The FDA approved Itvisma gene therapy for patients aged 2 years and older with spinal muscular atrophy and confirmed SMN1 ...

Vertex Pharmaceuticals said on Saturday its gene therapy helped children aged between 5 and 11 years with sickle cell disease ...

In a 15-patient, Phase 1, first-in-human trial, a one-time CRISPR-Cas9 gene-editing therapy safely reduced LDL cholesterol and triglycerides in people with difficult-to-treat lipid disorders, ...

The first study assessing the real-world commercial roll-out of gene therapies for sickle cell disease and beta thalassemia ...

Boston-based Life Biosciences is testing whether reprogramming human cells can slow or even reverse the effects of aging. Unsplash Life Biosciences, a Boston-based biotechnology company founded in ...

A new gene therapy can reverse the effects of heart failure and restore heart function in a large animal model. The therapy increases the amount of blood the heart can pump and dramatically improves ...

Biotechs in the charter city of Próspera aim to cure aging, but experts question the feasibility of their goals.

Regeneron Pharmaceuticals has teamed up with Tessera Therapeutics to develop and commercialise Tessera’s rare disease in vivo ...

After telegraphing an investigation in November, the FDA has moved to restrict the use of bluebird bio’s gene therapy Skysona in certain patients. The FDA has updated Skysona’s indication, allowing it ...

Data from a primate study suggested MGX-001 has potential as a hemophilia A treatment, moving the drug closer to clinical ...