Chantez Sanford Jr., 24, of Southfield, says his life has been transformed after undergoing Lyfgenia gene therapy at Children ...

With the FDA expecting to approve 10–20 Cell and Gene Therapies (CGTs) annually by 2025, it is evident that the field will continue to hold immense commercial and medical opportunities. To harness ...

Apertura Gene Therapy, a biotechnology company developing next-generation AAV capsids for delivering genetic medicines, and Viralgen, a leading contract development and manufacturing organization ...

BioCurie to Receive Up to $9.3 Million ARPA-H Award to Build AI Platform for Scalable Gene Therapy Manufacturing Key funding milestone will power the company's push to replace trial-and-error process ...

The last available hemophilia gene therapy is temporarily unavailable, according to CSL Behring.  | The company is working with regulators to ensure stable ongoing supply for the one-time gene therapy ...

Belief BioMed ("BBM") today announced that BBM-H901 (generic name: Dalnacogene Ponparvovec Injection), has been officially approved by the Pharmaceutical Administration Bureau of Macao Special ...

The special properties of methylcellulose foam could make it a vehicle for bedside genetic engineering, according to a proof-of-principle study from bioengineers at Fred Hutch Cancer Center. In the ...

New research shows epigenetic silencing may limit durability of AAV gene therapy in haemophilia by reducing long term transgene expression in liver cells.

A single one-time gene therapy could free patients with α-thalassemia, a rare and debilitating blood disorder, from the burden of lifelong transfusions. A single one-time gene therapy could free ...

Ultragenyx Pharmaceutical Inc. RARE shares are down on Thursday as the company reported data from its Phase 3 study of DTX301 ...

Researchers at Penn’s School of Veterinary Medicine have developed new mechanisms to treat advanced forms of inherited retinal degeneration, bridging a previous gap in patient treatments. Raghavi ...