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 ...

Thanks to in-utero blood transfusion technology, what was once a fatal diagnosis in the womb can now result in live births.

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 ...

Ocugen (NasdaqCM:OCGN) just gave investors something to talk about by announcing a new licensing agreement that hands Kwangdong Pharmaceutical the exclusive rights to commercialize its gene therapy ...

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 ...

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