In this article, we will take a look at some of the best biotech stocks that are currently offering attractive upside to investors.
On April 16, reports from J.P. Morgan’s biopharma and medtech venture and licensing studies indicated that the capital markets began the year 2026 with selective strength. Biopharma venture capital amounted to $5.2 billion in the first quarter of 2026, and licensing stood at $77.3 billion for the same period. The total biopharma M&A market was worth $15.6 billion for 19 deals. The six biopharma IPOs collected $1.8 billion, which was greater than the entire year 2025 tally. In medtech, venture funding totaled $2.2 billion across 66 rounds. Medtech M&A totaled $26.6 billion across 37 deals. IPO activity remained selective, with one IPO raising $600 million on Nasdaq.
The present market situation offers great opportunities for investors interested in making investments in biotechnology stocks. There is high activity in licensing and M&A deals, with venture capital going into late-stage ventures. Public market openings bring new possibilities. Licensing transactions on obesity and diabetes, along with the sourcing of assets originating from China, are becoming trends.
These trends indicate a change in strategic priorities in the biotech stock market. With that background, let’s explore our 10 Best Biotech Stocks with Highest Upside Potential.
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Our Methodology
To identify relevant stocks for this article, we conducted a screening of U.S.-listed biotechnology companies with market capitalizations above $2 billion. Also, we only shortlisted stocks with at least 75% upside potential according to consensus, as of April 24 closing. Finally, we selected 10 stocks with the highest upside and ranked them in ascending order.
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 498.7% since May 2014, beating its benchmark by 303 percentage points (see more details here).
10. Corcept Therapeutics Inc. (NASDAQ:CORT)
Corcept Therapeutics Inc. (NASDAQ:CORT) is one of the 10 best biotech stocks with highest upside potential.
On April 11, Corcept Therapeutics Inc. (NASDAQ:CORT) released the overall survival results from its Phase 3 ROSELLA study involving Lifyorli and nab-paclitaxel treatment for women with platinum-resistant ovarian cancer during the annual conference of the Society of Gynaecologic Oncology. The findings were concurrently published in The Lancet journal. The ROSELLA study achieved both of its primary objectives of overall survival and progression-free survival without compromising on safety. This backs our bullish views for the stock.
The combination of Lifyorli with nab-paclitaxel is now part of the National Comprehensive Cancer Network guidelines as a preferred treatment regimen. The drug received approval from the FDA in March 2026 for the treatment of platinum-resistant epithelial ovarian, fallopian tube, or primary peritoneal cancer.
Patients receiving Lifyorli with nab-paclitaxel showed 35% fewer chances of mortality. Median overall survival was 4.1 months higher. The patients had a 30% lower rate of disease progression. The combination therapy is safe. The common adverse effects include low levels of haemoglobin, neutrophils, fatigue, nausea, diarrhea, platelets, rash, and reduced appetite.
Corcept Therapeutics Inc. (NASDAQ:CORT) is a commercial-stage pharmaceutical company that is involved in developing therapies for severe conditions. These include endocrinologic, oncologic, metabolic, and neurologic disorders. It is developing medicines that target hyperglycemia, hypercortisolism, prostate cancer, and amyotrophic lateral sclerosis.
9. Beam Therapeutics Inc. (NASDAQ:BEAM)
Beam Therapeutics Inc. (NASDAQ:BEAM) is one of the 10 best biotech stocks with highest upside potential.
On April 2, Beam Therapeutics Inc. (NASDAQ:BEAM) released results from its Phase 1/2 BEACON study for ristoglogene autogetemcel. This therapy is being developed to treat severe vaso-occlusive crises associated with sickle cell disease, through the New England Journal of Medicine. Risto-cel is a potentially best-in-class autologous cell therapy. Results from the study indicated that the therapy was well-tolerated with safety, consistent with myeloablative conditioning, and showed highly promising efficacy results.
The study led to better parameters for hemolysis, anemia correction, and the absence of any complications with respect to vaso-occlusion after transplant. Rapid and complete bone marrow reconstitution was obtained in patients. The average values of hemoglobin F exceeded 60%, while those of hemoglobin S were below 40% in most patients.
All hemolysis-related measures were either normal or showed improvement after risto-cel therapy. All sickling measures declined after risto-cel therapy and were like those observed in people suffering from sickle cell trait. There were no major adverse events associated with risto-cel that were not related to busulfan conditioning, autologous hematopoietic stem cell transplantation, and SCD.
Beam Therapeutics Inc. (NASDAQ:BEAM) is a biotechnology company that develops precision genetic medicines and also engages in gene therapies and genome editing research. Since its launch, the company has been spearheading the CRISPR-based editing that facilitates the development of advanced genetic medicines.
