The biotechnology sector is where science, innovation, and investing intersect. Driven by groundbreaking medical research and life-changing therapies, biotech companies are shaping the future of healthcare. While the sector is known for its volatility and high-risk nature, it also offers significant upside for those who can identify companies with strong pipelines, strategic partnerships, and successful product launches.

As the global biotech market expands—projected to exceed $1 trillion by 2030—investors are paying close attention. From gene editing to rare disease treatment, a select group of biotech companies stands out for their contributions to medicine and their potential for delivering shareholder value. Below, this post explores some of the best biotech stocks to watch and why they’re generating buzz in the investing world.

Best Biotech Stocks to Watch

Here are some of the most compelling biotech companies currently making waves and worth watching for their growth potential.

1. Catalyst Pharmaceuticals (CPRX)

Catalyst Pharmaceuticals develops therapies for rare and ultra-rare diseases. Its key product, amifampridine, is approved for treating Lambert-Eaton myasthenic syndrome (LEMS), a condition with limited treatment options.

The company’s focus on niche markets with high unmet medical needs offers both societal value and commercial potential. With continued product development and potential expansion into related disorders, Catalyst is gaining attention as a focused and promising biotech stock.

2. Axsome Therapeutics (AXSM)

Axsome Therapeutics focuses on treatments for central nervous system (CNS) disorders, including depression, Alzheimer’s-related agitation, and migraines. Its drug, Auvelity, has been approved for treating major depressive disorder, making it the first oral NMDA receptor antagonist in this category.

Revenue projections for Axsome suggest robust growth, and analysts believe its innovative pipeline could position it well against industry peers. Despite some uncertainty around price targets, the company’s market position in mental health makes it one to watch in the coming years.

3. Biogen Inc. (BIIB)

Biogen is a long-established player in neuroscience, known for pioneering therapies for multiple sclerosis, spinal muscular atrophy, and, most recently, Alzheimer’s disease. Its FDA-approved drug, Aduhelm, has faced reimbursement hurdles, but it remains a significant step forward in Alzheimer’s treatment.

Biogen’s pipeline includes treatments for lupus, ALS, and other autoimmune and neurodegenerative disorders. With multiple assets in development and a track record of delivering neurological therapies, Biogen remains a heavyweight in the biotech sector.

4. CRISPR Therapeutics (CRSP)

CRISPR Therapeutics is at the forefront of gene-editing technology. Using CRISPR-Cas9, the company is developing treatments for genetic disorders, cancers, and blood diseases. Its gene-editing tools allow precise modification of DNA, offering new hope for previously untreatable conditions.

Though still early in commercializing its technology, CRISPR has formed strategic alliances with companies like Vertex Pharmaceuticals and Bayer. Its collaborative approach and innovative platform put it on the radar for investors looking to bet on the future of genomic medicine.

5. Genmab (GMAB)

Headquartered in Denmark, Genmab specializes in antibody-based cancer treatments. The company has formed partnerships with pharmaceutical giants like Johnson & Johnson and AbbVie, helping expand the reach and development of its therapies.

Darzalex, one of its leading products, has shown strong sales growth. Continued collaboration and a robust pipeline support Genmab’s growth narrative, positioning it as a reliable player in oncology-focused biotech investing.

6. Neurocrine Biosciences (NBIX)

Neurocrine Biosciences develops therapies for neurological and endocrine disorders. With several FDA-approved treatments, including Ingrezza for tardive dyskinesia, the company is steadily expanding its market presence.

Additional products in development target conditions such as Parkinson’s disease, uterine fibroids, and schizophrenia. Consistent earnings growth and a balanced portfolio of commercial products and pipeline candidates make Neurocrine an attractive long-term prospect.

7. Vertex Pharmaceuticals (VRTX)

Vertex Pharmaceuticals is best known for its dominance in cystic fibrosis treatment. With several CF therapies already on the market, Vertex continues to innovate in other areas, including kidney disease, diabetes, and muscular dystrophy. Its collaboration with CRISPR Therapeutics to develop gene-editing-based therapies for sickle cell disease and beta thalassemia highlights its forward-looking approach.

8. Regeneron Pharmaceuticals (REGN)

Regeneron Pharmaceuticals has built a strong reputation for its innovative pipeline and successful commercial products. Known for drugs like Eylea (used for age-related macular degeneration) and Dupixent (used for eczema, asthma, and nasal polyps), Regeneron consistently delivers strong financial performance and clinical advancements.

In addition to its marketed therapies, the company is actively expanding into oncology and genetic medicine through its collaborations with Alnylam and Intellia Therapeutics. With a solid foundation in immunology and ophthalmology and a forward-looking R&D pipeline, Regeneron continues to be one of the best biotech stocks to watch in 2025 and beyond.

9. Moderna Inc. (MRNA)

Moderna rose to prominence, but the company is far more than a pandemic play. It leverages messenger RNA (mRNA) technology to develop a wide range of vaccines and therapeutics, including candidates targeting flu, RSV, cancer, and rare diseases.

Moderna's deep pipeline and continuous investment in mRNA research suggest long-term relevance beyond COVID-19. With partnerships across biotech and government institutions and a focus on scalability and innovation, Moderna is transitioning from a one-hit wonder to a biotech mainstay with strong upside potential.

How to Choose the Right Biotech Stocks?

When evaluating biotech companies, investors should look beyond the hype and focus on the fundamentals:

• Approved Products: Companies with FDA-approved drugs are less speculative and more stable.
• Late-Stage Pipeline: A well-developed clinical pipeline can indicate future revenue streams.
• Strategic Partnerships: Collaborations with established pharmaceutical firms can help accelerate development and distribution.
• Financial Health: A strong cash position and consistent earnings or funding reduce risk during trial delays.

Diversifying across multiple biotech stocks or using biotech-focused ETFs can also help mitigate sector-specific volatility.

Conclusion

The biotech sector continues to lead innovations that redefine medicine and improve lives. Companies like Vertex, CRISPR Therapeutics, and Biogen are not only developing transformative therapies but are also reshaping how healthcare is delivered and understood.

For investors seeking exposure to this dynamic space, identifying firms with approved products, strategic partnerships, and promising pipelines is key. While the sector carries inherent risks, its potential for long-term growth and societal impact makes biotech investing an exciting opportunity for forward-thinking portfolios.