The Best Biotech Stocks to Buy

Investing in biotechnology stocks has always required a high risk tolerance and the patience to wait years, even decades, for results. These companies drive medical innovation, developing therapies that can revolutionize healthcare and offer significant long-term growth potential.

In the year to date, the Morningstar US Biotechnology Index rose 18.17%, while the Morningstar US Total Market Index gained 11.88%.

The 5 Best Biotech Stocks to Buy Now

These were the most undervalued biotech stocks that Morningstar’s analysts cover as of Sept. 15, 2026:

1. Intellia Therapeutics NTLA
2. Crispr Therapeutics CRSP
3. Ionis Pharmaceuticals IONS
4. BioMarin Pharmaceutical BMRN
5. Alnylam Pharmaceuticals ALNY

To come up with our list of the best biotech stocks to buy now, we screened for:

• Biotechnology stocks that are undervalued, as measured by our price/fair value metric.
• Stocks that earn narrow or wide
  Morningstar Economic Moat Ratings
  , as well as companies that do not have a moat. We think companies with narrow economic moat ratings can fight off competitors for at least 10 years; wide-moat companies should remain competitive for 20 years or more.
• Stocks that earn a Low, Medium, High, or Very High
  Morningstar Uncertainty Rating
  , which captures the range of potential outcomes for a company’s fair value.

Here’s a little more about each of the best biotech stocks to buy, including commentary from the Morningstar analysts who cover each company. All data is as of Sept. 15, 2026.

Intellia Therapeutics

• Morningstar Price/Fair Value: 0.42
• Morningstar Uncertainty Rating: Very High
• Morningstar Economic Moat Rating: None
• Industry: Biotechnology

Intellia Therapeutics is the most affordable stock on our list of the best biotech stocks to buy. Intellia Therapeutics is a gene-editing company focused on the development of Crispr/Cas9-based therapeutics. The stock is trading 58% below our fair value estimate of $27 per share.

Intellia Therapeutics is a gene-editing company focused on the development of Crispr/Cas9-based therapeutics. Intellia’s technology platform specializes in Clustered Regularly Interspaced Short Palindromic Repeats (Crispr)/Cas9, which precisely cuts DNA to disrupt, delete, correct, and insert genes to treat genetically defined diseases. Crispr/Cas9 has created a new class of medicines, which are well suited for targeting rare diseases or other disorders that are caused by genetic mutations.

Crispr/Cas9 works by having Crispr (pieces of DNA sequences) guide Cas9 (an enzyme that can cut and edit DNA) to edit, alter, or repair genes. Intellia is utilizing this gene knockout approach to remove unwanted proteins using its proprietary lipid nanoparticle delivery system. Intellia has leveraged its expertise in Crispr/Cas9 gene editing to advance a pipeline of in vivo and ex vivo therapies for diseases with high unmet medical needs.

We believe Intellia’s proprietary technology has the potential to build blockbusters in rare diseases with limited treatment options available. Intellia currently has no approved drugs and a relatively early-stage pipeline, so we refrain from awarding the company an economic moat.

Intellia’s most advanced in vivo candidates are Nex-z (formerly NTLA-2001) for the treatment of transthyretin amyloidosis, or ATTR, and Lonvo-z (formerly NTLA-2002) for the treatment of hereditary angioedema, or HAE. Nex-z is part of a co-development and co-promotion agreement with narrow-moat Regeneron, in which Intellia is the clinical and commercial lead party, and Regeneron is the participating party. Regeneron shares in 25% of worldwide development costs and commercial profits for the ATTR program. We like that Intellia will retain 75% of the economic profits of Nex-z, if approved, and the company also has the expertise and financial support of Regeneron to offset some of the development costs. In addition, we appreciate that Lonvo-z is wholly owned by Intellia.

While Intellia does not currently have approved products, the company provides long-term investors with pure-play exposure to gene-editing technology.

Read more about Intellia Therapeutics here.

Crispr Therapeutics

• Morningstar Price/Fair Value: 0.49
• Morningstar Uncertainty Rating: Very High
• Morningstar Economic Moat Rating: None
• Industry: Biotechnology

Crispr Therapeutics is a gene-editing company focused on the development of Crispr/Cas9-based therapeutics. This cheap stock looks 51% undervalued and has a fair value estimate of $106 per share.

Crispr Therapeutics is an emerging gene-editing company focused on the development of Crispr/Cas9-based therapeutics. The company’s proprietary platform specializes in clustered regularly interspaced short palindromic repeats (Crispr)/Cas9, which precisely cuts DNA to disrupt, delete, correct, and insert genes to treat genetically defined diseases. Crispr’s emerging technology has led to a new class of therapies, which are well-suited for targeting rare diseases or other disorders that are caused by genetic mutations.

