In this article, we will be taking a look at the 12 most promising biotech stocks according to Wall Street analysts.
Biotech Sector Set for Growth Amid Innovation and Investor Interest
The biotechnology sector is gaining new momentum with better market conditions, cutting-edge innovations, and growing investor attention. The industry has recovered from a challenging 2024 and is poised for significant expansion led by advances in personalized medicine, AI-driven drug discovery, and growing demand for biologics. MarketsandMarkets predicted that the global biotech market would expand by a strong 13%, from $483.0 billion in 2024 to $546.0 billion in 2025. This expansion demonstrates the sector’s tenacity and promise for growth.
The anticipated shift in the Federal Reserve’s interest rate policies is a key driver of this upturn. Biotech is sensitive to changing rate patterns since it requires a large amount of money for costly R&D and clinical testing. Lower rates make more funding available, which helps biotech companies expand, draw in venture capital, and expedite drug development, according to Genetic Engineering and Biotechnology News. A rate drop, according to analysts, may release billions of dollars in investment funds that have been put aside for fledgling biotech firms looking for steady funding.
Investor interest in biotech equities is growing. Biotech is still a high-growth industry with many chances for risk-takers, despite brief ups and downs. The comeback of biotech has been noted by leading investment banks. Citing solid fundamentals, improved clinical outcomes, and a supportive regulatory environment, Goldman Sachs referred to it as an “undervalued opportunity.” According to the firm, biotech equities have tremendous upside potential and an “option-like structure,” particularly when interest rates decline. Analysts at JPMorgan anticipate a recovery in biotech funding at the same time, pointing to indications of stability in manufacturing and research sectors that were negatively impacted by earlier funding shortages. Industry analysts predict that declining interest rates and advantageous investment circumstances may reopen the IPO window for businesses looking for institutional support, even though biotech IPOs have been quiet since 2021.
As gene editing, AI-powered drug discovery, and precision medicine transform how we treat uncommon genetic illnesses, autoimmune diseases, and cancer, scientific advances are also propelling the expansion of biotech. Biotech firms are meeting medical demands in ways that were unthinkable a decade ago because of developments in CRISPR gene editing and cell treatments. Given this, we will take a look at some of the best promising stocks from the biotech sector.
A biotechnologist in a lab wearing an apron and safety glasses, working on a biopharmaceutical process.
Our Methodology
For this article, we shortlisted stocks that met the following criteria: a market capitalization of over $2 billion, coverage by at least 15 analysts, and a price target upside of more than 20%. We then ranked the selected stocks based on their price target upside, as determined by Wall Street analysts, as of March 21, 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 12 most promising biotech stocks according to Wall Street analysts.
12. Insmed Incorporated (NASDAQ:INSM)
Analyst Upside: 22.66%
Insmed Incorporated (NASDAQ:INSM) is a global biopharmaceutical company focused on developing treatments for rare diseases, particularly those affecting the lungs. Patients with few other treatment options are treated for Mycobacterium avium complex (MAC) lung illness with its most effective drug, ARIKAYCE (amikacin liposome inhalation suspension). The business is also creating new treatments, such as TPIP, which treats pulmonary hypertension associated with lung disorders, and Brensocatib, a medication for bronchiectasis.
Insmed Incorporated (NASDAQ:INSM) reported record revenue growth of 19% to $363.7 million for the year in its Q4 2024 financial report. The corporation performed well in all regions: revenue increased by 14% in the US, 33% in Japan, and 39% in Europe and the rest of the world. Its growth is still being driven by its ARIKAYCE, with sales estimates for 2025 ranging from $405 million to $425 million, solidifying its position among the Most Promising Stocks.
Despite reporting a $191 million cash burn in Q4 as a result of investments in the launch of Brensocatib, Insmed Incorporated (NASDAQ:INSM) ended 2024 with a healthy cash position of over $1.4 billion. Despite these charges, the business spent $599 million on research and development (R&D) and $462 million on selling general, and administrative (SG&A) costs. Supporting Brensocatib’s Phase III outcomes also increases the likelihood that the FDA will approve the medication and that it will be successful commercially in the future.
