CRISPR / Gene Editing News

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Weekly gene editing clinical trial progress, company moves, and ethics debates

CRISPR / Gene Editing News2026/05/29 22:36:07

Gene Editing Weekly — May 22–29, 2026

Cardiovascular base editing dominates the week: Lilly/Verve's VERVE-102 Phase 1b paper in NEJM reports up to 88% PCSK9 reduction and 62% LDL-C lowering in a single dose, flanked by a China Phase 1 trial in Nature Medicine and Editas EDIT-401 NHP data — the densest PCSK9 base-editing data week yet. CBER installed its fourth acting director since January 2025. Charles River's CGT CDMO exit and an AAV-linked pediatric brain tumor in Nature Medicine round out the week's key signals.

CRISPR / Gene Editing News2026/05/22 22:35:09

Gene Editing Weekly — May 17–22, 2026

REGENXBIO's pivotal Phase 3 RGX-202 data in Duchenne muscular dystrophy dominates the week: the primary endpoint was met (93% microdystrophin expression, first-ever expression-function correlation in DMD), but two SAEs and FDA’s RCT suggestion triggered a 43% stock crash and cast the 2027 approval target in serious doubt. Intellia’s lonvo-z advanced into rolling BLA — becoming the first in vivo CRISPR therapy under active FDA review — while Beam presented 29-patient BEAM-302 durability data at ATS 2026 and announced a pivotal cohort for H2 2026. The new CBER acting director Karim Mikhail takes over as three gene editing BLAs converge on FDA, and the April NGS off-target guidance sets the technical baseline for what will be entirely novel regulatory territory.

CRISPR / Gene Editing News2026/05/17 22:54:06

Gene Editing Weekly — May 10–17, 2026

ASGCT 2026 week delivered two gene-editing clinical readouts: Vor Biopharma's trem-cel achieved 100% Day-28 engraftment in all 30 AML/MDS patients in a final Phase 1/2a report in Nature Medicine; Fate Therapeutics' FT819 iPSC CAR-T produced 100% SRI-4 response in 3 lupus patients. FDA leadership disruption — Commissioner Makary resigned, CDER director Høeg departed — creates near-term uncertainty for three gene-editing BLAs in progress. Science Translational Medicine published a coordinated prime/base-editing preclinical trio for epilepsy and metabolic disease; NEJM reported the first AAV integration-induced tumor in a human patient.

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