gene therapy

Gene therapy benefits for retinal dystrophies persist through trialResults from 3 years of follow-up in the phase III trial investigating treatment with adeno-associated viral vector delivery of human RPE65 (voretigene neparvovec, Spark Therapeutics) show this gene therapy has an acceptable safety profile.
Tisagenlecleucel (Kymriah) for Acute Lymphoblastic Leukemia
Tisagenlecleucel (Kymriah) for Acute Lymphoblastic LeukemiaA new era of treatment has arrived—here’s what you need to know.
Game-changing gene therapies present cost problems for health execsCAR-T therapies have high price tags so it’s important to understand your patient population and budget impact.
Four ways health execs can prep for costly CAR-T cell therapiesWith the high efficacy and dire diagnosis, there will be demand for both Kymriah and Yescarta. Here’s how to manage the monetary impact.
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.