Repeal of the Orphan Drug Tax Credit Would Stifle Promising Medical Research That Could be Life-Changing for 30 Million Americans, says FFB

The Foundation Fighting Blindness (FFB), the world’s leading private funder of research on treatments and cures for vision-robbing inherited retinal diseases, strongly opposes recent U.S. Senate and House of Representatives actions which would slow or stop research on treatments for Americans impacted by rare diseases.

Stem-Cell Therapy Clinics Remain Inadequately Regulated, Pose Risk to Patients

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 Doses First Participant in Its LCA10 Therapy Clinical Trial

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.

MeiraGTx Treats First Patient in XLRP Gene-Therapy Trial

MeiraGTx, a gene-therapy company in London and New York City, has treated its first patient in a gene-therapy clinical trial for people with X-linked retinitis pigmentosa (XLRP) caused by mutations in the gene RPGR. The Phase I/II study is taking place at Moorfields Eye Hospital in London. The safety-oriented trial will enroll 36 participants. Three dose levels of the therapy will be evaluated.

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