Giuliana Grossi

Experts shared opinions about the future of gene therapy in sickle cell and other rare diseases.

A look at global access to gene therapy and an overview of the progress in hemophilia treatments.

The latest data build on previous research that showed on-target effects analyzed across multiple measurements and a favorable safety profile.

The investigational therapy has received orphan drug designation from the FDA for Stargardt disease as well as retinitis pigmentosa.

Investigators from IRCCS San Raffaele analyzed results from a phase 1/2 study on atidarsagene autotemcel.

The FDA has placed a partial clinical hold on the program for patients that are under the age of 18 after an adolescent patient developed persistent anemia.