oncodaily - Rabi Hanna, Pediatric Blood and Marrow Transplant Program Director, and Chairman at the Department of Pediatric Hematology-Oncology and BMT at Cleveland Clinic, shared a post on LinkedIn: “I am excited […]
AI Summary: A first‑in‑human CRISPR/Cas12a therapy for sickle cell disease has treated initial patients, building on preclinical gene‑editing successes that reversed severe disease in models. The advance underscores rapid translation from lab to clinic and renews hope for durable cures—while reminding everyone that the safety and long‑term durability questions remain firmly on the table.
FDA clears AstraZeneca’s new blood-pressure drug baxdrostat / 13 days
Antiviral ensitrelvir dramatically cuts household COVID transmission risk / 15 days
Gene therapy pioneers win Breakthrough Prize for CRISPR sickle‑cell work / 6 wks
Denali drug receives accelerated approval for rare CNS indication / 2 months
First-in-human trial primes immune system to accept donor livers / 6 wks
Experimental therapy significantly reduces Parkinson’s toxic protein in early trial / 7 wks
Therapeutic HPV vaccine slows throat tumors in preclinical tests / 3 months
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