For the first time, the tools to rewrite human biology are moving from speculative fiction into regulated clinics and ...

Plan to resume MAGNITUDE-2 patient enrollment and dosing FDA engagement ongoing regarding clinical hold on MAGNITUDE Phase 3 clinical trial in ...

Genetic disorders occur due to alterations in the primary genetic material—deoxyribonucleic acid (DNA)—of an organism.

The gene editing tool known as CRISPR-Cas9 is changing what's possible for treating a wide range of diseases caused by genetic mutations. But so far, attempts to use the technology to address ...

After a patient safety signal and then death, the FDA in October 2025 placed holds on two of the company’s CRISPR programs for hereditary transthyretin amyloidosis.

Picture CRISPR-Cas9, a gene editing technology, as a GPS-guided scalpel: gRNA directs the Cas9 enzyme, a protein that cuts ...

CRISPR–Cas9-based therapies are widely investigated for their clinical applications. However, there are limitations ...

Gene editing is rewriting the future of medicine, as seen in the case of a baby named KJ Muldoon who got a fully personalised ...

Beam Therapeutics BEAM is engaged in developing investigational gene therapy candidates that are currently in early-to-mid-stage development for treating certain hematology and genetic diseases. The ...