FDA approves first gene therapy for inherited disease



Dr. Gottlieb also commented: "Today's approval marks another first in the field of gene therapy, both in how therapy works and in expanding the use of gene therapy beyond cancer treatment for the treatment of vision loss. … The culmination of decades of research has resulted in three gene therapy approvals this year for patients with rare and serious diseases.I believe that gene therapy will become a fundamental pillar to treat, and perhaps cure, many of our most common diseases. devastating and intractable. "

Next year, the FDA intends to begin launching a series of what it calls disease-specific guidance documents the development of specific gene therapy products. This will "establish modern and more efficient parameters – including new clinical measures – for the evaluation and review of gene therapy for high priority diseases where the platform is being attacked," said Dr. Gottlieb. "We are at a turning point when it comes to this new form of therapy and, at the FDA, we are focused on establishing the right policy framework to capitalize on this scientific openness."

Biallelic RPE65 Associated Mutation Retinal dystrophy affects 1000 to 2000 patients in the USA. UU and is part of a group of disorders of the retina caused by more than 220 different genes that lead to progressive visual dysfunction and potentially blindness. In the case of retinal dystrophy badociated with the biallelic RPE65 mutation, affected individuals have inherited mutations in both copies of the RPE65 gene, which results in reduced levels or a complete lack of the RPE65 protein in the cells of the retina. The disorder leads to progressive loss of vision, often during childhood or adolescence, and eventual blindness.

Administered as a subretinal injection, Luxturna uses an AAV vector to deliver a normal copy of the RPE65 gene to viable retinal cells in patients with confirmed disease. Gene therapy has been tested in two open phase I studies and one open controlled phase III study, involving 41 participants aged 4 to 44 years at the time of treatment.

Luxturna will be administered to patients with genetically confirmed disease, by specially trained surgeons in selected US centers. UU Spark anticipates that gene therapy will be available during the first quarter of 2018. The firm will manufacture Luxturna at its facility in West Philadelphia, which it says is the first manufacturing facility in the US. UU licensed for a gene therapy to treat an inherited disease.

"During more than 12 years of innovative research with dedicated collaborators near and far, I have witnessed dramatic improvement in vision in many patients who would otherwise have lost their sight," commented Jean Bennett, MD Ph. D., FM Kirby Professor of Ophthalmology at the Perelman School of Medicine at the University of Pennsylvania and the Scheme Eye Institute of Penn. "I believe that the success of Luxturna's clinical development program will pave the way for the development of other genetic therapies that can help the millions of patients with genetic diseases who currently have limited or no treatment options."

"This approval is a milestone in the watershed," said Benjamin Yerxa, Ph.D., executive director of Foundation Fighting Blindness (FFB), a non-profit organization focused on research to prevent and treat blindness caused for inherited retinal diseases. "For people with an inherited retinal disease and for other patient communities, this decision can create an important boost for research genetic therapies."


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