This account is pending registration confirmation. Please click on the link within the confirmation email previously sent you to complete registration.
Need a new registration confirmation email? Click here
Stocks Under $10 with 50-100% upside potential - 14 days FREE!

Sangamo BioSciences Presents Data From Non-Human Primates Providing Proof Of Concept For Disruptive And Broadly Leverageable Platform For Protein Replacement Therapies

RICHMOND, Calif., Dec. 10, 2013 /PRNewswire/ -- Sangamo BioSciences, Inc. (Nasdaq: SGMO) announced the presentation of new pre-clinical data demonstrating therapeutic levels of gene modification in non-human primates (NHPs) from its In Vivo Protein Replacement Platform. Based on Sangamo's zinc finger DNA-binding protein (ZFP) genome-editing technology, the platform enables the permanent production of therapeutic proteins from a specific genomic site in the liver with a single systemic treatment, potentially providing curative treatments for a range of monogenic diseases such as hemophilia and lysosomal storage disorders (LSD) including Gaucher and Fabry disease. Such diseases are currently treated by regular infusions of protein or enzyme replacement therapy (ERT) throughout the patient's life. The data were presented at the 55 th Annual Meeting of the American Society of Hematology (ASH) which is being held in New Orleans.

(Logo: http://photos.prnewswire.com/prnh/20130102/SF35903LOGO)

"These data provide proof of concept for this broadly applicable genome editing strategy by demonstrating that our process is scalable to large animals and by validating the use of the albumin safe harbor as a site for expression of therapeutic proteins," said Philip Gregory. D. Phil., Sangamo's vice president of research and chief scientific officer. "We have further optimized Sangamo's ZFN system and demonstrated that a single systemic treatment enables stable liver-specific production of replacement protein. Early data in primates suggest that we can achieve circulating levels of protein above the threshold for therapeutic effect, which we believe are sufficient for the correction of a range of monogenic diseases. Our data demonstrate expression of replacement enzymes for multiple different proteins, including those deficient in lysosomal storage disorders, which serves to demonstrate the potential of this approach for a broad range of other monogenic diseases."

Sangamo's In Vivo Protein Replacement Platform makes use of a highly expressed and liver specific genomic "safe-harbor site" that can be edited with ZFP nucleases (ZFNs) to accept and express any therapeutic gene and thus permanently produce high levels of the missing protein with a single treatment. The gene encoding albumin, the most abundant protein in blood serum, was chosen as a safe harbor site because it is active exclusively in the liver.  The albumin promoter is highly active, continuously producing large amounts of albumin protein (approximately 15g/day) which is in excess of the body's requirements. With such a large capacity for protein production, targeting and co-opting a very small percentage of the albumin gene's production capacity is sufficient to safely produce the needed replacement protein at therapeutically relevant levels with no significant effect on albumin production. The study was performed as part of Sangamo's collaboration with Shire and in the laboratory of Katherine A. High, M.D., director of the Center for Cellular and Molecular Therapeutics at The Children's Hospital of Philadelphia. Dr. High is a Howard Hughes Medical Institute Investigator, and Professor of Pediatrics at the Perelman School of Medicine at the University of Pennsylvania.

"These data represent an important milestone in the progress of our monogenic disease programs towards the clinic," stated Edward Lanphier, Sangamo's president and chief executive officer. "Our In Vivo Protein Replacement Platform is a highly leverageable and disruptive application of our ZFN-mediated genome editing technology and can potentially be applied to any disease-relevant gene where enabling the liver to provide a stable source of corrective replacement protein will be therapeutic. The NHP data presented at ASH represent a significant de-risking step and provide important proof of concept and validation for this entire strategy."

The data were presented in an oral presentation:

1 of 3

Select the service that is right for you!

COMPARE ALL SERVICES
Action Alerts PLUS
Try it NOW

Jim Cramer and Stephanie Link actively manage a real portfolio and reveal their money management tactics while giving advanced notice before every trade.

Product Features:
  • $2.5+ million portfolio
  • Large-cap and dividend focus
  • Intraday trade alerts from Cramer
  • Weekly roundups
TheStreet Quant Ratings
Try it NOW
Only $49.95/yr

Access the tool that DOMINATES the Russell 2000 and the S&P 500.

Product Features:
  • Buy, hold, or sell recommendations for over 4,300 stocks
  • Unlimited research reports on your favorite stocks
  • A custom stock screener
  • Upgrade/downgrade alerts
Stocks Under $10
Try it NOW

David Peltier, uncovers low dollar stocks with extraordinary upside potential that are flying under Wall Street's radar.

Product Features:
  • Model portfolio
  • Stocks trading below $10
  • Intraday trade alerts
  • Weekly roundups
Dividend Stock Advisor
Try it NOW

Jim Cramer's protege, David Peltier, identifies the best of breed dividend stocks that will pay a reliable AND significant income stream.

Product Features:
  • Diversified model portfolio of dividend stocks
  • Alerts when market news affect the portfolio
  • Bi-weekly updates with exact steps to take - BUY, HOLD, SELL
Real Money Pro
Try it NOW

All of Real Money, plus 15 more of Wall Street's sharpest minds delivering actionable trading ideas, a comprehensive look at the market, and fundamental and technical analysis.

Product Features:
  • Real Money + Doug Kass Plus 15 more Wall Street Pros
  • Intraday commentary & news
  • Ultra-actionable trading ideas
Options Profits
Try it NOW

Our options trading pros provide daily market commentary and over 100 monthly option trading ideas and strategies to help you become a well-seasoned trader.

Product Features:
  • 100+ monthly options trading ideas
  • Actionable options commentary & news
  • Real-time trading community
  • Options TV
To begin commenting right away, you can log in below using your Disqus, Facebook, Twitter, OpenID or Yahoo login credentials. Alternatively, you can post a comment as a "guest" just by entering an email address. Your use of the commenting tool is subject to multiple terms of service/use and privacy policies - see here for more details.
Submit an article to us!
DOW 16,967.37 -15.22 -0.09%
S&P 500 1,981.97 +3.06 0.15%
NASDAQ 4,463.6640 +18.7550 0.42%

Brokerage Partners

Rates from Bankrate.com

  • Mortgage
  • Credit Cards
  • Auto

Free Newsletters from TheStreet

My Subscriptions:

After the Bell

Before the Bell

Booyah! Newsletter

Midday Bell

TheStreet Top 10 Stories

Winners & Losers

Register for Newsletters
Top Rated Stocks Top Rated Funds Top Rated ETFs