In 2023, the global biotechnology market size was estimated at $1.55 trillion. This number is projected to reach $3.88 trillion by 2030, representing an anticipated CAGR of 13.96% from 2024 to 2030, according to estimates from Grand View Research. The growth of big data, AI, and ML technologies is fueling demand for scalable and high-performance cloud infrastructure. Reports indicate that China is quickly gaining an edge over the US in the biotechnology sector, beyond its advancements in AI. Earlier on June 5, the Harvard Belfer Center for Science and International Affairs, titled “Critical and Emerging Technologies Index,” identified biotech as the area where China has the most immediate opportunity to surpass the US among 5 critical tech sectors, which also include AI, semiconductors, space, and quantum. While the US currently leads in all 5, the narrow gap in biotech suggests a potential shift in global power.
Growing concerns in Washington support this assessment. The US National Security Commission on Emerging Biotechnology, following 2 years of research, stated in an April report that a “ChatGPT moment for biotechnology” is inevitable. If China achieves it first, the US may never catch up. The commission recommended a two-track strategy: accelerating US innovation and slowing down China, proposing that the US government invest ~$15 billion over the next 5 years to support its domestic biotech sector. China’s biotech industry has advanced to the point where, in recent months, US and European pharmaceutical giants have spent billions to acquire China-developed drugs for potential cancer treatment.
That being said, we’re here with a list of the 13 best biotech stocks to invest in now.
A specialized research team in white lab coats working together on a breakthrough biotechnological discovery.
Methodology
We used the Finviz stock screener to compile a list of the top biotech stocks. We then selected the 13 best stocks that were the most popular among elite hedge funds and that analysts were bullish on. The stocks are ranked in ascending order of the number of hedge funds that have stakes in them, as of Q1 2025. The hedge fund data was sourced from Insider Monkey’s database which tracks the moves of over 1000 elite money managers.
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).
13 Best Biotech Stocks To Invest In Now
13. Travere Therapeutics Inc. (NASDAQ:TVTX)
Number of Hedge Fund Holders: 42
Travere Therapeutics Inc. (NASDAQ:TVTX) is one of the best biotech stocks to invest in now. On June 10, Travere Therapeutics launched a new awareness campaign called ‘Play It Forward’ to support the focal segmental glomerulosclerosis/FSGS community. FSGS is a rare kidney disease that affects both children and adults and is a leading cause of kidney failure.
FSGS is a rare proteinuric kidney disorder that is estimated to affect 40,000+ patients in the US, with similar numbers in Europe. It is characterized by the progressive scarring of the kidneys, often leading to kidney failure. The campaign debuted on the first-ever FSGS Awareness Day and features an anthem created by Grammy-winning musician Brian Kennedy and singer-songwriter David Rush, both of whom live with FSGS. The song celebrates the strength and unity of the FSGS community.
Kennedy and Rush encourage the FSGS community to use this soundtrack to share their creative expressions on social media. The campaign also includes a hero video where Kennedy and Rush discuss their FSGS journeys and the creation of their anthem. Through their shared experiences, they aim to foster connection and empower others living with FSGS. To engage with the campaign, individuals can search using the hashtag #PlayitForwardFSGS or visit RKDandMe.com/PlayItForwardFSGS.
Travere Therapeutics Inc. (NASDAQ:TVTX) is a biopharmaceutical company that identifies, develops, and delivers therapies to people living with rare kidney and metabolic diseases in the US.
12. Amylyx Pharmaceuticals Inc. (NASDAQ:AMLX)
Number of Hedge Fund Holders: 42
Amylyx Pharmaceuticals Inc. (NASDAQ:AMLX) is one of the best biotech stocks to invest in now. On June 3, Amylyx Pharmaceuticals announced that the US FDA granted Fast Track designation to AMX0114. AMX0114 is an investigational antisense oligonucleotide/ASO that is specifically designed to target calpain-2. This protein is believed to be a significant contributor to axonal degeneration, which is a key driver in the progression of amyotrophic lateral sclerosis/ALS.
ALS is a rapidly progressive and fatal neurodegenerative disorder with limited therapeutic options. The Fast Track designation is a crucial step for Amylyx in its mission to develop treatments for ALS. The FDA’s decision acknowledges both the severity of ALS and the encouraging preclinical data supporting AMX0114’s potential.
Calpain-2 is a calcium-activated protease. Research has indicated that overactive calpain-2 activity might accelerate disease progression in ALS and other neurodegenerative conditions by causing the degeneration of axons, which are vital for transmitting signals between neurons and muscles. Preclinical studies of AMX0114 have demonstrated improved neuronal survival and reduced levels of extracellular neurofilament light (NfL), which is a biomarker for neuronal damage across various disease models. AMX0114 was also well-tolerated in preclinical safety studies.
