Modality
PR NewswirePress Release: Sangamo BioSciences, Inc. – Tue, Dec 13, 2011 7:00 AM EST
RICHMOND, Calif. , Dec. 13, 2011 /PRNewswire/ -- Sangamo BioSciences, Inc. (Nasdaq: SGMO - News) announced the presentation of new data demonstrating the ability to permanently correct a disease gene in an adult mouse model of hemophilia B using systemic delivery of zinc finger nucleases (ZFNs) at the 53nd Annual Meeting of the American Society of Hematology (ASH).
"We have demonstrated functional correction of a human gene for the clotting factor, Factor IX, with a single, systemic administration of ZFNs in an animal model of disease," said Geoff Nichol , M.B, Ch.B., Sangamo's executive vice president of research and development. "Our approach enables permanent correction of the genetic defect responsible for hemophilia B. This circumvents the problems of traditional gene-addition approaches that uncouple the gene from its normal regulatory mechanism and which may result in gene silencing and random gene insertion and potentially lead to malignancy or other unintended consequences."
Abst. No.668 - Robust Factor IX Expression Following ZFN-mediated Genome Editing in an Adult Mouse Model of Hemophilia B (Oral Session: 801)
In this study, scientists demonstrated efficient ZFN-mediated gene correction in an adult mouse model of hemophilia B with a single systemic administration of ZFNs and a donor DNA sequence encoding the corrected human Factor IX gene. Stable levels of protein made from the corrected human gene could be measured in the plasma of the treated animals and resulted in the restoration of normal rates of blood clotting for the period of the study. This work expands upon earlier studies, published in Nature*, that demonstrated similar results in neonatal mice. Data described at the ASH meeting demonstrate that growth of liver cells, as is observed in neonates, is not required for efficient ZFN-mediated repair of the human Factor IX gene, substantially expanding the potential of the approach.
The study was conducted in the laboratory of Katherine High , M.D., Investigator, Howard Hughes Medical Institute, Professor of Pediatrics, University of Pennsylvania School of Medicine and Director, Center for Cellular and Molecular Therapeutics at The Children's Hospital of Philadelphia , in collaboration with Sangamo scientists.
In a second study presented at the meeting, ZFN gene disruption was used to generate a next generation cancer immunotherapy by enhancing the targeted killing activity and safety profile of the product.
Abst. No. 667 - TCR Gene Editing Results in Effective Immunotherapy of Leukemia without the Development of GvHD (Oral Session: 801)
Cancer immunotherapy uses CD8 T-cells that have been engineered to express high avidity T-cell receptor (TCR) genes isolated from tumor-specific lymphocytes. The engineered CD8 T-cells are then able to attack the tumor. Problems can arise with this approach because the expression of the CD8 T-cell's own TCR gene interferes with expression of the inserted tumor-specific TCR gene. This interaction limits the potency of this cellular therapy but, more importantly, it can also make the cells "self-reactive" leading to graft versus host disease (GvHD).
In this study, ZFNs were used to disrupt the native TCR genes in these tumor-directed CD8 T-cells resulting in an enhanced immunotherapeutic product with potent cancer cell-killing activity and the elimination of GvHD in a mouse model. These studies were performed in the laboratory of Chiara Bonini , M.D., Head of the Experimental Hematology Unit, San Raffaele Hospital, Milan , in collaboration with Luigi Naldini , Head of TIGET, San Raffaele Hospital, and Sangamo scientists.
"We continue to develop our ZFP Therapeutic pipeline and, on the strength of our success in mouse models, have advanced our hemophilia B program into preclinical studies in a large animal model of the disease," stated Edward Lanphier , Sangamo's president and chief executive officer. "As these presentations demonstrate, our ZFN gene-editing platform has broad applicability in that it can be applied to any disease-relevant gene enabling permanent gene modification and has the potential to provide a valuable therapeutic approach to a variety of unmet medical needs."
* Nature. 2011 Jun 26;475 (7355):217-21. doi: 10.1038/nature10177. "In vivo genome editing restores haemostasis in a mouse model of haemophilia".
The SB-509 trial was questionable from the start IMHO.
Diabetic Neuropathy is affected by many variables of which VEGF is only one. So there was always the chance that the ZF treatment would have the expected direct result intended (increase of VEGF) but that that increase would not have the expected results for it. And that, unfortunately, was exactly what happened. "The treatment was a success but the patient died."
