11 Best Performing Biotech Stocks So Far in 2025

3. CRISPR Therapeutics AG (NASDAQ:CRSP)

Year-to-Date Performance: 37.45% 

CRISPR Therapeutics AG (NASDAQ:CRSP) is a pioneering biotech company focused on gene-editing medicines using CRISPR/Cas9 technology. Its flagship therapy, Casgevy, is an ex vivo CRISPR-based treatment for sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT), with global adoption expanding rapidly. As of mid-2025, over 75 authorized treatment centers are operational, and more than 90 patients have undergone cell collection, with new initiations expected to grow throughout the year.

In 2025, CRISPR Therapeutics AG (NASDAQ:CRSP) also reported encouraging Phase 1 results for CTX310, an in vivo gene-editing candidate targeting ANGPTL3, a gene involved in regulating LDL cholesterol and triglycerides. The therapy demonstrated dose-dependent reductions of up to 86% in LDL and 82% in triglycerides with a favorable safety profile, addressing a significant unmet need in cardiovascular disease. Full Phase 1 data are anticipated in the second half of 2025, potentially validating the business’ in vivo liver editing platform. These advances have helped CRISPR Therapeutics emerge among the best performing stocks in the biotech sector.

Beyond blood disorders and cardiovascular disease, CRISPR Therapeutics AG (NASDAQ:CRSP) is advancing its oncology and autoimmune pipeline. Two next-generation CAR-T candidates, CTX112, targeting CD19, and CTX131, targeting CD70, are progressing through clinical development, with updates expected later this year. These programs reflect the business’s broader ambition to extend gene-editing therapies to cancer and immune-mediated conditions.