In this article, we will look at the Top 10 Gene Therapy Stocks to Buy According to Hedge Funds.
On February 23, Lauren Goodwin, New York Life Investments, appeared on CNBC’s ‘Closing Bell’ to talk about the state of equity markets and tariff uncertainties. Talking about the situation around tariffs, tech, and software, she stated that we are finally seeing a moment of the market looking at the wall of worry and feeling some indigestion. This makes sense to her, as we have seen a lot of inconsistency around AI and software, and a lot of the risks related to geopolitics are getting very binary for investors.
That said, she stated that she has a very constructive outlook for this year, with earnings growth last quarter of 13% and the forecast for this year looking very similar to that, policy rate moving lower, and a 10-year yield that is just above 4%. All of these factors, according to her, make it very hard to be overly pessimistic.
READ ALSO: 11 Best Transportation Stocks to Buy According to Wall Street Analysts AND 10 Small-Cap Stocks With Huge Growth Potential.
Talking about tariffs, Goodwin stated that from a macroeconomic perspective, not a lot has changed, as we have an average effective tariff rate that looks pretty similar. The story on tariffs is really on the micro level, according to her, spanning across how these new tariffs are put into position, and where the sector and company winners and losers play out. That is likely to be challenging for certain names. We talked about the healthcare sector in a recently published article on 11 Best Strong Buy Healthcare Stocks to Invest In. Here is an excerpt from the article:
“On February 23, Steven Wieting, CIO Group chief investment strategist, appeared on CNBC’s ‘The Exchange’ to talk about the stock market and the recent price action in stocks. CNBC reported that staples, healthcare, and utilities are all leading, outperforming since January 1 and showing that this is not a one-day trend but has rather been going on since the turn of the calendar. Wieting stated that a lot is going on in the market, with trends showing AI hardware eating software, its effects on the lending industry for software, and several other undercurrents, with healthcare and energy up, the probability of a US strike on Iran, and oil prices picking up sharply at the expense of growth sentiment. He also cited tariffs at 15% and the situation surrounding them.
Talking about healthcare, Wieting stated that he is strongly overweight in healthcare because of how badly it performed and how much policy concern played a role in its deep underperformance, which is now catching up. While it is not a wonderful year-to-date performer, he thinks that there is more there, and cited its low correlation to tech and the circumstances there.
Wieting also talked about the shifting AI sentiment, stating that the market narrative has generally gone quite negative on the broader impact of AI, with the market pricing in the ongoing AI infrastructure boom while associated applications drive considerable declines in business costs. He further said that there are likely to be mistakes and exaggerations within the market sentiment associated with AI.”
With these broader market trends in view, let’s narrow down and look at the top gene therapy stocks to buy according to hedge funds.
Our Methodology
We sifted through stock screeners and financial media reports to compile a list of the best gene therapy stocks and selected the top 10 most popular among elite hedge funds as of Q3 2025. We sourced the hedge fund data from Insider Monkey’s database. The stocks are ranked in ascending order of hedge fund sentiment.
Note: All data was recorded on February 26.
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 427.7% since May 2014, beating its benchmark by 264 percentage points (see more details here).
Top 10 Gene Therapy Stocks to Buy According to Hedge Funds
10. Beam Therapeutics Inc. (NASDAQ:BEAM)
Beam Therapeutics Inc. (NASDAQ:BEAM) is one of the top gene therapy stocks to buy according to hedge funds. On February 25, RBC Capital adjusted the price target on Beam Therapeutics Inc. (NASDAQ:BEAM) to $26 from $22 and maintained a Sector Perform rating on the shares. The rating update came after the company reported its fiscal Q4 financial results, with the firm telling investors that Beam Therapeutics Inc. (NASDAQ:BEAM) is continually targeting BLA submission this year on sickle cell disease, and announced a non-dilutive debt deal with Sixth Street to support potential commercialization.
Beam Therapeutics Inc. (NASDAQ:BEAM) reported its fiscal Q4 and full-year 2025 results on February 24 and also announced the expansion of its liver-targeted genetic disease franchise with a new program, BEAM-304, for the treatment of phenylketonuria (PKU). PKU has a considerable unmet need and affects around 20,000 individuals in the United States. The company’s plans for 2026 include a focus on execution across its most advanced programs, including reporting updated Phase 1/2 data, further defining the pivotal path forward for BEAM-302, and preparing for a potential BLA submission for risto-cel as early as year-end.
Beam Therapeutics Inc. (NASDAQ:BEAM) also announced that it entered into a strategic financing agreement with Sixth Street for significant, long-term, non-dilutive capital to fund the potential launch of risto-cel in sickle cell disease (SCD). The company reported cash, cash equivalents, and marketable securities of $1.25 billion as of December 31, 2025, compared to $850.7 million as of December 31, 2024.
