Atsena Therapeutics, a clinical-stage gene therapy company focused on using the life-changing power of genetic medicine to reverse or prevent blindness, today announced that the U.S. Food and Drug ...

The gene therapy BBM-H901 (dalnacogene ponparvovec) has been approved in China for adults with moderate to severe hemophilia ...

The prospects for a one-time treatment that reduces the risk of heart disease for the rest of a person’s life just got ...

INmune Bio (INMB) has partnered with the Cell and Gene Therapy Catapult to establish large-scale, commercial-ready manufacturing for its cell ...

But a new genetic test appears capable of sussing out which men are at greater risk of urinary side effects from radiation ...

Feb. 7, 2025 — A research team has made a major discovery on how the CUL5 gene affects CAR-T cell therapy, an advanced cancer therapy. The study reveals that reducing the activity of the CUL5 ...

RAG-17 therapy slowed functional decline and stabilized or improved breathing in people with ALS who carry mutations in the SOD1 gene.

The new bid surpasses an offer SK Capital and Carlyle Group made last month for the gene therapy developer, which Bluebird’s board still supports.

The death of a young boy with Duchenne muscular dystrophy (DMD) who was treated with Pfizer’s experimental gene therapy in a phase 2 trial has prompted the company to pause dosing in another study.

A case report in Japan shows Spinraza helped a boy with SMA type 0 sit without support for a short time at age 2 and stand ...