The zinc finger therapy did indeed result in a higher production of VEGF, but that did not have the expected results. (Another variable affecting the condition was more important)
Thus the trial was, in a very real sense, a success for zinc fingers but a failure for their use to increase VEGF as a therapy for DN. (There are other conditions that increasing VEGF might be therapeutically useful but they have a similar risk as the DN trial. So IMHO SGMO was wise to drop SB-501 for now (later when it has more money it could do trials for its use in other conditions.)
Time to go after the "low hanging fruit" (monogenic diseases, etc.)
RE: SGMO adding sites to SB-728-0902 HIV Study, a total of 7 sites now.
There are two reasons why it may be difficult to find enough patients for this study:
1) the obvious one is that only a small percentage of HIV patients will be heterozygous (one copy) for the CCR-5 receptor deletion allele (genetic variation)(the study is for such patients).
Only about 10% of the population have one copy of this deletion. But the number of HIV patients with it will be much smaller, a fact I suspect that few are appreciating. Two copies of this genetic variation render one immune to HIV because it requires the CCR-5 receptor (that the variation results in the lack of) to enter and infect cells.
The result of one copy is not a lack of the receptors but a smaller number of them. This considerably delays the progression of HIV virus with the result that SOME carriers of one copy of the variant can go 25 or 30 years after infection before AIDS develops. I don;t know how the figures work out on that exactly. So the percentage of patients close to the onset of AIDS among those with one copy of the variant will be lower than 10% because those will be people infected considerably longer ago(than the other patients with AIDS) when the numbers of people with HIV infections was much lower than now. And the mainstream thinking on the treatment of HIV has been to wait until close to the onset of AIDS to start the drug regimen. (That attitude has started to change due to recent studies showing that starting treatment far earlier is much better but attitudes in the medical community change slowly Countering that is that people with HIV tend to be VERY up to date on treatment.
But the treatment is VERY expensive and those with an apparent option to delay it might be tempted (or required) to do so for financial reasons.
Because of that, many of those with one copy of the variation who know that they are HIV positive may be far more complacent about getting treated, especially if they haven't been positive that long. Some may well think that they can afford to just wait a few years for better treatments to be developed.
HOWEVER, there is also the consideration that being a carrier of the virus (which they very much still are) would have very negative implications for their sex lives. Certainly some would not be overly concerned about that but many (I would think most) would. And I would think that having a chance to become non-infective would be very attractive to them. But it might take a fair bit of education among the HIV community to get them to realize this and the other benefits that the treatment could potentially confer Eventually time passes and the prospect of having to eventually take the very expensive multidrug regimen with considerable side effects etc is not pleasant. And, as with most groups of people, there will certainly be many altruistic people among them who would like to help advance the work for a cure as well. While they may be "safe" for a period because of their variant they will have many friends who are not.
BOTTOM LINE: The motivations for entering the study are far more complex than they might have appeared at first. I therefore do not find it surprising that SGMO may have some delay in filling the trial.
However they only need 20 patients. While it may take longer than hoped for IMHO the trial will get filled without too much delay. The community of those affected is far too active and concerned for that not to happen. I'm sure that if some of the active members of the community realize that this important trial is having trouble getting filled they will work hard to drum up support.
IMHO setting up more sites is a very good move: it may cost a bit more but will be well worth it if it helps to speed things up.
It will happen. It just may take a bit longer than hoped for for the above reasons.
(OT to milwalkeebucks: thanks for posting my KBLB post on IV SGMO board. I'm not a member so I can't post there, but I have followed that board for many years and been held SGMO shares since the last millennium! (And I'll never be able to say THAT about another stock!)
Strange silence on IV SGMO board.
Although it's had about the usual number of posters and roughly the usual number of readers (and at times considerably more) there has been a markedly lower number of posts than the usual on the Investor Village SGMO board.
IMHO that's because a large number of the most active voters have been waiting for what they consider to be a bottom (after the severe drop in SP due to the DN drug disappointment and SGMO (wisely*, IMHO) dropping that drug.
