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

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

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 ...

Its multi-platform development approach applies the lentiviral vector (LVV) and adeno-associated viral vector (AAV) gene therapy platforms. The firm’s clinical program is a LVV-based gene therapy for ...

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 ...

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, ...

Genascence Corporation ("Genascence"), a clinical-stage biotechnology company revolutionizing the treatment of prevalent ...

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 ...

StitchR, a new gene therapy technique, delivers large genes in two parts to treat muscular dystrophies by restoring critical proteins in animal models.

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