Amylyx Pharmaceuticals Inc. (NASDAQ:AMLX) is a clinical-stage pharmaceutical company that discovers and develops treatments for neurodegenerative diseases and endocrine conditions in the US.
11. Centessa Pharmaceuticals (NASDAQ:CNTA)
Number of Hedge Fund Holders: 43
Centessa Pharmaceuticals (NASDAQ:CNTA) is one of the best biotech stocks to invest in now. On June 16, Centessa Pharmaceuticals announced that the US FDA cleared its Investigational New Drug/IND application for ORX142. The clearance allows Centessa to initiate a Phase 1 clinical study of ORX142 in healthy volunteers.
ORX142 is a novel, highly potent, and selective orexin receptor 2 (OX2R) agonist, and it represents the second drug candidate from the company’s multi-asset orexin franchise. The company’s most advanced OX2R agonist, ORX750, is currently in the Phase 2a CRYSTAL-1 study for the treatment of narcolepsy type 1 (NT1), narcolepsy type 2 (NT2), and idiopathic hypersomnia (IH). With ORX142, Centessa aims to explore its potential for several neurological and neurodegenerative disorders that have unmet needs.
The Phase 1 study for ORX142 will evaluate its safety, tolerability, and pharmacokinetics. Centessa plans to begin the first-in-human clinical study imminently, with early proof-of-concept data expected in 2025. This data will help in selecting the appropriate dose for future studies involving patients. Orexin is a neuropeptide for regulating the sleep-wake cycle. Targeting the orexin pathway with OX2R agonists is a promising strategy to address issues like excessive daytime sleepiness/EDS, impaired attention, cognitive deficits, and fatigue.
Centessa Pharmaceuticals (NASDAQ:CNTA) is a clinical-stage pharmaceutical company that discovers, develops, and delivers medicines.
10. Gossamer Bio Inc. (NASDAQ:GOSS)
Number of Hedge Fund Holders: 45
Gossamer Bio Inc. (NASDAQ:GOSS) is one of the best biotech stocks to invest in now. On June 16, Gossamer Bio announced the completion of enrollment for its global registrational Phase 3 PROSERA Study. The study is evaluating seralutinib in patients with Functional Class II and III PAH. Seralutinib is being jointly developed by Gossamer Bio and the Chiesi Group under a global collaboration agreement.
Enrollment for the PROSERA study officially closed on June 11. The study is a double-blind, placebo-controlled clinical trial designed to assess seralutinib’s efficacy when administered in addition to standard PAH therapy. Patients in the trial are randomly assigned in a 1:1 ratio to either the seralutinib or placebo groups and will receive blinded treatment for up to 48 weeks.
The primary endpoint for the PROSERA Study is the change in six-minute walk distance (6MWD) from baseline compared to placebo at Week 24. Key secondary endpoints include time to clinical worsening, as compared to placebo, up to Week 48, along with other secondary and exploratory endpoints, and an evaluation of safety and tolerability. Topline results from the Phase 3 PROSERA Study are anticipated in February 2026.
Gossamer Bio Inc. (NASDAQ:GOSS) is a clinical-stage biopharmaceutical company that develops and commercializes seralutinib for the treatment of pulmonary arterial hypertension/PAH in the US. Chiesi Farmaceutici is a pharmaceutical firm that researches, develops, produces, and markets medicines for respiratory, neonatology, rare diseases, and special care therapeutic areas.
9. Dyne Therapeutics Inc. (NASDAQ:DYN)
Number of Hedge Fund Holders: 46
Dyne Therapeutics Inc. (NASDAQ:DYN) is one of the best biotech stocks to invest in now. On June 17, Dyne Therapeutics announced that the FDA granted Breakthrough Therapy Designation to DYNE-101 for treating myotonic dystrophy type 1 (DM1). The designation expedites the development and review of drugs for serious conditions that show preliminary clinical evidence of improvement over existing therapies.
Benefits of this designation for DYNE-101 in the US include enhanced FDA support, early and frequent communication with reviewers, and eligibility for rolling and Priority Review, potentially reducing the Biologics License Application/BLA review timeline from 12 to 8 months. Following a Type C meeting with the FDA in May 2025 and an analysis of new long-term functional data, Dyne Therapeutics also unveiled an updated plan for obtaining US Accelerated Approval for DYNE-101 in DM1.