Most of them probably feel, as do I, that the SP had already completely discounted SB501 before the results of the failed clinical trial came in and that therefore the subsequent drop in SP was grossly overdone, leading to a price that reflected little more than cash on hand and little or nothing for the DAS, SIAL and other deals that have immense potential for bringing large amounts of royalty income (starting slowly at first but with potential for very large amounts in a few years) with no further input of time or resources from SGMO.
IMHO they are effectively keeping silent (most of them) in order to not impede the SP from possibly dropping even lower so they can get even more shares at less money.
IMHO that is grossly short sighted, but investors often do such things. I am accumulating all I can get my hands on at these levels. While the price may drop significantly somewhat lower, it may not and therein lies the danger, IMHO: If SGMO should wind up failing (which I consider exceedingly unlikely given all the deals which require no further input from SGMO that could be generating large royalties in a few years and continually increasing ones after that, plus the two years cash on hand plus the potential in the monogenetic diseases.
What the market appears to be missing is that in the SB501 trial THE ZINC FINGERS MADE EXACTLY THE GENETIC MODIFICATIONS INTENDED AND THOSE GENETIC MODIFICATIONS HAD EXACTLY THE PRIMARY EFFECT INTENDED: the increase of the production of VEGF. It was the failure of that increase of production of VEGF to have the anticipated effects that caused the failure of the clinical trials. DN is a complex condition affected by many factors and that an increase in VEGF would not, BY ITSELF, be sufficient to generate adequate therapeutic effect was always a strong possibility. IMHO SGMO chose that condition not because of the likelihood of success but because of the size of the potential market. (NOT good thinking, IMHO. But the only instance of poor thinking I can see by SGMO so far.)
My attitude is this: if the company ultimately fails it will make little difference whether I bought shares at the current price or even lower. If it succeeds, my regret at having paid more by buying now when I could have bought even lower (IF that chance arises) would be vastly less than regret at having not bought more at all (or at much higher prices) due to waiting for a further price drop that never came. In any case I plan to continue to accumulate whether or not the price continues to drop.
I don't keep my mouth shut while trying to accumulate simply because doing so would mean being silent for years! (I can't post on IV because I don't have a subscription).
* at least for that indication! IMHO diabetic neuropathy was always a very poor choice of conditions to do a clinical trial on for that drug (SB501): VEGF has much more PRIMARY effects in a number of other conditions (in DN it is only one of a number of factors that have considerable influence on the condition) and likely could be a useful therapeutic for some of those other conditions. But for the FIRST drugs to test IMHO it makes far more sense to test drugs that directly address the underlying cause of conditions (as, has been noted on IV SGMO board the monogenetic diseases). That initial poor choice of direction has apparently been corrected.
Sangamo BioSciences Reports Third Quarter 2011 Financial Results
Date : 10/25/2011 @ 4:00PM
Source : PR Newswire
Stock : Sangamo Biosciences (SGMO)
Quote : 3.2 -0.22 (-6.43%) @ 5:35PM
Sangamo BioSciences Reports Third Quarter 2011 Financial Results
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Sangamo Biosciences (NASDAQ:SGMO)
Intraday Stock Chart
Today : Wednesday 26 October 2011
Sangamo BioSciences, Inc. (Nasdaq: SGMO) today reported third quarter 2011 financial results and accomplishments.
For the third quarter ended September 30, 2011, Sangamo reported a consolidated net loss of $9.6 million, or $0.18 per share, compared to a net loss of $8.7 million, or $0.19 per share, for the same period in 2010. As of September 30, 2011, the Company had cash, cash equivalents and marketable securities of $85.0 million.
Revenues for the third quarter of 2011 were $1.9 million, compared to $2.9 million for the same period in 2010. Third quarter 2011 revenues were generated from the Company's collaboration agreements with Dow AgroSciences (DAS), Sigma-Aldrich Corporation (Sigma) and agreements related to protein production and research grants. The revenues recognized for the third quarter of 2011 consisted of $0.8 million in collaboration agreements and $1.1 million related to research grants, compared to $2.2 million in collaboration agreements and $0.7 million related to research grants for the same period in 2010.
The decrease in collaboration agreement revenues was primarily due to the completion of the research term of our commercial license agreement with Sigma in July 2010. The increase in research grant revenues was primarily due to funding from the CHDI Foundation, Inc. to support research using zinc finger DNA-binding (ZFP) technology for a novel therapeutic for Huntington's disease as well as Sangamo's portion of the Disease Team Research Award from the California Institute for Regenerative Medicine.
