N=1 Collaborative Seminar – Project Butterfly: from drug development to clinical trial. A personalized antisense oligonucleotide medicine for a rare form of CLN3 Batten Disease
VirtualThe presentation will review our development of a personalized antisense oligonucleotide (ASO) medicine for two children with CLN3 Batten disease. We will detail the collaborative process of designing the ASO therapy, achieving FDA IND approval, and establishing a clinical trial protocol.