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Something seems to be brewing at SGMO. Recent price runup. Directors exercising shares. Biogen funding & neurogenetic research starting. ZFN 2.0. Lots of positive signs.
$SGMO Today"s volume , 5.5 mil, is the 5th highest volume day for the last 12 months, and shorts only backed it up a penny today. There's not only retail and pro shorts, but the market makers as well are short, today leading up to tomorrow's huge conference. Squeeze is on This is a $100.00 stock in a year or two. Special Announcement will dropped another partnership with a new blue chip biotech
Biogen just bought 17% of the company in one of the largest biotech deals in history
https://finance.yahoo.com/news/sangamo-therapeutics-announces-participation-upcoming-133000246.html
Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, announced today that management will present at the following investor conferences:
Cowen 40th Annual Health Care Conference
Presentation Date: Wednesday, March 4th at 10:00 a.m. Eastern Time
Location: Boston, MA
Barclays Global Healthcare Conference
Presentation Date: Wednesday, March 11th at 8:30 a.m. Eastern Time
Location: Miami, FL
Presentations will be webcast live and may be accessed via a link on the Sangamo Therapeutics website in the Investors and Media section under Events and Presentations. The presentation will also be available on the Sangamo website after the event.
About Sangamo Therapeutics
Sangamo Therapeutics is committed to translating ground-breaking science into genomic medicines with the potential to transform patients’ lives using gene therapy, ex vivo gene-edited cell therapy, and in vivo genome editing and genome regulation. For more information about Sangamo, visit www.sangamo.com.
$SGMO
The Deal With Biogen Boosts The Recommendation To Buy Sangamo Shares
Mar. 2, 2020 8:51 AM ET|9 comments | About: Sangamo Therapeutics, Inc. (SGMO), Includes: BIIB
The new estimate for cash runway goes to five years.
Biogen will own 17% of Sangamo, future buyout potential emerges.
Sangamo's undervalued condition is even more apparent.
The recommendation update
Biogen Inc. (BIIB) and Sangamo's Therapeutics Inc. (SGMO) agreement have eclipsed my recommendation found in my recent article. Despite adding 24M to 116M outstanding shares, Biogen as the largest shareholder of Sangamo offers a strong hint that the company is, in fact, on the radar of big pharma. Substantial equity ownership by Biogen could serve as a springboard to the acquisition of the company under the right financial circumstances.
A central nervous system (NYSE:CNS) partnership, which I thought initially would take place in 2021, adds confidence to the investment thesis. Since Biogen is buying shares at $9.21, this is a very comfortable upper limit to be used for accumulation. I expect shares to trade in double digits, even before Pfizer's phase 3 trial or SB-525 updates are publicized.
The deal for Alzheimer's and Parkinson's, plus one not identified target, adds $350M to Sangamo's coffers, which had about $385M at the end of 2019, based on the Q4 release. In the past, the cash runway was expected to last to the end of 2021, and with the new money, I assume, Sangamo can operate until the end of 2023. Pfizer's objective to commercialize an SB-525 program as gene therapy for hemophilia A is expected to deliver $275M in milestones by 2022. Provided milestone payments are coming from Gilead Inc. (GILD) and Sanofi (SNY), which just paid for dosing of a first patient in the sickle cell disease trial, Sangamo appears to be funded for the next five years.
Sangamo's partners, Gilead, Pfizer Inc (PFE), Takeda Pharmaceutical Company Limited (TAK), Sanofi, and now Biogen, paid for collaboration with Sangamo because of the compelling science, offering the best form of validation for retail investors. Still, what is apparent, besides now broken thesis that the company is running out of cash, Sangamo continues to be the most undervalued member of the edit and gene therapy' group of companies (table below).
Adjusted for new shares, Sangamo is just below the market capitalization of Editas (EDIT), a CRISPR gene edit company, which has not dosed a single patient yet, and already discussed in my previous article uniQure (QURE).
The Biogen deal illustrates a deep discount
Biogen purchased a genome regulation platform to produce a cure for a patient population of six million. A page from the gene therapy book on pricing produces, at $1M per treatment, a $6 trillion market opportunity, of course, when the treatment works. Even the middle of the road royalty at 9% would offer Sangamo a mind-boggling $540B in payments. If the application is better than the current standard of care but has limitations, and the condition has reduced the market opportunity by 90%, Sangamo would still receive around $50.4B in royalties.
Sangamo's war chest of possible milestone payments, worth about $6B, does not have a much better understanding. On February 28th, Sangamo's market capitalization neared $1.2B, after I included Biogen shares in the calculation. In this scenario, after the deduction of $605M in cash or equivalents expected by the end of Q2 2020, the $6B associated with partnered programs would be worth only $590M, and those without the partnership, like Fabry or MPS II, would have zero value.
Other updates
The company held a Q4 call on February 28th. We have learned that the Fabry program will deliver results by the end of 2020 or early 2021. While the company was tight-lipped on details, the entire process appears much more disciplined in the design and preparation of the study. The company has not enrolled patients yet, because the process rejected more patients than anticipated. Bettina M. Cockroft, Senior V.P. & Chief Medical Officer Sangamo, offered following on the Fabry trial:
"So we've initiated six sites in the U.S. and actually participated in the initiation of the seventh site in London on Monday this week. So we're currently screening patients. And it's my priority, above all, to ensure the quality of the study by ascertaining that the right patients are included. And so we have screened several patients, some of whom had borderline exclusion criteria and who are the screen failed. Indeed, we've had more screen failures than we were expecting at the outset of the trial, but other patients continue to be in the screening phase. And just as a reminder, screening can take up to 2 months to complete. So we've also introduced protocol amendments that will optimize the inclusion criteria and will allow us to optimize those as well as address the FDA's recent guideline on - guidance on Fabry disease."
Separately, Pfizer has replaced Sangamo as a sponsor of the phase 2 trial of SB-525 on clincialtrials.gov, and it is recruiting two more patients, mirroring the seven patients' count of BioMarin's phase 2 trial. This specific move helps my belief that Pfizer will take the same approach to BLA as BioMarin. I expect the progression to reach the accelerated review four months sooner than BioMarin, based on the rapid uptake of SB-525. My anticipated date for Pfizer to file BLA is February 2021.
