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harlem,
While it is possible to enroll a patient prior to submitting protocol to clinicaltrials.gov it seems many start recruitment after submission for possibly the reason below. No info at the site ..yet.
From January 3 ACT PR,
"Specific inclusion and exclusion requirements and Investigator contact information will be posted shortly at clinicaltrials.gov. Patients and their caregivers should refer to this source rather than contacting ACT or its representatives."
____________________________________________________________________
the registration of certain “applicable clinical trials” (see Definitions) in ClinicalTrials.gov no later than 21 days after the first subject is enrolled
http://grants.nih.gov/Clinicaltrials_fdaaa/index.htm?print=yes&
ClinicalTrials.gov facilitates registration of trials in accordance with the International Committee of Medical Journal Editors (ICMJE) initiative requiring prior entry of clinical trials in a public registry as a condition for publication.
The International Committee of Medical Journal Editors (ICJME) has established a requirement that all clinical trials be entered in a public registry before the onset of patient enrollment, as a condition of consideration for publication.
http://prsinfo.clinicaltrials.gov/
Why can I not find my trial in ClinicalTrials.gov?
After a protocol record has been entered (or modified) and marked as ‘Complete’, it must be approved and released by a PRS administrator. From the time the record is released, it normally takes between 2 and 5 working days for internal quality assurance review and processing for publication on the ClinicalTrials.gov website. Records that contain Results may take up to 30 days.
http://healthcare.partners.org/phsirb/ct.gov_faqdetails.htm
Advanced Cell Technology Interim Chairman and CEO Gary Rabin to Present at OneMedForum Finance Conference in San Francisco
Advanced Cell Tech (OTCBB:ACTC)
Intraday Stock Chart
Today : Wednesday 12 January 2011
Advanced Cell Technology, Inc. (“ACT”; OTCBB:ACTC), a leader in the field of regenerative medicine, announced today that Interim Chairman and Chief Executive Officer Gary Rabin will be presenting at the fourth annual OneMedForum Business Development and Finance Conference for Emerging Healthcare, in San Francisco. Mr. Rabin’s presentation is scheduled for today at 11:15AM PST, at the Sir Francis Drake Hotel, Renaissance Room.
OneMedForum features presentations by over 100 of the world’s most promising emerging life science public and private companies. Over 1,000 investors attended last year’s conference. This year’s event features a keynote speech by Nathan Mhryvold, co-founder of Intellectual Ventures and former Chief Technical Officer of Microsoft. To learn more about the forum, visit http://www.onemedplace.com.
A live, streaming webcast of the ACT presentation will be made available at http://www.ustream.tv/channel/onemedforum. The slide presentation will be made available on the company’s website at http://advancedcell.com/news-and-media/act-investor-and-stem-cell-conference-presentations/
ysung,
the following link may help if you are not already aware of how the JV with SCRMI is structured and CHA aquired the hemangioblast based technology which todays PR is based on.
http://investorshub.advfn.com/boards/read_msg.aspx?message_id=54716921
Hemangioblast Technology agreement
http://www.sec.gov/Archives/edgar/data/1140098/000114420409040583/v156251_ex10-126.htm
Hemangioblast Program. Hemangioblasts are a newly-characterized stem cell capable of differentiating into both hematopoietic, meaning blood cell-forming, and angiogenic, meaning blood vessel endothelium-forming, cells. We believe it will be possible to utilize hemangioblast cells in engraftment to repair age-related endothelial dysfunction associated with numerous significant age-related diseases, including cardiovascular disease, stroke, and perhaps even cancer. In 2006 we successfully derived hemangioblast cells generated from the company's blastomere-derived human embryonic stem cell lines. In 2007, we published data reporting that through utilization of hemangioblast based therapy we generated function in vivo with respect to the repair of ischemic retinal vasculatures and restoration of blood flow in ischemic limbs. In addition, we also reported increased survival rates of animals suffering from myocardial infarction. The hemangioblast program is currently in preclinical development.
ysung,
The paragraph below from todays PR best describes my response .
The first sentence tells you this an important progressional step towards their goal and is not a near term event.
The second sentence describes the magnitude of the potential.
"More research is clearly needed before this technology can advance into the clinic," stated Gary Rabin, Interim CEO and Chairman of ACT. "However, once this technology is perfected, we believe pluripotent stem cells -- both embryonic and iPS cells -- will play an important role in developing a renewable, donorless source of transfusable platelets."
http://custom.marketwatch.com/custom/tdameritrade-com/html-story.asp?guid=%7B54f58731-8a62-49a6-bde7-0fdac7a5dcef%7D
To understand the similarities and differences between the
EMEA and FDA requirements for initial submissions of new
medicinal products
• To take home practical suggestions for preparing the
EU IMPD CTA versus the US IND
• IMPD = Investigational Medicinal Product Dossier
• CTA = Clinical Trial Application
• IND = Investigational New Drug
http://www.pharmatek.com/pdf/PTEKU/Sept232008.pdf
2010 Blood/SCRMI/funding refresher links,
PATIENT-SPECIFIC AND UNIVERSAL DONOR BLOOD CELL FROM PROTEIN-INDUCED IPS CELLS
Project funding link from NIH site:
http://projectreporter.nih.gov/project_info_details.cfm?aid=8045716&icde=5465234
The NIH Award provides McLean Hospital and ACT's joint venture with CHA Biotech, Stem Cell & Regenerative Medicine International (SCRMI), $1.9 million to explore the potential of protein-induced pluripotent stem (iPS) cells as a source of universal red blood cells and platelets for transfusion.
http://investorshub.advfn.com/boards/read_msg.aspx?message_id=54681106
Interview with Dr Shi-Jiang Lu
Dr Lu is a senior director at Stem Cell and Regenerative International Inc.,
http://investorshub.advfn.com/boards/read_msg.aspx?message_id=50569793
"The firm had a solicitation at the Department of Defense for $5.6 million to create universal blood, but none of the Bush lines were O negative, and ACT was unable to get the White House to sign off on permitting the use of the company's single blastomere technique."
