NHS Scotland has approved a one-time CRISPR gene therapy for severe sickle cell disease, offering durable reduction in vaso-occlusive crises for eligible patients.
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 ...
The FDA AMT designation verifies NanoMosaic platform's ability to enhance efficiency, product quality, and scalability across AAV gene therapy manufacturing.
Universal gene‑edited CAR‑T therapies turn donor T cells into off‑the‑shelf cancer treatments, cutting delays and costs while ...
Gazi University in Ankara has officially launched the production process for Türkiye's first local and national gene therapy research product. The project, which targets rare genetic diseases, is ...
NanoMosaic's technology is designed to support upstream and downstream process development, quality control, and release testing.
Under the agreement, participating states will receive “discounts and rebates” from the drugmakers if the treatments don’t ...
Data represents up to 8.5 years of follow-up and are consistent across age, dose, and genotypeBLA resubmitted to U.S. FDA in January 2026; ...
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 ...
What Is Waskyra, and Why Does It Matter? Waskyra (etuvetidigene autotemcel) is a new gene therapy approved to treat Wiskott-Aldrich syndrome (WAS), a rare inherited condition that affects the immune ...
As the controversy over the safety of Sarepta Therapeutics’ gene therapies comes to a head, the biotech is standing firm behind its approved Duchenne muscular dystrophy (DMD) treatment. The FDA, for ...
Just before the Covid-19 pandemic, it seemed gene therapies were a few years away from curing all sorts of diseases with a quick, simple infusion. The idea was that drugmakers could stuff some genetic ...
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