13 Biggest Biotech Companies In Boston

Boston and neighboring Cambridge anchor one of the most concentrated biotech hubs in the world, with more than 1,000 life-science companies clustered around neighborhoods like the Seaport, Kendall Square, the Longwood Medical Area, and Alewife.

Fueled by research powerhouses such as Harvard, MIT, and the Mass General Brigham health system, the region consistently ranks among the top U.S. markets for NIH funding, venture capital investment, and life-science lab space.

In recent years, Greater Boston’s biotech ecosystem has evolved from a primarily early-stage, venture-backed scene to a dense mix of scaled public companies, commercial-stage drug makers, and big-pharma acquirers.

Kendall Square in Cambridge has become synonymous with biotech innovation—including neurology and RNA-based medicines—anchored by companies like Biogen and Moderna, while the Boston Seaport and nearby Fort Point host major headquarters and lab towers for firms such as Vertex and Ginkgo Bioworks. Longwood and the surrounding Fenway–Kenmore area connect directly into major teaching hospitals and academic medical centers, creating a short feedback loop between basic science, clinical trials, and commercial pipelines.

Against this backdrop, the region’s largest biotech players span cell and gene therapy, RNA medicines, precision oncology, and synthetic biology, and together represent tens of billions of dollars in market capitalization and annual revenue. The 13 companies highlighted below are among the most prominent anchors of this ecosystem—whether through commercial products, late-stage pipelines, or strategic acquisitions that continue to redraw Boston’s biotech map.

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Sector: Biopharmaceuticals and pharmaceuticals

HQ: 50 Northern Ave, Boston, MA

Year Founded: 1989

Key Leaders: Reshma Kewalramani, CEO and President.

Number of Employees: 6,100

Stage: Public

Financial Snapshot: $108.13 billion market cap

Notable Investors: The Vanguard Group, BlackRock, Capital World / Capital Research

Key Products: Vertex markets Trikafta, Symdeko, Orkambi, and Kalydeco for cystic fibrosis, all of which are CFTR modulators. The company is also developing VX-548 for pain, as well as gene-editing therapies for Type 1 diabetes, sickle cell disease, and Duchenne muscular dystrophy.

Recent Highlights: In April 2024, Vertex Pharmaceuticals announced it would buy Alpine Immune Sciences for about $4.9 billion. The main motivation for the merger agreement was Vertex's access to Alpine's drug, povetacicept, which shows promise in treating IgA nephropathy. Vertex hit another milestone a few months later in August 2024 when the UK's NHS agreed to cover CASGEVY, its gene-editing therapy for beta thalassemia.

Sector: Biotechnology and synthetic biology

HQ: The Innovation and Design Building at 27 Drydock Ave, Boston, MA

Year Founded: 2008

Key Leaders: Jason Kelly, Co-Founder and CEO

Number of Employees: 800–900

Stage: Public

Financial Snapshot: $709.80 million market cap

Notable Investors: Viking Global Investors, General Atlantic, and Cascade Investment

Key Products: Ginkgo Bioworks operates a cell programming platform that engineers microbes and cells to produce pharmaceuticals and other products, such as chemicals and food ingredients. Companies leveraging Ginkgo Bioworks' technology can create a variety of bio-based products. Additionally, the company provides services like pathogen monitoring.

Recent Highlights: Ginkgo has grown its synthetic biology platform through a series of impactful new deals. It received $9 million from a biologics manufacturing collaboration with Merck and another $26 million from a European contract for rapid point-of-care diagnostics. The company focuses on "cell programming as code" and has a broad platform covering diagnostics, manufacturing, and biosecurity, serving multiple markets rather than just one disease.

Sector: Genomic diagnostics and precision oncology

HQ: 400 Summer St, Boston, MA

Year Founded: 2010

Key Leaders: Daniel Malarek, CEO.

