In this article, we will be taking a look at the 11 best gene therapy stocks to buy according to analysts.
Gene therapy is a medical approach that entails changing or modifying genes to treat or prevent disease. By repairing, replacing, or changing genes within a person’s cells, the therapy aims to address the underlying genetic cause of a condition. According to industry experts, this novel therapy may be used to treat cancer, certain genetic problems, and other illnesses.
According to IMARC, the US healthcare system changed to accommodate gene treatments. In 2023, 114 gene therapy trials were started, according to IQVIA’s count. Approximately 77% of these were financed by the healthcare sector. There are currently 38 cell and gene therapies that have received FDA approval, according to Kella Kapnisi, Head of Cell and Gene Therapy at Team Consulting, a medical technology design and development agency. Interestingly, a large number of these have been brought to market mostly using manual laboratory manufacturing techniques.
Growth and Trends in the US Gene Therapy Market
The size of the US gene therapy market is estimated by IMARC to be US$1,312.2 million in 2024. By 2033, the market is expected to reach US$3,697.8 million. The rising incidence of genetic illnesses, developments in genetic engineering, a strong emphasis on strategic partnerships, encouraging legal environments, and increased R&D expenditures are all anticipated to contribute to this extraordinary expansion.
The FDA has recognized more than 1,000 medications for rare diseases as orphan pharmaceuticals since the program’s inception, many of which are gene treatments. Additionally, as the regulatory body demonstrated its commitment to advancing the development of these medications in 2023, the excitement surrounding the gene therapy sector increased. The FDA has approved three new gene therapies for diseases like hemophilia and spinal muscular atrophy, according to IMARC.
According to Kella Kapnisi, the necessity for specially designed technologies is crucial because the cell and gene therapy sector is constantly changing. Real-time process control with the aid of sophisticated inline and online analytics, small batch manufacturing systems tailored for personalized therapies and decentralized manufacturing, and sophisticated data analysis and management systems utilizing machine learning technology continue to be significant trends that could propel the expansion of the manufacturing of cell and gene therapies.
According to IMARC, partnerships among biotech businesses, pharmaceutical corporations, and university research institutes can provide the necessary funding to tackle the difficulties associated with developing and promoting gene therapy. The rising incidence of chronic illnesses and genetic abnormalities, together with a rise in strategic alliances and partnerships, are significant developments in the US gene therapy business, according to IMARC. One of the main drivers of the gene therapy market is the rising incidence of genetic diseases and cancer. Because gene therapy can accurately identify and target cancerous cells to stop additional harm, it is an essential part of cancer treatment.
A scientist holding a Petri dish filled with a gene therapy sample.
Our Methodology
For this article, we first filtered gene therapy companies with a market cap of over $2 billion. From that list, we selected stocks with an analyst upside of over 20% and strong institutional ownership. We then ranked these stocks based on their analyst upside as of March 30, 2025.
Why are we interested in the stocks that hedge funds pile into? The reason is simple: our research has shown that we can outperform the market by imitating the top stock picks of the best hedge funds. Our quarterly newsletter’s strategy selects 14 small-cap and large-cap stocks every quarter and has returned 373.4% since May 2014, beating its benchmark by 218 percentage points (see more details here).
Here is our list of the 11 Best Gene Therapy Stocks to Buy According to Analysts.
11. Catalyst Pharmaceuticals, Inc. (NASDAQ:CPRX)
Price Target Upside: 32.50%
Catalyst Pharmaceuticals, Inc. (NASDAQ:CPRX) is a commercial-stage biopharmaceutical company specializing in developing and commercializing treatments for rare neurological and neuromuscular diseases. Its flagship product, Firdapse, is an FDA-approved treatment for Lambert-Eaton Myasthenic Syndrome (LEMS).
