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How to advance AAV-based gene therapies

Drug Discovery World

Adeno-associated virus (AAV) vector-based gene therapies hold exceptional promise across a range of disease areas. We’re beginning to see their potential come to fruition, with the FDA having approved three treatments as of January 2023 — for retinal dystrophy 1 spinal muscular atrophy, 2 and haemophilia B. She received a Ph.D.

Therapies 163
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Who are the AACR Scientific Achievement Award winners?

Drug Discovery World

He is being recognised for his revolutionary contributions to developing the first gene-edited cell-based therapy for cancer that involves the genetic re-engineering of a patient’s own T cells to combat their disease, and for demonstrating that adoptive T-cell therapy can induce remission and in some cases cure patients with advanced cancer.

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A research team searches for every gene that helps tumors evade immunotherapy

Broad Institute

By Allessandra DiCorato October 11, 2023 In 2011, Robert Manguso was working in a cell biology lab when his mother was diagnosed with Merkel cell carcinoma, a rare and aggressive skin cancer. His mother had a presentation of the disease that suggested her immune system was already on the job.

Research 137