Beam Therapeutics says risto-cel met its pivotal endpoint of at least 12 months free of severe pain crises in the BEACON trial, and plans to seek FDA approval as early as the end of 2026.

A new FDA draft guidance would let one license cover a personalized gene editor and its patient-specific versions. New mutations could be added with lab data instead of new clinical trials, a path built for cases like KJ Muldoon's.

Cornell researchers fused two bacterial proteins, TldR and TniQ, into a compact RNA-guided system that inserts large pieces of DNA at a chosen site without a CRISPR-Cas protein. It works only in bacteria so far, but it targets one of gene editing's biggest gaps.

An arbitration tribunal confirmed Prime Medicine's rights to PM647, a prime-editing therapy for alpha-1 antitrypsin deficiency, ending a dispute with Beam Therapeutics. Here's the science and what comes next.

A June 2026 FDA draft guidance lets developers of genome-editing therapies reuse prior manufacturing, nonclinical and clinical knowledge. Here's why the platform approach could speed rare-disease cures.

Phase 1 data for VERVE-102, a one-time base-editing treatment that switches off PCSK9, showed LDL cholesterol cuts of up to 62% lasting up to 18 months. Here's what it means for heart disease.

As CRISPR-based therapies edge closer to becoming mainstream, groundbreaking treatments for blood disorders and cancer are making their mark. One of the most exciting developments is Casgevy, a gene-editing therapy that has shown remarkable success in treating sickle-cell disease and β-thalassaemia. Approved in both the UK and US, Casgevy works by reactivating fetal hemoglobin, offering patients a chance at long-term relief with fewer blood transfusions and hospital visits. W