gene therapy

First gene therapy for cancer approved: What health execs should knowThe first therapy based on gene transfer has been approved by the FDA. What’s next for healthcare executives?
Gene therapy trial for blistering skin condition advancing to next stage
Gene therapy trial for blistering skin condition advancing to next stageFCX-007 could be the first major medical advance for recessive dystrophic epidermolysis bullosa, or RDEB, in decades.
FDA Approves First Gene TherapyWhy the agency says the approval of Novartis’ new therapy is historic.
Unmasking retinal structure, function in X-linked retinoschisis“There is a compelling need to explore gene therapy in the eye,” according to Paul A. Sieving, MD, PhD.
ARVO 2016 meeting paves path for personalized medicineEmerging trends and hot topics in eye and vision research filled the sessions at this year’s meeting of the Association for Research in Vision and Ophthalmology.
Surgical gene therapy study for wet AMD yields mixed resultsIn a phase IIa study of patients with neovascular age-related macular degeneration, a single subretinal injection of rAAV.sFlt-1 gene therapy demonstrated acceptable safety, but not a complete or durable anti-VEGF response. Additional preclinical research is under way.
Gene therapy for wet AMD shows promising long-term resultsThree-year follow-up from the phase I/IIa trial of rAAV.sFlt-1 subretinal injection is encouraging for gene therapy for exudative age-related macular degeneration.
Researchers explore promise of gene therapyScientists explore innovative delivery systems that might one day treat hemophilia and other blood diseases
Gene therapy improves functional vision in Leber congenital amaurosisA phase III gene therapy trial for Leber congenital amaurosis showed that patients’ mean mobility test scores promptly improved after treatment, and the benefit was sustained at 1 year, said Albert M. Maguire, MD.
Gene therapy new frontier for ocular disorders
Gene therapy new frontier for ocular disordersThe advent of gene therapy poses a new area of drug development for inherited and acquires ocular disorders.