Beam Therapeutics Inc. (NASDAQ:BEAM) is a biotechnology company that develops precision genetic medicines through base editing, a technology that allows efficient and predictable single-base changes at targeted genomic sequences. The company’s lead programs focus on sickle cell disease and alpha-1 antitrypsin deficiency. Beam Therapeutics Inc. (NASDAQ:BEAM) is advancing programs in other genetic diseases as well, along with oncology and immunology. Its primary programs include BEAM-101, Engineered Stem Cell Antibody Paired Evasion (ESCAPE), BEAM-302, BEAM-301, and BEAM-201.
9. Krystal Biotech, Inc. (NASDAQ:KRYS)
Krystal Biotech, Inc. (NASDAQ:KRYS) is one of the top gene therapy stocks to buy according to hedge funds. On February 25, Guggenheim revised the price target on Krystal Biotech, Inc. (NASDAQ:KRYS) to $284 from $224 and reiterated a Buy rating on the shares following the company’s fiscal Q4 report. Krystal Biotech, Inc. (NASDAQ:KRYS) announced its fiscal Q4 and full year 2025 financial results on February 17, reporting that it recorded $107.1 million and $389.1 million in VYJUVEK net product revenue for fiscal Q4 and full year 2025, respectively.
The company further reported that it has secured over 660 reimbursement approvals for VYJUVEK in the United States and is continuing to maintain strong access nationwide. Steady VYJUVEK uptake is being driven by high patient demand since launch in Germany, France, and Japan, and Krystal Biotech’s (NASDAQ:KRYS) estimates show that over 90 patients have been prescribed VYJUVEK therapy across the three countries. It is advancing pricing discussions with the reimbursement authorities in Italy and is currently on track for a potential launch in the second half of 2026. Krystal Biotech, Inc. (NASDAQ:KRYS) also reported a strong balance sheet, ending the quarter with $955.9 million in cash and investments.
Krystal Biotech, Inc. (NASDAQ:KRYS) is a biotechnology company that develops and commercializes pharmaceutical products and offers the product VYJUVEK, which is a topical gel that treats wounds in adult and pediatric patients (from birth) with dystrophic epidermolysis bullosa (DEB).
8. CRISPR Therapeutics AG (NASDAQ:CRSP)
CRISPR Therapeutics AG (NASDAQ:CRSP) is one of the top gene therapy stocks to buy according to hedge funds. On February 17, Morgan Stanley lifted the price target on CRISPR Therapeutics AG (NASDAQ:CRSP) to $33 from $32 and maintained an Underweight rating on the shares. The firm stated that it refreshed its estimates on the stock and reviewed incremental pipeline progress after partner Vertex Pharmaceuticals recently reported fiscal Q4 Casgevy sales.
CRISPR Therapeutics AG (NASDAQ:CRSP) also received a rating update from JPMorgan on February 16. The firm cut the price target on the stock to $67 from $69 while maintaining an Overweight rating on the shares. It told investors that the company’s fiscal Q4 results came broadly in line with expectations, and that it looks forward to the company’s pipeline updates later this year.
The same day, Citi also adjusted the price target on CRISPR Therapeutics AG (NASDAQ:CRSP), raising it to $80 from $77 and reaffirming a Buy rating on the shares. The firm stated that the company’s Casgevy revenue exceeded estimates in fiscal Q4, and that it sees CRISPR Therapeutics AG’s (NASDAQ:CRSP) clinical and preclinical updates in 2026 driving potential upside in the shares.
Headquartered in Zug, Switzerland, CRISPR Therapeutics AG (NASDAQ:CRSP) develops transformative gene-based medicines for serious diseases through its proprietary CRISPR/Cas9 platform. The CRISPR/Cas9 platform allows precise changes to genomic DNA by employing gene editing technology. The company’s product portfolio spans therapeutic programs across various disease areas, including oncology, rare diseases, regenerative medicine, etc.
7. Intellia Therapeutics, Inc. (NASDAQ:NTLA)
Intellia Therapeutics, Inc. (NASDAQ:NTLA) is one of the top gene therapy stocks to buy according to hedge funds. Intellia Therapeutics, Inc. (NASDAQ:NTLA) announced its fiscal Q4 and full year 2025 financial results on February 26, reporting cash, cash equivalents, and marketable securities of $605.1 million as of December 31, 2025, compared to $861.7 million in the prior year period. The company expects its cash, cash equivalents, and marketable securities as of December 31, 2025, to fund operations into the second half of 2027, as well as through lonvo-z’s anticipated U.S. commercial launch for HAE.
Management further reported that the collaboration revenue for the quarter was $23.0 million, compared to $12.9 million for the prior year period. The increase was attributed to the recognition of $9.0 million in revenue related to the termination of the license and collaboration agreement with SparingVision SAS, along with an increase in cost reimbursements related to the company’s collaboration with Regeneron. Research and development expenses for the quarter came up to $88.7 million, compared to $116.9 million for the prior year period.