Crispr/Cas9 works by having Crispr (pieces of DNA sequences) guide Cas9 (an enzyme that can cut and edit DNA) to edit, alter, or repair genes. We think Crispr Therapeutics’ proprietary technology has the potential to build blockbusters in rare diseases with limited treatment options available.

Crispr Therapeutics is focused on developing and commercializing novel therapies to treat severe, genetic diseases and currently possesses a sizable, yet mostly early-stage pipeline. Its first approved product, Casgevy, was developed in collaboration with narrow-moat Vertex Pharmaceuticals to treat transfusion-dependent beta-thalassemia and sickle-cell disease. Crispr Therapeutics and Vertex have received regulatory approvals for Casgevy in several geographies, including the European Union, the UK, and the US. We think Casgevy’s high selling price and the significant unmet medical need to treat SCD and TDT will help it achieve blockbuster global sales over our 10-year forecast period. The rest of Crispr Therapeutics’ pipeline is in relatively early stages of development and carries very high uncertainty related to regulatory approvals, so we refrain from awarding the company an economic moat. However, Crispr Therapeutics provides long-term investors with pure-play exposure to gene editing technology.

Read more about Crispr Therapeutics here.

Ionis Pharmaceuticals

• Morningstar Price/Fair Value: 0.56
• Morningstar Uncertainty Rating: High
• Morningstar Economic Moat Rating: Narrow
• Industry: Biotechnology

Next on our list of the best biotech stocks to buy is Ionis Pharmaceuticals. Ionis Pharmaceuticals is the leading developer of antisense technology to discover and develop novel drugs. The stock is trading at a 44% discount to our fair value estimate of $84 per share.

Ionis Pharmaceuticals is a leader in RNA-based therapies. Its spinal muscular atrophy drug Spinraza, marketed by partner Biogen, is the first RNA-based therapy to achieve blockbuster status. The firm’s antisense oligonucleotide, or ASO, technology faces strong competition from RNA interference technology emerging from Alnylam, Arrowhead, and Novo Nordisk (Dicerna), as well as gene-editing and gene therapy pipelines at multiple firms. However, Ionis has built a massive pipeline of promising new drugs that are rapidly moving toward the market, securing a narrow moat.

Ionis’ therapies alter production of a given protein in the body, typically reducing production of a toxic mutant version. Therefore, Ionis can tackle diseases that are difficult to treat effectively with other methods, as its therapies are targeted (avoiding safety issues with off-target effects of small-molecule drugs), can act inside the cell (unlike antibody therapies), and are reversible (unlike gene therapy). Ionis has a broad pipeline and strong collaboration partners to help usher to market drugs for large indications, requiring large clinical trials and sales forces. Ionis spun out cardiovascular-focused Akcea in 2017 but reacquired full ownership again in 2020, given the advancement and increasing attractiveness of Akcea’s late-stage cardiology pipeline.

While first-generation ASOs had side effects that limited their commercial potential, we’re more enthusiastic about next-generation ASOs, which require much smaller doses and are easier to administer. AstraZeneca-partnered Wainua launched in 2024 in amyloidosis patients with polyneuropathy. Ionis has full rights to the high-triglyceride drug olezarsen, which received US approval in December 2024 in a rare disorder and launched in 2026 in a broader population of severe hypertriglyceridemia. Ionis also holds US rights to hereditary angioedema drug Dawnzera, which launched in the US in 2025. Partnered programs in neurology (Biogen), cardiology (Novartis), and the complement pathway (Roche) are also advancing, with multiple data readouts and regulatory milestones expected in 2026.

Read more about Ionis Pharmaceuticals here.

BioMarin Pharmaceutical

• Morningstar Price/Fair Value: 0.72
• Morningstar Uncertainty Rating: High
• Morningstar Economic Moat Rating: Narrow
• Industry: Biotechnology

BioMarin is a global biotechnology company focused on developing and commercializing therapies for rare genetic diseases. The firm earns a narrow economic moat rating, and the shares of its stock look 28% undervalued relative to our $90 fair value estimate.

BioMarin is amassing a portfolio of rare genetic-disease therapies, making historical comparisons with Genzyme (acquired by Sanofi) difficult to avoid. Commercialization and research and development expenses kept BioMarin in the red for years despite multiple approved products, but we’re confident in the durable, profit-generating power of its current portfolio. With a deep in-house pipeline and the ability to supplement growth with strategic acquisitions, BioMarin is in a strong position.