Fourteen Wall Street analysts set a 12-month price target for Insmed Incorporated (NASDAQ:INSM), averaging $98 (high: $110, low: $90), a 22.62% increase from its last price of $79.92.
11. Moderna, Inc. (NASDAQ:MRNA)
Analyst Upside: 27.83%
Moderna, Inc. (NASDAQ:MRNA) is a biotech company that develops vaccines based on messenger RNA (mRNA) and stands eleventh on our list among the 12 most promising biotech stocks according to Wall Street analysts. The business has solid foundations, despite poor sales and slow market uptake of its new goods. It has a positive outlook because of its complex pipeline of pharmaceuticals undergoing clinical testing.
The company is developing its late-stage candidate pipeline to preserve its market share in 2025 for COVID-19 vaccinations. In the respiratory vaccine space, Moderna, Inc. (NASDAQ:MRNA) is also awaiting FDA approval for an expanded indication of its RSV vaccine. Additionally, it has applied for licensure of a combination influenza-COVID-19 vaccine supported by encouraging phase 3 research results.
Analysts are also optimistic about Moderna, Inc. (NASDAQ:MRNA) because of its non-respiratory portfolio, which includes cytomegalovirus (CMV) and norovirus vaccines. Additionally, mRNA-4157, which is being studied in partnership with Merck, exhibits promise across a range of malignant tumor types. The corporation’s capacity to introduce its mRNA technology into new markets is demonstrated by this varied pipeline, which positions it as a more established and varied commercial-stage biotech business.
Wall Street analysts have provided 12-month price targets for the stock, with an average target of $41.75. The forecasts range from a high of $78.00 to a low of $25.00. This suggests a potential 27.83% increase from Moderna, Inc. (NASDAQ:MRNA)’s current price of $32.66.
10. BioMarin Pharmaceutical Inc. (NASDAQ:BMRN)
Analyst Upside: 39.51%
BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) develops and markets specialized therapies for rare genetic diseases, focusing on enzyme replacement treatments and other biopharmaceuticals. The business is notable for its commitment to rare diseases; it has successfully introduced eight innovative medications to the market, including therapies for lysosomal storage diseases, severe hemophilia A, and achondroplasia.
The Q4 2024 financial results of BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) demonstrated robust development in several important areas. Q4 revenue surged by 16% to $747 million, and its overall revenue grew by 18% to $2.85 billion for the year. Increased acceptance for achondroplasia led to a 56% increase in VOXZOGO sales to $735 million, while enzyme treatments contributed over $1.9 billion, a 12% increase from 2023.
The non-GAAP operating margin increased to 28.6% for the year and 31.1% in Q4, indicating a considerable improvement in the company’s profitability. Revenue growth and operational efficiency were the main drivers of the 69% increase in earnings per share (EPS) to $3.52 and the 88% increase in Q4 EPS to $0.92. Additionally, operating cash flow increased by a staggering 260% to $573 million, giving investors financial flexibility for upcoming projects.
With a 10% growth projection, BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) projects revenue in 2025 to reach between $3.1 billion and $3.2 billion. With non-GAAP operating margins between 32% and 33% and EPS between $4.20 and $4.40, margins are expected to continue to expand which suggests sustained profitability and efficiency. Given its strong financial performance and future growth potential, many analysts consider BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) one of the Most Promising Stocks in the biotech sector.
In addition to its financials, the company is developing its therapeutic pipeline; later this year, early clinical data for BMN 333 and BMN 351 (Duchenne Muscular Dystrophy) are anticipated. These changes may spur additional expansion and have a favorable effect on the stock.
9. Jazz Pharmaceuticals plc (NASDAQ:JAZZ)
Analyst Upside: 39.68%
Jazz Pharmaceuticals plc (NASDAQ:JAZZ) is a biopharmaceutical company specializing in neuroscience and oncology, focusing on treatments for serious diseases with limited therapeutic options, such as narcolepsy, epilepsy, and certain cancers.
