Avidity Biosciences Receives Investment

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."

Avidity will present its Duchenne specific research during a webinar hosted by CureDuchenne on November 16, 2018 at 1:00 PT/4:00 ET. To register for the webinar, please click here: https://bit.ly/2OryjSI.

About Avidity Biosciences

Avidity Biosciences is a privately held biotech company pioneering Antibody Oligonucleotide Conjugates (AOC), which combine the tissue selectivity of monoclonal antibodies and the precision of oligonucleotide-based therapeutics to overcome barriers to the delivery of oligonucleotides and target genetic drivers of disease. Avidity is advancing a pipeline of therapeutic programs focused on rare muscle disorders and other serious diseases. Avidity has raised $30 million in venture financing from a top-tier group of sophisticated healthcare investors. More information about Avidity can be found on the company's website at www.aviditybio.com.

About CureDuchenne

CureDuchenne is the nation's leading nonprofit organization dedicated to finding a cure for Duchenne, the most common and lethal form of muscular dystrophy. As the leading genetic killer of young boys, Duchenne affects more than 300,000 boys living today. CureDuchenne has garnered international attention for its efforts to raise funds and awareness for Duchenne through venture philanthropy. For more information on how to help raise awareness and funds needed for research, please visit www.cureduchenne.org.