Catalyst Pharmaceuticals, Inc. (NASDAQ:CPRX) reported unprecedented financial growth during Q4 2024, with a 23.5% increase, leading to $491.7 million in total annual revenue. The fourth-quarter revenue amounted to $141.8 million, which reflected a 28.3% growth compared to the previous year. In 2024, the company generated $517.6 million in cash revenue, maintaining a debt-free financial position. During the year, the company generated $276.3 million in non-GAAP income, resulting in $2.33 per basic share, while GAAP income amounted to $163.9 million, or $1.38 per basic share. As one of the best genomic stocks, the company is well-positioned for further growth
Sales projections for Catalyst Pharmaceuticals, Inc. (NASDAQ:CPRX) suggest a potential range of $545 million to $565 million for 2025. A favorable settlement with Teva granted the company the right to prevent the entry of generic Firdapse into the market until 2035. The company is committed to developing collaborations and acquisitions within the orphan drug market while exploring potential immunosuppressive applications for Agamri in rare disease conditions.
10. Denali Therapeutics Inc. (NASDAQ:DNLI)
Price Target Upside: 37.57%
Denali Therapeutics Inc. (NASDAQ:DNLI) focuses on producing and marketing its product candidate portfolio for treating neurodegenerative diseases. The company implements gene therapy development programs for central nervous system diseases through partnerships with top organizations. Considered among the best genomic stocks, the business is working to create SIRION Biotech AAV-based gene therapy vectors specifically aimed at treating neurodegenerative diseases such as Parkinson’s, Alzheimer’s, and ALS. The company is also continuing the development of granulin replacement treatment for frontotemporal dementia and enzyme replacement drugs for lysosomal storage disorders, including Hunter syndrome.
Denali Therapeutics Inc. (NASDAQ:DNLI) has developed LRRK2 inhibitors and eIF2B activators aimed at treating Parkinson’s disease, ALS, and FTD under collaborative partnerships to accelerate their development. Tividenofusp alfa for Hunter syndrome will undergo its first BLA submission, with plans for the company’s commercial market entry. The corporation is also working with the FDA for expedited approval of its second program, DNL126, which treats Sanfilippo syndrome.
Denali Therapeutics Inc. (NASDAQ:DNLI) continues to expand its product lineup with TV-supported therapeutic products, including enzymes, oligonucleotides, and antibodies. The company intends to initiate 1-2 advanced TV programs annually over the next three years. The TV platform at the corporation provides promising opportunities for barrier-crossing therapeutics while attracting positive investor expectations for the company’s future growth.
9. Apellis Pharmaceuticals, Inc. (NASDAQ:APLS)
Price Target Upside: 45.53%
Apellis Pharmaceuticals, Inc. (NASDAQ:APLS) is a biopharmaceutical company focused on developing therapies targeting the complement system, specifically the C3 protein, to treat rare and serious diseases. The company generates revenue through its FDA-approved products, SYFOVRE for geographic atrophy (GA) and EMPAVELI for paroxysmal nocturnal hemoglobinuria (PNH).
Beam Therapeutics is collaborating with Apellis Pharmaceuticals, Inc. (NASDAQ:APLS) to develop gene-editing solutions for complement-driven diseases. Beam’s base-editing technology enables researchers to make specific one-base changes, targeting diseases linked to complement misregulation in the eyes, as well as the liver and brain. Through their joint effort, the organizations aim to create lasting treatment options for inflammatory and autoimmune illnesses caused by complement pathway overactivation.
Apellis Pharmaceuticals, Inc. (NASDAQ:APLS)’s complement C3 inhibitor, APL-9, advances as a gene therapy delivery enhancer that reduces immune response interference caused by AAV vectors. The Phase I trials of APL-9 demonstrate its potential by controlling immune activity and showing good tolerance in test subjects.
The company establishes its position as a leader in complement-focused therapies with approved therapies such as Pegcetacoplan (EMPAVELI and SYFOVRE) within its robust pipeline. Apellis Pharmaceuticals, Inc. (NASDAQ:APLS)’s growth prospects will be strengthened through its expanding development pipeline and strategic collaborations, particularly its agreement with Sobi to expand their international presence.