Tag: #genetherapy

FDA approves Otarmeni, first U.S.-cleared gene therapy for genetic hearing loss

FDA approves Otarmeni, the first U.S. gene therapy for OTOF-linked deafness; Regeneron's dual-AAV treatment wins accelerated approval, follow-up data required.

#fda, #regeneron, #genetherapy, #hearingloss, #otology

FDA Issues Draft Guidance on Using Next-Generation Sequencing to Assess Genome-Editing Safety

FDA's draft guidance explains how developers should use next-generation sequencing to detect off-target edits and chromosomal damage before human trials.

#geneediting, #fda, #ngs, #genetherapy

Engineered ‘living’ cell implant normalizes blood pressure in mice, study shows

Engineered human cell implants sense angiotensin II and secrete soluble ACE2 to restore normal blood pressure in hypertensive mice.

#syntheticbiology, #hypertension, #genetherapy, #ace2

Iranian researchers use AI to design kidney-targeted viral shells for gene therapy

A preprint describes AAVGen, an AI system that generates adeno-associated virus capsids predicted to better target kidneys—so far only in silico.

#genetherapy, #ai, #kidney, #aav