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.
At Achillion, we are driven to transform the lives of patients and families affect...
At Achillion, we are driven to transform the li...
Sol-Gel is a clinical-stage specialty pharmaceutical company focused on developing...
Sol-Gel is a clinical-stage specialty pharmaceu...
Patient Access Solutions Inc provides administrative, management, and facility ser...
Patient Access Solutions Inc provides administr...
STRATA Skin Sciences is a medical technology company focused on the therapeutic an...
STRATA Skin Sciences is a medical technology co...
Together, a potent new drug and a blood test to guide its use can be a life change...
Together, a potent new drug and a blood test to...
Anthera Pharmaceuticals, Inc., a development stage biopharmaceutical company, focu...
Anthera Pharmaceuticals, Inc., a development st...
Adverum is a clinical-stage gene therapy company targeting unmet medical need in o...
Adverum is a clinical-stage gene therapy compan...
Join the National Investor Network and get the latest information with your interests in mind.