ONCE Onconetix Inc

Spark Therapeutics (NASDAQ:ONCE), a fully integrated, commercial gene therapy company dedicated to challenging the inevitability of genetic disease, today announced that the Galien Foundation has awarded the prestigious 2019 Prix Galien USA Award for Best Biotechnology Product to LUXTURNA® (voretigene neparvovec-rzyl). The award, announced at the Prix Galien USA Awards Ceremony on Oct. 24, 2019 at the American Museum of Natural History in New York City, recognizes the development of the world’s most innovative medicines that help improve the overall human condition.

“We are honored that LUXTURNA, the first gene therapy for an inherited disease in the U.S., was awarded the prestigious 2019 Prix Galien USA Award for Best Biotechnology Product,” said Katherine A. High, M.D., president and head of research & development at Spark Therapeutics. “We hope the historic approval of LUXTURNA continues to serve as a catalyst for the field of gene therapy, improving our collective capabilities so that we may together, someday, conquer the realm of inherited disease. This recognition would not have been possible without the many contributions of the people who work at Spark Therapeutics, as well as the courage and commitment of patients and their families, and expertise and insights of the investigators, who continue to participate in the clinical development program.”

LUXTURNA is first gene therapy for a genetic disease approved in both the U.S. and EU. On Dec. 19, 2017, the U.S. Food and Drug Administration (FDA) approved LUXTURNA, a one-time gene therapy indicated for the treatment of patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy and viable retinal cells. On Nov. 23, 2018, the European Commission granted marketing authorization for LUXTURNA. Novartis has exclusive rights to pursue development, registration and commercialization rights for LUXTURNA in all countries outside the U.S. Spark Therapeutics previously entered into a separate agreement with Novartis to manufacture and supply LUXTURNA to Novartis.

For more information about LUXTURNA and prescribing information in the U.S. please visit www.LUXTURNA.com.

About Spark Therapeutics At Spark Therapeutics, a fully integrated, commercial company committed to discovering, developing and delivering gene therapies, we challenge the inevitability of genetic diseases, including blindness, hemophilia, lysosomal storage disorders and neurodegenerative diseases. We currently have four programs in clinical trials. At Spark, we see the path to a world where no life is limited by genetic disease. For more information, visit www.sparktx.com, and follow us on Twitter and LinkedIn.

Investor Relations Contact:	Media Contact:
Ryan Asay	Monique da Silva
Ryan.asay@sparktx.com	communications@sparktx.com
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