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

A team of doctors at the Children's Hospital of Philadelphia and Penn Medicine treated a baby with a gene editing technology ...

May 15, 2025 — A research team has developed and safely delivered a personalized gene editing therapy to treat an infant with a life-threatening, incurable genetic disease. The infant ...

This technique streamlines quality control in gene therapy vector production, potentially accelerating manufacturing and improving clinical outcomes. This summary was automatically generated using ...

The 28th American Society of Gene & Cell Therapy (ASGCT) meeting kicks off in mid-May in New Orleans. The annual event, which is on pace toward a goal of 8,000 attendees, will be a whirlwind of ...

A Johnson & Johnson gene therapy in development for an inherited vision-loss disorder has failed a Phase 3 study, the latest clinical research setback for a rare eye disease that so far has no FDA ...

Neurocrine Biosciences has handed back two discovery-stage gene therapy programs to Voyager Therapeutics, although the companies’ wider central-nervous-system-focused collaboration remains on track.

A new study in Human Gene Therapy describes a machine learning (ML) model that can be used as a surrogate for laborious in vitro experiments. This in silico approach aims to increase the fitness ...

Beacon Therapeutics may have found a guiding light in a disease area where other companies have stumbled with its laruparetigene zovaparvovec (laru-zova), a gene therapy that proved early ...

The FDA has approved Abeona Therapeutics’ prademagene zamikeracel (Zevaskyn) gene therapy for recessive dystrophic epidermolysis bullosa (DEB), a rare genetic skin disease. Prademagene ...

About Skyline Therapeutics Skyline Therapeutics is an innovation-driven, clinical-stage gene therapy company focused on developing unique and novel therapeutic solutions for rare and severe diseases ...

High dose and low dose AMT-130 demonstrated an 80% and 30% slowing of disease progression vs external control, respectively. The Food and Drug Administration has granted Breakthrough Therapy ...