We applaud the news from our friends at ForeBatten Foundation that the FDA has provided IND clearance for the clinical investigation for Zebronkysen (FBF-001) under the N-of-1 guidance for individualized Antisense Oligonucleotide (ASO) drug products. Two patients with the unique CLN3 c.569dupG mutation began treatment in June 2024.
To read more about this significant milestone, please click here.
To learn more about ASOs, check out this 2-minute clip from Harvard Medical School.