At the American Epilepsy Society 2024 conference on December 6, 2024, Jazz Pharmaceuticals plc (NASDAQ:JAZZ) presented fresh Epidiolex (cannabidiol) research that demonstrated its advantages beyond seizure management. Research has shown that patients with epilepsy and tuberous sclerosis complex (TSC) had improved emotional well-being, communication, and cognitive abilities. Most nurses and caregivers continued treatment, reporting better quality of life and fewer seizures. Epidiolex’s broad-spectrum efficacy was further supported by the EpiCom trial, which revealed that it also decreased behavioral problems in TSC patients.
With over $4 billion in revenue and significant growth across its sleep, epilepsy, and oncology portfolios—all of which generate over $1 billion annually—Jazz Pharmaceuticals plc (NASDAQ:JAZZ) had a successful 2024. The FDA’s expedited approval of Ziihera for HER2+ biliary tract cancer and encouraging developments in late-stage pipeline assets were among the company’s significant accomplishments. With $3 billion in cash at the end of 2024 and a voluntary $750 million debt payoff, the company strengthened its position for further expansion in 2025.
Wall Street analysts maintain a Strong Buy rating on the stock. Over the past three months, 18 analysts have set a 12-month average price target of $193.82, with estimates ranging from $150.00 to $230.00. This suggests a potential 39.68% increase from the current price of $138.76.
8. Regeneron Pharmaceuticals, Inc. (NASDAQ:REGN)
Analyst Upside: 45.22%
Regeneron Pharmaceuticals, Inc. (NASDAQ:REGN) stands eighth among the most promising stocks according to Wall Street analysts. The company is focused on developing life-changing medicines for serious diseases, including eye conditions, cancer, inflammation, and rare genetic disorders. The company’s cutting-edge VelociSuite technology, which speeds up drug research and genetic medicine development and aids in converting scientific discoveries into practical therapies, is what sets it apart.
Regeneron Pharmaceuticals, Inc. (NASDAQ:REGN) reported $3.8 billion in revenue in Q4 2024, a 10% increase over the same period the year before. With $14.2 billion in revenue for the entire year, it earned $1.4 billion in net income. The company’s strong financial position was demonstrated by its $3.7 billion in free cash flow and its remarkable 86% gross margin.
The corporation’s expansion was driven by important products like EYLEA, Libtayo, and Dupixent. With over a million patients treated globally, dupixent is still a huge success and may find new applications. In the meantime, the business is developing novel therapies for lymphoma and multiple myeloma that may increase its market share in oncology.
Notwithstanding its achievements, Regeneron Pharmaceuticals, Inc. (NASDAQ:REGN) still faces difficulties in the anti-VEGF market, as EYLEA sales are being impacted by growing biosimilar competition. The business must manage these demands while keeping its attention on growth and innovation.
7. Blueprint Medicines Corporation (NASDAQ:BPMC)
Analyst Upside: 46.53%
Blueprint Medicines Corporation (NASDAQ:BPMC) is a biopharmaceutical company focused on developing targeted therapies for cancers and blood disorders. Its primary source of income is AYVAKIT (avapritinib) which is a systemic mastocytosis (SM) medication that is marketed in the United States and Europe. Its areas of expertise are allergy/inflammation and oncology/hematology.
Strong financial results were published by Blueprint Medicines Corporation (NASDAQ:BPMC) for the fourth quarter and the entire year 2024. AYVAKIT generated $479 million in global revenue, including $144.1 million in Q4 alone. With a projected revenue of $680–$710 million in 2025—a 45% increase from the previous year—the corporation anticipates tremendous growth. With AYVAKIT expected to reach $2 billion by 2030, it envisions a $4 billion peak revenue opportunity for its systemic mastocytosis (SM) business.
