President Trump signed an executive order on the day of his inauguration that created doubts about the future of a program to ...

Immvention Therapeutix is partnering with Novo Nordisk to develop oral treatments for sickle cell disease (SCD) and other chronic conditions.

Discover the latest addition to Children's Hospital Los Angeles' 10 cell and gene therapy treatments, including Casgevy for sickle cell disease ...

Since two gene therapies were approved for sickle cell disease in late 2023, only a handful of patients have started the expensive treatments.

This story was originally published on BioPharma Dive. To receive daily news and insights, subscribe to our free daily BioPharma Dive newsletter. As 2025 begins, emerging biotechnology firms can ...

Bluebird bio is a case study. The company has three approved gene therapies: Zynteglo for beta thalassemia, Skysona for cerebral adrenoleukodystrophy and Lyfgenia for sickle cell. Yet the firm has ...

The company, which secured $200 million from investors in a private funding deal last week, learned Tuesday of a serious side effect experienced by one participant on a high dose of its Rett therapy.

Story continues below this ad 4. Casgevy and Lyfgenia: In December 2023, the FDA approved two groundbreaking treatments, Casgevy and Lyfgenia, the first cell-based gene therapies to treat sickle cell ...

The FDA is scrutinising reports of haematological cancers in patients treated with bluebird bio's Skysona gene therapy for neurological disease CALD The US FDA has cleared two novel gene therapies ...