We are a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined diseases in areas of high unmet medical need, with an initial focus on rare diseases. We have developed a proprietary product engine that we employ to systematically identify and validate cellular drug targets that can potentially modulate gene expression to treat the known root cause of genetically defined diseases. We are using our product engine to identify targets that can be drugged by small molecules regardless of the particular underlying mechanism of gene mis-expression. We have identified drug targets to treat the root causes of facioscapulohumeral muscular dystrophy (FSHD) and certain hemoglobinopathies, namely sickle cell disease (SCD) and b-thalassemia. Source
No articles found.
ACS Global, Inc. engages in the collection, processing, and long-term storage of s...
ACS Global, Inc. engages in the collection, pro...
Intersect ENT (NASDAQ: XENT) is a medical technology company dedicated to advancin...
Intersect ENT (NASDAQ: XENT) is a medical techn...
Nymox Pharmaceutical Corporation specializes in the research and development of th...
Nymox Pharmaceutical Corporation specializes in...
Over a century ago in Melbourne, Australia, we made a promise to save lives and pr...
Over a century ago in Melbourne, Australia, we ...
Dicerna is a biopharmaceutical company focused on the discovery and development of...
Dicerna is a biopharmaceutical company focused ...
We are developing nalbuphine ER to treat serious neurologically mediated condition...
We are developing nalbuphine ER to treat seriou...
Join the National Investor Network and get the latest information with your interests in mind.