Fellow investor and S.A. author, Marty Chilberg, analyzed the most recent FDA guideline on the sameness of gene therapy. A different vector in the same viral class would be reviewed on a case-by-case basis to determine the condition of unique treatment. Sangamo's adeno-associated virus combination of AAV2/AAV6 is likely differentiated enough from AAV5 used by BioMarin to support such a status. Still, the mechanism of uptake and hopefully durability will be equally speaking for it.
Adrian Woolfson, Head of Research and Development for Sangamo, confirmed Pfizer would be updating on progress in hemophilia A:
"We were in regular contact with Pfizer about the hemophilia A study. They remain incredibly enthusiastic, as do we. Pfizer will provide the next update, and it's probably best if we leave it for them to announce when that happens."
The CEO, Sandy Macrae also commented:
"I know they are eager to share the data when it reaches the important time points because they want to make sure that we are all aware of the advantages of this asset."
I prefer Sangamo focused on delivering work in the clinic instead of a narrative of the possibilities and the giveaway of time for updates. The element of surprise, like the announcement of the Biogen deal, is much healthier, and I favor this approach over the road map of conferences and updates handed to short sellers.
Conclusion
I recommend Sangamo as a buy with $9.21 as an upper limit for accumulation, even though I believe this number is quite conservative. I continue to expect $17 to $18 by the end of the year, based on already described milestones and also including added shares. I still see Fabry adding $1B to the market capitalization. If all pieces fall into the right place, a 2021 target of $25 per share or the market capitalization of $3.5B is very realistic. Also, I feel Sangamo could make another surprise announcement for the pre-clinical program like Colitis or Multiple Sclerosis within the next 12 months; however, I do not expect equity offering as part of it.
$SGMO
Gilead, Sangamo strike $3B off-the-shelf CAR-T deal
Kite is paying Sangamo $150 million upfront and committing to $3 billion in milestones.
Gilead’s Kite has struck a $3 billion deal to access Sangamo Therapeutics’ zinc finger nuclease (ZFN) technology. Kite will use the ZFN gene editing platform to create next-generation, off-the-shelf CAR-T therapies to cement its position as a long-term front-runner in the growing cell therapy space.
Sangamo is giving Kite the exclusive right to use its ZFN platform to create allogeneic and autologous anticancer cell therapies. For Kite, that expands the toolkit it can apply to its R&D projects while also serving to keep the technology out of the hands of Novartis, Celgene’s Juno and the other CAR-T runners and riders.
In return, Kite is paying Sangamo $150 million upfront and committing to $3 billion in milestones. The milestones are tied to the success of 10 or more products that could potentially use Sangamo’s technology.
Full details of how those pipeline prospects will differ from the first wave of CAR-T therapies are yet to emerge. But Gilead’s release put a discussion of the potential to use the gene-editing technology in off-the-shelf cell therapies up top. As Gilead noted, off-the-shelf cell therapies eliminate the need to process a patient’s own cells, thereby cutting the time it takes for them to receive an infusion.
Cellectis, with the support of Pfizer and Sevier, is betting that benefit, plus the simplified logistics and linked effect on cost of production, will enable its off-the-shelf CAR-T cell therapies to come from behind and capture the market. The French biotech built its R&D strategy around the TALEN editing platform, while Novartis and Juno are using CRISPR/Cas9 to power their next-generation pipelines.
RELATED: Kite's CAR-T medication, cancer candidates draw choosy Gilead
ZFN has been around for longer than CRISPR/Cas9 and was put in the shade by the breathless hype around the latter technology. But Sangamo has continued plugging away at the platform, bringing it to the point that it has a ZFN drug in the clinic and a landmark deal with Gilead. The deal gives Sangamo near and potentially long-term cash boosts, while also validating its belief in ZFN.
“We believe Sangamo’s zinc finger nucleases provide the optimal gene editing platform, and we look forward to working with Sangamo to accelerate our efforts to develop next-generation autologous cell therapies, as well as allogeneic treatments that can be accessed more conveniently in the hospital setting for people living with cancer,” Gilead CEO John Milligan, Ph.D., said in a statement.
$SGMO
We will see a bidding war for this company Pfizer and Biogen Also partnered with KITE, Sanofi & Takedo
https://investor.sangamo.com/news-releases/news-release-details/sangamo-announces-early-completion-transfer-pfizer-sb-525
Biogen puts down massive $350M upfront for Sangamo preclinical assets
https://www.fiercebiotech.com/biotech/biogen-puts-down-a-massive-350m-upfront-for-sangamo-preclinical-assets
https://www.sangamo.com/collaborations
$SGMO
More clinical updates and Analysts upgrades coming Biogen now owns% of SGMO $350 Million up front with possible 2-3 Billion royalties. This is just on a few of sgmo trials there is a wide array of trials in gene editing field. Cash runway into 2023 Biogen will buy them before that.
https://www.sangamo.com/pipeline
$SGMO
SGMO - BIIB Global Neuro Deal Validates ZNF Gene Editing - BUY - In one of the largest preclinical deals in biotech history, Biogen will pay Sangamo $350 million upfront and up to very achievable $2.37 billion in potential milestones. The deal vindicates and validates SGMO’s ZNF platform in gene editing, which most investors have written off. With current cash ~$700 million, SGMO is cheaper than ever to us on multiple valuation methodologies, either as a standalone and even so when compared to other gene editing stocks.
$SGMO
SGMO - BIIB Global Neuro Deal Validates ZNF Gene Editing - BUY - In one of the largest preclinical deals in biotech history, Biogen will pay Sangamo $350 million upfront and up to very achievable $2.37 billion in potential milestones. The deal vindicates and validates SGMO’s ZNF platform in gene editing, which most investors have written off. With current cash ~$700 million, SGMO is cheaper than ever to us on multiple valuation methodologies, either as a standalone and even so when compared to other gene editing stocks.
