We are a vertically integrated, clinical stage gene therapy company with four ongoing clinical programs and a broad pipeline of preclinical and research programs. We have core capabilities in viral vector design and optimization and gene therapy manufacturing, as well as a potentially transformative gene regulation technology. Led by an experienced management team, we have taken a portfolio approach by licensing, acquiring and developing technologies that give us depth across both product candidates and indications. Though initially focusing on the eye, salivary gland and central nervous system, we intend to expand our focus in the future to develop additional gene therapy treatments for patients suffering from a range of serious diseases. Source
No articles found.
Allenaâs lead product candidate, reloxaliase, is a first-in-class, oral enzyme t...
Allenaâs lead product candidate, reloxaliase,...
Prothena Corporation plc (Nasdaq: PRTA) is a clinical-stage neuroscience company f...
Prothena Corporation plc (Nasdaq: PRTA) is a cl...
Dermira is a biopharmaceutical company dedicated to bringing biotech ingenuity to ...
Dermira is a biopharmaceutical company dedicate...
Cellect Biotechnology Ltd. was founded in 2011 by Kasbian Nuriel Chirich, Dr. Shai...
Cellect Biotechnology Ltd. was founded in 2011 ...
Aeolus Pharmaceuticals, Inc. is a biopharmaceutical company. The Company focuses o...
Aeolus Pharmaceuticals, Inc. is a biopharmaceut...
DBV Technologies is developing ViaskinÂŽ, an investigational proprietary technolog...
DBV Technologies is developing ViaskinÂŽ, an in...
Spark Therapeutics, a fully integrated company, strives to challenge the inevitabi...
Spark Therapeutics, a fully integrated company,...
Join the National Investor Network and get the latest information with your interests in mind.