Based on FDA feedback and new 6-month and 12-month efficacy data, Dyne submitted a revised protocol for the ongoing Registrational Expansion Cohort of the ACHIEVE trial in June 2025. Dyne expects to complete enrollment in the Registrational Expansion Cohort in Q4 2025. Data from this cohort are planned for mid-2026 to support a potential US Accelerated Approval submission in late 2026. Additionally, Dyne plans to initiate a confirmatory Phase 3 clinical trial in Q1 2026 and is pursuing expedited approval pathways for DYNE-101 globally.
Dyne Therapeutics Inc. (NASDAQ:DYN) is a clinical-stage neuromuscular disease company that discovers and develops therapeutics for neuromuscular diseases in the US.
8. Madrigal Pharmaceuticals Inc. (NASDAQ:MDGL)
Number of Hedge Fund Holders: 46
Madrigal Pharmaceuticals Inc. (NASDAQ:MDGL) is one of the best biotech stocks to invest in now. On June 20, Madrigal Pharmaceuticals moved closer to introducing the first treatment for metabolic dysfunction-associated steatohepatitis/MASH in Europe. The European Medicines Agency’s Committee for Medicinal Products for Human Use issued a positive opinion recommending the approval of Rezdiffra (resmetirom).
Although the European Commission is not obligated to follow this recommendation, a final decision is anticipated in August 2025. Following this announcement, shares of Madrigal (MDGL) opened 0.5% higher on June 20. The Pennsylvania-headquartered company has a market capitalization of ~$6.3 billion and also has a therapy portfolio for cardiovascular and metabolic diseases.
Rezdiffra is already approved and available in the US for adults with noncirrhotic MASH who have moderate to advanced liver fibrosis. If approved in Europe, Rezdiffra would hold the same indication. Madrigal secured accelerated approval from the US FDA for Rezdiffra in March 2024, making it the first MASH treatment in the US market. MASH is a form of non-alcoholic fatty liver disease/NAFLD and is a significant health concern, being the leading cause of liver transplantation in women and the second leading cause of all liver transplantations in the US.
Madrigal Pharmaceuticals Inc. (NASDAQ:MDGL) is a biopharmaceutical company that delivers novel therapeutics for metabolic dysfunction-associated steatohepatitis/MASH in the US.
7. Amicus Therapeutics Inc. (NASDAQ:FOLD)
Number of Hedge Fund Holders: 47
Amicus Therapeutics Inc. (NASDAQ:FOLD) is one of the best biotech stocks to invest in now. On June 3, Amicus Therapeutics announced the publication of a post-hoc analysis of data from the ERT-experienced cohort of the PROPEL study in Muscle and Nerve. The analysis focused on the efficacy of Pombiliti (cipaglucosidase alfa-atga) + Opfolda (miglustat) (cipa+mig) in adults with late-onset Pompe disease/LOPD.
The results indicated that subjects who switched from alglucosidase alfa to cipa+mig generally achieved improvements or stability in most measured outcomes. The PROPEL study was a 52-week, double-blind, randomized global study that evaluated the efficacy, safety, and tolerability of cipa+mig against non-US-approved alglucosidase alfa + placebo.
Pombiliti + Opfolda is a two-component therapy. Pombiliti (cipaglucosidase alfa-atga) is a bis-M6P-enriched rhGAA designed for high-affinity uptake and efficient processing. Opfolda (miglustat) is an oral enzyme stabilizer that helps reduce the loss of enzyme activity in the blood. Late-onset Pompe disease is an inherited lysosomal disorder caused by a deficiency of the enzyme acid alpha-glucosidase/GA, leading to glycogen accumulation in cells. This progressive disease causes debilitating muscle weakness throughout the body, particularly affecting skeletal muscles and those involved in breathing.
Amicus Therapeutics Inc. (NASDAQ:FOLD) is a biotechnology company that discovers, develops, and delivers novel medicines for rare diseases in the US and internationally.
6. Syndax Pharmaceuticals Inc. (NASDAQ:SNDX)
Number of Hedge Fund Holders: 51
Syndax Pharmaceuticals Inc. (NASDAQ:SNDX) is one of the best biotech stocks to invest in now. On June 12, Syndax Pharmaceuticals announced that data from the BEAT AML trial involving revumenib were published in the Journal of Clinical Oncology and simultaneously presented at the 30th European Hematology Association/EHA Annual Congress Meeting, which was held from June 12-15 in Milan, Italy, and virtually.