$SGMO
https://investor.sangamo.com/news-releases/news-release-details/biogen-and-sangamo-announce-global-collaboration-develop-gene
Biogen and Sangamo Announce Global Collaboration to Develop Gene Regulation Therapies for Alzheimer’s, Parkinson’s, Neuromuscular, and Other Neurological Diseases
February 27, 2020 at 4:06 PM EST
Download PDF
Broad collaboration for gene regulation therapies in neurology, initially focused on development of ST-501 for tauopathies including Alzheimer’s disease, ST-502 for synucleinopathies including Parkinson’s disease, and a neuromuscular target, with exclusive rights for nine additional undisclosed neurological targets
Biogen will pay Sangamo $350 million upfront, including a license fee and an equity investment in Sangamo stock
Sangamo is eligible to receive up to $2.37 billion in potential milestones, as well as royalties on potential net commercial sales
Biogen’s access to Sangamo's gene regulation therapies complements its expanding efforts in gene therapy across diverse neurological diseases
CAMBRIDGE, Mass. & BRISBANE, Calif.--(BUSINESS WIRE)-- Biogen Inc. (Nasdaq: BIIB) and Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, today announced that they have executed a global licensing collaboration agreement to develop and commercialize ST-501 for tauopathies including Alzheimer’s disease, ST-502 for synucleinopathies including Parkinson’s disease, a third undisclosed neuromuscular disease target, and up to nine additional undisclosed neurological disease targets. The companies will leverage Sangamo’s proprietary zinc finger protein (ZFP) technology delivered via adeno-associated virus (AAV) to modulate the expression of key genes involved in neurological diseases.
This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20200227005961/en/
“As a pioneer in neuroscience, Biogen will collaborate with Sangamo on a new gene regulation therapy approach, working at the DNA level, with the potential to treat challenging neurological diseases of global significance. We aim to develop and advance these programs forward to investigational new drug applications,” said Alfred Sandrock Jr., M.D., Ph.D., Executive Vice President, Research and Development at Biogen.
“There are currently no approved disease modifying treatments for patients with many devastating neurodegenerative diseases such as Alzheimer’s and Parkinson’s, creating an urgency for the development of medicines that will not just address symptoms like the current standards of care, but slow or stop the progression of disease,” said Sandy Macrae, CEO of Sangamo. “We believe that the promise of genomic medicine in neuroscience is to provide a one-time treatment for patients to alter their disease natural history by addressing the underlying cause at the genomic level.”
Sangamo’s genome regulation technology, zinc finger protein transcription factors (ZFP-TFs), is currently delivered with AAVs and functions at the DNA level to selectively repress or activate the expression of specific genes to achieve a desired therapeutic effect. Highly specific, potent, and tunable repression of tau and alpha synuclein has been demonstrated in preclinical studies using AAV vectors to deliver tau-targeted (ST-501) and alpha synuclein-targeted (ST-502) ZFP-TFs.
“The combination of Sangamo’s proprietary zinc finger technology, Biogen’s unmatched neuroscience research, drug development, and commercialization experience and capabilities, and our shared commitment to bring innovative medicines to patients with neurological diseases establishes the foundation for a robust and compelling collaboration,” said Stephane Boissel, Head of Corporate Strategy at Sangamo. “This collaboration exemplifies Sangamo’s commitment to our ongoing strategy to partner programs that address substantial and diverse patient populations in disease areas requiring complex clinical trial designs and commercial pathways, therefore bringing treatments to patients faster and more efficiently, while deriving maximum value from our platform.”
Under the terms of the collaboration, Biogen has exclusive global rights to ST-501 for tauopathies including Alzheimer’s disease, ST-502 for synucleinopathies including Parkinson’s disease, and a third undisclosed neuromuscular disease target. In addition, Biogen has exclusive rights to nominate up to nine additional undisclosed targets over a target selection period of five years. Sangamo will perform early research activities, costs for which will be shared by the companies, aimed at the development of the combination of proprietary CNS delivery vectors and ZFP-TFs targeting therapeutically relevant genes. Biogen will then assume responsibility and costs for the investigational new drug-enabling studies, clinical development, related regulatory interactions, and global commercialization.
Sangamo will be responsible for GMP manufacturing activities for the initial clinical trials for the first three products of the collaboration and plans to leverage its in-house manufacturing capacity. Biogen will assume responsibility for GMP manufacturing activities beyond the first clinical trial for each of the first three products.
Upon closing of this transaction, Sangamo will receive $350 million comprised of $125 million in a license fee payment and $225 million from the sale of new Sangamo stock, or approximately 24 million shares at $9.21 per share. In addition, Sangamo may receive up to $2.37 billion in other development, regulatory, and commercial milestone payments, including up to $925 million in pre-approval milestone payments and up to $1,445 million in first commercial sale and other sales-based milestone payments. Sangamo will also be eligible to receive from Biogen tiered high single-digit to sub-teen double-digit royalties on potential net commercial sales of products arising from the collaboration. Closing of the transaction is contingent on completion of review under antitrust laws, including the Hart-Scott-Rodino (HSR) Antitrust Improvements Act of 1976 in the U.S.
About Biogen
At Biogen, our mission is clear: we are pioneers in neuroscience. Biogen discovers, develops, and delivers worldwide innovative therapies for people living with serious neurological and neurodegenerative diseases as well as related therapeutic adjacencies. One of the world’s first global biotechnology companies, Biogen was founded in 1978 by Charles Weissmann, Heinz Schaller, Kenneth Murray, and Nobel Prize winners Walter Gilbert and Phillip Sharp. Today Biogen has the leading portfolio of medicines to treat multiple sclerosis, has introduced the first approved treatment for spinal muscular atrophy, commercializes biosimilars of advanced biologics, and is focused on advancing research programs in multiple sclerosis and neuroimmunology, Alzheimer’s disease and dementia, neuromuscular disorders, movement disorders, ophthalmology, immunology, neurocognitive disorders, acute neurology, and pain.
Biogen routinely posts information that may be important to investors on its website at www.biogen.com. To learn more, please visit www.biogen.com and follow Biogen on social media – Twitter, LinkedIn, Facebook, YouTube.
About Sangamo Therapeutics
Sangamo Therapeutics is committed to translating ground-breaking science into genomic medicines with the potential to transform patients’ lives using gene therapy, ex vivo gene-edited cell therapy, and in vivo genome editing and gene regulation. For more information about Sangamo, visit www.sangamo.com.
$SGMO
SGMO has major potential just signed huge deal with Biogen giving them cash into 2023 Deep pipeline top Gene Editing company in the world will be worth more than Crisper soon
One needs to think of the patient population. The R&D presentation had 5M Alzheimer's patients and Parkinson's was 1M. A genome regulation would be likely priced as gene therapy, at $1M. The market potential is about $6 Trillion. At 9% royalty, think $540B to Sangamo. agreement means to Sangamo. BTW Biogen shares are in an escrow, cannot be sold for one year, and then they may be sold in 50% drops until 5% is reached.