"They let the clock run out on it," Lanza said. If the NIH revises it guidelines and approves ACT's five single-blastomere lines, it would open the possibility that the firm could potentially get the DoD to reconsider, he added
http://investorshub.advfn.com/boards/read_msg.aspx?message_id=46950661
Actually, SCRMI has two focused objectives and todays PR is one of them.
Transfusable Blood Components:For decades, donor-derived supplies of transfusable blood components have failed to keep up with increasing demand. This continual shortfall has prompted efforts to develop safe and effective blood substitutes which can be produced from non-immunoreactive sources and in limitless quantities. We are exploring the utility of pluripotent stem cells to serve as an alternative source for producing various life-saving components of the blood including red blood cells (RBCs), megakaryocytes/platelets, and bone marrow repopulating cells.
Vascular Repair Cells:Several diseases, in various organ systems, are characterized by the destruction of vascular tissues and/or irreversible damage to specific endothelial cell populations. Major research efforts at SCRMI are focused on developing cell based-therapies for the repair and regeneration of damaged tissues associated with a wide variety of age-related conditions. Areas of research currently include retinal vascular, cardiovascular, and ischemic vascular repair
http://www.steminternational.com/our-focus/
Per request, links and info re: Orphan Desig-Europe
(Announced Dec.23,2010)
Advanced Cell Technology Files in Europe for Orphan Drug Designation For Its RPE Cells for Treatment of Stargardt’s Disease
http://www.businesswire.com/news/home/20101223005366/en/Advanced-Cell-Technology-Files-Europe-Orphan-Drug
Submission deadlines table:
http://www.ema.europa.eu/ema/index.jsp?curl=pages/regulation/general/general_content_000037.jsp&murl=menus/regulations/regulations.jsp&mid=WC0b01ac0580024c5d&jsenabled=true
Note: In accordance with Article 5.5 of Regulation (EC) No 141/2000, the COMP will reach an Opinion on a valid application for orphan designation within 90 days. Please note that the dates provided above correspond to the COMP meeting falling on or just prior to day 90. Opinions may be reached earlier than day 90 if no questions are raised by COMP.
Compassionate Use: (Questions, Guidelines)
http://www.ema.europa.eu/ema/index.jsp?curl=pages/regulation/general/general_content_000293.jsp&murl=menus/regulations/regulations.jsp&mid=WC0b01ac058007e691&jsenabled=true
slide 18 and 20..still the approx. 2 yr. timeframe,
http://www.thechairmansblog.com/william-caldwell/wp-content/uploads/2010/12/ACT-Macular-Degeneration-Program.pdf
It's IRB and here is an FDA link to them but you won't find any progress report on ACTC. When you see clinical trial data submitted to to the following site you will know the IRB has approved the start..http://clinicaltrials.gov/ct2/search
ACT Myoblast Phase 2 is here, hard to believe it has sat almost 3 years..
http://clinicaltrials.gov/ct2/show/NCT00626314?term=NCT00626314&rank=1
IBC? Do you mean IRB(institutional review board )?
JP,
Your question seemed like a good time to address a topic that according to the mail I get , needs clarification.
The FDA approval you speak of means to me FDA approval for SMD trials that are slated to start first. The trial has not yet been started. IMO, we are looking at a couple years at minimum before an NDA(New Drug Approval) would be possible. I base this on an expedited review of Orphan designations being about half of the normal 5-7 years time for "regular trials and NDA approval". The safety and efficacy are all the same as other trials and huge challenges exist as only 16-17% of all trials(orphan and non-orphan) receive FDA marketing approval. What the sp might be that far out is nothing but a WAG...thanks
A couple notables from FDA slide show in 2009 on Orphan Status,
(slide 14)
Marketing Application Fee for
Orphan Designated Products
In the Orphan Drug Act, Congress exempted
designated orphan products from the user fee
requirement
How does this help the company?
In FY 2009, the company would save the user fee of
$1,247,200 per application, which would otherwise
be paid to FDA whether their product is approved
or not
(slide 15)
Successes Since 1983
2870 Designation requests submitted
Approximately 2006 products have received
orphan-drug designation.
335 orphan-designated products have received
FDA approval for marketing.
Approved orphan-designated products are
available to treat patient populations totaling 25
million in the U.S.
http://www.fsma.org/UploadedFiles/FSMACommunity/Conference/2009Presentations/Linda%20Ulrich,%20M.D.%20OOPD%20FDA.pdf
chot,
Analysis is a strong word, it's just one of many ways to measure a companies value. Enterprise value is actually more what I would use verses just looking at the market cap figures. It puts the dilutive shares in place and can uncover large debt. If EV was my only criteria for making an investment I wouldn't be in ACT either.
