Gene therapy for cystic fibrosis is advancing fast. Explore key biotech players, clinical progress, and its potential to ...

A new paper published in Gene Therapy raises serious concerns about the effectiveness of gene therapy for Duchenne muscular ...

Lexeo Therapeutics has posted updated data in Friedreich ataxia (FA) patients, linking a gene therapy to improvements on ...

The HFpEF Summit is a biannual, two-day meeting bringing together internationally recognized leaders in the clinical, biological, and translational study of HFpEF. This summit focused on the latest ...

Despite significant advancements in spinal muscular atrophy (SMA) management, unmet needs persist, especially for patients ...

In response to a request from the European Medicines Agency (EMA) following the death of a 16-year-old patient in the U.S., ...

Durham-based Atsena Therapeutics has raised $150 million to advance gene therapies for preventing or reversing types of ...

Beam Therapeutics Inc. (NASDAQ:BEAM) on Saturday presented additional data from the Phase 1/2 clinical trial of BEAM-302 in ...

Opus Genetics (IRD) announced one-month clinical data from the first pediatric patient treated with its investigational gene therapy, ...

None of the cell and gene therapies that reached the final regulatory submission stage were rejected by the FDA.

Atsena Therapeutics has raised $150 million in a round led by Bain Capital, a private investment firm based in Boston. The ...