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.
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.
7. Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM)
Price Target Upside: 58.55%
Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM) focuses on developing therapies for rare liver diseases caused by bile acid buildup, including progressive familial intrahepatic cholestasis (PFIC) and Alagille syndrome (ALGS). Its flagship product, Livmarli (maralixibat), is approved for treating cholestatic pruritus in pediatric ALGS patients, contributing to the company’s revenue.
Vivet Therapeutics collaborates with the company to develop AAV gene therapies VTX-803 and VTX-802, which treat PFIC subtypes 3 and 2. These therapeutic approaches aim to restore defective MDR3 transporter and bile salt export pump (BSEP) functions, allowing bile secretion to be restored and preventing serious liver damage. Tests on VTX-803 showed significant improvements in PFIC3 biomarker levels, and the drug has earned Orphan Drug Designation from both the FDA and EMA.
Total net product sales for Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM) reached $99.4 million during Q4 2024, which reflects a 43% increase from the previous year. Sales of Livmarli increased by 50% during the year, with the product generating $64.1 million in the fourth quarter and $213.3 million in annual sales. During the period, the company spent $140.6 million on R&D expenses and $202.2 million on SG&A expenses. The corporation’s cash position stood at $293 million, with product sales projected to be between $420 million and $435 million for 2025.
The FDA approved the drug CTEXLI (chenodeoxycholic acid) for the treatment of cerebrotendinous xanthomatosis (CTX), providing the drug with seven years of market protection. Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM) also plans to begin a Phase 2 trial of MRM-3379 to treat Fragile X syndrome, thereby increasing potential expansion opportunities.
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.
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.
4. Avidity Biosciences, Inc. (NASDAQ:RNA)
Price Target Upside: 68.25%
Avidity Biosciences, Inc. (NASDAQ:RNA) is developing a new class of RNA-based therapies called Antibody Oligonucleotide Conjugates (AOCs). These therapies aim to treat rare muscle disorders like myotonic dystrophy type 1 (DM1), Duchenne muscular dystrophy (DMD), and facioscapulohumeral muscular dystrophy (FSHD). The company is preparing for a significant expansion in 2025, including filing its first Biologics License Application (BLA), broadening its pipeline, and accelerating preparations for potential product launches.
Avidity Biosciences, Inc. (NASDAQ:RNA)’s AOC platform allows for the targeted delivery of RNA therapeutics to specific tissues, such as muscle cells, overcoming traditional delivery limitations. This breakthrough technology enables efficient gene modification or silencing, marking significant progress in treating neuromuscular diseases. The business’s pipeline includes AOC 1001 for DM1, AOC 1020 for FSHD, and AOC 1044 for DMD, each showing promising clinical results.
Avidity Biosciences, Inc. (NASDAQ:RNA) also aims to expand its pipeline into precision cardiology and immunology, with partnerships, such as one with Eli Lilly, to apply AOCs to these areas. The company has achieved several regulatory milestones, including FDA orphan drug designations, which support rapid progression toward commercialization. With a strong cash position of $1.6 billion, the corporation is well-positioned for growth and global expansion.
3. Bio-Techne Corporation (NASDAQ:TECH)
Price Target Upside: 81.25%
Bio-Techne Corporation (NASDAQ:TECH), headquartered in Minneapolis, Minnesota, is a leading life sciences company that develops, manufactures, and sells biotechnology products and scientific tools for research, diagnostics, and bioprocessing. The company offers over 500,000 products, including antibodies, proteins, analytical instruments, and molecular diagnostics tools.
Bio-Techne Corporation (NASDAQ:TECH) plays a significant role in gene therapy by providing essential products such as GMP-grade recombinant proteins, specialized cell culture media, and reagents optimized for gene editing tools like CRISPR-Cas9. Their offerings also include analytical tools for characterizing gene therapy products and support for AAV vector production, although this is not a primary focus. The company targets research institutions, biotech companies, and pharmaceutical firms involved in gene therapy, making it one of the most promising companies among the best genomic stocks in the biotech sector.
In its Q4 2024 financial results, Bio-Techne Corporation (NASDAQ:TECH) reported a 2% increase in net sales to $306.1 million, with organic revenue growing 1%. However, GAAP EPS decreased to $0.25 per diluted share, down from $0.47 the previous year, while adjusted EPS also declined. The company’s Protein Sciences segment saw a 4% decrease in sales, while the Diagnostics and Genomics segment grew by 15%. Cash flow from operations increased by 18%, reaching $299.0 million, reflecting strong operational cash generation.
The corporation’s acquisition of Lunaphore, a spatial biology firm, is a strategic move to enhance its position in this rapidly growing field. However, the acquisition and other factors, like unfavorable volume leverage in Protein Sciences, contributed to a decline in profitability, with operating margins for both GAAP and adjusted results decreasing.
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.
1. Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE)
Price Target Upside: 95.96%
Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) tops our list for being one of the best genomic stocks. It focuses on developing and commercializing treatments for genetic diseases. Its product portfolio includes Crysvita, Mepsevii, Dojolvi, and Evkeeza, with plans to expand in 2025 through several products in launch mode worldwide. The company exceeded expectations in 2024, reporting $560 million in total revenue and forecasting $640 million to $670 million in 2025. Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) is targeting full-year GAAP profitability by 2027, and analysts remain bullish, with Buy ratings and price targets ranging from $104.00 to $115.00, implying significant upside potential.
The company employs in vivo gene replacement therapy using adeno-associated viral (AAV) vectors to deliver therapeutic genes into specific cells. This one-time treatment aims to target the underlying cause of genetic diseases, potentially halting or slowing progression. The corporation’s gene therapy pipeline includes promising candidates such as UX111 (for Mucopolysaccharidosis Type IIIA), DTX301 (for Ornithine Transcarbamylase deficiency), DTX401 (for Glycogen Storage Disease Type Ia), and a gene therapy for Wilson disease.
Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) has developed the Pinnacle PCL Platform, which is a proprietary manufacturing system designed to enhance scalability and reduce production costs. This platform uses engineered Producer Cell Lines (PCLs) to produce high yields of AAV vectors, ensuring safety and quality while enabling faster therapy delivery. The company is also advancing regulatory progress, which includes the submission of a Biologics License Application (BLA) for UX111, with a surrogate endpoint supporting long-term cognitive benefits.
Overall, Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) ranks first among the 11 best gene therapy stocks to buy according to analysts. While we acknowledge the potential of gene therapy companies, our conviction lies in the belief that AI stocks hold greater promise for delivering higher returns, and doing so within a shorter time frame. If you are looking for an AI stock that is more promising than RARE but that trades at less than 5 times its earnings, check out our report about the cheapest AI stock.
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