CRISPR / Gene Editing News

CRISPR / Gene Editing News

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NeoDrop Official
NeoDrop Official

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

CRISPR / Gene Editing News
CRISPR / Gene Editing News

Gene Editing Weekly - Jun 26-Jul 3

This issue leads with FDA’s Casgevy pediatric label expansion to age 2+ and its 53-day CNPV review, then ties that to CBER personnel churn, Capricor’s upcoming advisory committee, and the substantial-evidence guidance read-through. It also covers REGENXBIO’s NAAVIGATE start and AbbVie milestone, Beam’s BEAM-304 IND clearance, Voyager’s VY1706 AAIC marker, Sangamo’s fresh 8-K deterioration, and the week’s science/governance signals.

CRISPR / Gene Editing News
CRISPR / Gene Editing News

Gene Editing Weekly — June 19–26, 2026

This week’s briefing centers on Sangamo’s Chapter 11 filing and asset-sale process, FDA/CBER’s renewed flexibility around rare-disease gene therapy pathways, and a new embryo base-editing paper that intensified governance pressure. It also covers REGENXBIO’s NAVSUNLI and RGX-202 updates, Prime Medicine’s PM359 RMAT designation, Serapha’s reverse merger and financing, BEAM-304 IND clearance, and the EU’s NGT crop regulation.

CRISPR / Gene Editing News
CRISPR / Gene Editing News

Gene Editing Weekly — June 12–19, 2026

The week's lead story is the full Phase 3 HAELO readout for Intellia's lonvo-z — 87% HAE attack reduction, 62% attack-free at six months in 80 patients, no serious adverse events, simultaneous NEJM publication — the first in vivo CRISPR therapy to complete Phase 3. On June 18, Beam Therapeutics received FDA IND clearance for BEAM-304 (PKU base editing) and Prime Medicine received New Zealand's first-ever clinical authorization for in vivo prime editing (PM577a, Wilson disease), both citing FDA's June 2 platform-knowledge guidance. UniQure's AMT-130 FDA reversal marks the third documented CBER flexibility case. Two SCD gene editing papers (risto-cel + reni-cel) appeared in NEJM. Research highlights include the Doudna lab's 49% in vivo liver prime editing efficiency (Nature Nanotechnology) and LEAPER 2.0's 1.5-year DMD NHP durability and first-in-human data (Cell). The EU Parliament passed a gene-edited crops fast-track law.

CRISPR / Gene Editing News
CRISPR / Gene Editing News

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 News
CRISPR / Gene Editing News

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 News
CRISPR / Gene Editing News

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 News
CRISPR / Gene Editing News

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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