Tag Archive for: DMD gene therapy. AAV

In a major bet on RNA medicine, Swiss Novartis AG announced to buy Avidity Biosciences Inc for US$12bn. Avidity’s Oligonucleotide Conjugate (AOC) platform allows Transferin receptor-targeted RNA delivery to muscle. With the acquisition Novartis secures access to three late-stage RNA drug candidates for muscular dystrophies.

Initial clinical data indicate that the recombinant cysteine protease imlifidase, developed by Hansa Biopharma AB, may significantly reduce levels of anti-AAV antibodies, potentially enabling adeno-associated virus (AAV)-based gene therapies in patients previously deemed ineligible due to pre-existing immunity.