9 Best Small Cap Stocks with Biggest Upside Potential

7. Dyne Therapeutics Inc. (NASDAQ:DYN)

Market Capitalization as of August 15: $1.76 billion

Number of Hedge Fund Holders: 46

Average Upside Potential as of August 15: 91.16%

Dyne Therapeutics Inc. (NASDAQ:DYN) is one of the best small cap stocks with biggest upside potential. On August 4, Dyne Therapeutics announced that its investigational therapy, called DYNE-251, received Breakthrough Therapy Designation from the US FDA. This designation is for treating Duchenne muscular dystrophy/DMD patients whose condition is amenable to exon 51 skipping.

This marked the second such designation for the company, as its other lead program, called DYNE-101, also received the designation earlier in 2025 for myotonic dystrophy type 1/DM1. This designation is based on data from the ongoing DELIVER clinical trial. DYNE-251 has shown sustained functional improvements over 18 months, measured by key metrics like time to rise and stride velocity.

The therapy also demonstrated the production of near-full-length dystrophin protein. The FDA grants this designation to expedite the development and review of drugs for serious conditions that show preliminary evidence of a substantial improvement over existing treatments. The benefits for DYNE-251 include enhanced FDA support, early and frequent communication with reviewers, and eligibility for rolling and Priority Review, which could reduce the Biologics License Application/BLA review period from 12 to 8 months.

Dyne Therapeutics Inc. (NASDAQ:DYN) is a clinical-stage neuromuscular disease company that focuses on discovering and developing therapeutics for neuromuscular diseases in the US.