Biogen is also forbidden to buy shares on the open market, just in case they want to take over, or worse sell short own holding.
Pfizer is adding two patients to phase 2 to copy BioMarin 7 patients and have a mirror image. Judging by the moves of BioMarin, Pfizer could be filing BLA by February 2021. Between now and then, data drops to keep BioMarin at bay would be likely. At $10, including Biogen investment, the stock is worth $1.4B. Biogen did not select CRSP for it, as the method cannot make precise regulation. Vertex sold half shares of CRSP, but WS barrel fish bought up CRSP to over $4.2B. That's $33 for Sangamo share. None of the CRSP deals are like the ones for Sangamo, and Vertex seems to be moving out, not because it needs money.
Keep your shares, people,
17% shares in Biogen's hands. That is the point. Shorts are not getting those shares. Equity investment is much more attractive than selling to the markets, and shorts to cover. Sangamo is getting $350M, and secures cash runway for another two years, in worse case 2023.
https://www.sangamo.com/pipeline
$SGMO
* * $SGMO Video Chart 02-28-2020 * *
Link to Video - click here to watch the technical chart video
More like earth shattering. In a better environment we'd double on this news. Often these generous statements of confidence are a precursor to a buyout.
https://finance.yahoo.com/news/biogen-sangamo-announce-global-collaboration-210610139.html
Excellent! Partnership with Biogen!
IMO they'd (PFE) be getting a steal at these prices
The CEO (of whom I am not fond of) stated years ago they were not for sale.
Who knows with bio-pharmas. Stuff you think will never be considered gets sold for huge valuations, and those that you think would/should be candidates just languish.
SGMO
Some coments in stocktwits about PFIZER BUYOUT
PFIZER could buy SGMO?
Any opinion?
Unfortunately
The CEO and the IR/PR dude need to be booted .... this has happened too often ....
well, that was a big fizzle.
Awesome background on ASH and SGMO here
https://twitter.com/_B_I_O_T_E_C_H_
"Top Notch Program, Platform and Leadership
The SB 525 dataset provides evidence that Sangamo has a best in class:
Therapy
Platform
Leadership"
https://www.investorvillage.com/smbd.asp?mb=1933&mn=138068&pt=msg&mid=20064375
SGMO and PFE HUGE News on Drug Trial
https://www.businesswire.com/news/home/20191207005019/en/
Good stuff, thanks
Are you reading the other SGMO site I sent you in private message? Check your mailbox.
Market really doesn't like Sangamo's PR history
Overpromise underdeliver is not shareholder friendly. Not holding $11.50 looks ominous, price of last dilution
Biomarin needs to go commercial as soon as possible
valoctocogene dose 6e13 vg/kg dose
(ALT) elevation (11 participants, 73%); arthralgia, (10 participants, 67%); aspartate aminotransferase elevation (8 participants, 53%); headache (7 participants, 47%); back pain, fatigue, and upper respiratory tract infection (6 participants, 40%), insomnia (5 participants, 33%), and pain in extremity (4 participants, 27%).
SB-525 - showing a more potent response at 3e13 vg/kg cohort
elevated alanine aminotransferase (30%) and aspartate aminotransferase (10%), pyrexia (30%), fatigue (10%), hypotension (10%), myalgia (10%), and tachycardia (10%)
Expecting doctors to lean their patients towards the (Pfizer) trial.
Sangamo has begun the tech transfer for the manufacturing processes to Pfizer.
Pfizer getting serious as this step is finally in progress. A big endorsement for the Sangamo platform.
“Jefferies: SGMO providing reasons for pts+docs to hesitate to use Valrox.
New ISTH HemeA Data Stand Out W/ Consistent
Efficacy, Durability, and Safety
July 7, 2019
Key Takeaway
SGMO reported new '525 hemeA data at ISTH that were in-line w/ our expectations here
-- data showed durable F8 in follow-up for two high-dose (3E13) pts and consistent F8
efficacy in two new 3E13 pts w/ favorable safety. Data add more support to case for
'525 being differentiated -- tracking superior to Valrox and SPK-8011; more data at ASH.
SGMO noted in their PR they are working w/ PFE to move '525 into a registrational trial. RMAT
announced too.
Recall, normal F8 levels were achieved rapidly for the two initial pts dosed at 3E13 (last
cut reported on 4/2); the ISTH cut shows that F8 levels were maintained in the longerterm
follow up. For these two pts both reached a normal F8 range (50-170%) w/in 6 wks
w/ F8 levels tracking better than both BMRN's Valrox and Roche/ONCE's SPK-8011 w/
in this timeframe; longer-term data showed normal F8 was maintained out to 24 and 19
wks for the two pts (#7 and #8, respectively). Notably, the follow-up data also showed at
the 1E13 dose pt #6 had F8 durable to wk 52 and #5 some durability to wk 32.
First look at two new expansion pts who received high-dose 3E13 (pts #9 and 10) showed
consistent F8 kinetics w/ two initial 3E13 pts. W/ new pts #9 and 10 we saw F8 ramp
up rapidly post tx; notably, pt #9 had the lowest baseline F8 (of the 4 high-dose pts), and
showed rapid F8 increase after wk 1, which continued to ramp up at wks 2-5. The slide
deck noted after the ISTH cut pt #9 reached normal F8 at wk 7. Pt #10 has only been on tx
for 3 wks, and this pt is showing a similar trajectory. Together, the data from the two new
pts support consistency, and total follow-up support durability, giving us greater confidence.
Also, the total '525 safety profile so far looks good, even w/ no prophylactic steroid used.
No new notable safety events were observed; in the two new pts (according to the PR)
there was one add'l transient Gr1 ALT (both ALTs managed w/ oral tapering) and a new
Gr2 pyrexia. We view the overall safety profile so far as a favorable.
What is SGMO doing differently to stack up better vs the rest? Safety and efficacy may
go hand-in-hand -- better SGMO profile could be driven in part by the low total viral load
used (vs Valrox), a benefit that resonates w/ experts... and has manufacturing advantages
too, which may reflect a better transgene/expression system w/ '525 that enables '525
to come out on top of SPK-8011. SGMO has highlighted their modified F8 transgene
including optimized liver-specific promoter and cassette, which led to better preclin NHP
data vs BMRN and ONCE -- seems to be panning out in the clinic. Could also be related
to the different AAV serotypes used, which may improve '525 tropism/transduction.
