Gene Therapy: The Future of Rare Disease Treatment
Experts shared opinions about the future of gene therapy in sickle cell and other rare diseases.
Replacement, Non-Factor, and Gene Therapies: A Look at Current Options for Hemophilia Treatment
A look at global access to gene therapy and an overview of the progress in hemophilia treatments.
Preclinical Data Show Promise for Adrenomyeloneuropathy Gene Therapy
The latest data build on previous research that showed on-target effects analyzed across multiple measurements and a favorable safety profile.
Stargardt Disease Gene Therapy IND Cleared to Proceed
The investigational therapy has received orphan drug designation from the FDA for Stargardt disease as well as retinitis pigmentosa.
Stem Cell Gene Therapy Efficacious in Metachromatic Leukodystrophy
Investigators from IRCCS San Raffaele analyzed results from a phase 1/2 study on atidarsagene autotemcel.
bluebird bio Sickle Cell Gene Therapy On Hold Following Adverse Event
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.
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