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Spark share value increases following historic treatment

Spark Therapeutics has celebrated the first FDA-approved application of its US$425,000 (AU$547,550) Luxturna gene therapy treatment.

The groundbreaking procedure was the first time any FDA-approved gene therapy had been given to a patient for an inherited disease and the company’s stock price has jumped to more than US$77 (AU$99.20) in the weeks following the successful treatment.

The procedure was performed on a 13-year-old boy in March by specialists from Massachusetts Eye and Ear (MEE) led by Dr Jason Comander, who said it was an honour to take part in medical history.

"I am so excited the time has come when we can offer this groundbreaking therapy to our patients, who are truly in need of our help."
Dr Jason Comander, Massachusetts Eye and Ear

“This project has been underway for 20 years, and the early successes with this therapy demonstrated by Jean Bennett, Al Maguire, and others, inspired me to dedicate my career toward helping patients with inherited retinal diseases,” Comander said.

“I am so excited the time has come when we can offer this groundbreaking therapy to our patients, who are truly in need of our help.”

The treatment involves injecting a modified virus into a patient’s eyes to correct a deficiency caused by mutations in the RPE65 gene. Without treatment, these mutations prevent the production or function of a protein needed for proper functioning of the retina.

Clinical trials found 93% of the participants reported improvement in functional vision, by measuring their ability to navigate through various controlled levels of light.

Chief of ophthalmology at MEE Dr Joan Miller said the success of the treatment had brought gene therapy from science fiction to clinical reality for patients with inherited retinal disease.

“Our hope is that our ophthalmology community can leverage Luxturna’s success to accelerate the development of similar gene therapies for the many blinding retinal diseases that still afflict our patients,” Miller said.

Spark has entered into a US$105 million (AU$135.3 m) licensing and supply agreement with Novartis, giving it the exclusive rights to commercialise Luxturna in markets outside the US. The company’s books contained US$540.2 million (AU$696 m) at the end of last year, excluding the money received from Novartis related to the agreement.


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