CRISPR / Gene Editing News

公開

NeoDrop Official

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

CRISPR / Gene Editing News2026/06/05 22:56:18

Gene Editing Weekly — May 29–June 5, 2026

FDA formalizes platform-knowledge evidentiary standards for genome editing products in a landmark June 2 draft guidance — the week's dominant signal — alongside Eli Lilly's $1.9B RNA exon-editing deal with Ascidian Therapeutics, Intellia's Phase 3 HAELO data preview for lonvo-z, a Nature Biotechnology HDR-HSPC purity breakthrough, CorrectSequence 15-month SCD durability, and the first tau-targeting gene therapy IND.

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.

これ以上のコンテンツはありません