Gene therapy can effectively treat various diseases, but for some debilitating conditions like muscular dystrophies there is a big problem: size. The ...

Carlos Quezada-Ruiz, M.D., FASRS, SVP, Therapeutic Area Head, Ophthalmology ...

Ascend Advanced Therapies, which has been on an expansion tear since debuting three years ago, inked a deal with EW ...

MeiraGTx Holdings plc (Nasdaq: MGTX), a vertically integrated, clinical stage genetic medicines company, today announced financial and operational results for the third quarter ended September 30, ...

Ascend’s fill/finish capacity in the US will also be expanded. “We founded Ascend with a vision to quilt together the best operators and technologies within the advanced manufacturing field. The ...

Bank of America Securities analyst Jason Zemansky maintained a Buy rating on Rocket Pharmaceuticals (RCKT – Research Report) today. The ...

This study presents a valuable finding on the molecular mechanisms that govern GABAergic inhibitory synapse function. The authors propose that Endophilin A1 serves as a novel regulator of GABAergic ...

Analysts did not seem very concerned by the treatment-related serious adverse event, noting that NGN-401 was well-tolerated ...

The multicenter Phase1b open-label dose escalation study (NCT05973630) will evaluate safety, pharmacodynamics, efficacy, and immunogenicity in children receiving intravenous ATA-200, a single-dose ...