Foundation Fighting Blindness and ProQR enter into a partnership to develop QR-421a for Usher syndrome type 2A, targeting mutations in exon 13 of the causative USH2A gene.
… ProQR Therapeutics Teams Up with the Foundation Fighting Blindness and Blueprint Genetics to Support the My Retina … today its participation in the Foundation Fighting Blindness My Retina Tracker Program, a collaborative, open … Chief Scientific Officer of the Foundation Fighting Blindness. “As part of the program, the My Retina Tracker …
2017 may become a very exciting year for Patricia Biasutto, sepofarsen captain. Her work, developing a novel RNA therapy for Leber congenital amaurosis 10, has progressed steadily. The team expects to start a first clinical trial this year.
ARVO 2020 clinical trial results presentation by Stephen Russel et al. Sepofarsen had a manageable safety profile and showed improvement in mean visual acuity, and full-field stimulus testing.
Top-line results expected H1 2022
Sepofarsen is a potential first-in-class RNA therapy for the treatment of LCA10, a rare inherited retinal disorder that leads to blindness
LEIDEN, Netherlands & CAMBRIDGE, Mass., Jan.