VIDEO: RNAi for LCA10 — a project defeated by regulatory challenges

ByBart Leroy, MD, PhD

ByMichela Cimberle

Fact checked byChristine Klimanskis, ELS

ROME — In this Healio Video Perspective from the FLORetina-ICOOR meeting, Bart Leroy, MD, PhD, speaks about RNA interference technology for the treatment of Leber congenital amaurosis type 10.

LCA10 is the worst type of Leber congenital amaurosis, with the lowest level of best corrected visual acuity. Leroy explains the genetic basis of this disease, the rationale for antisense oligonucleotide therapy, and the problems encountered with EMA and FDA when trying to design a phase 3 study. The project was eventually abandoned.

“Unfortunately, that does show you that you can actually end up having a good product that ends up in the bin, and that is something I would certainly like to not have happen to [inherited retinal disease] patients in the future,” he said.

Published by:

Sources/Disclosures

Collapse

Source:

Leroy B. Regulatory challenges for genetic therapies for IRDs. Presented at: FLORetina-ICOOR; Nov. 30-Dec. 3, 2023; Rome.

Disclosures: Leroy reports having received travel grant and research support from ProQR Therapeutics.

FLORetina-ICOOR meeting

Read more

VIDEO: RNAi for LCA10 — a project defeated by regulatory challenges

Related Content