3 decent reads on Orphan Status/approval and off-label drug use
Below is a decent read of "highlights" and the report is about one year old. No, I am not paying $3800 to get full report..lol
1)Opportunities in Orphan DrugsStrategies for developing maximum
returns from niche indications
http://www.globalbusinessinsights.com/content/rbhc0251m.pdf
2)Orphan Status Designations(Some FDA Stats)
The 1983 US Orphan Drug Act (ODA) established fiscal incentives for companies that produced promising drugs to treat rare diseases, defined as a disease that occurs in <200,000 people in the US. The OOPD recently published two articles assessing the characteristics and distribution of orphan designations and approvals by the US FDA. The first, by Braun et al, determined that the FDA granted 1,892 orphan designations from 1983 to August 2008, of which 326 received marketing approval. More than one drug can be designated for each orphan disease, and more than 200 diseases are represented among the approvals. Cancer was the most commonly designated category of disease and also had the greatest number of marketing approvals. The largest number of approvals for any condition was growth hormone deficiency (nine) and the second largest was AIDS (eight), which also had the largest number of orphan designations (57). Orphan drugs can be designated for more than one disease, with 1,243 drugs representing the 1,892 designations. In addition, there were 247 different drugs among the 326 orphan designated drugs that received marketing approval, 36 of which received two or more marketing approvals. Approximately half of the approvals occurred by four years after being granted designation, and the most common patient population size was fewer than 10,000 patients. In summary, there were only ten drugs that received FDA approval for the treatment of a rare disease in the 8-10 years before the ODA, thus the ODA may have contributed to a large number of drugs being developed for a variety of rare diseases, which has implications on future development and marketing of rare disease therapeutics.
http://www.fda.gov/ForIndustry/DevelopingProductsforRareDiseasesConditions/ucm217869.htm
3)Subject: Off-Label Drug and Approved Orphan Drug Use
Current Effective Date: 04/21/2010
Clinical UM Guideline
http://www.anthem.com/medicalpolicies/guidelines/gl_pw_a048553.htm
ison,
below are the perks(incentives) of Orphan Desig, all but the 7 year exclusive could be or going into play anytime.
NDA,
http://www.fda.gov/Drugs/DevelopmentApprovalProcess/HowDrugsareDevelopedandApproved/ApprovalApplications/NewDrugApplicationNDA/default.htm
(perks)
"The 1983 Orphan Drug Act guarantees the developer of an orphan product seven years of market exclusivity following the approval of the product by the FDA. As a result of the Orphan Drug Act, the following procedures are administered by the Office of Orphan Products Development:
Overseeing the orphan product program that gives sponsors seven years of exclusive marketing for orphan products.
Coordinating research study design assistance for sponsors of drugs for rare diseases.
Encouraging sponsors to conduct open protocols, allowing patients to be added to ongoing studies.
Awarding grant funding to defray costs of qualified clinical testing incurred in connection with the development of drugs for rare diseases and conditions."
http://investorshub.advfn.com/boards/read_msg.aspx?message_id=41946204&txt2find=orphan
Part 3,
Summary: I tried to lay out some info as it relates to our future based on acceptable and positive SMD trials under Orphan Designation and the eventual FDA NDA(new drug approval) that follows if everything goes as planned.
As prior info shows some factual examples of how one orphan designation prospered in a big way for many companies by expanding into nonorphan indications(which ACT has done with AMD)and the very possible and potential area of off-label usage. There are over 200 diseases of the retina so given prior examples of blockbuster sucess cited earlier it would seem to me that if the SMD Orphan trial is successful and declared safe using the Ma09-hRPE cell line
in humans we are in a great position to capitalize just as the other examples did and maybe more.
All of this implies to me that ACT can go 7 years from market approval without the FDA considering any more appovals for same indication. In the mean time, ACT has built a formidable barrier around the RPE as they have received 3 patents in the last 7 months protecting development and process of RPE cells.
Whether the WARF commercial license agreement is or is not in ACT's possesion seems less important, at least to me, for the next several years. By the time the market exclusive 7 years has expired and provided ACT exists in the present form, maybe someone else(partner or PHARMA) can take it to the final commercial end game as ACT by then will certainly be in a large leveraging position with the possibility of some great revenues to call the shots and produce shareholder value whether by buyout or partnership or whatever form..
The Orphan Drug ACT has put into place a system where many companies have and are capitalizing on. The FDA sights "abuse" of the system with regards to AIDS as billions have been made. They may call it that but it is their system and many companies are busy going to the bank and big Pharmas step up to the plate when trials or technology is proven.
The KEY for ACT, imo, is to start treating patients, show safety of the Ma-09 hRPE in humans, produce positive results through all phases, receive NDA for marketing and then apply some or all of the Orphan advantages, move forward with nonorphan indications and use or take advantage of off label usage.
I certainly welcome all opinions whether you disagree or agree...Food for thought with actual examples of how lucrative it could be without commercializing and using the Oprhan process..thanks
Part 2,
Back in November of 2009, ACT filed their first IND announcing the Orphan Drug route in the SMD area with the explanation they felt it would be the right first step in advancing towards the much larger trial involving AMD. This in fact turned out to be the right move, the SMD IND took one year to gain safety clearnace from the FDA and the much more lucrative IND for AMD "dovetailed" quickly into the clearance from FDA as much of the same info and same cell line were involved.
As we all know the Orphan status is set up to supply incentives for companies to work/research these rare diseases. The one I will key on here is the 7 year market exclusive. FWIW, if designation is granted in Europe they provide 10 year marketing exclusive.
