This multi-center, open-label, dose-escalation clinical trial in the U.S. will assess the safety, tolerability and efficacy of KH658 as a single suprachoroidal space administration of gene therapy for ...

Pacira BioSciences, Inc. announced that it will present findings from a Phase 1 study of its gene therapy candidate, PCRX-201 (enekinragene inzadenovec), for knee osteoarthritis at the 28th Annual ...

The HHS released a report advocating for behavioral therapy and against gender-affirming care for transgender youth. The report, commissioned by a Trump executive order was authored by unnamed ...

A boy with an ultra-rare immune disease which can carry a “death sentence” is living a normal life after taking part in a groundbreaking gene therapy trial. Four-year-old Eisa Hussain now ...

Red, orange, yellow, green, blue, indigo, and violet: the rainbow. But what if that wasn’t it? Sophomore Ryan Evert ...

Many diseases are caused by a missing or defective copy of a single gene. For decades, scientists have been working on gene therapy treatments that could cure such diseases by delivering a new copy of ...

Martin, 20, was the first patient in St. Louis and among the first across the country to receive a new gene therapy for sickle ... He doesn’t yet have the green light to attend a Cardinals ...

Designer Mitesh Trambadia’s (@desimeetsdesign) home, built in the 2000s, features a 165-square-foot bathroom with a clunky soaker tub and green walls — neither of which he wanted anymore. “I also did ...

Caribou was one of the first biotech companies formed to turn CRISPR gene editing into medicines for humans ... The company is now pinning its hopes on CB-010, a therapy for non-Hodgkin’s lymphoma, ...

Intas Pharmaceuticals has announced a series of leadership appointments across several core areas, including gene therapy, information technology, quality control, and learning and development. The ...

Ray Therapeutics’ approach uses an optimized optogenetic gene therapy to deliver light-sensitive proteins to the retina of the eye to restore visual function to patients with RP, regardless of ...

Benefits of the gene therapy persisted over several years of follow-up. Presymptomatic children with MLD appeared to have the best outcomes. Severe motor impairment or death was significantly less ...