Research and development expenses were $7.8 million for the third quarter of 2011, compared to $8.8 million for the same period in 2010. The decrease was primarily due to reduced clinical expenses associated with our diabetic neuropathy program, partially offset by higher expenses for our HIV/AIDS program. General and administrative expenses were $3.6 million for the third quarter of 2011, compared to $2.9 million for the same period in 2010. The increase was primarily due to increased professional services expenses.
Total operating expenses for the third quarter of 2011 were $11.4 million, compared to $11.7 million for the same period in 2010.
Nine Months Results
For the nine months ended September 30, 2011, the consolidated net loss was $29.4 million, or $0.59 per share, compared to a consolidated net loss of $16.5 million, or $0.37 per share, for the nine months ended September 30, 2010. Revenues were $5.6 million for the nine months of 2011, compared to $16.1 million in the same period in 2010, with the decrease primarily due to the completion, in July 2010, of the amortization period for the commercial and research license fee received from Sigma under the expanded agreement of October 2009. Total operating expenses were $35.0 million for the nine months of 2011 and $32.7 million for the same period in 2010.
Financial Guidance
Cash, cash equivalents and marketable securities of at least $85 million at the end of 2011, exclusive of new funding from potential future partnerships.
Operating expenses for 2011 to be relatively flat compared to 2010, in the range of $43 to $47 million.
Revenues and related cash proceeds in the range of $10 to $12 million in 2011.
Recent Events
Announcement that Sangamo's Phase 2b Trial (SB-509-901) in Subjects with Moderately Severe Diabetic Neuropathy Did Not Meet Key Endpoints. Based on the data, the Company announced that it has discontinued further development of SB-509 and will focus resources on its pipeline including ZFP Therapeutics for HIV and monogenic diseases.
Presentation at ICAAC 2011 of Groundbreaking Clinical Data from Sangamo's Ongoing Phase 1 Clinical Trials of SB-728-T. Data from its Phase 1 clinical programs to develop SB-728-T, a novel ZFP nuclease (ZFN)-based therapeutic approach for the treatment of HIV/AIDS, were discussed in two presentations at the 51st Interscience Conference on Antimicrobial Agents and Chemotherapy (ICAAC). Based on these data, the Company expects to initiate two new clinical trials designed to maximize the engraftment of CD4+ T-cells that are have undergone biallelic ZFN-mediated modification of the CCR5 gene.
Appointment of Dr. Geoffrey Nichol, M.B., Ch.B., as Executive Vice President, Research and Development. In this newly created position, Dr. Nichol oversees all of the Company's research and clinical development activities and operations.
Publication of Studies Demonstrating the Use of ZFN Technology for the Correction of the Alpha1-Antitrypsin Gene in induced Pluripotent Stem Cells (iPSCs) and the Efficient Generation of Transgenic Pigs. Sangamo scientists published a preclinical study in Nature demonstrating highly specific, functional correction of the alpha1-antitrypsin (A1AT) gene defect in patient-derived iPSCs using ZFNs. The work highlights the precision and broad applicability of ZFN-based genome-editing for the development of ZFP Therapeutics® for the treatment of monogenic diseases. In a second publication in the Proceedings of the National Academy of Sciences (PNAS), scientists demonstrated the use of ZFNs to produce genetically modified pigs. This work represents a significant advancement in the development of improved, less immunogenic, animal tissue as a source for transplant into humans.
Conference Call
Sangamo will host a conference call today, October 25, 2011 at 5:00 p.m. ET, which will be open to the public. The call will also be webcast live and can be accessed via a link on the Sangamo BioSciences website in the Investor Relations section under "Events and Presentations" http://investor.sangamo.com/events.cfm. The webcast replay will also be available for two weeks after the call. During the conference call, the Company will review these results, discuss other business matters, and provide guidance with respect to the remainder of 2011.
The conference call dial-in numbers are 877-377-7553 for domestic callers and 678-894-3968 for international callers. The passcode for the call is 20389876. For those unable to listen in at the designated time, a conference call replay will be available for one week following the conference call, from approximately 8:00 p.m. ET on October 25, 2011 to midnight ET on November 1, 2011. The conference call replay numbers for domestic and international callers are 855-859-2056 and 404-537-3406, respectively. The conference ID number for the replay is 20389876.