Once we learned of the Orphan route from ACT, I posted reference to an article that I deemed very important to explaining the ins and outs. I went back and re-read that a couple times this weekend and it was the catalyst for posting what I am today. Below, in bold, is the part that flipped a light switch on with regards to current and future possibilities without commercializing, at least for several years or more. Please read the entire link as ALL the info is applicable and important, imo.
"Once established as an orphan drug, off-label usage and expansion to nonorphan indications can lead to an increase in sales. Genentech’s Rituxan, for example, became a blockbuster drug within a short span of time as it was prescribed for many off-label indications. Drugs such as Centocor’s Remicade have become blockbusters due to expansion to nonorphan indications including rheumatoid arthritis. In fact, 12 out of 19 leading orphan drugs had just one approval for an orphan indication but became blockbusters by expanding to nonorphan indications or by off-label usage"
http://www.genengnews.com/gen-articles/biomarket-trends-orphan-drug-arena-driven-by-biologics/2318/
KEY WORDS: off label usage and nonorphan indications
Scroll down on this FDA LINK and read definition of
On-Label Uses and Off-Label Uses
Orphan Drug Act (ODA) of 1983
I deem this passage as the point of my posts here today.
In fact, 12 out of 19 leading orphan drugs had just one approval for an orphan indication but became blockbusters by expanding to nonorphan indications or by off-label usage
Upcoming Part 3 will try to summarize my ramblings. Putting this together in a coherent fashion was difficult today for some reason, old age or maybe being a weekend ware part of it..:)
Part 1,
For those that may not have been following the discussion on the WARF license and whether or not ACT currently is licensed or not under the(supposedly) broad patent protection of WARF is the basis for which I went back in time to try understanding ACT's strategy with the clinical trials and the possibilities therof.
My interest in doing so was sparked by a question I posed yesterday.
Is it essential at this time or in the near future(years)that we have such broad patent protection or not?
IMO, no. The WARF contract gives the right to use their technology and patents for research but the main emphasis is on commercializing. On many occasions Caldwell mentioned "Freedom to Operate" and it is descried quite well in the following link.
"In such cases, without prior authorization of the patent owners, the risks of being accused of infringement are extremely high. This is why many companies, prior to launching a new product, and often even prior to initiating a new line of research that may lead to the development of a new product, seek to minimize the risk of infringement by securing their "freedom to operate" (FTO), i.e. ensuring that the commercial production, marketing and use of their new product, process or service does not infringe the intellectual property rights of others."
http://www.wipo.int/sme/en/documents/freedom_to_operate.html
Does ACT have in their plans being able to commercialize?
I don't believe so and have stated that on several occasions and Caldwell saw it as a stretch also about a year ago.(quote below)
"For a small biotechnology company with limited resources, commercialization is challenging. If we reach that point, it is possible that the Company would either have been acquired or have partnered with a larger company more financially capable of commercializing the technology."
http://www.sec.gov/Archives/edgar/data/1140098/000101376209001648/form14a.htm
With all of the above in mind, what or where does that leave us in the upcoming years? Part 2 describes a unique situation with many examples of factual results.
asif and 3Dicon,
welcome, the recent discussions here on the WARF license topic had me doing some digging the past couple days and I will be throwing out some "thinking outside the box" posts that possibly have some merit and hopefully will draw discussion from all. btw asif, I am not a moderator on this board..thanks
MA09-hRPE cell line listings
BioCentury is the essential source of intelligence for a global audience of biotech and pharmaceutical executives and investors who demand deep industry knowledge, data-driven analysis, independent perspectiveand a commitment to accuracy.
http://www.biocentury.com/products/ma09-hrpe_cells
The portal for rare diseases and orphan drugshttp://www.orpha.net/consor/cgi-bin/Drugs_Search.php?lng=EN&data_id=76142&search=Drugs_Search_Simple&data_type=Status&Typ=Sub
"Even without Patent No. 7,029,913, Geron has significant patent protections regarding use of eSCs and probably, more importantly, induced pluripotent SCs"
http://www.robindayne.com/newsletter-article-options-moore/305
pwise,
welcome to the board. Currently, to be honest, I have no definitive answers to the fundamental hESC patent deal. I do know there are many companies licensed under WARF but as you know, only 2 have been cleared for hESC trials, ACT(2) and Geron(1). Geron has an exclusive as I posted earlier and currently I don't know if ACT has a license with them. I guess the questions I seek answers to are 1)do we need it? 2)if not, what is in our arsenal to replace or overide?
a caveat, pwise, just because I don't have the answers yet doesn't mean they are not there and it doesn't mean ACT doesn't have what they need and are comfortable with it. These are things that I feel could be easily coordinated on an RPE section of their website as I mentioned in last post..
The fundamental hESC WARF/WiCell discussion has prompted some of you to ask about the RPE side of things. First of all, YES, we did receive a couple patents covering our RPE cell programs back in September. You folks that mailed are correct, there is NO reason not to have this info listed under the "Press Releases" section of Company website and NO reason the new patents are not listed under "IP" section....Currently ACT has two approved RPE trials and agressively pursuing Europe for the same. There is a lot of marbles riding on the RPE TRIALS and therefore, IMO, a large portion of their site should be RPE and the very least UPDATED...enough on that. here is the info you folks were asking about.