SGMO's high-dose data are early and small #s, but looking relatively good so far. BMRN
ahead, but SGMO providing reasons for pts+docs to hesitate to use Valrox.
RMAT, add'l data at ASH, and talk about a registrational path. SGMO is expected to
present add'l data (follow-up+new pts) at ASH -- PR notes pt 11 is expected to be treated
soon (may treat two more at 3E13 for 7 total). The PR also announced RMAT, which we
view as an important positive that should help w/ advancement to a registrational.”
“WF - SGMO: Hem A Update, Emerging Best-In-Class (Outperform PT 34)
We are reiterating our OUTPERFORM rating on shares of Sangamo
Therapeutics (SGMO) following data update for its hemophilia A gene
therapy, SB-525, with partner Pfizer (PFE). Overall data suggest
stable Factor VIII activity in the normal range between weeks 19-24
for the highest dose cohort and well above the 68% level mean level
from competitor Biomarin (BMRN) at 24 weeks. With Biomarin data
suggesting waning efficacy from 64% to 36% between 12 months
and 24 months, additional follow up will be important but at this
stage data appear best-in-class, in our view, and supportive of
significant upside potential given Pfizer investment in late stage
development and manufacturing.
? Sangamo Therapeutics (SGMO) and partner Pfizer (PFE) reported
over the weekend updated data from the phase 1/2 Alta study of
their gene therapy product candidate SB-525 in hemophilia A at the
International Society on Thrombosis and Haemostasis (ISTH)
meeting, and also announced that FDA has granted SB-525 the
RMAT (regenerative medicine advanced therapy) designation.
? The Alta study treated 10 patients at four dose levels (9E11, 2E12,
1E13, and 3E13 vg/kg). The highest dose cohort has treated 4
patients, including 2 patients (P7 and P8) for whom initial data were
reported in April 2019, and two new patients (P9 and P10). The
factor VIII (FVIII) levels in P7 and P8 remained in normal range
(measured by a chromogenic assay) at 24 and 19 weeks of follow-
up, respectively. The two new patients, P9 and P10, had 6 and 4
weeks of follow-up, respectively, and they demonstrated rapid FVIII
increase kinetics that appear consistent with P7 and P8 at similar
time points (though with slightly lower starting levels). SGMO noted
that, after the data cut-off for the ISTH presentation, P9 reached
normal FVIII levels at week 7. Overall, it was noted that patients in
the highest dose cohort reached normal FVIII levels (50-150%)
within 5-7 weeks of treatment, with sustained FVIII levels and no
bleeding episodes and no factor replacement use. SGMO also
reported that the two patients treated in the second highest dose
cohort (1E13 vg/kg) have demonstrated durability of FVIII levels
through weeks 52 and 32, respectively.
? SB-525 was described as generally well tolerated. Patients in the
Alta study were not treated with prophylactic steroids. There was
one case of treatment-related SAE as previously reported
(hypotension and fever 6 hours after SB-525 infusion), and no
similar hypotension event was observed in the three subsequent
patients dosed. ALT and AST elevation occurred in 30% and 10% of
patients, respectively, though no patients experienced ALT elevation
associated with loss of FVIII expression. In the highest dose cohort,
2 patients experienced Grade 1 ALT elevation (above 1.5x baseline)
managed with a tapering course of oral steroids.
? SGMO expects to treat the 5th patient (P11) in the 3E13 vg/kg dose cohort soon, and SGMO and PFE are working on plans to advance SB-525 to a registrational study. PFE is to assume responsibility for SB-525 late-stage development and manufacturing, and transfer of the SB-525 manufacturing process from SGMO to PFE has been initiated.
? SGMO announced that, based on data from the Alta study, the FDA has granted RMAT designation for SB-525 to treat severe hemophilia A. RMAT designation, granted to regenerative medicine therapies with preliminary clinical evidence indicating the potential to address an unmet medical need with a serious condition, includes all the benefits of fast track and breakthrough therapy designation programs, including early interactions with FDA.”
“BREAKING NEWS - Sangamo (SGMO) - SB-525 Looks Highly Competitive At ISTH; FDA Grants RMAT & Pfizer Planning Registration Trials - With the updated ALTA study presented at ISTH, Sangamo has firmly made its case for SB-525 as a leading hemophilia A gene therapy treatment. The FDA appears to agree - it has granted the Company RMAT status - the first one given for a hem A gene therapy. Partner Pfizer (included in the press release headline) will be taking over the program as it heads into Phase III clinical trials. REITERATE BUY........
Efficacy - Fast Acting, Consistent & Durable Responses To Date - The two original 3E13 patients have been treated for 19 and 24 weeks, respectively (as of 5/30/19). Two more patients have since been treated at the higher dose (as per the extension cohort) and are showing the same rapid response in Factor VIII activity to SB-525 as the first two - hence 4 subjects have now been treated at the “3e13” dose. Since the cutoff date, one of those two (subject 9) has also since attained normal Factor VIII levels by week 7. As the chart above shows, Factor VIII levels of the first two have stayed well in the “normal” range with no diminution, while the other two appear on their way, too. As a comparison, it took ~16 weeks for BMRN’s ValRox subjects to reach normal FVIII levels versus 7 weeks for SB-525. And the ValRox normal levels reached were below that of SB-525, too.
No Spontaneous Bleeds - In two additional tables below, at the high dose there has been no bleeding episodes in any of the 4 patients after 3 weeks post-treatment (the fourth had yet to reach the >3 weeks period at the cutoff).
No Need For FVIII Infusions - Prophylactic Factor VIII usage in the 3e13 dose, also went to zero after the >3 week treatment period. The prophylactic FVIII regimen of the four subject prior to SB-525 treatment was an infusion either every other day, every 3 days or every 4 days.........
