11 Best Gene Therapy Stocks to Buy According to Analysts

8. Xenon Pharmaceuticals Inc. (NASDAQ:XENE)

Price Target Upside: 57% 

Xenon Pharmaceuticals Inc. (NASDAQ:XENE) stands eighth among the best genomic stocks. It is a biopharmaceutical company focused on developing treatments for neurological and psychiatric disorders, including epilepsy and major depressive disorder (MDD). The company addresses high, unmet medical needs in the field of neuroscience.

Xenon Pharmaceuticals Inc. (NASDAQ:XENE) targets genetically proven ion channel genes to create specific drugs for epilepsy and major depressive disorder (MDD). The company’s extensive genetic knowledge of ion channels positions it to potentially develop advanced gene therapy solutions, despite currently focusing on small molecule drug candidates.

Azetukalner serves as the company’s leading drug candidate and is undergoing Phase 3 trials targeting focal onset seizures (FOS), primary generalized tonic-clonic seizures (PGTCS), and major depressive disorder (MDD). The company plans to explore bipolar depression (BPD) research in the future. Xenon Pharmaceuticals Inc. (NASDAQ:XENE) continues to develop both Kv7 and Nav1.7 modulators, with IND submissions expected in 2025. The business is also collaborating with Neurocrine Biosciences to develop epilepsy medication based on Nav1.2/Nav1.6 sodium channel inhibition.

As of Q4 2024, the corporation holds $754.4 million in cash, suggesting its operations will be funded through 2027. Research and development expenses during Q4 totaled $59.5 million, primarily supporting the Azetukalner clinical development program and Phase 3 trials. General and administrative costs increased to $18 million during the fourth quarter. The fourth-quarter net loss amounted to $65.7 million, but Xenon Pharmaceuticals Inc. (NASDAQ:XENE) demonstrated growth through the advancement of Azetukalner in Phase 3 trials and ongoing Phase 3 research in bipolar depression.