ACT Obtains Broad Patent Covering Use of Stem Cell-Derived RPE Cells to Treat Eye Disorders
http://investorshub.advfn.com/boards/read_msg.aspx?message_id=54368736
patent number 7,794,704 broadly cover methods for treating retinal degeneration using human RPE cells differentiated from human embryonic stem cells (hESCs).
http://www.freepatentsonline.com/7794704.html
Patent 7,795,025 contribute to the development of the company’s protection of the processes for manufacturing RPE cells from human ES cells.
http://www.freepatentsonline.com/7795025.html
rumit,
at this stage I am not sure if anyone knows the final outcome of patent battles that are in play and the ones that will surely arise. Of course Geron has an "exclusive" with WARF as they funded the original work at University of Wisconsin-Madison. I will post to this board anything I can find out directly relating to WARF/WiCell and ACT agreements, I would be suprised if they give me the info but you never know...
rumit,
logically they must have resolved this licensing agreement with WARF
As of right now rumit I am not convinced that is the case. The 2009 10K was filed March 16 of 2010. So it is obvious as of that date at least nothing was done or they never would have pulled the info.
No S-1 or any S-1A's or POSAM filings have updated either and the last one was 12-21-2010. That in itself is not proof positive of anything, but there is no doubt they were in breach." We are currently behind on our payments of the license fees, since 2008, and as such we are in breach of the license agreement."
rumit, it's possible Rabin can help. I am not sure if WARF will disclose anything of that nature regarding license agreements but I have an e-mail out to them, left a recorded message at WARF licensing Dept. and if all else fails to deliver I will drive there and see what I can muster up, I am only 15 minutes from them.
On another note, the timing of this possible breach had me checking other events with WARF. Pfizer..then Pfizer+Coffey+ACT mentioned
May 05, 2009 02:32 PM Eastern Time
Pfizer And Wisconsin Alumni Research Foundation (WARF) Sign License Agreement For Human Embryonic Stem Cells
http://www.businesswire.com/news/home/20090505006451/en/Pfizer-Wisconsin-Alumni-Research-Foundation-WARF-Sign
Pfizer Licensing Deal with WARF Allows Firm to Develop hESC-based Therapies, Discovery Tools
published on May 12.
Pfizer and the Wisconsin Alumni Research Foundation said last week that they have inked a licensing agreement that gives Pfizer the right to work with human embryonic stem cells for drug research and discovery.
With the deal, WARF has now inked 35 licensing deals with 27 corporate partners for its portfolio of patents related to isolating and differentiating hESCs in the late 1990s, Janet Kelly, WARF's director communications, wrote in an e-mail to BTW.
Pfizer is also the largest company and only pure-play pharmaceutical company that WARF has disclosed as a licensee of its hESC patent portfolio, Kelly said.
As such, the pharma giant's recent foray into regenerative medicine and subsequent need to license WARF's hESC patents for freedom to operate could symbolize a growing industry trend and additional pharma licenses for WARF's most lucrative IP asset.
WARF, the non-profit technology-licensing arm of the University of Wisconsin-Madison, "has licensed stem cell technologies to other pure-play pharma companies," Kelly said. However, those licensees have requested that their deals remain confidential.
Kelly said that other pharmaceutical companies have also expressed interest in licensing WARF's stem-cell patents, but their identities likewise could not be disclosed.
Other licensees of the patent that have been disclosed include Invitrogen, BioTime, Cellartis, CellCura, ES Cell International, Aruna Biomedical, Cellular Dynamics International, Becton Dickinson, ProteoSys, Geron, Stem Cell Technologies, Chemicon (a subsidiary of Millipore), Advanced Cell Technology, and Stemina Biomarker Discovery.
For Pfizer, the agreement gives the company's recently formed Pfizer Regenerative Medicine unit the freedom to pursue its goal of developing new stem cell-based therapies for a variety of diseases, as well as tools for drug-discovery research, the company said.
Under the terms of the agreement, the financial details of which have not been disclosed, WARF has granted Pfizer a license to "work with hES cells for drug research and discovery," the company said in a statement.
"Our license with WARF provides us with new information and materials that will allow us to use their cell lines to explore a whole new range of therapies," Ruth McKernan, chief scientific officer of Pfizer Regenerative Medicine, said in a statement.
Pfizer said that its stem-cell R&D program has three components: to use human cells as tools in its drug-discovery research; to improve the safety of new treatments; and "most importantly, to move towards cell therapy."
In an e-mail to BTW, McKernan elaborated that Pfizer Regenerative Medicine "bases its stem-cell research and drug-discovery efforts on a full set of drug-discovery capabilities, including in vivo and translational stem cell research, cell and molecular biology, and biological chemistry."
Pfizer launched its regenerative medicine unit in November to explore "an exciting array of promising technologies," McKernan said. "This unit examines novel biologics, small-molecule therapies, antibody technologies, and a variety of cell and tissue replacement strategies."
McKernan added that the unit will grow to approximately 70 colleagues by next year, with efforts focused at two locations: researchers based at the company's Research Technology Center in Cambridge, Mass., will focus on using stem cells to develop therapies for diabetes, she said.
Meantime, scientists at Pfizer's Cambridge, UK, location will focus on age-related and degenerative disorders with "a particular interest in common cellular mechanisms and disorders of the central and peripheral nervous system," McKernan said.
Both locations will achieve their goals "through in-house research and a vast array of alliances and collaborations," McKernan added.
