Perspective from Stephen Rose, PhD
December 20, 2017
2 min read
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Spark aims for availability of new gene therapy early in 2018

Perspective from Stephen Rose, PhD
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Newly FDA-approved Luxturna is expected to be available in selected treatment centers in the first quarter of 2018, according to a press release from Spark Therapeutics.

Luxturna (voretigene neparvovec) is the first directly administered gene therapy approved in the United States to target a disease caused by mutations in a specific gene, in this case biallelic RPE65 mutation-associated retinal dystrophy.

Jeffrey D. Marrazzo, CEO at Spark Therapeutics, called the approval of the one-time gene therapy a “landmark” moment. According to the release, the therapy represents a trio of firsts: the first FDA approved gene therapy for a genetic disease, the first pharmacologic treatment for an inherited retinal disease and the first adeno-associated virus vector gene therapy approved in the U.S.

Luxturna will be manufactured at Spark Therapeutics’ manufacturing facility in West Philadelphia, the first licensed manufacturing facility in the country for a gene therapy treating an inherited disease, according to the release. Selected retinal surgeons will receive surgical training provided by the company on the administration of the procedure.

“This one-time gene therapy for an inherited disease represents a first-of-its-kind breakthrough that may lay the groundwork for the development of gene therapies for other conditions that are not adequately addressed today. We offer our sincere gratitude to the patients and their families as well as the expert investigators who continue to participate in Luxturna’s clinical development program,” Marrazzo said in the release.

Disclosure: Rose reports no relevant financial disclosures.