11 Best Gene Therapy Stocks to Buy According to Analysts

2. BioMarin Pharmaceutical Inc. (NASDAQ:BMRN)

Price Target Upside: 94% 

BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) focuses on developing specialized therapies for rare genetic diseases, with a strong emphasis on enzyme replacement treatments and gene therapies. The company has introduced eight innovative medications, including treatments for lysosomal storage diseases, severe hemophilia A, and achondroplasia.

BioMarin Pharmaceutical Inc. (NASDAQ:BMRN)’s gene therapy pipeline includes Roctavian, a one-time treatment that aims to reduce or eliminate the need for regular factor VIII infusions. Despite slow commercial uptake, long-term safety and efficacy data are being collected. The company is also advancing gene therapies for phenylketonuria (PKU), with BMN-307 currently under FDA Fast Track designation, and exploring gene therapies for genetic cardiovascular diseases in collaboration with Skyline Therapeutics.

In Q4 2024, the company reported a 16% increase in revenue to $747 million, contributing to an 18% rise in annual revenue, reaching $2.85 billion. Sales of VOXZOGO for achondroplasia surged by 56% to $735 million, and enzyme treatments saw a 12% increase, totaling over $1.9 billion. This growth, along with improved operational efficiency, led to a 69% increase in earnings per share (EPS) to $3.52 for the year and an 88% increase in Q4 EPS to $0.92. Operating cash flow also rose by 260%, reaching $573 million. Looking ahead, BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) projects a 10% growth in 2025, with anticipated revenue between $3.1 billion and $3.2 billion and non-GAAP operating margins between 32% and 33%.

The corporation continues to refine its strategic approach, including narrowing the geographic scope of Roctavian and adjusting clinical trial enrollment. The company remains patient with its gene therapies, acknowledging the challenges of slow adoption and regulatory hurdles. Collaborative efforts, like its partnership with Skyline Therapeutics, are integral to expanding its gene therapy capabilities, particularly in cardiovascular diseases.