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Jack Morris is in the 9th grade at MICDS - Mary Institute and St. Louis Country Day School. Despite being more challenging, he really enjoys high school thus far. Jack has been excelling in his school’s wrestling program since seventh grade.
Spark Therapeutics’ vision-restoring RPE65 gene therapy has received marketing approval from the U.S. Food and Drug Administration, becoming the first gene therapy to gain regulatory approval in the U.S. for the eye or any inherited condition.
Though the U.S. Food & Drug Administration (FDA) is more tightly regulating U.S. clinics that tout stem-cell trials and treatments for inherited retinal diseases (IRDs) and a host of other conditions, many significant loopholes and enforcement limitations remain.
ProQR, a biotech company in the Netherlands, has treated its first patient in a Phase 1/2 clinical trial for QR-110, a therapy for people with Leber congenital amaurosis 10 (LCA10), which is caused by the p.Cys998X mutation in the CEP290 gene. The mutation is estimated to affect about 2,000 people in the Western world.