Tag Archives: The Stem Cell Blog

Spark grabs FDA nod for Luxturna, a breakthrough gene therapy likely bearing a pioneering price – FiercePharma

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2017-12-19 NEWS

Spark grabs FDA nod for Luxturna, a breakthrough gene therapy likely bearing a pioneering price
FiercePharma
Luxturna is now Spark’s only approved med and works by delivering a gene called RPE65 into a patient’s retinal cells, which then produce a protein to restore vision loss. The one-time treatment uses a naturally occurring adeno-associated virus to

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2017-12-19 NEWS

FDA approves Spark’s gene therapy for rare blindness pioneered at CHOP
Philly.com
The treatment, developed by Philadelphia drug maker Spark Therapeutics and researchers at Children’s Hospital of Philadelphia, and represents the first gene therapy for a genetic disease in the United States. “Today’s approval marks another first in

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2017-12-19 NEWS

FDA Approves Gene Therapy for Rare Vision Loss Disease
Specialty Pharmacy Times
“Today’s approval marks another first in the field of gene therapy—both in how the therapy works and in expanding the use of gene therapy beyond the treatment of cancer to the treatment of vision loss—and this milestone reinforces the potential of