gene-editing

A single infusion of VERVE-102 reduced LDL-C by up to 62% and PCSK9 by up to 88% in a 35-patient trial, with effects sustained up to 18 months.

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.

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.

Eli Lilly's deal with Bezos-backed Profluent funds AI-designed recombinases that aim to insert entire genes, going beyond CRISPR's localized cuts.

Two companion Nature Biotechnology papers map over 30,000 CRISPR edits across sorghum's regulatory DNA, achieving unprecedented control over photosynthesis genes with implications for crop yields and carbon capture.

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 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.

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.

The RUBY trial of renizgamglogene autogedtemcel (reni-cel), a one-time CRISPR-Cas12a gene-editing therapy, showed that 27 of 28 patients with severe sickle cell disease had no painful crises after treatment, with hemoglobin levels normalizing to 13.8 g/dL by six months.

Researchers at the University of California San Diego have developed pPro-MobV, a second-generation CRISPR-based gene drive that spreads through bacterial populations via conjugal transfer to disable antibiotic resistance genes, demonstrating effectiveness even within biofilms.

Precision BioSciences receives FDA Fast Track for PBGENE-DMD, a gene-editing therapy that excises faulty exons to restore near full-length dystrophin in up to 60 percent of Duchenne patients.

A Nature paper describes INSTALL, a gene editing platform using circular single-stranded DNA to insert therapeutic genes without triggering fatal immune responses, keeping mice healthy at doses where conventional DNA proved lethal.