Spark up: US FDA approves first gene therapy for genetic disease

By Dan Stanton

- Last updated on GMT


Related tags Gene therapy Gene Genetics Dna

Spark Therapeutics has received US approval for Luxturna (voretigene neparvovec-rzyl), its one-time gene therapy treatment for an inherited form of vision loss.

The US Food and Drug Administration (FDA) approved Luxturna for patients with biallelic RPE65 ​mutation associated retinal dystrophy, making it the first gene therapy product to receive regulatory the thumbs up for a genetic disease.

The therapy is a recombinant adeno-associated viral (AAV) vector serotype 2, expressing the gene for human retinal pigment epithelial 65 kilodalton protein (AAV2-hRPE65v2) which was found to be 93% effective in improving vision in patients with inherited retinal dystrophy (IRD) in Phase III trials.

Spark’s CEO Jeffrey Marrazzo declared the approval as “a moment decades in the making for the field of gene therapy” ​which “may lay the groundwork for the development of gene therapies for other conditions that are not adequately addressed today.”

Spark will make Luxturna at its 48,000-square foot facility in West Philadelphia, Pennsylvania, which it says is the first licensed manufacturing plant in the US for a gene therapy treating an inherited disease.

“The gene therapy will be administered at selected treatment centers in the US by leading retinal surgeons, who will receive surgical training provided by Spark Therapeutics on the administration procedure,”​ the firm said.

Cell therapies and gene therapies

The news comes in a landmark year for personalised medicines in the US, following the approvals of Novartis’ Kymriah (tisagenlecleucel)​​ and Gilead/Kite’s Yescarta (axicabtagene ciloleucel)​.

​However, both are chimeric antigen receptor (CAR) T cell therapies, which differ from gene therapies such as Luxturna.

Cell therapy transplants whole cells into a person while gene therapies manipulate cells by bringing genetic material into cells that are not working correctly or could work better, delivered using a vector.

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