BioMarin specializes in enzyme replacement therapies for ultra-rare diseases, often with only a few thousand patients globally, yet high pricing power and barriers to entry drive annual sales in the hundreds of millions. BioMarin partnered with Genzyme to launch its first drug, Aldurazyme, for mucopolysaccharidosis I. BioMarin’s MPS VI therapy, Naglazyme, continues to grow due to higher (more expensive) dosing as young patients mature, with peak sales expected near $630 million.

In addition, BioMarin treats patients with phenylketonuria. While generic versions of Kuvan (mild to moderate PKU) launched in the US in 2020, the more potent drug Palynziq launched in 2018 in the US to serve adult patients with PKU, including those with more severe disease. PKU is generally well-diagnosed thanks to newborn screening programs, and no alternative drug therapies exist.

Voxzogo, launched in late 2021, has shown the ability to restore growth rates in young patients with achondroplasia, the most common form of dwarfism. Additional trials could extend Voxzogo’s use to other growth disorders, and we model total sales around $2 billion at peak.

In December 2025, BioMarin announced plans to acquire Amicus Therapeutics for $4.8 billion, adding two US- and EU-approved rare-disease therapies, Galafold (Fabry disease) and Pombiliti + Opfolda (Pompe disease). We see strong growth potential from leveraging BioMarin’s scale, global footprint, and commercial capabilities. We project Galafold and Pombiliti + Opfolda to each exceed $1 billion in annual sales by the end of our 10-year forecast, representing about 30% of total company revenue in 2034.

Read more about BioMarin Pharmaceutical here.

Alnylam Pharmaceuticals

• Morningstar Price/Fair Value: 0.80
• Morningstar Uncertainty Rating: High
• Morningstar Economic Moat Rating: Narrow
• Industry: Biotechnology

Alnylam Pharmaceuticals rounds out our list of best biotech stocks to buy. Alnylam Pharmaceuticals is a leader in the study of RNA interference therapeutics. The stock is 20% undervalued relative to our fair value estimate of $299 per share.

Alnylam Pharmaceuticals is a pioneer in designing therapeutics based on RNA interference, a mechanism that occurs naturally in cells and silences overexpressed genes, which often cause protein misfolding and/or protein accumulation. Alnylam seeks to capitalize on the therapeutic potential of RNAi by creating small interfering RNA, or siRNA, that can treat diseases at the genetic level. When siRNA reaches its intended target, it can silence the gene that it contains code for, making it a potentially game-changing technology for difficult- to-treat diseases caused by genetic mutations.

After over a decade of research, Onpattro, the first RNAi therapy, was approved in 2018 for polyneuropathy in hereditary ATTR amyloidosis. This positioned Alnylam as a leader among RNA-based biotech companies. Alnylam is one of only a few biotechnology companies with a systematic methodology for identifying targets and developing drug candidates. In addition to Onpattro’s 2018 approval, Alnylam received approval in 2019 for Givlaari for acute hepatic porphyria, or AHP. In 2020, Oxlumo was approved for the treatment of primary hyperoxaluria type 1 in all age groups. In June 2022, Alnylam received FDA approval for Amvuttra for the treatment of ATTR amyloidosis with polyneuropathy. In December 2021, Leqvio received approval for high cholesterol, which was developed with Alnylam’s partner Novartis. In March 2025, the FDA approved Amvuttra in ATTR-CM and Qfitlia for hemophilia A or B.

Alnylam competes with a few biotech firms that focus on RNA-based therapies, including Ionis, which received approval of Tegsedi for polyneuropathy in hATTR amyloidosis in 2018, about two months after Alnylam. Onpattro’s safety profile is much more attractive, since Tegsedi carries a black-box warning due to severe safety risks, but we expect both companies to produce next-generation candidates that could be more competitive.

Strong patient uptake and international expansion have supported Alnylam’s sales growth, but rising ATTR-CM competition from BridgeBio’s Attruby and Pfizer’s Vyndaqel and Vyndamax will likely pressure Amvuttra’s market share and pricing later in our 10-year forecast period.

Read more about Alnylam Pharmaceuticals here.

How to Find More of the Best Biotech Stocks to Buy

Investors who’d like to extend their search for top biotech stocks can do the following:

• Review Morningstar’s comprehensive list of biotechnology stocks to investigate further.
• Read Morningstar’s Guide to Stock Investing to learn how our approach to investing can inform your stock-picking process.
• Use the Morningstar Investor screener to build a shortlist of biotech stocks to research and watch.