Intellia Therapeutics, Inc. (NASDAQ:NTLA) is a clinical-stage genome editing company involved in the development of curative therapeutics using the CRISPR/Cas9 system.
6. Novartis AG (NYSE:NVS)
Novartis AG (NYSE:NVS) is one of the top gene therapy stocks to buy according to hedge funds. Novartis AG (NYSE:NVS) announced on February 25 plans to establish a new 46,000-square-foot radioligand therapy manufacturing site in Denton, Texas. It stated that the purpose-built RLT site would mark the company’s fifth in the United States and first manufacturing facility in Texas, and reflects further progress in its $23 billion US investment. Construction is scheduled to begin in 2026, and the site is anticipated to become fully operational in 2028.
Novartis AG (NYSE:NVS) further stated that the new site is expected to create new jobs in advanced manufacturing, bioengineering, quality, and operations, supporting economic growth in Denton and surrounding communities. Vas Narasimhan, CEO of Novartis AG (NYSE:NVS), stated that RLT holds the potential to revolutionize cancer care and that the addition of a fifth RLT manufacturing site in the United States bolsters the company’s ability to meet growing demand and build the capabilities needed to deliver these next-generation treatments with the required speed and precision.
Headquartered in Basel, Switzerland, Novartis AG (NYSE:NVS) develops, markets, and manufactures a range of healthcare and pharmaceutical products, and is also involved in immuno-oncology research. Its operations span the Innovative Medicines, Sandoz, and Corporate segments.
5. 4D Molecular Therapeutics, Inc. (NASDAQ:FDMT)
4D Molecular Therapeutics, Inc. (NASDAQ:FDMT) is one of the top gene therapy stocks to buy according to hedge funds. 4D Molecular Therapeutics, Inc. (NASDAQ:FDMT) announced enrollment completion for 4FRONT-1, the first Phase 3 clinical trial evaluating 4D-150 in patients with wet age-related macular degeneration (wet AMD), on February 9. 4FRONT-1 is a Phase 3 multicenter, randomized, double-masked, aflibercept 2 mg comparator-controlled study of intravitreal 4D-150 in wet AMD, and the primarily endpoint is non-inferiority in the mean change from baseline in best corrected visual acuity at 52 weeks.
David Kirn, M.D., Co-founder, President, and Chief Executive Officer of 4D Molecular Therapeutics, Inc. (NASDAQ:FDMT), stated that the 4D-150 4FRONT-1 Phase 3 trial enrolled in 11 months in a treatment-naïve large market patient population, and is a landmark moment for the company, for wet AMD patients, and for its targeted and locally delivered genetic medicines.
In a separate development, Barclays assumed coverage of 4D Molecular Therapeutics, Inc. (NASDAQ:FDMT) with an Overweight rating on January 27, setting a price target of $33. The firm initiated coverage of 12 biotech stocks and assumed coverage of 11 with a positive view of the industry, telling investors in a research note that it likes the setup for the group in 2026. It further stated that several biotech stocks remain undervalued, and it anticipates “significant tailwinds”, including continued mergers and acquisitions, “strong” underlying fundamentals, and less of a focus on drug pricing.
4D Molecular Therapeutics, Inc. (NASDAQ:FDMT) is a clinical-stage genetic medicines company that invents and develops genetic medicines to treat large market diseases in ophthalmology, pulmonology, and cardiology. The company develops genetic medicines using its proprietary invention platform, Therapeutic Vector Evolution, and is currently advancing five clinical-stage and two preclinical product candidates.
4. BioMarin Pharmaceutical Inc. (NASDAQ:BMRN)
BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) is one of the top gene therapy stocks to buy according to hedge funds. On February 25, Bernstein lifted the price target on BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) to $94 from $90 and maintained an Outperform rating on the shares. It noted that revenues of $875 million beat by 5%, while EPS of 46c missed by 16%, primarily due to the consensus not modeling Roctavian write-down. The firm further stated that the total revenue guidance missed by 6%, while EPS missed by 3%, with product sales guidance looking soft on Voxzogo and solid on Enzymes.
The rating update came after BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) reported its fiscal Q4 and full-year 2025 financial and operating results on February 23. Full year total revenues rose 13% year-over-year to $3.2 billion, led primarily by a 9% revenue growth for enzyme therapies and 26% revenue growth for VOXZOGO®. Total revenues for fiscal Q4 grew 17% year-over-year, led by 13% revenue growth for enzyme therapies and 31% revenue growth for VOXZOGO. BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) also announced a definitive agreement to acquire Amicus Therapeutics, which is expected to considerably accelerate and diversify revenues.
BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) develops and commercializes therapies for serious and life-threatening medical conditions and rare diseases. The company’s product pipeline includes Valoctocogene roxaparvovec, Vosoritide, and BMN 307.
3. Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX)
Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) is one of the top gene therapy stocks to buy according to hedge funds. Canaccord lifted the price target on Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) to $441 from $411 on February 17 and maintained a Hold rating on the shares, telling investors that it updated its model after the fiscal Q4 results delivered the familiar top and bottom line beat. The firm also stated that Trikafta revenue came in slightly below consensus and Alyftrek slightly above. The same day, Barclays also adjusted the price target on Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) to $607 from $606 and maintained an Overweight rating on the shares after the fiscal Q4 report.
In another development, H.C. Wainwright lifted the price target on Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) to $591 from $518 on February 17, reaffirming a Buy rating on the shares and stating that it views povetacicept as “the best-in-class contender” in primary membranous nephropathy following Roche’s Phase 3 MAJESTY study.
The rating updates came after Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) released its fiscal Q4 and full year 2025 earnings on February 12, reporting a full year total revenue of $12.0 billion, reflecting a 9% growth compared to full year 2024. Total revenue for fiscal Q4 came up to $3.19 billion, up 10% compared to the prior year period.
Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) is a global biotechnology company that develops medicines for rare, serious diseases. The company’s product pipeline includes clinical-stage programs for various disorders, including TDT, SCD, CF, acute and neuropathic pain, APOL1-mediated kidney disease, type 1 diabetes, myotonic dystrophy type 1, and alpha-1 antitrypsin deficiency.
2. UniQure (NASDAQ:QURE)
UniQure (NASDAQ:QURE) is one of the top gene therapy stocks to buy according to hedge funds. Wolfe Research initiated coverage of UniQure (NASDAQ:QURE) with a Peer Perform rating on February 23, without assigning a price target. The firm stated that it launched coverage of the neuroscience biotech sector with a “portfolio of high conviction names across multiple therapeutic areas”, adding that it requires additional clarity on UniQure’s (NASDAQ:QURE) lead program AMT-130 before recommending the shares.
UniQure (NASDAQ:QURE) also received a rating update from H.C. Wainwright on February 10, with the firm reiterating a Buy rating on the stock and setting a $70.00 price target. It supported the optimistic rating with several factors, including the strengthening of uniQure’s gene therapy platform as demonstrated by new Fabry disease data.
The firm also considers the emerging safety profile and clearer dose window as constructive, with the observed liver enzyme elevations being manageable and helping refine future dosing instead of derailing development. According to H.C. Wainwright, the Fabry data, along with the ongoing progress in the CNS pipeline and a solid balance sheet, are not yet near-term regulatory catalysts. However, they do add meaningful, longer-term value that is not fully reflected in the current share price.
UniQure (NASDAQ:QURE) discovers, develops, and commercializes innovative gene therapies, with its discoveries focused on treating Huntington’s disease, glybera, hemophilia, and cardiovascular problems.
1. Regeneron Pharmaceuticals, Inc. (NASDAQ:REGN)
Regeneron Pharmaceuticals, Inc. (NASDAQ:REGN) is one of the top gene therapy stocks to buy according to hedge funds. Regeneron Pharmaceuticals, Inc. (NASDAQ:REGN) announced on February 24 that the U.S. Food and Drug Administration (FDA) approved Dupixent® (dupilumab) for the treatment of adult and pediatric patients aged 6 years and older with allergic fungal rhinosinusitis with a history of sino-nasal surgery.
It reported that the FDA evaluated Dupixent under Priority Review for the treatment of AFRS, reserved for medicines that hold the potential to offer considerable improvements in the diagnosis, treatment, or prevention of serious conditions. Management stated that the approval expands Regeneron Pharmaceuticals, Inc.’s (NASDAQ:REGN) indications in sino-nasal diseases to now include AFRS, alongside chronic rhinosinusitis with nasal polyps.
Kenneth Mendez, President and CEO, Asthma and Allergy Foundation of America (AAFA), stated that as the first treatment specifically approved for AFRS, Dupixent offers the potential for relief to adults and children six years and older struggling with potentially debilitating symptoms.
In another development, Regeneron Pharmaceuticals, Inc. (NASDAQ:REGN) announced on February 19 the FDA’s acceptance for Priority Review of the Biologics License Application (BLA) for garetosmab to treat adults with fibrodysplasia ossificans progressiva. The target action date for the FDA decision is August 2026. Management stated that garetosmab is a monoclonal antibody that blocks Activin A, a protein discovered by the company to be crucial in the development of heterotopic ossification (HO) lesions in people with FOP.
Regeneron Pharmaceuticals, Inc. (NASDAQ:REGN) is a pharmaceutical company that develops, discovers, and commercializes therapies for several diseases, including cancer, eye disorders, and allergic conditions.
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