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How to advance AAV-based gene therapies

Drug Discovery World

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.

Therapies 163
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Who are the AACR Scientific Achievement Award winners?

Drug Discovery World

He is being recognised for his fundamental discoveries dedicated to characterising the genetic drivers of human B-cell lymphomas and for uncovering new avenues for cancer prevention and therapy that have been routinely exploited clinically to diagnose and determine novel therapeutic strategies for B-cell lymphoma.

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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. By then, a few cancer immunotherapies had entered clinical trials and only a handful had been approved by the FDA.

Research 137