Even though the company reported a net loss of $50 million in the fourth quarter and $67.1 million for the year, these numbers represent a significant improvement over 2023, when losses were far greater. This points to a route toward profitability for Blueprint Medicines Corporation (NASDAQ:BPMC) and improved financial management. The company’s revenue is still being driven by strong demand for AYVAKIT, making it one of the Most Promising Stocks which indicates successful commercialization and long-term growth potential.
Blueprint Medicines Corporation (NASDAQ:BPMC) attracts investors due to its remarkable expansion, promising therapeutic pipeline, which includes BLU-808, a possible treatment for inflammatory and allergic illnesses, and sound financial management.
6. BioNTech SE (NASDAQ:BNTX)
Analyst Upside: 47.23%
BioNTech SE (NASDAQ:BNTX) is a German biotechnology company specializing in mRNA-based vaccines and cancer immunotherapies. It is best known for its COVID-19 vaccine developed with Pfizer and focuses on personalized treatments tailored to genetic profiles.
BioNTech SE (NASDAQ:BNTX) is transitioning from reliance on COVID-19 vaccine revenues to focusing on its core cancer therapeutics. In Q4 2024, the company reported revenues of €1,190.0 million, down from €1,479.0 million in Q4 2023, while full-year revenues fell to €2,751.1 million from €3,819.0 million in 2023. The decline was mainly due to reduced demand for COVID-19 vaccines. Consequently, the company posted a net profit of €259.5 million for Q4 but reported a full-year net loss of €665.3 million compared to a €930.3 million profit in 2023.
Despite the revenue decline, BioNTech SE (NASDAQ:BNTX) maintains a strong financial position with €17.4 billion in cash and investments which provides stability for future growth. The corporation’s oncology pipeline is advancing rapidly, with over 20 active Phase 2 and Phase 3 trials. Its key programs include BNT327, a bispecific antibody targeting PD-L1 and VEGF-A, and mRNA cancer immunotherapies leveraging its expertise in mRNA technology.
Analysts remain optimistic about BioNTech SE (NASDAQ:BNTX)’s future, with projected 2025 revenues between €1.7 billion and €2.2 billion. The company has made significant strides in oncology, initiating global Phase 3 trials for BNT327 in lung cancer and presenting promising data for triple-negative breast cancer at SABCS 2024. Additionally, the business expanded its pipeline through the acquisition of Biotheus, securing full control of BNT327. These strategic developments position the company for long-term growth in the oncology sector.
5. ACADIA Pharmaceuticals Inc. (NASDAQ:ACAD)
Analyst Upside: 55.69%
The fifth stock on our list of the most promising stocks according to Wall Street analysts is ACADIA Pharmaceuticals Inc. (NASDAQ:ACAD). It specializes in developing therapies for central nervous system disorders, focusing on neuropsychiatric symptoms. Its flagship product, NUPLAZID, is the only FDA-approved treatment for Parkinson’s disease psychosis, while its recently launched DAYBUE is the first approved treatment for Rett syndrome in the U.S. and Canada. With a strategic focus on unmet medical needs in neuroscience, the company has established itself as a leader in developing first-in-class therapies for complex disorders, setting it apart in the biotech industry.
ACADIA Pharmaceuticals Inc. (NASDAQ:ACAD) delivered strong financial results for Q4 and full-year 2024, showcasing significant revenue growth and strategic advancements. Total revenue reached $259.6 million in Q4 and $957.8 million for the year, driven by strong sales of its key products. NUPLAZID generated $162.9 million in Q4 and $609.4 million for the year, benefiting from increased unit sales and pricing. DAYBUE, the company’s novel treatment for Rett syndrome, contributed $96.7 million in Q4 and $348.4 million for the full year, which reflected rising market adoption.