One of those alliances, announced last month, is a research collaboration between Pfizer Regenerative Medicine and the University College London to develop stem cell-based therapies for ophthalmologic diseases (see BTW, 4/29/2009).
Led by Peter Coffey, a professor at UCL's Institute of Ophthalmology, the collaboration will primarily explore differentiating stem cells into retinal pigment epithelium cells to develop therapies for wet and dry age-related macular degeneration and other retinal diseases. Pfizer is providing an undisclosed amount of funding to support the project.
McKernan said that Pfizer's deal with WARF "does not preclude" the company from exploring a research collaboration with Thomson or another researcher at UWM; however, the recently announced agreement is strictly a licensing deal, she said.
In a statement, Carl Gulbrandsen, managing director of WARF, said that the organization was "pleased to enter into this licensing agreement with Pfizer," whose "access to this key intellectual property may have far-reaching benefits for both public health and Wisconsin's growing biotechnology industry."
http://www.genomeweb.com/biotechtransferweek/pfizer-licensing-deal-warf-allows-firm-develop-hesc-based-therapies-discovery-to
investing,
fwiw, the mother non human patent for the same that came in a month or so ago took another 36 days from the point you describe now to being issued...
Patent Number: 7,838,727
Patent: 11-23-2010
11-23-2010 Recordation of Patent Grant Mailed
11-03-2010 Issue Notification Mailed
11-23-2010 Patent Issue Date Used in PTA Calculation
10-18-2010 Dispatch to FDC
10-18-2010 Application Is Considered Ready for Issue
The 2009 10 K while absent WiCell and WARF are showing UMASS with the same notation the others had..Hopefully that has been taken care of, the 10K in March will tell the story.
Licenses of Intellectual Property to Us
The following summarizes technology licensed to us.
UMass License - On February 1, 2002 and April 16, 1996, we entered into exclusive license agreements (indefinite license period) with the University of Massachusetts. The 1996 Agreement has been amended by amendments dated September 1, 1997, May 31, 2000 and September 19, 2002. Pursuant to these agreements, the University of Massachusetts, referred to as UMass, exclusively licensed to us certain biological materials, patent rights and related technology for commercialization in specified fields. The license agreements require us to use diligent efforts to develop licensed products and licensed services and require us to pay certain royalties, minimum annual royalties, milestone payments and sublicense income to UMass. UMass received 73,263 shares of common stock of ACT as partial consideration of the license granted. We are currently behind on our payments of all UMass license fees, since 2008, and as such we are in breach of the license agreement.
rumit,
This is the "something" I wanted to check out.
License agreements with both Wicell and WARF appear in the 2008 10K
but neither are listed in 2009 10K. I have checked and re-checked rumit, it appears for now we may have breached and lost those agreements due to the info below.
I posted this as NEW back in July 2009,
"We are not in full compliance with some of our license agreements. We are not in full compliance with some of our licenses (see Our Intellectual Property in the BUSINESS section of this 10k) and due to limited financial resources we cannot guarantee that we will regain full compliance status." http://investorshub.advfn.com/boards/read_msg.aspx?message_id=39323481
(page 15) WiCell 2002 License
We are currently behind on our payments of the license fees, since 2008, and as such we are in breach of the license agreemen
http://www.sec.gov/Archives/edgar/data/1140098/000114420409036193/v154150_10k.htm
I checked for prior year(2008)10K and they were listed.
WiCell 2002 License - In March 2002, we entered into an industry research license and material transfer agreement (indefinite license period) with WiCell Research Institute, Inc., referred to as WiCell, pursuant to which WiCell granted to us a non-exclusive license, with no right to sublicense, to make, use and sell or otherwise transfer certain primate embryonic stem cells and derivatives thereof for internal research purposes and to receive such primate embryonic stem cells or derivatives from third parties for internal research purposes. In consideration of the license granted to us by WiCell, we agreed to pay a license fee of $100,000 and an annual maintenance fee of $25,000. The license includes a grant from us to WiCell of a non-exclusive, royalty-free, irrevocable, paid-up research license under any inventions made by or for us to the extent that such inventions are a modification of an invention described in the licensed patent rights. We are currently behind on our payments of the license fees, since 2008, and as such we are in breach of the license agreement.
WARF and WiCell 2007 License - On May 2, 2007, we entered into a commercial products addendum (indefinite period) with the Wisconsin Alumni Research Foundation ("WARF") and its subsidiary, WiCell Research Institute, Inc., ("WiCell"). The addendum amends in certain respects the industry research license and material transfer agreement discussed above. The addendum (i) grants to us a non-exclusive, world-wide commercial license to 23 issued patents and 123 patents pending to pursue therapeutic and research products utilizing human embryonic stem cell technology and (ii) provides for certain sublicensing rights to enable us to further the development and commercialization of products. The addendum requires us to make certain royalty payments and pay license fees to WARF as set forth in the addendum. The maintenance fees required under the 2002 WiCell License are waived during the term of the 2007 License.