The data showcased revumenib in combination with venetoclax and azacitidine (ven/aza) for newly diagnosed mutant NPM1 (mNPM1) and KMT2A-rearranged (KMT2Ar) acute myeloid leukemia/AML patients. The BEAT AML Phase 1b trial was part of The Leukemia & Lymphoma Society’s Beat AML Master Clinical Trial (NCT03013998) and evaluated the safety and clinical activity of this combination in newly diagnosed older adults (aged ≥60 years) with mNPM1 or KMT2Ar AML.
Revuforj (revumenib) is an oral, first-in-class, selective menin inhibitor that is FDA-approved for relapsed or refractory (R/R) acute leukemia with a lysine methyltransferase 2A gene (KMT2A) translocation in adult and pediatric patients aged one year and older. Revumenib is also in development for R/R AML with a nucleophosmin 1 mutation (mNPM1). Positive pivotal data from the AUGMENT-101 trial for this indication were recently published, and Syndax completed the submission of a supplemental New Drug Application (sNDA) for revumenib in R/R mNPM1 AML in April 2025.
Syndax Pharmaceuticals Inc. (NASDAQ:SNDX) is a commercial-stage biopharmaceutical company that develops therapies for the treatment of cancer.
5. Blueprint Medicines Corporation (NASDAQ:BPMC)
Number of Hedge Fund Holders: 52
Blueprint Medicines Corporation (NASDAQ:BPMC) is one of the best biotech stocks to invest in now. On June 12, Blueprint Medicines announced new data reinforcing the sustained clinical efficacy and well-tolerated safety profile of long-term AYVAKIT/AYVAKYT (avapritinib) treatment. These findings were presented at the 2025 European Hematology Association Hybrid Congress, which was held from June 12-15, in Milan, Italy, and the European Academy of Allergy and Clinical ImmunologyCongress 2025, held from June 13-16, in Glasgow, UK.
The presentations highlighted over a decade of collaboration to transform systemic mastocytosis/SM treatment. The data were derived from large patient populations in the PIONEER, PATHFINDER, and EXPLORER trials and featured follow-up periods of up to 5 years in Indolent Systemic Mastocytosis/ISM and up to 6.5 years in advanced SM. The extensive follow-up demonstrates the favorable long-term benefits of AYVAKIT and the significant datasets amassed by Blueprint Medicines.
SM is a rare disease primarily driven by the KIT D816V mutation in ~95% of cases. It leads to uncontrolled mast cell proliferation and activation, which results in chronic, severe, and unpredictable symptoms across multiple organ systems. AYVAKIT (avapritinib) is the first and only medicine approved by the US FDA to treat the root cause of SM. It received FDA approval for advanced SM in June 2021 and for ISM in May 2023.
Blueprint Medicines Corporation (NASDAQ:BPMC) is a precision therapy company that develops medicines for genomically defined cancers and blood disorders in the US and internationally.
4. Roivant Sciences Ltd. (NASDAQ:ROIV)
Number of Hedge Fund Holders: 53
Roivant Sciences Ltd. (NASDAQ:ROIV) is one of the best biotech stocks to invest in now. On June 16, Pulmovant, a Roivant company, announced the publication of Phase 1 pharmacokinetic/PK data for inhaled mosliciguat in healthy male volunteers. The peer-reviewed manuscript, titled “Pharmacokinetics and Lung Deposition After Administration of Inhaled Mosliciguat (BAY 1237592): Results from Randomized Phase I Studies in Healthy Men,” was published in the journal Clinical Pharmacokinetics.
The findings support the potential for once-daily inhaled administration of mosliciguat as a treatment option for patients with pulmonary hypertension/PH, suggesting a longer half-life compared to oral and IV administration, which could lead to limited systemic side effects. The published manuscript details three Phase 1 studies that characterized the PK profile of mosliciguat following inhalation via a low-resistance device using a lactose carrier-based dry powder formulation.
In all three studies, mosliciguat was well tolerated, with no major systemic effects observed on heart rate or blood pressure. The data indicate a long residence time for the drug in the lungs and a slow, continuous release into the systemic circulation. PH is a progressive and debilitating condition characterized by high blood pressure in the lungs’ blood vessels, which leads to symptoms like shortness of breath and fatigue.
Pulmovant is a clinical-stage biotechnology company and a Roivant company. Roivant Sciences Ltd. (NASDAQ:ROIV) is a clinical-stage biopharmaceutical company that discovers, develops, and commercializes medicines and technologies.
3. Argenx (NASDAQ:ARGX)
Number of Hedge Fund Holders: 54
Argenx (NASDAQ:ARGX) is one of the best biotech stocks to invest in now. On June 20, Argenx announced that the European Commission/EC approved VYVGART (efgartigimod alfa) 1000mg for subcutaneous/SC injection. The approval is for its use as a monotherapy in adult patients with progressive or relapsing active chronic inflammatory demyelinating polyneuropathy/CIDP who have previously been treated with corticosteroids or immunoglobulins.