7:04AM Sangamo BioSci announces publication in nature of gene correction strategy for Alpha 1-Antitrypsin deficiency (SGMO) 3.38 : The study, published in Nature, further highlights the precision and broad applicability of ZFN-based genome-editing for the development of ZFP Therapeutics for the treatment of monogenic diseases. "These data demonstrate the potential of combining human iPSCs with ZFN-driven gene correction to generate differentiated cell-based therapies," stated Philip Gregory, D. Phil., Sangamo's vice president of research and chief scientific officer. "Importantly, analysis of the entire coding sequence of a ZFN-corrected iPSC line revealed that the only modification attributable to ZFN activity was the intended repair of the A1AT gene. This demonstrates the singular specificity that can be achieved using Sangamo's ZFP technology
Ouch. Quite the crater today. That news could not have been much worse. I wonder if they will abandon that product altogether.
Halted @ 3.43 upon negative trial result:
Sangamo BioSci announces Phase 2b trial of SB-509 in diabetic neuropathy did not meet key study endpoints (SGMO) 4.35 : Co announced that its Phase 2b study did not meet its primary or secondary clinical endpoints in subjects with moderate severity diabetic neuropathy as compared to placebo. SB-509 treatment did not show statistically significant improvements from baseline compared with placebo at 180 days in the primary endpoint, sural nerve conduction velocity the secondary endpoint, neuropathy impairment score in the lower limb or intraepidermal nerve fiber density. SB-509 was generally well-tolerated. There were three serious adverse events in the SB-509 treated group compared with three serious adverse events in the placebo group. The remaining adverse events were mild and reversible and were generally equivalent in both groups. (stock halted)
7:03AM Sangamo BioSci announces Phase 2b trial of SB-509 in diabetic neuropathy did not meet key study endpoints (SGMO) 4.35 : Co announced that its Phase 2b study did not meet its primary or secondary clinical endpoints in subjects with moderate severity diabetic neuropathy as compared to placebo. SB-509 treatment did not show statistically significant improvements from baseline compared with placebo at 180 days in the primary endpoint, sural nerve conduction velocity the secondary endpoint, neuropathy impairment score in the lower limb or intraepidermal nerve fiber density. SB-509 was generally well-tolerated. There were three serious adverse events in the SB-509 treated group compared with three serious adverse events in the placebo group. The remaining adverse events were mild and reversible and were generally equivalent in both groups. (stock halted)
Question from the multiple-challenged investor??
Would anyone be willing to help out WE who are challenged by math (stat sig?), science, FDA study speak, and just plain old senility??
What sort of numbers/percentages on the primary and secondary endpoints would be considered
A) Super
B) Adequate
C) Borderline/ambiguous
D) Awful.
TIA
It is not a zero-sum game. Other patients had both CCR5 copies disrupted but the heterozygote had larger percentages given that he had only one copy of the CCR5 gene to begin before the procedure. One non-heterozygote had a 2 log reduction in viral load during the 12 week treatment interruption.
The basic proof of concept has been demonstrated and the cells clearly have an antiviral effect. It is now a matter of optimization to get the percentage of biallelically modified cells high enough to reduce the virus to undetectable levels.
More good news for the price to go down. Just means it is on sale.
This stock has potential from many angles.
HIV
Diabetes
SAIL
KBLB
and I am sure more.
Should be a good one to hold. IMHO.
Good luck all
I think Motley Fool got it right:
"The patient who has been essentially cured of HIV already had one non-functional copy of CCR5. By knocking out the functional copy, the cells were left with no CCR5 genes.
To make this work beyond the 5% to 10% of HIV patients with a genetic mutation in one copy of CCR5, Sangamo needs to figure out a way to delete both copies of the gene in the T-cells."
Zinc what do you think is causing SGMOs drop sincer their announcement? I have not even begun to scrape the surface of SGMOs abilitys but I thought the results were good. Why the down trend ever since?
