11 Best Gene Therapy Stocks to Buy According to Analysts

5. PTC Therapeutics, Inc. (NASDAQ:PTCT)

Price Target Upside: 64% 

PTC Therapeutics, Inc. (NASDAQ:PTCT) is a biopharmaceutical company focused on developing treatments for rare and serious diseases, specializing in small molecule drugs and gene therapies. Its notable products include treatments for Duchenne muscular dystrophy, spinal muscular atrophy, and AADC deficiency. The company is unique for its groundbreaking approval of Kebilidi, which is the first brain-delivered gene therapy for AADC deficiency. The business also stands out with innovative drug delivery methods and a robust pipeline targeting rare diseases like Huntington’s disease and mitochondrial disorders. Through strategic partnerships with companies like Roche and Novartis and a strong global commercial infrastructure, the corporation efficiently develops and distributes therapies worldwide, earning its spot as the fifth stock on our list among the best genomic stocks.

PTC Therapeutics, Inc. (NASDAQ:PTCT) reported strong financial results for Q4 2024, with revenue of $213 million and full-year revenue of $807 million, surpassing guidance. The growth was primarily driven by the success of its Duchenne muscular dystrophy (DMD) franchise, which generated $547 million in revenue for 2024, and royalties from Evrysdi, contributing $204 million. The company ended the year with over $1.1 billion in cash, bolstered by an additional $1 billion from its Novartis PTC518 transaction in January 2025, providing significant financial flexibility.

For 2025, the company provided revenue guidance of $600 million to $800 million, reflecting uncertainty due to regulatory decisions. The corporation also projected non-GAAP R&D and SG&A expenses of $730 million to $760 million. PTC Therapeutics, Inc. (NASDAQ:PTCT)’s solid cash position and diverse pipeline of rare disease therapies, including the anticipated launches of sepiapterin for PKU and vatiquinone for Friedreich’s ataxia, position the company for future growth. Strategic collaborations, particularly with Novartis for the Huntington’s disease program, further strengthen its outlook.

The company’s key developments include the successful execution of clinical and regulatory milestones in 2024 and the ongoing collaboration with Novartis, which included a $1 billion upfront payment. These achievements reinforce the business’s potential for continued commercial success.