The company has started enrolling patients into a run-in period for the Phase III study, and plans to begin dosing later this ...

Few companies stand better poised than Beam Therapeutics to reap the first fruits of the FDA’s promised flexibility toward cell and gene therapies. | Riding a regulatory win for its base editing ...

New one-dose treatment edits a gene in the liver, disabling the ANGPTL3 protein and significantly lowers cholesterol.

Opus Genetics has shared data on its gene therapy in children with an ultrarare eye disease, setting the biotech up to talk to the FDA about the next steps for the program. North Carolina-based Opus ...

Yvette Crawley, 71, from Apex, N.C., started noticing changes to her vision around the time she turned 60. She described it as if she were “looking through a dirty window.” She was diagnosed with a ...

Opus Genetics Inc. will be sitting down with the U.S. FDA to talk about positive three-month data from the pediatric cohort of its ongoing phase I/II trial called OPGx-LCA5-1001 – partially funded by ...

Youth with congenital deafness treated with gene therapy showed better progress on certain hearing measures than counterparts treated with cochlear implantation in a cohort study from China. Gene ...

Astellas Gene Therapies remains intent on treating X-linked Myotubular Myopathy (XLMTM) through a gene therapy—but instead of AAV-based resamirigene bilparvovec (AT132), Astellas is now partnering ...

Ocugen has announced the publication of positive results from its phase 1 GARDian1 trial of OCU410ST, a gene therapy for ...

A preclinical study uncovered a new gene therapy that targets pain centers in the brain while eliminating the risk of ...

The new method is designed to focus specifically on pain-related signals, without interfering with normal activity in other parts of the brain. A new preclinical study has identified a gene therapy ap ...