RNA therapy specialists Avidity Biosciences to be acquired by Novartis for USD 12 billion

Avidity Biosciences is developing the next generation of RNA therapeutics: antibody oligonucleotide conjugates (AOCs). Antibodies are a widely used therapeutic modality that are excellent at identifying cells with specific surface markers, but cannot directly interfere with cellular processes. Oligonucleotides, on the other hand, are effective at influencing how DNA is converted into proteins by interacting with messenger RNAs. This enables them to correct mistakes that cause disease. However, delivering oligos to specific tissues has been a challenge for this technology. Avidity has successfully addressed this issue by combining oligonucleotides with antibodies to ensure targeted RNA delivery to muscle tissue, yielding improved therapeutic outcomes for various diseases.

Avidity has three programs in the pivotal phase, all in rare genetic muscle disorders.

• Their most advanced programme, Del-Zota, addresses Duchenne muscular dystrophy (DMD). Due to an error in the dystrophin protein, the muscles weaken and become more easily damaged, leaving patients (usually young boys) wheelchair-bound from their teens. Avidity’s drug has been proven to deliver to the muscle and has led to clinically meaningful increases in dystrophin levels, resulting in functional improvements as measured by endpoints such as stair climbing and time to rise. The company plans to apply for FDA approval in the first quarter of 2026.
• Avidity’s second programme, del-desiran, addresses myotonic dystrophy type 1 (DM1), which is another rare muscle disease. The company demonstrated improvements in MBNL levels (an important biomarker sequestered by faulty mRNA in this disease, which is needed for the normal production of multiple muscle proteins). This led to further encouraging functional improvements. The company expects to receive phase 3 data from this programme in the second quarter of next year, after which it will file for drug approval.
• Similarly, Avidity expects to see pivotal results from its facoscapulohumeral muscular dystrophy (FSHD) programme next year, having demonstrated similar improvements in biomarkers and functional outcomes (mobility and strength) in this muscle disease.

Through this acquisition, Novartis has obtained a comprehensive platform, positioning itself at the forefront of RNA innovation. Avidity is a good fit with the company’s neuromuscular disease strategy, in which Novartis already has a presence through the gene therapy Zolgensma, and has experience of developing and marketing RNA therapies with its cardiovascular medicine Leqvio. Interestingly, Avidity’s preclinical cardiology pipeline will not be part of the deal; these assets will instead be spun out as a separate company.

Overall, this deal will also benefit the entire biotech sector, further improving sentiment and strengthening the upward trend in biotech acquisitions this year.