CRISPR

Scribe's ELXR platform silences PCSK9 in the liver without cutting DNA; a single low dose sustained LDL reductions for roughly 18 months in primates.

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.

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.

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.

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.

A two-vector system inserts a CAR transgene at the TRAC locus inside the body, generating functional cancer-killing T cells without ex vivo manufacturing or lymphodepletion.