rumit,
here is the whole license agreement with WARF, fee schedule and royalties below..still need to check on that "sometthing"
A. License Fee.
In consideration of the rights granted under this Agreement, Licensee agrees to pay to WARF a license fee of Six Hundred Thousand Dollars ($600,000) (the “Initial License Fee”) in accordance with the payment schedule set forth herein this Section 4; provided, however, Licensee shall be entitled to a credit in the amount of Two Hundred Twenty Five Thousand Dollars ($225,000) representing the license fees paid to WARF by Licensee as required under the Research Agreement. Licensee shall pay to WARF the Initial License Fee in twelve (12) installments of $31,250 each. The installments shall be due on a quarterly basis as follows:
B. Royalty.
Licensee also agrees to pay to WARF as “earned royalties” a royalty of (i) four percent (4.0%) of the Net Selling Price of Therapeutic Products, and (ii) two percent (2.0%) of the Net Selling Price of Diagnostic Products and Research Products, sold or transferred under this Agreement. The royalty is deemed earned as of the earlier of the date the Licensed Product is actually sold, leased or otherwise transferred for consideration, the date an invoice is sent by Licensee, or the date a Licensed Product is transferred to a third party for any promotional reasons. Licensee acknowledges and agrees that the royalty paid hereunder is provided as additional consideration for the rights afforded under the Research Agreement which led to the discovery and/or development of the Licensed Products to be commercialized under this Agreement. Licensee is required to pay only one royalty with respect to a Licensed Product regardless of the number of patents or patent applications included within the Licensed Patents whose claims cover that Licensed Product.
http://www.sec.gov/Archives/edgar/data/1140098/000110465907062594/a07-18956_1ex10d1.htm
rumit,
Here is some info, I will be back at you soon as I need to check something that may or may not be relevant..
Advanced Cell Technology and Wisconsin Alumni Research Foundation Announce Commercial License Agreement
http://www.sec.gov/Archives/edgar/data/1140098/000110465907037066/a07-13392_1ex99d1.htm
ITEM 1.01 Entry into a Material Definitive Agreement.
Agreement with WARF and WiCell
On May 2, 2007, Advanced Cell Technology, Inc., a Delaware corporation (the “Company”), entered into that certain Commercial Products Addendum (the “Agreement”) with the Wisconsin Alumni Research Foundation (“WARF”) and its subsidiary, WiCell Research Institute, Inc, (“WiCell”). The Agreement amends in certain respects that certain Industry Research License and Material Transfer Agreement dated March 1, 2002 by and among the Company, WARF and WiCell.
The Agreement (i) grants the Company a non-exclusive, world-wide commercial license to 23 issued patents and 123 patents pending to pursue therapeutic and research products utilizing human embryonic stem cell technology and (ii) provides for certain sublicensing rights to enable the Company to further the development and commercialization of products. The Agreement requires the Company to make certain royalty payments and pay license fees to WARF as set forth in the Agreement.
The Agreement described above will be filed as an exhibit to our Quarterly Report on Form 10-QSB for the quarter ending June 30, 2007, with portions omitted and filed separately with the Secretary of the Securities and Exchange Commission pursuant to a request for confidential treatment.
http://www.sec.gov/Archives/edgar/data/1140098/000110465907037066/a07-13392_18k.htm
wild4,
" My Mom in law who is 75 ( but great physical shape ) is suffering from Mac Deg and has lost practically all the sight in one eye and most in the other"
Sorry for confusion,
I misunderstood, the previous info was for SMD.
More applicable would be the latest cleared trial for AMD. This is about one month behind the SMD so the IRB process is still ongoing and will come in at a later time. You are right, at least for now, on the sites being in the west.
The Phase I/II trial will be a prospective, open-label study that is designed to determine the safety and tolerability of the RPE cells following sub-retinal transplantation into patients with Dry AMD. Twelve patients will be enrolled in the study at multiple clinical sites. Sites currently under consideration are the Jules Stein Eye Institute at UCLA, and the Ophthalmology Department at Stanford University School of Medicine. Additional sites may be considered.
wild4,
welcome and good luck with your purchases. Below are a couple sites out east that were mentioned in PR. Which ones were actually decided upon I am not sure. The trials are yet to open for recruitment, info will be posted at this link to signify all the details and who to contact. Management expects this info to be posted "shortly"....http://clinicaltrials.gov/
The sites which are currently under consideration are the Jules Stein Eye Institute at UCLA (headed by Dr. Steven Schwartz); the Casey Eye Institute in Portland, Oregon (headed by Dr. Peter Francis of the Oregon Health Sciences University); the University of Massachusetts Memorial Medical Center in Worcester, Massachusetts (headed by Dr. Shalesh Kaushal, Chair of the Department of Ophthalmology); the UMDNJ – New Jersey Medical School in Newark, New Jersey (headed by Dr. Marco Zarbin, Chair, Institute of Ophthalmology and Visual Science); additional sites may be considered.
http://www.advancedcell.com/news-and-media/press-releases/advanced-cell-technology-receives-fda-clearance-for-the-first-clinical-trial-using-embryonic-stem-cel/
(Jan 3)
Specific inclusion and exclusion requirements and Investigator contact information will be posted shortly at clinicaltrials.gov. Patients and their caregivers should refer to this source rather than contacting ACT or its representatives.
http://www.advancedcell.com/news-and-media/press-releases/advanced-cell-technology-receives-fda-clearance-for-clinical-trials-using-embryonic-stem-cells-to-tre/
To all,
I will copy and paste what I just sent to our friend louisa,
"you are so welcome. I just finished convincing myself so why not share? All that boring reading has me in need of some eye relief...maybe an RPE injection would help?..lol "
The following is the long term safety/function report using the MA01 and MA09 cell lines to create the hRPE clinical grade cells.
This is a fine read of the development process and protections that ACT has taken to satisfy FDA requirements.
One of the Genetic tests were Polymerase Chain Reaction, or PCR.