The company’s profitability improved considerably, with a net income of $226.5 million in 2024, reversing a net loss of $61.3 million in 2023. This turnaround was fueled by higher revenues, cost management, and a one-time gain of $146.5 million from the sale of a Rare Pediatric Disease Priority Review Voucher. ACADIA Pharmaceuticals Inc. (NASDAQ:ACAD) ended the year with $756 million in cash and investments, up from $438.9 million in 2023, providing financial flexibility for future growth.
Looking ahead, the corporation projects 2025 revenues between $1.03 and $1.095 billion, with expected NUPLAZID sales of $650–$690 million and DAYBUE sales of $380–$405 million. The company is also making strides in regulatory and clinical development, including a European regulatory submission for trofinetide and pipeline advancements in Prader-Willi syndrome, Alzheimer’s disease psychosis, and Lewy Body Dementia psychosis.
4. CRISPR Therapeutics AG (NASDAQ:CRSP)
Analyst Upside: 73.65%
CRISPR Therapeutics AG (NASDAQ:CRSP) uses its patented CRISPR/Cas9 platform to create revolutionary gene-based medications for severe illnesses. Gene editing technique is used by the CRISPR/Cas9 platform to enable precise modifications to genomic DNA. The company’s product portfolio includes therapeutic projects in oncology, rare diseases, regenerative medicine, and other medical fields.
The main goal of CRISPR Therapeutics AG (NASDAQ:CRSP) is the strategic advancement of its clinical trial portfolio in the areas of autoimmune, diabetes, cancer, and cardiovascular diseases. For the company, the FDA’s approval and introduction of CASGEVY, a gene therapy for sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT) in patients aged 12 and up, has generated significant momentum. Additionally, the business has acquired regulatory permission for the medicine in several countries worldwide, including the European Union, Canada, Switzerland, and Great Britain, among others, and product launches are currently in progress. The drug’s continuous rollout is accelerating, and in 2025, fresh cell patient collection initiations are predicted to rise sharply. Globally, more than 50 CASGEVY-approved treatment centers (ATCs) have been operational.
CRISPR Therapeutics AG (NASDAQ:CRSP)’s balance sheet is robust as of December 31, 2024, with approximately $1.9 billion in marketable securities, cash, and cash equivalents. Analysts have a Moderate Buy rating on the stock. Over the past three months, 20 Wall Street analysts have set a 12-month price target with an average of $71.58. The high forecast is $120.00, while the low is $32.00. This average target reflects a potential upside of 73.65% from the current price of $41.22.
3. Apellis Pharmaceuticals, Inc. (NASDAQ:APLS)
Analyst Upside: 75.14%
Apellis Pharmaceuticals, Inc., (NASDAQ:APLS) is a biopharmaceutical company focused on developing therapies targeting the complement system, specifically at the C3 protein level, to treat rare and serious diseases. The company generates revenue through its FDA-approved products, SYFOVRE for geographic atrophy and EMPAVELI for paroxysmal nocturnal hemoglobinuria.
Apellis Pharmaceuticals, Inc. (NASDAQ:APLS) reported strong financial results for Q4 and the full year 2024, highlighting significant revenue growth. Q4 2024 revenue reached $212.5 million, up from $146.4 million in Q4 2023, while full-year revenue surged 97% to $781.4 million from $396.6 million in 2023. This growth was primarily driven by the strong sales of SYFOVRE, the first approved treatment for geographic atrophy (GA), which generated $167.8 million in Q4 and $611.9 million for the year. EMPAVELI also contributed with $23.4 million in Q4 and $98.1 million for the full year.
Despite a net loss of $36.4 million for Q4 2024 and $197.9 million for the year, Apellis Pharmaceuticals, Inc., (NASDAQ:APLS) showed significant improvement in its financial performance compared to 2023, where losses were higher. R&D expenses decreased, reflecting improved operational efficiency, and SG&A expenses remained stable. As of December 31, 2024, the company had $411.3 million in cash and cash equivalents, positioning it to continue operations until profitability.
