Positive preliminary data from InSight extension study of sepofarsen for LCA10 reported – consistent with benefit seen in Phase 1/2 and building confidence in Phase 2/3 Illuminate trial;
Strategic convertible debt financing extends runway into 2023 if fully draw
Phase 2/3 pivotal Illuminate trial of sepofarsen for LCA10 enrollment complete with top-line data anticipated in H1 2022; Brighten pediatric trial of sepofarsen to begin in 2021; additional data from Phase 1/2 InSight extension study to be reported in
… and presented positive pre-clinical data at two European scientific conferences. WINGS, a Phase 1/2 safety and … Appointed several thought leaders to ScientificAdvisoryBoard (Drs. Phil Zamore, Cy Stein, Scott Armstrong and …
… Maraganore, PhD, as Strategic Advisor to the Supervisory Board LEIDEN, Netherlands & CAMBRIDGE, Mass., March 07, 2022 … alliances. He continues to serve on the Alnylam ScientificAdvisoryBoard. Prior to Alnylam, he served as an officer and …
Axiomer™ ADAR-mediated RNA editing pipeline advancing across liver and CNS programs, with CTA filing on track for Q2 2025 for lead program AX-0810 targeting NTCP for Cholestatic diseases Company expects up to four clinical data readouts in 2025 and 2026 across multi