11 Best Gene Therapy Stocks to Buy According to Analysts

6. Ionis Pharmaceuticals, Inc. (NASDAQ:IONS)

Price Target Upside: 60.63% 

Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) specializes in RNA-targeted therapeutics for neurological, cardiovascular, and rare diseases. The company generates revenue through royalties from drugs like SPINRAZA and sales of TEGSEDI and WAYLIVRA. Its innovative approach uses modified RNA to address genetic causes of diseases, providing potential breakthroughs for conditions with limited treatments, making it one of the best genomic stocks to watch.

Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) has a strong pipeline that includes ION582 for Angelman Syndrome, which has shown improvements in communication, cognition, and motor function in Phase I/II trials, with Phase III trials planned for 2025. Other programs include Huntington’s Disease gene-lowering therapy and Myotonic Dystrophy Type 1 treatment.

The company has developed FDA-approved drugs, such as SPINRAZA for spinal muscular atrophy, TEGSEDI and WAYLIVRA for rare genetic disorders, and TRYNGOLZA for familial chylomicronemia syndrome (FCS).

Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) reported $227 million in Q4 2024 revenue, with $705 million for the year, which was driven by product launches like WAINUA and TRYNGOLZA. Despite a decrease in R&D revenue, the company maintains a strong cash position of $2.3 billion. Operating expenses totaled $337 million for Q4, which reflects ongoing commercialization efforts. Their recent developments include the FDA approval of TRYNGOLZA in December 2024 and progress on WAINUA for ATTRv-PN. Additionally, ION582 for Angelman syndrome is on track for Phase 3 in 2025.