Safety - Pretty Clean - SB-525 was generally well tolerated. Unlike other gene therapies for hem A, patients in the Alta study were not treated with prophylactic steroids. One treatment-related serious adverse event (SAE) was reported. This patient experienced hypotension and fever six hours after completion of SB-525 infusion and this fully resolved with treatment and the patient was discharged as planned within 24 hours. No similar hypotension event was observed in the three subsequent patients dosed. Adverse events observed in 10% (n=1) or more patients included: increased alanine aminotransferase (30%) and aspartate aminotransferase (10%), pyrexia (30%), fatigue (10%), hypotension (10%), myalgia (10%), and tachycardia (10%). No patients treated with SB-525 have experienced an alanine aminotransferase (ALT) elevation associated with a loss of Factor VIII expression. In the 3e13 vg/kg cohort, two subjects experienced a transient grade 1 ALT elevation (>1.5 x baseline) managed with a tapering course of oral steroids. This safety profile suggests that SB-525 could be the cleanest of the three (ValRox, SPK-800, SB-525).....
FDA Grants SB-525 RMAT Status - Based on the accumulating results from the Alta study, the FDA has granted regenerative medicine advanced therapy (RMAT) designation for SB-525 gene therapy to treat severe hemophilia A. RMAT designation is granted to regenerative medicine therapies intended to treat, modify, reverse, or cure a serious condition, for which preliminary clinical evidence indicates that the medicine has the potential to address an unmet medical need. The RMAT designation includes all the benefits of the fast track and breakthrough therapy designation programs, including early interactions with FDA. In our view, the RMAT designation will accelerate the clinical and approval timelines for SB-525.
PFE Is About To Take Over - Seng Cheng, Senior Vice President and Chief Scientific Officer of Pfizer’s Rare Diseases Research Unit said, “We are encouraged by the initial clinical data suggesting safety, tolerability, and efficacy of SB-525 and are beginning preparations, including manufacturing, to potentially advance into a registrational study. We are also encouraged by our interactions with regulators and by the FDA’s recent RMAT designation. If FVIII levels are sustained, and patients continue to have no bleeding episodes and remain off factor replacement therapy, we believe that this gene therapy may potentially represent a transformative treatment paradigm for severe hemophilia A.” The PR also mentions that Pfizer will soon assume responsibility for SB-525 late-stage development and manufacturing.Transfer of the SB-525 manufacturing process from Sangamo to Pfizer has been initiated.
SGMO - Now A Bonafide HEM A GT Player - Before ISTH, SGMO was off of investors’ radar screens for hem A gene therapy. While the number of patients is small and time on SB-525 is still relatively short compared with BMRN and ONCE (acquired by Roche recently for $5 billion), the new updated data, along with the RMAT designation and PFE’s increased participation in the program has no doubt placed the Company among the leaders in the race for blockbuster hemophilia A gene therapy treatments.
SMGO is a BUY under 20 with a TARGET PRICE of 30”
* * $SGMO Video Chart 07-08-2019 * *
Link to Video - click here to watch the technical chart video
$11.50, raised $145 million
And an impressive update on the Factor VIII trial as Pfizer is now taking on the manufacturing and trial. New here and got in at $11.40
Biomarin has inferior results and a $14 billion market cap that is sliding as Sangamo results are much more impressive with a much lower vector dose
RICHMOND, Calif., April 3, 2019 /PRNewswire/ -- Sangamo Therapeutics, Inc. (Nasdaq: SGMO) today announced that it has commenced an underwritten public offering of shares of its common stock. All of the shares are being offered by Sangamo. In addition, Sangamo expects to grant the underwriters of the offering a 30-day option to purchase additional shares of its common stock at the public offering price, less the underwriting discounts and commissions. The offering is subject to market and other conditions, and there can be no assurance as to whether or when the offering will be completed, or as to the actual size or terms of the offering.
Sangamo anticipates using the net proceeds from the offering for working capital and other general corporate purposes, including support for its own and its partnered gene therapy, genome editing, cell therapy and gene regulation product candidates and research programs, its manufacturing facilities and other business development activities.
https://investor.sangamo.com/news-releases/news-release-details/sangamo-therapeutics-announces-proposed-public-offering-common-1
* * $SGMO Video Chart 04-02-2019 * *
Link to Video - click here to watch the technical chart video
Sangamo price target raised to $34 from $24 at Wells Fargo. Wells Fargo analyst Jim Birchenough raised his price target for Sangamo Therapeutics to $34 from $24 following this morning's clinical update on the company's gene therapy, gene editing and cell therapy pipeline. The stock in morning trading is up 35%, or $3.35, to $12.88. The update was better than expected, particularly for hemophilia gene therapy and beta thalassemia ex vivo gene editing, Birchenough tells investors in a research note. The analyst is "encouraged" by Sangamo's progress against the higher technical hurdle of in vivo genome editing. He believes direct evidence of genome editing, "strong" fetal hemoglobin response and early signs of transfusion independence bode well for ultimate success and should translate into improved likelihood of success in the larger opportunity of sickle cell disease. The analyst reiterates an Outperform rating on Sangamo Therapeutics.
Read more at:
https://thefly.com/landingPageNews.php?id=2887507
ciciagt...
Thanks for posting...
":>)JL
“After getting slammed two months ago after rolling out the first in vivohuman data available from a rare disease study using its gene editing tech, the players at Sangamo have earned some hard won respect on Wall Street today after posting a snapshot of promising, though very early stage, results for their hemophilia A gene therapy, partnered with Pfizer.
Focusing on the high-dose cohort — with only 2 patients getting a 3e13 vg/kg dose of SB-525 — researchers spotlighted FVIII activity of 140% and 94%, or 93% and 65% of normal, depending on which assay they used. That’s very competitive in a field dominated up to now by BioMarin $BMRN and Spark, now being acquired by Roche, so long as it holds up.
One ex vivo gene editing program also gathered some initial responses in beta thalassemia and Sangamo outlined plans to beef up its AAV manufacturing operations with a new contract signed with Brammer Bio while its own facility is under construction.
Sangamo’s shares $SGMO slowly swelled in pre-market trading as the positive message sunk in. Then it soared. Its stock was up more than 50% before the bell......”
https://www.investorvillage.com/smbd.asp?mb=1933&mn=124063&pt=msg&mid=19300692
Nice update. I have been intrigued by what this Company has to offer for years. Cheap valuation down here IMO.
Sangamo (SGMO)
Gene Editing company with one of the largest pipelines in the sector.
400 Million in cash
Partnerships: Gilead(Kite)-Pfizer-Shire and Sanofi
JPM Update – WORLD, 5 Trials Underway, 4 More To Start, Etc.
