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 ...
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 ...
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.
Preliminary results from two trials of the gene therapy exagamglogene autotemcel (exa-cel) suggest the therapy offers an effective cure for beta-thalassemia and sickle cell disease in children younger ...
Tenaya Therapeutics remains pre-revenues, and it’s a thinly traded microcap. Its tight cash runway is also a concern. Read ...
Research links variations in the gene GRIN2A to a higher risk of developing schizophrenia and other forms of mental illness.
Two years ago, he was diagnosed with a terminal illness called Batten disease -- often leading to blindness, seizures, and ...
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