Vertex Pharmaceuticals said on Saturday its gene therapy helped children aged between 5 and 11 years with sickle cell disease ...
Abeona Therapeutics Inc. (Nasdaq: ABEO) today announced the first commercial patient treatment with FDA-approved ZEVASKYN ...
A cutting-edge base-edited CAR T-cell therapy is opening a revolutionary new frontier for patients battling aggressive T-cell leukemia.
Over the last decade, the number of cell and gene therapy clinical trials has more than tripled. In this article, several ...
Novartis’ Itvisma wins FDA approval as the first gene replacement therapy for spinal muscular atrophy (SMA) patients age 2 ...
Itvisma contains the active ingredient in Zolgensma approved in 2019 for children younger than 2 years but is reformulated ...
Operational real-world data analyzed from patients prescribed ZYNTEGLO™ and LYFGENIA™Data demonstrates the value proposition of one-time, durable gene therapy for the treatment of sickle cell disease ...
The new intrathecal formulation means the one-shot gene therapy is now available to all SMA patients, regardless of their age ...
Please refer to the ASH 2025 online program for full session details and visit the Genetix booth (#1261) onsite.
Biotechs in the charter city of Próspera aim to cure ageing, but experts question the feasibility of their goals.
14don MSN
California boy, 3, receives first-ever gene therapy for his rare disease: ‘It’s just so exciting’
A California toddler is the first person in the world to receive gene therapy to treat his devastating disease. Three-year-old Oliver Chu was born with a rare, genetic condition called Hunter syndrome ...
Pharmaceutical Technology on MSN
Regeneron and Tessera join forces to forge in vivo AATD gene therapy
If approved, Regeneron and Tessera's TSRA-196 could become the first curative therapy to reach patients with AATD, who have ...
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