Sangamo presented an overview of its clinical and technological gene therapy/editing leadership. With 5 trials underway and data from all five clinical results due this year, to us 2019 will be the year when SGMO gaps ahead of the gene editing crowd. The upcoming WORLD meeting (2/7) will begin to show that. REITERATE BUY.
WORLD To Include IDS Levels – The upcoming WORLDSymposium on February 7 in Orlando (https://www.worldsymposia.org) will demonstrate the most up-to-date CHAMPIONS Phase I/II trial of SB-913 in MPSII. The data will include 24-week safety and changes in GAG /IDS levels on 6 patients of data. The company will also be using a much higher-sensitivity IDS assay. This is rather important, as SMGO shares dropped sharply after the first look at CHAMPIONS data last September showed a dose-dependent drop in GAG but not a material and corresponding rise in IDS levels. IDS levels are the study’s primary endpoint and what the Company believes is being produced – and that will lead to patient’s withdrawing from Enzyme Replacement Therapy (ERTs).
SGMO shares fell after the breakout session when someone asked what is the main way to know if SB-913 is working? McCrae said that is when a patient comes off ERTs. Although that is not the primary endpoint and knowing that it is just too early for patient’s to have begun to withdraw from ERTs (or maybe they have started), that data point will not be at WORLD – even though it was never supposed to be there in the first place. This was a major over-reaction in our view. Sometime in 2019, liver biopsy and ERT withdrawal data will be released/updated which should prove the efficacy. That is and has always been the plan.
The key event for us is the IDS levels – and learning that the new assay will be presented at WORLD is a major positive, to prove that SB-913 is indeed producing therapeutic IDS levels (that will eventually lead to ERT withdrawal). Producing and measuring therapeutic IDS are the next logical step in the clinical program of SB-913.
MPS I Update Also At WORLD – The first look at the second SGMO in-vivo gene editing trial will include 4-week safety on 3 patients.
Hemophilia A Gene Therapy – SB-525 with Pfizer – The next SMC meeting is due soon and a further recommendation to begin dosing the second patient in the fourth cohort is likely. SGMO believes this is probably the last dose for SB-525. Updated SB-525 data are anticipated sometime in 2019.
New Zinc Finger 2.0 Publication – An upcoming zinc finger publication is due in early 2019, describing ZNF 2.0 and how the nuclease technology can be optimized to generate higher specificity/lower off target cutting.
INDs/Updates For 2019 – Include Fabry’s disease, Beta-thalassemia (BIVV/SNY), sickle cell disease and the first TReg trial from GILD (renal transplants). Further partnerships may include CNS (tau/Alzheimer’s)/Immunology.
If any one of the above studies is successful, both the SGMO market cap and takeover potential will skyrocket. With the current update, we remain confident that it will.
TARGET PRICE of 40.00
* * $SGMO Video Chart 02-07-19 * *
Link to Video - click here to watch the technical chart video
Bought some on the dip today hoping to average down. Looking good so far
You can't hug you children with ZINC fingers !
Bioverativ & Sangamo Announce FDA Acceptance of IND Application for Gene-Edited Cell Therapy BIVV003 to Treat Sickle Cell Dis...
Source: Business Wire
Bioverativ Inc., a Sanofi company dedicated to transforming the lives of people with rare blood disorders, and Sangamo Therapeutics, Inc. (NASDAQ:SGMO) announced today that the U.S. Food and Drug Administration (FDA) has accepted the Investigational New Drug (IND) application for BIVV003, a gene-edited cell therapy candidate for the treatment of people with sickle cell disease. Bioverativ and Sangamo are developing BIVV003 as part of an exclusive worldwide collaboration to develop and commercialize gene-edited cell therapies for sickle cell disease and beta thalassemia.
This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20180516005404/en/
“This acceptance marks the second IND for this gene-editing approach in less than a year, and the first for a gene-edited therapy in sickle cell disease,” said Ken Huttner, M.D. Ph.D., Vice President, Clinical Development at Bioverativ. “It represents our commitment to advancing cutting-edge science and offers hope to the many people who have been waiting generations for an effective way to treat sickle cell disease. We look forward to advancing the program into clinical trials.”
“Sickle cell disease is a lifelong blood disorder with serious, painful and debilitating complications, and patients deserve new, more effective treatment options,” said Ed Conner, M.D., Chief Medical Officer at Sangamo. “Gene-edited cell therapy has the potential to provide patients living with sickle cell disease a lifelong treatment with a single administration. We believe the precision, efficiency and specificity of zinc finger nuclease technology differentiate BIVV003 from other genomic therapies in development.”
BIVV003 is a non-viral approach utilizing zinc finger nuclease (ZFN) gene-editing technology. By modifying a short sequence of the BCL11A gene in a patient’s red blood cell precursors, sickle hemoglobin production is suppressed, and the production of fetal hemoglobin is reactivated to levels that may protect patients against the progression of their sickle cell disease.
This IND enables Bioverativ to initiate a Phase 1/2 clinical trial to assess the safety, tolerability, and efficacy of BIVV003 in adults with sickle cell disease, and Bioverativ expects to open several clinical sites across the United States this year. Currently, Sangamo is enrolling patients with transfusion-dependent beta thalassemia in a Phase 1/2 trial evaluating the safety, tolerability, and efficacy of ST-400, which uses the same gene-editing approach as BIVV003.
About Sickle Cell Disease
Globally, 300,000 people are born with sickle cell disease every year, and approximately 100,000 people are living with sickle cell disease in the United States.1 People with sickle cell disease have a mutation that alters hemoglobin, the protein in red blood cells that carries oxygen to cells throughout the body. The sickle mutation causes red blood cells to have an abnormal sickle or crescent shape, which makes them inefficient in their oxygen-carrying capacity and leads to chronic anemia, vaso-occlusive crises with severe pain, multi-organ damage, complications like stroke, and a shortened life expectancy.
About BIVV003 and the Phase 1/2 Clinical Trial
BIVV003 is an autologous cell therapy that involves gene editing of a patient’s own hematopoietic stem cells (HSCs) using zinc finger nuclease (ZFN) technology. As part of the clinical trial protocol, a patient’s HSCs are isolated from the blood and then undergo non-viral, ex vivo gene editing using ZFNs to modify the erythroid enhancer of the BCL11A gene, which is a key regulator of the level of fetal hemoglobin. Following a bone marrow conditioning regimen, patients are infused with their own modified HSCs, with the goal of producing red blood cells that have increased production of fetal hemoglobin. Using the patient’s own cells reduces the risk of graft failure, and eliminates the risk of graft-versus-host disease and the need for immunosuppression that is associated with transplant from a donor.
