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.
Athenex is a global oncology focused biopharmaceutical company dedicated to the di...
Athenex is a global oncology focused biopharmac...
Madrigal Pharmaceuticals is a clinical-stage biopharmaceutical company pursuing no...
Madrigal Pharmaceuticals is a clinical-stage bi...
AGTC is a clinical-stage biotechnology company that uses a proprietary gene therap...
AGTC is a clinical-stage biotechnology company ...
Achieve is a specialty pharmaceutical company committed to advancing cytisinicline...
Achieve is a specialty pharmaceutical company c...
PLx Pharma Inc. (Nasdaq: PLXP), is a late-stage specialty pharmaceutical company i...
PLx Pharma Inc. (Nasdaq: PLXP), is a late-stage...
ImmunoGen is developing the next generation of antibody-drug conjugates (ADCs) to ...
ImmunoGen is developing the next generation of ...
Join the National Investor Network and get the latest information with your interests in mind.