Looking ahead, Apellis Pharmaceuticals, Inc., (NASDAQ:APLS) benefits from a first-mover advantage with SYFOVRE in the GA market, and its pipeline expansion, including potential approvals for C3G and IC-MPGN, could further boost revenues. Strategic collaborations, such as with Sobi, enhance global reach, and the company’s path to profitability is becoming clearer with strong revenue growth and controlled expenses. Most promising stocks are often those that show significant potential for growth, and Apellis is positioning itself to be one of them.
2. Ionis Pharmaceuticals, Inc. (NASDAQ:IONS)
Analyst Upside: 77.30%
Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) develops RNA-targeted therapeutics for neurological, cardiovascular, and rare diseases. It generates revenue from royalties on drugs like SPINRAZA, sales of TEGSEDI and WAYLIVRA, and research collaborations. The company’s innovative approach uses modified RNA to target genetic causes of diseases, offering potential breakthroughs for conditions with limited treatments.
Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) reported strong financial results for Q4 and full-year 2024, with total revenue of $227 million for Q4 and $705 million for the year. The increase in commercial revenue was mainly driven by the launch of new products like WAINUA and TRYNGOLZA. Despite a decrease in R&D revenue due to the winding down of joint development for WAINUA, the business maintains a solid cash position of $2.3 billion.
The corporation’s operating expenses for Q4 2024 were $337 million, with a total of $1.18 billion for the year, reflecting the company’s ongoing commercialization efforts for new products. R&D expenses remained stable as Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) continued to invest in its promising pipeline. Key target markets include Familial Chylomicronemia Syndrome (FCS), Hereditary Transthyretin-Mediated Amyloidosis (ATTRv-PN), Spinal Muscular Atrophy (SMA), and Severe Hypertriglyceridemia (sHTG). Notably, TRYNGOLZA became the first-ever treatment for FCS, and WAINUA is gaining traction for ATTRv-PN.
Recent developments include the FDA approval of TRYNGOLZA in December 2024, Ionis Pharmaceuticals, Inc. (NASDAQ:IONS)’s first independent commercial product, and significant progress for WAINUA with regulatory milestones in Europe. Additionally, ION582, a treatment for Angelman syndrome, is on track for Phase 3 development in 2025.
1. Cytokinetics, Incorporated (NASDAQ:CYTK)
Analyst Upside: 79.24%
Cytokinetics, Incorporated (NASDAQ:CYTK) tops our list for being one of the most promising stocks. It is a biopharmaceutical company focused on developing muscle activators and inhibitors to treat diseases with impaired muscle function, such as heart failure and ALS. With over 25 years of expertise in muscle biology, the company stands out for its specialized approach to drug development, resulting in first-in-class muscle activators and next-in-class muscle inhibitors, distinguishing it from other biotech firms.
Cytokinetics, Incorporated (NASDAQ:CYTK) reported strong financial results for Q4 2024, with revenue reaching $16.9 million which is a significant increase from $1.7 million in Q4 2023. Their full-year revenue for 2024 was $18.5 million, up from $7.5 million in 2023. The increase was largely driven by a $15.0 million upfront payment from Corxel related to the assignment of aficamten rights to Sanofi in Greater China.
Despite the revenue growth, the company posted a net loss of $150.0 million for Q4 2024 and a full-year net loss of $589.5 million, reflecting ongoing investments in R&D. R&D expenses rose to $93.6 million in Q4 2024 and $339.4 million for the full year, as the company continues to advance its muscle biology-focused pipeline.
Looking ahead, Cytokinetics, Incorporated (NASDAQ:CYTK) expects operating expenses for 2025 to range from $670 million to $710 million and anticipates topline results from its MAPLE-HCM trial in Q2 2025. The company is also preparing for the potential US launch of aficamten in the second half of 2025, pending FDA approval.
Overall, Cytokinetics, Incorporated (NASDAQ:CYTK) ranks first among the 12 most promising biotech stocks according to Wall Street analysts. While we acknowledge the potential of biotech 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 CYTK but that trades at less than 5 times its earnings, check out our report about the cheapest AI stock.
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