The master cell bank was cytogenetically analyzed by G-banding karyotype analysis.
Long-Term Safety and Function of RPE from Human Embryonic
Stem Cells in Preclinical Models of Macular Degeneration
BIN LU,a CHRISTOPHER MALCUIT,b SHAOMEI WANG,a SERGEJ GIRMAN,a PETER FRANCIS,a LINDA LEMIEUX,b
ROBERT LANZA,b RAYMOND LUND
http://www.advancedcell.com/documents/0000/0237/ACT_esRPE_Stem_cell_June_11__2009.pdf
(Page 5)
Pathogen Testing and Stability of hRPE
The most important criterion to consider in release of a product
for clinical application is product safety. To ensure that our hRPE product was free of contamination during the extensive
culture and differentiation process, we carried out the following
testing according to U.S. Food and Drug Administration
and International Conference on Harmonization
guidelines for applicable microbial and viral agents on our
master cell bank: United States Pharmacopeia membrane filtration
sterility, fluorochrome-based mycoplasma, transmission
electron microscopy for viral particles, in vitro tissue culture
safety testing for adventitious agents, in vivo inapparent virus
detection, PCR-based reverse transcriptase detection, HIV-1,
HIV-2, HBV, HCV, CMV, HTLV-1 and -2, parvovirus B19,
Epstein-Barr virus, and herpesvirus 6. Additionally, the master cell bank was cytogenetically analyzed by G-banding karyotype
analysis. Results confirmed that these cell lines are karyotypically
stable with no presence of infectious pathogens teratoma and/or tumor formation.
DISCUSSION
We showed the long-term safety and efficacy of hESC-derived
RPE cells produced under manufacturing conditions applicable
for use in human clinical trials. In addition to the development
of assays with qualified range limits (which constituted the
‘‘identity’’ of the final hRPE product), extensive pathogen testing was carried out to ensure that the manufacturing procedure
did not introduce any infectious diseases or adventitious agents
into the final product. To confirm the functionality of these
GMP-compliant cells, both dose-response and long-term efficacy
were evaluated in homologous models of human retinal
disease. Because of the proliferative nature of hESCs, evidence
of safety under Good Laboratory Practice conditions
(21CFR58) is imperative for translating hESC-derived cellular
products into the clinic. The extensive characterization
detailed above provides assurance of cellular identity, whereas
the long-term tumorigenicity study presented here provides
strong evidence that the hESC-RPE cells are safe and do not
form teratomas and/or tumors during the lifetime of NIH III
immune-deficient mice.
Another decent read in general if you haven't seen already,
STEM CELLS: An Industry Overview..March 4, 2010
Page 4 snippet is pertinent to recent discussion on "report"
Possibly ACT is monitoring their cell bank in the same fashion?
"Athersys also passages cells in culture over 30 times but performs genome-wide analysis, SNP analysis and expression profiling routinely in order to ensure the working cell banks remain identical to the master cell bank."
Page 24 is ACTC Info(they are mentioned several times in overview)
http://www.nationalsecurities.com/research/Stem%20Cells%20National.3.4.2010.pdf
SNP Analysis+ACT,
digging around I found the following SNP applications in some of ACT research papers..If you take the time and read(boring?) you will see that the SNP analysis was used extensively.
2009
Reprogramming of Human Somatic Cells Using
Human and Animal Oocytes
(Page 4)
Genomic single nucleotide polymoprhism (SNP) genotyping
and mtDNA resequencing of human SCNT embryos.
All 262K SNP genotypes were compared
to generate % similarities for all possible comparisons
of embryonic WGA with donor total DNA. SNP microarray
Genomic SNP genotyping and mtDNA PCR
for iSCNT embryos
(page 7)
Each SNP was assigned a value from 0
to 5, and the overall chromosome was assigned a copy number
based on the majority of SNP copy number assignments
(full karyotypes were based on the copy number of each
chromosome). This technique has been previously used to
examine the karyotype of stable cell lines with various established
karyotypes
(page 12)
SUPPLEMENTARY TABLE 2. KARYOTYPE OF SCNT
EMBRYOS USING SNP MICROARRAYS
SUPPLEMENTARY TABLE 3. SNP CALL RATE AND
CHROMOSOME KARYOTYPING OF ISCNT EMBRYOS
USING SNP MICROARRAY
http://www.advancedcell.com/documents/0000/0003/Human-Cloning.pdf
FWIW, These reports can be expected on many fronts in the future,imo. Lanza's report of early aging of iPSC's caused some agnst too. Biotime fired back a few months later saying they solved the problem. Take them in stride until something says different..
(Lanza)
http://www.newsweek.com/2010/02/10/still-no-truce-in-the-stem-cell-wars.html
(Biotime)
http://nextbigfuture.com/2010/04/biotime-reverses-aging-of-human-cells.html
investing,
MA09 Cell line: is a pluripotent cell line that is derived from early stage human embryos
the hesc-RPE: The RPE cells were generated from the MA09 cell line. A working bank of RPE cells were then created and characterized, purity tested to the point they are "terminally differentiated", meaning they can no longer revert back to an embryonic state. So yes, the RPE cell product is no longer pluripotent.
Report: The only issue, if there even is one, is the report is stating some of the problems happen when in culture during differentiating process and cannot be detected by "normal" means. This may mean nothing to the MA09- RPE cells we will use in both trials but may have some effect on the MA09 cell line itself..jmo