Few companies stand better poised than Beam Therapeutics to reap the first fruits of the FDA’s promised flexibility toward cell and gene therapies. | Riding a regulatory win for its base editing ...

Gene therapy treatments for rare diseases are being developed, but getting them out of the lab has proved challenging.

People looking to lose weight and lower their blood sugar may someday be able to get a single injection that turns their cells into tiny factories churning out a protein that is essentially the active ...

Ocugen, Inc. gene therapy outlook: key 2026 catalysts, Phase 2/3 eye-disease data, BLA plans to 2028, and risks. Click for ...

A preclinical study uncovered a new gene therapy that targets pain centers in the brain while eliminating the risk of addiction from narcotics treatments, a breakthrough which could provide hope for ...

Osaka, Japan - A research team at The University of Osaka has unveiled the molecular mechanism behind genome ejection from adeno-associated virus (AAV) vectors, a crucial delivery vehicle in gene ...

Cell and gene therapies use the body's building blocks - cells and genetic material - to boost the immune system or correct ...

A preclinical study uncovered a new gene therapy that targets pain centers in the brain while eliminating the risk of ...

Abu Dhabi achieved a medical milestone on January 5, 2026, with the UAE's first gene-therapy injection for inherited blood disorders. CASGEVY, utilizing CRISPR-Cas9, corrects faulty DNA in conditions ...