About the Bioverativ and Sangamo Collaboration
Bioverativ and Sangamo have an exclusive worldwide collaboration to develop and commercialize ZFN-mediated gene-edited cell therapies for the treatment of beta thalassemia and sickle cell disease. Based on the terms of the agreement, Bioverativ is responsible for conducting the BIVV003 Phase 1/2 clinical trial and subsequent worldwide clinical development, manufacturing, and commercialization.
About Bioverativ, a Sanofi company
Bioverativ, a Sanofi company, is dedicated to transforming the lives of people with hemophilia and other rare blood disorders through world-class research, development, and commercialization of innovative therapies. Bioverativ is committed to actively working with the blood disorders community, and its hemophilia therapies when launched represented the first major advancements in hemophilia treatment in more than two decades. For more information, visit www.bioverativ.com or follow @bioverativ on Twitter.
About Sangamo Therapeutics
Sangamo Therapeutics, Inc. is focused on translating ground-breaking science into genomic therapies that transform patients’ lives using the Company’s platform technologies in genome editing, gene therapy, gene regulation and cell therapy. The Company has open Phase 1/2 clinical trials in hemophilia A and hemophilia B, lysosomal storage disorders MPS I and MPS II, as well as beta thalassemia. Sangamo has an exclusive, global collaboration and license agreement with Kite, a Gilead company, for engineered cell therapies for oncology, with Pfizer Inc. for gene therapy programs for Hemophilia A, with Bioverativ for hemoglobinopathies, including beta thalassemia and sickle cell disease, and with Shire International GmbH to develop therapeutics for Huntington’s disease. For more information about Sangamo, visit the Company’s website at www.sangamo.com.
Sangamo’s Forward-Looking Statements
This press release contains forward-looking statements regarding Sangamo’s current expectations. These forward-looking statements include, without limitation, references to the potential for Sangamo’s gene-edited cell therapy to provide patients living with sickle cell disease a long-lasting treatment with a single administration and the potential for the precision, efficiency and specificity of zinc finger nuclease technology to differentiate BIVV003 from other genomic therapies in development. These statements are not guarantees of future performance and are subject to certain risks, uncertainties and assumptions that are difficult to predict. Factors that could cause actual results to differ include, but are not limited to, the dependence on the success of the clinical trials of lead programs, the lengthy and uncertain regulatory approval process, uncertainties related to the initiation and completion of clinical trials, whether clinical trial results will validate and support the safety and efficacy of Sangamo's product candidates, and the reliance on partners and other third-parties to meet their obligations. Actual results may differ from those projected in forward-looking statements due to risks and uncertainties that exist in Sangamo’s operations and business environments. These risks and uncertainties are described more fully in Sangamo’s Quarterly Report on Form 10-Q as filed with the Securities and Exchange Commission. Forward-looking statements contained in this announcement are made as of this date, and Sangamo undertakes no duty to update such information except as required under applicable law.
References:
1. World Health Organization; Piel et al. 2013. Lancet 381: 142-51.
View source version on businesswire.com: https://www.businesswire.com/news/home/20180516005404/en/
Media:
Bioverativ Inc.
Tracy Vineis, +1 781-663-4376
media@bioverativ.com
or
Sangamo Therapeutics, Inc.
McDavid Stilwell, 510-970-6000 x219
mstilwell@sangamo.com
or
Varant Shirvanian, 510-970-6000 x205
vshirvanian@sangamo.com
This is from the Motley Fool five days ago:
Sangamo’s stock has been red-hot over the past 12 months due to the growing interest in all-things gene therapy. The backstory is that Sangamo struck a series of licensing deals with biopharma heavyweights Gilead Sciences (NASDAQ: GILD) and Pfizer (NYSE: PFE) to develop next-generation therapies for cancer and rare diseases like the bleeding disorder hemophilia.
The Gilead partnership centers around creating adoptive cell therapies that can be manufactured at scale using Sangamo's zinc-finger nuclease (ZFN) gene-editing platform. The current generation of adoptive cell therapies require extensive manufacturing processes that can delay treatment, and create backlogs of patients.
That being said, Gilead's decision to tap Sangamo's ZFN platform did surprise most Wall Street analysts and industry insiders. The consensus was that Gilead would ultimately go the CRISPR route for its gene-editing needs. After all, Sangamo's ZFN platform is more labor-intensive and time-consuming than CRISPR. With serious questions currently swirling around CRISPR's safety in human subjects, however, Sangamo's ZFN tech was apparently the more compelling choice for Gilead at this stage in the game.
The big ticket item for investors here is that this gene-editing deal with Gilead could eventually translate into a whopping $3 billion in total milestone payments for Sangamo -- that is, if things go according to plan. That's a staggering opportunity for a company with a market cap of less than half that amount at present.
Sangamo's severe hemophilia A partnership with Pfizer is also on track to produce preliminary data by mid-year. If successful, this partnership may trigger buyout talks between the two companies. Pfizer, after all, has deep interests in novel treatments for rare diseases, and the biopharma is currently in the process of ramping up its adoptive cell therapy pipeline as well. Put simply, Sangamo would be a near-perfect fit for Pfizer.
Aside from the possibility of a buyout from one of its partners, Sangamo's deep and robust gene therapy platform also offers up another compelling reason to own this stock right now. Sangamo could run into some bad luck in the clinic, but the sheer breadth of its cutting-edge pipeline should ultimately land the company a commercial-stage product at some point.
So, like its clinical-stage peer Jounce, Sangamo's risk-to-reward ratio is arguably heavily skewed toward the upside as things stand now. The biotech's 10.8% drop last week therefore comes across as an incredible buying opportunity for investors with a long-term mind-set.
From The Motley Fool
Yes, I also have to thank Zincfinger for an early heads up on this one.
depends on phase trials---$50-100 ??
Fill me in guys. What is your 3 month target?
Somebody named Zincfingers told KBLB shareholders about this stock--glad i bot some.
Zinc if u r out there thanks and come visit the KB board sometime.
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http://www.sangamo.com/index.php
http://finance.yahoo.com/q/ks?s=SGMO+Key+Statistics
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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