Jesse Gelsinger died after receiving a gene therapy treatment to treat a liver disease. The death sparked an investigation ...

The indication has been expanded to include use in newborns and allows patients and caregivers to apply the treatment at home ...

At the European Cell & Gene Therapy Summit 2025, funding challenges and innovation drove discussions on the sector’s future.

A new analyst survey suggests that doctors are still prescribing Sarepta’s Elevidys, even after a series of deaths in certain ...

Prominent critics say Sarepta’s Elevidys Duchenne therapy may overstate how much gene it delivers—raising new questions about ...

Fresh treatments for rare skin diseases shift the focus from symptom management to repair and help children with such ...

Sangamo Therapeutics hasn’t convinced a pharma company to buy its experimental gene therapy for Fabry disease after two years ...

Kriya Therapeutics has closed an oversubscribed Series D funding round, raising $320m, aimed at propelling its gene therapy ...

University of Iowa researchers developed a gene therapy that could prevent craniosynostosis, a condition where babies’ skull ...

The commercial success of existing lipid-lowering medications highlights the enormous market potential for effective ...

The biotech has paused its Phase I trial of CAP-002, pending investigation into the “root cause” of the death.