8. Praxis Precision Medicines Inc. (NASDAQ:PRAX)
Praxis Precision Medicines Inc. (NASDAQ:PRAX) is one of the 10 best biotech stocks with highest upside potential.
As of the close of play on April 24, the broader consensus sentiments towards Praxis Precision Medicines Inc. (NASDAQ:PRAX) remained strongly bullish. The stock received coverage from 16 analysts, 14 of whom assigned Buy ratings and 1 gave a Hold call. With just 1 Sell rating, it carries a median 1-year target price of $625.31, leading to an upside potential of more than 81% for investors. This makes it one of the standout names within the biotech space.
On April 10, Raymond James assigned a Strong Buy rating to Praxis Precision Inc. (NASDAQ:PRAX). The firm set a target price of $815, which leads to a lucrative upside potential of more than 136% at the current level.
The firm reflected on the prevailing bearish sentiment towards the stock, which it believes is based on a predetermined conclusion completely devoid of empirical evidence. It emphasized that the expected FDA approval of ulixacaltamide during early 2027 could act as a definitive commercial catalyst for the stock. The firm also believes that at the current valuation level, investors are significantly undervaluing the likelihood of formal regulatory success.
Praxis Precision Medicines Inc. (NASDAQ:PRAX) is involved in developing therapies to cure disorders related to the central nervous system, which are characterized by neuronal excitation-inhibition imbalance. These disorders include epilepsy, movement disorders, and depression. The company utilizes its proprietary platforms like Cerebrum and Solidus to target such imbalances.
7. Denali Therapeutics Inc. (NASDAQ:DNLI)
Denali Therapeutics Inc. (NASDAQ:DNLI) is one of the 10 best biotech stocks with highest upside potential.
On April 6, Denali Therapeutics Inc. (NASDAQ:DNLI) revealed that it has received notice from Takeda (NYSE: TAK) about the termination of the collaboration agreement on developing and commercializing DNL593. This move was due to some strategic reasons and is unrelated to the results on the efficacy or safety of the drug.
DNL593 is a progranulin replacement therapy that involves the use of Denali’s Protein Transport Vehicle technology to deliver progranulin to the brain to treat frontotemporal dementia-granulin. Denali has been responsible for all the developmental activities and is going to resume full control over DNL593 and the intellectual property portfolio.
As pointed out by Chief Executive Officer Ryan Watts, although the collaboration was important, full ownership of DNL593 is good news. The firm is still certain that the scientific principle and the evidence collected so far are strong. According to the plan, Denali will present the findings from its phase I/II clinical trials by the end of 2026.
As the first approved means of transporting through the blood-brain barrier, the TransportVehicle technology offers great possibilities for all forms of neurodegeneration, such as frontotemporal dementia.
Denali Therapeutics Inc. (NASDAQ:DNLI) is a biotechnology company that utilizes proprietary technology to address neurodegenerative diseases. Through their TransportVehicle™ platform, and by leveraging the power of biotherapeutics, they develop medicines for some very serious conditions, including lysosomal storage.
6. Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE)
Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) is one of the 10 best biotech stocks with highest upside potential.
On April 16, Morgan Stanley increased the price target for Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) from $50 to $67, resulting in an adjusted upside potential in excess of 178% at the prevailing level. The firm also reiterated its Outperform rating on the stock.
Morgan Stanley revised the price target upward based on a strict new probability-of-success approach to the forthcoming GTX-102 Phase 3 Angelman trial. This major clinical readout is anticipated in the second half of 2026. The resulting risk profile is rather asymmetrical. The firm has projected a 50% to 70% upside, which significantly offsets a 20% downside risk of a clinical failure.
Back on March 30, Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) announced clearance from the US Food & Drug Administration for the Investigational New Drug Application for UX016. It is a prodrug of sialic acid under investigation for use as a substrate replacement therapy for GNE Myopathy. GNEM is a rare neuromuscular disease characterized by severe disability due to mutations in the GNE gene, which impairs sialic acid biosynthesis.
The UX016 program is supported externally via proof-of-concept clinical trials, which include a Phase 1/2 trial scheduled for initiation in the latter half of 2026. Commenting on the progress of UX016, CEO Emil Kakkis observed that moving the drug into the clinic is a significant step for the GNE myopathy community, and it demonstrates the company’s effort to create a novel method for improving sialic acid utilization by the muscles. The first-in-human study will involve around 24 patients between 18 and 55 years of age suffering from GNEM, who will be recruited in the US.
Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) develops novel therapies, with a focus on identifying, acquiring, and commercializing products for rare and ultra-rare genetic diseases. It has a strong emphasis on gene therapy and covers various stages of clinical trials. The company is currently going through high cash burn with the aim of turning profitable from 2027 onwards.
While we acknowledge the potential of RARE 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 RARE and that has 100x upside potential, check out our report about the cheapest AI stock.
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