Gene Therapy
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Encoded's ETX101 Cuts Dravet Seizures 76% at Dose Level 3 and Tracks Toward Neurotypical Development in Children Treated Before Age 2
An AAV9 gene-regulation therapy delivered durable, dose-dependent seizure control and early signs of neurodevelopmental rescue through 52 weeks, just as the pivotal study begins dosing.
Two teams publish DNA-guided CRISPR-Cas12 systems in Nature Biotechnology, inverting the RNA-guide paradigm for RNA diagnostics and antivirals
Researchers at the University of Florida and the Hong Kong University of Science and Technology each report a DNA-guided CRISPR-Cas12 platform that targets RNA, reducing off-target effects and enabling attomolar-level pathogen detection.
United Therapeutics Wins FDA Clearance for EXPRESS, the First Pig-Heart Xenotransplant Trial Designed to Support a BLA
The FDA cleared an IND for UHeart, a 10-gene-edited pig heart, opening a single-center phase 1/2/3 trial that will enroll up to two patients with end-stage heart failure before any expansion.
RNA-Triggered CRISPR System Selectively Kills Cancer and Viral Cells by Shredding Their DNA, Researchers Report in Nature
A University of Utah-led international team reports a new CRISPR nuclease, Cas12a2, that uses RNA guides to trigger widespread DNA shredding and cell death specifically in diseased cells such as KRAS-mutant cancers and HPV-infected cells, while leaving healthy cells unharmed.
Regenxbio's RGX-202 Hits Primary Endpoint in Pivotal Duchenne Trial, Clearing Path to a 2027 Accelerated-Approval Filing
Regenxbio's gene therapy RGX-202 met the primary endpoint of its pivotal Phase III AFFINITY DUCHENNE trial, with 93% of participants reaching the microdystrophin expression threshold.
CRISPR Therapeutics and Vertex post $43 million CASGEVY revenue in Q1 2026 as pediatric submission lands with FDA Priority Voucher
First-quarter results from both partners show CASGEVY past 500 treatment starts, a US filing for ages 5 to under 12, and CRISPR Therapeutics' in vivo pipeline advancing across four liver-directed programs.
Lilly Bets Up to $2.25 Billion on Profluent's AI-Designed Recombinases, Pushing Gene Editing Beyond CRISPR Into Kilobase-Scale Territory
Eli Lilly's deal with Bezos-backed Profluent funds AI-designed recombinases that aim to insert entire genes, going beyond CRISPR's localized cuts.
FDA Approves Regeneron's Otarmeni as First Gene Therapy for Genetic Hearing Loss, with the Drugmaker Pledging Free Access in the U.S.
Regeneron's one-time intracochlear gene therapy for OTOF-related deafness cleared the FDA 61 days after BLA filing, the first gene therapy product approved under the Commissioner's National Priority Voucher pilot program.
Intellia Therapeutics to Release World's First In Vivo CRISPR Phase 3 Data on April 27
Intellia Therapeutics will report topline Phase 3 results for lonvoguran ziclumeran in hereditary angioedema on April 27, 2026 — the first Phase 3 readout for any in vivo CRISPR candidate.
NIH-Funded Team Engineers a Miniature CRISPR Nuclease That Fits Inside Viral Vectors, Clearing a Major Hurdle for In Vivo Gene Therapy
UT Austin and Metagenomi researchers engineered Al3Cas12f RKK, a compact CRISPR enzyme that achieves over 80% editing efficiency while fitting inside AAV delivery vehicles.
Prime Medicine Seeks FDA Accelerated Approval for First-Ever Prime Editing Therapy After Two Patients Achieve Durable Immune Restoration
Prime Medicine is pursuing accelerated approval for PM359, an autologous prime editing therapy that restored immune function in both patients treated for chronic granulomatous disease, marking the first clinical use of prime editing technology.
UCSF and Berkeley Scientists Engineer CAR-T Cells Directly Inside the Body, Clearing Tumors in Mice With a Single Injection
A two-particle gene-editing system published in Nature creates functional CAR-T cells in vivo, eliminating the costly weeks-long manufacturing process and clearing leukemia, myeloma, and solid tumors in preclinical models.