11 Best Gene Therapy Stocks to Buy According to Analysts

4. Avidity Biosciences, Inc. (NASDAQ:RNA)

Price Target Upside: 68.25% 

Avidity Biosciences, Inc. (NASDAQ:RNA) is developing a new class of RNA-based therapies called Antibody Oligonucleotide Conjugates (AOCs). These therapies aim to treat rare muscle disorders like myotonic dystrophy type 1 (DM1), Duchenne muscular dystrophy (DMD), and facioscapulohumeral muscular dystrophy (FSHD). The company is preparing for a significant expansion in 2025, including filing its first Biologics License Application (BLA), broadening its pipeline, and accelerating preparations for potential product launches.

Avidity Biosciences, Inc. (NASDAQ:RNA)’s AOC platform allows for the targeted delivery of RNA therapeutics to specific tissues, such as muscle cells, overcoming traditional delivery limitations. This breakthrough technology enables efficient gene modification or silencing, marking significant progress in treating neuromuscular diseases. The business’s pipeline includes AOC 1001 for DM1, AOC 1020 for FSHD, and AOC 1044 for DMD, each showing promising clinical results.

Avidity Biosciences, Inc. (NASDAQ:RNA) also aims to expand its pipeline into precision cardiology and immunology, with partnerships, such as one with Eli Lilly, to apply AOCs to these areas. The company has achieved several regulatory milestones, including FDA orphan drug designations, which support rapid progression toward commercialization. With a strong cash position of $1.6 billion, the corporation is well-positioned for growth and global expansion.