Webcast on webpage
Sangamo BioSciences Announces Presentation at the Sixth Annual JMP Securities Healthcare Conference
print
Sangamo Biosciences (NASDAQ:SGMO)
Historical Stock Chart
1 Month : August 2011 to September 2011
Sangamo BioSciences, Inc. (Nasdaq: SGMO) announced today that Edward Lanphier, Sangamo's president and CEO, will provide an update on the progress of Sangamo's ZFP Therapeutic® development programs and an overview of the company's business strategy at 3:30 pm ET on Tuesday, September 27, 2011 at the Sixth Annual JMP Securities Healthcare Conference which will be held in New York City.
The presentation will be webcast live and may be accessed via a link on the Sangamo BioSciences website in the Investor Relations section http://investor.sangamo.com/index.cfm under Events and Presentations. The presentation will be archived on the Sangamo website for two weeks after the event.
Thanks for your replay. I asked my sister about this topic. She is the director of the women and infant center in RI and a professor at Brown University. She sounded interested, but would like to see more studies as well as an article in in a science review.She also explained that gene splicing has been tried for years with little to no luck. I will still keep my eye on it. thanks again.
IMHO SGMO's HIV therapy should be fine for children (far better than other approaches)
It takes immune cells removed from the patient and removes their CCR5 receptors which HIV must use to enter the cells.
Because the rest of the patients immnune system is left intact, children treated this way should develop the same pattern of resistance to diseases that they otherwise would (if no HIV and no CCR5 removal).
It would also be better because compliance is not a problem: no need to take huge numbers of pills/day and no frequent injections either. And, of course none of the side effects that current HIV drugs have. I don''t know whether current HIV drugs would have adverse effects on child development but suspect that they might, There should be no problem in that respect with SGMO's HIV therapy either. (IMHO the stem cell branch is the one that would be best)
If you can post on the investorvillage SGMO board adenylyl (by a very wide margin the most knowledgeable (he's a research scientist in the field) could give you a very definitive answer if he's still there (have to be a paid subscriber to post)
I think I have the email address of a couple of posters there. If i can find one I'll see if I can get one of them to post your question there (you can read without a subscription).
Sorry for the late reply - been behind a bit on email etc.
Sangamo SGMO and their zinc finger IP was added to Patent Play Stocks
ZincFinger,
I have seen your post on KBLB and I have found you very knowledgable on the topic. My sister is the director of the women's and infant center in R.I and a professor at Brown university. I will see her this weekend and wanted to discuss SGMO 's HIV /Aids project. She is in charge of over 30 newborns everyday. I was wondering SGMO's process could be geared for children. Any thoughts? Thanks in advance.
Can anyone tell me if the zinc fingers and the HIV project would work on childhood HIV/AIDS?
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Sangamo Therapeutics, Inc., a clinical stage biopharmaceutical company, focuses on translating ground-breaking science into genomic therapies that transform patients' lives using platform technologies in genome editing, gene therapy, gene regulation, and cell therapy. The company?s proprietary zinc finger DNA-binding protein (ZFP) technology enables specific genome editing and gene regulation. The ZFPs could be engineered to make ZFP nucleases (ZFNs), proteins that could be used to specifically modify DNA sequences by adding or knocking out specific genes; and ZFP transcription factors (ZFP TFs), proteins that can be used to turn genes on or off. Its therapeutic products include SB-728-T, a ZFN-mediated autologous T-cell product for human immunodeficiency virus and acquired immunodeficiency syndrome (HIV/AIDS), which is in Phase II and Phase I clinical trials; and SB-728-HSPC that is in Phase I/II clinical trials for HIV/AIDS. The company also engages in Phase I/II studies of in vivo genome editing applications of ZFP Therapeutics for hemophilia B, Hemophilia A, and Mucopolysaccharidosis I (MPS) and MPS II, which are lysosomal storage disorder (LSD); proprietary preclinical programs in other LSDs; and research stage programs in certain central nervous system disorders and cancer immunotherapies. It has collaborative partnerships with Biogen Inc. to develop therapeutic genome editing products in hemoglobinopathies; and with Shire International GmbH to develop the preclinical development program in Huntington?s disease, as well as license agreement with Sigma-Aldrich Corporation to develop ZFP-based laboratory research reagents and Dow AgroSciences, LLC to modify the genomes or alter protein expression of plant cells, plants, or plant cell cultures. The company was formerly known as Sangamo BioSciences, Inc. and changed its name to Sangamo Therapeutics, Inc. in January 2017. Sangamo Therapeutics, Inc. was founded in 1995 and is headquartered in Richmond, California.
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