RARE Daily

Avidity Advances RNA Programs into New Therapeutic Areas

February 27, 2023

Rare Daily Staff

Avidity Biosciences said it has advanced new experimental therapies in its pipeline extending its platform technology of antibody oligonucleotide conjugates into skeletal muscle and cardiology indications.

The preclinical programs have been engineered using Avidity’s proprietary AOC platform technology, which is designed to combine the specificity of monoclonal antibodies with the precision of oligonucleotide therapies to target the root cause of diseases previously untreatable with RNA therapeutics. The expansion of the pipeline reflects the success in demonstrating targeted delivery of RNA into muscle.

“We are now pursuing new programs in skeletal muscle and cardiology and believe that there are many more opportunities where AOCs can address debilitating diseases previously unreachable with existing RNA therapies,” said Sarah Boyce, president and CEO of Avidity.

Avidity continues to advance its three distinct rare disease phase 1/2 programs in the clinic: AOC 1001 for myotonic dystrophy type 1 (DM1), AOC 1020 for the treatment of facioscapulohumeral muscular dystrophy (FSHD) and AOC 1044 for the treatment of Duchenne muscular dystrophy (DMD) mutations amenable to exon 44 skipping (DMD44). Avidity has decided not to pursue research and development programs in muscle atrophy and Pompe disease at this time.

In addition to its own internal research programs, Avidity said it continues to explore the full potential of its AOC platform through collaborations and partnerships. Avidity is working with Eli Lilly on a collaboration focused on new medicines in immunology and other select indications outside of muscle. Avidity also has a research collaboration with MyoKardia, a wholly owned subsidiary of Bristol Myers Squibb, to demonstrate the potential utility of AOCs in cardiac tissue.

Photo: Sarah Boyce, president and CEO of Avidity

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