The first human applications of gene therapy before birth are expected within the next decade, pending rigorous safety and ethical evaluations. This new era also brings new questions. If we ...

Scientists successfully restarted inactive genes by bringing them closer to genetic switches on the DNA called enhancers. The ...

The results of gene therapy for hemophilia B contrast with those of factor replacement therapies, where lifelong infusions ...

The biotech plans to use one-year follow-up data from the Phase II/III trial to support a biologics license application for OCU410ST in 2027.

Gene therapy experts and advocates warning of the sector’s many challenges found a receptive audience in FDA leadership at a ...

RNA gene scissors (CRISPR-Cas13) are gaining significant attention as a next-generation gene therapy with fewer side effects.

Trump health officials signal eased FDA rules for rare disease gene therapy, but concerns linger over safety, oversight, and ...

Showcased 7 presentations at ISSCR 2025, including a featured oral presentation on first-in-human gene therapy for heart ...

Gene editing could be used to treat millions of glaucoma patients. Glaucoma is a leading cause of blindness, characterized by ...

Glioblastoma has been deadly and hard to treat, but researchers say they’ve made progress with bivalent CAR T-cell therapy ...

Ocugen has announced the US Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) amendment to ...