VYVGART SC is available as a vial or prefilled syringe and offers a flexible administration option, which allows it to be given by a patient, caregiver, or healthcare professional. Treatment begins with a weekly dose regimen, which can be adjusted to every other week based on clinical evaluation. This marks the first approval of an IgG Fc-antibody fragment specifically targeting the neonatal Fc receptor (FcRn) for CIDP in Europe, and it represents the first novel mechanism of action for CIDP treatment in 30+ years.
The EC’s decision follows a positive recommendation from the Committee for Medicinal Products for Human Use/CHMP and is supported by the results of the ADHERE clinical trial, which is the largest study conducted to date on CIDP patients. CIDP is a rare, debilitating, and often progressive immune-mediated neuromuscular disorder affecting the peripheral nervous system. The EC approval covers all 27 European Union Member States, as well as Iceland, Liechtenstein, and Norway.
Argenx (NASDAQ:ARGX) is a commercial-stage biopharma company that develops various therapies for the treatment of autoimmune diseases in the US, Japan, China, the Netherlands, and internationally.
2. Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX)
Number of Hedge Fund Holders: 60
Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) is one of the best biotech stocks to invest in now. On June 20, Vertex Pharmaceuticals announced the simultaneous presentation and publication of updated data from the Phase 1/2 portion of the Phase 1/2/3 FORWARD-101 clinical trial of zimislecel (VX-880). Zimislecel is an investigational stem cell-derived, fully differentiated islet cell therapy for individuals with type 1 diabetes (T1D) who experience impaired hypoglycemic awareness and severe hypoglycemic events (SHEs).
The data were presented at the American Diabetes Association/ADA annual conference in Chicago, specifically during the symposium “Innovation and Progress in Stem Cell-Derived Islet-Cell Replacement Therapy” and were simultaneously published online by the New England Journal of Medicine. The presented data included 12 patients who received a full dose of zimislecel as a single infusion and were followed for at least one year, as of October 2024.
The results consistently demonstrated the transformative potential of zimislecel, showing durable patient benefit with longer follow-up. All 12 participants exhibited engraftment with glucose-responsive endogenous C-peptide production, which remained durable throughout one year of follow-up. Zimislecel was generally well-tolerated, with most adverse events being mild or moderate. Type 1 diabetes (T1D) is an autoimmune disease where the body’s insulin-producing beta cells in pancreatic islets are destroyed, leading to insulin deficiency and hyperglycemia.
Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) is a biotechnology company that develops and commercializes therapies for treating cystic fibrosis/CF.
1. SpringWorks Therapeutics Inc. (NASDAQ:SWTX)
Number of Hedge Fund Holders: 71
SpringWorks Therapeutics Inc. (NASDAQ:SWTX) is one of the best biotech stocks to invest in now. On June 20, SpringWorks Therapeutics announced that the European Medicine Agency’s Committee for Medicinal Products for Human Use/CHMP adopted a positive opinion recommending the approval of nirogacestat. The oral gamma secretase inhibitor is proposed as a monotherapy for adults with progressing desmoid tumors who require systemic treatment.
The European Commission/EC is expected to review the CHMP opinion and make a decision regarding approval in Q3 2025. If approved, nirogacestat will be the first and only therapy with marketing authorization in the European Union for desmoid tumors. Nirogacestat previously received Orphan Drug designation from the EC for soft tissue sarcoma. The positive CHMP opinion was based on the Marketing Authorization Application/MAA for nirogacestat, which utilized results from the Phase 3 DeFi trial.
The results were published in The New England Journal of Medicine. In the DeFi trial, nirogacestat met its primary endpoint by improving progression-free survival/PFS, which showed a 71% lower risk of disease progression compared to placebo. The trial also showed a significant improvement in objective response rate and early, sustained improvements in patient-reported outcomes, including pain, physical functioning, and overall quality of life. The safety and tolerability profile of nirogacestat was manageable.
SpringWorks Therapeutics Inc. (NASDAQ:SWTX) is a commercial-stage biopharmaceutical company that develops and commercializes medicines for underserved patient populations suffering from rare diseases and cancer.
While we acknowledge the potential of SWTX to grow, our conviction lies in the belief that some AI stocks hold greater promise for delivering higher returns and have limited downside risk. If you are looking for an AI stock that is more promising than SWTX and that has 100x upside potential, check out our report about this cheapest AI stock.
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