The approval of Waskyra for Wiskott–Aldrich syndrome demonstrates that gene therapies can successfully reach regulatory approval when developed by non-profit organizations. This milestone may accelerate future therapeutic innovation, broaden investment in rare disease research, and expand access to curative-intent treatments for underserved patient populations.

For decades, Huntington’s disease had no treatment that could alter its course. The new AMT 130 gene therapy trial shows promise in slowing disease progression, marking a historic milestone for patients, families, and the broader rare disease community.

Explore the reality of Gene Therapy in rare conditions. Is it a genetic fix or false hope?

Tragic deaths in Sarepta’s gene therapy trials have deeply impacted the rare disease community, raising concerns about liver risks, FDA responses, and treatment access.

In a groundbreaking first, doctors developed a custom gene- editing therapy in just seven months to treat a baby with a deadly and ultra-rare metabolic disorder.

New research on "jumping genes" could lead to safer, more precise gene-editing techniques.

Retinitis Pigmentosa (RP) is a group of rare, inherited eye disorders leading to progressive vision loss.

Explore the FDA's extension of the priority review period for Kresladi, a groundbreaking gene therapy for severe LAD-1

Explore the FDA's groundbreaking approval of CASGEVY™ the treatment of transfusion-dependent beta-thalassemia (TDT).