Fractyl Health is developing a shot that would program the body to make more of the GLP-1 hormone naturally, a risky bet that it can provide a longer-lasting benefit than blockbuster weight-loss drugs ...

GENE202 is a single dose gene therapy for patients with rare metabolic disease, methylmalonic acidaemia.

Orphan Drug Designation applies to approximately 50,000 Patients who predominantly suffer from severe chronic pain due ...

Nanoscope Therapeutics has received Pioneering Regenerative Medical Product (Sakigake) and Orphan Drug designations for ...

At a Food and Drug Administration cell and gene therapy (CGT) roundtable last June, most speakers pushed for the agency to ...

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.

NEW YORK and TRUMBULL, Conn., April 30, 2025 /PRNewswire/ -- Apertura Gene Therapy, a biotechnology company focused on innovative gene therapy solutions, and the Rett Syndrome Research Trust (RSRT), ...

Universal gene‑edited CAR‑T therapies turn donor T cells into off‑the‑shelf cancer treatments, cutting delays and costs while ...

The FDA granted fast track status to VectorY’s VTx-002, an antibody gene therapy targeting TDP-43 protein aggregates in ALS.

Clinical and commercial quality controls are eased as the FDA looks to expedite therapy approvals in areas of unmet need.

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ELRIG, a not-for-profit, volunteer-led organisation dedicated to the global drug discovery community, today announced the ...