NVS' phase III study on experimental gene therapy candidate Intrathecal onasemnogene abeparvovec (OAV101IT) meets its primary goal.

PFE ends deal with SGMO for hemophilia A gene therapy candidate. NVS' gene therapy for SMA meets goal in study.

Hoth stock soars as a mid-stage study meets the goal, showing significant improvement in skin toxicities related to EFGR inhibitor therapy in all...

Hoth stock soars as a mid-stage study meets the goal, showing significant improvement in skin toxicities related to EFGR inhibitor therapy in all...

Study published in iHuman Genome Therapy/i hypothesizes that adeno-associated virus (AAV)-based gene therapy can deliver anti-human eosinophil...

NMRA stock falls after its experimental depression treatment failed to meet the primary and secondary endpoints in the first of three late-stage...

Neumora Therapeutics, Inc., a clinical─stage biopharmaceutical company with a therapeutics pipeline consisting of seven clinical and pre─clinical...

Denali obtains Breakthrough Therapy Designation for its experimental candidate, tividenofusp alfa (DNL310), for the treatment of pateints with Hunter...

DNLI's DNL343 fails to achieve the primary and secondary endpoints, as observed from an analysis of Regimen G of the phase II/III HEALEY ALS platform...

Solid Biosciences rises as the FDA clears SGT-212, a gene therapy candidate, for clinical studies for the treatment of Friedreich's ataxia.