Avidity Biosciences Receives Investment from CureDuchenne
Investment advances pre-clinical development of novel exon skipping therapies for treatment of patients with Duchenne muscular dystrophy
LA JOLLA, Calif.–(BUSINESS WIRE)–Avidity Biosciences, a privately held biotech company pioneering a new class of precision medicines based upon antibody-oligonucleotide conjugates, announced today that CureDuchenne has made an equity investment in the company. Funding will help to advance pre-clinical development of potential therapies to treat patients with Duchenne muscular dystrophy (DMD).
“We are grateful for an investment from CureDuchenne Ventures to advance our antibody-oligonucleotide technology for treatment of patients with Duchenne muscular dystrophy,” said Arthur A. Levin, Ph.D., EVP of research and development, Avidity Biosciences. “Cure Duchenne has a history of supporting groundbreaking research in Duchenne, and we look forward to working with them, as well as their community of patients, parents, scientists and scientific experts to advance our pipeline of new therapies for boys with this serious disease.”
The AOC™ technology being advanced by Avidity addresses a key issue of oligonucleotide therapeutics for Duchenne: namely, delivery of these powerful therapeutic agents for exon skipping to muscle, diaphragm and heart. Using antibody-mediated uptake in muscle cells, AOCs increase the activity of splice skipping oligonucleotides dramatically (a 100-fold increase in potency in the MDX mouse model), which should allow for reduced dose levels and reduced dosing frequency. This ground-breaking science provides a great opportunity to address Duchenne muscular dystrophy, and other muscle diseases.
“CureDuchenne is excited about the future of Avidity’s science and committed to supporting exon skipping development to treat the entire Duchenne population,“ said Debra Miller, founder and CEO, CureDuchenne. “Currently only 13% of Duchenne patients are served with an approved treatment for exon 51. Avidity’s programs have the potential to enable effective therapies to treat a broad set of Duchenne patients. We hope it won’t be long before every Duchenne patient has a viable therapy.”
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