Number of Employees: 1,700+

Stage: Acquired (by Roche)

Financial Snapshot: $5.3 billion valuation at the time of acquisition (2018)

Notable Investors: Early investors included Third Rock Ventures, which led their Series A, and various VC backers before their acquisition.

Key Products: Foundation Medicine markets three different FDA-approved tests for cancer genomic profiling: FoundationOne CDx, FoundationOne Liquid CDx, and FoundationOne Heme. Each has its own purpose, and together, they help doctors create treatment plans for their patients.

Recent Highlights: Foundation Medicine has been part of Roche since 2015, which fully acquired the company in 2018 and provided over $150 million in potential R&D funding. In 2020, Foundation Medicine acquired Lexent Bio to expand its liquid biopsy capabilities using DNA methylation and low-pass whole-genome sequencing. The most significant leadership change occurred in 2023, with Dan Malarek joining as CEO of the company and Troy Schurr coming in as Chief BioPharma Business Officer. Most recently, Foundation Medicine teamed up with Manifold to accelerate drug discovery.

Sector: Genetic medicines and gene therapy HQ: 645 Summer St, Suite 200, Boston, MA Year Founded: 2016 Key Leaders: Emmanuel J. (Manny) Simons, PhD MBA, CEO and Co-Founder Number of Employees: 100+ Stage: Acquired (by Eli Lilly) Financial Snapshot: Acquisition valued at $610 million ($487 million in cash, plus CVR) Notable Investors: 5AM Ventures, New Enterprise Associates (NEA) Key Products: Akouos does not yet market any products; however, it is developing AK-OTOF, an AAV-based gene therapy for otoferlin-related hearing loss. Additional gene therapies for other forms of inherited deafness are also in development.

Recent Highlights: Eli Lilly and Company acquired Akouos in 2022 for up to approximately $610 million ($487 million in cash, plus CVR) , focusing on its inner-ear gene therapy pipeline. The lead asset, AK-OTOF, has shown early data suggesting hearing restoration in a participant just 30 days after administration. Akouos focuses exclusively on hearing and balance disorders, using AAV gene therapies that target the inner ear—a space with very few specialized competitors.

Sector: Biopharmaceuticals, with a focus on rare diseases HQ: 222 Berkeley St, Boston, MA Year Founded: 2008 Key Leaders: David Meeker, MD, Chairman, President, and CEO Number of Employees: 250+ Stage: Public Financial Snapshot: $7.57 billion market cap Notable Investors: Perceptive Advisors, HealthCare Royalty Partners, Ipsen Key Products: Rhythm markets IMCIVREE (setmelanotide) for rare genetic forms of obesity caused by POMC, PCSK1, or LEPR deficiencies. The company is also studying setmelanotide for Bardet-Biedl syndrome and other MC4R pathway-related disorders.

Recent Highlights: Rhythm reported positive topline results from its Phase 3 TRANSCEND trial of setmelanotide for acquired hypothalamic obesity, paving the way for potential approval in late 2025. The company also raised approximately $75 million through its ATM program, extending its operating runway into 2027. Rhythm focuses on ultra-rare genetic and hypothalamic forms of obesity, offering branded therapies rather than competing in the crowded general obesity market.

Sector: Pharmaceuticals, with a focus on GI and rare diseases

HQ: 100 Summer St, Suite 2300, Boston, MA

Year Founded: 1998

Key Leaders: Thomas McCourt, CEO

Number of Employees: 200–300

Stage: Public

Financial Snapshot: $540 million market cap

Notable Investors: The Vanguard Group, BlackRock, Armistice Capital

Key Products: Ironwood markets Linzess (linaclotide) for irritable bowel syndrome with constipation (IBS-C) and chronic idiopathic constipation (CIC). It is also developing IW-3300 for the treatment of abdominal pain and other gastrointestinal disorders.

Recent Highlights: Ironwood reported about $351 million in revenue for 2024, with adjusted EBITDA around $100 million. Its next-generation GLP-2 drug, Apraglutide, has initiated a rolling NDA submission after study data showed that more patients were able to wean off full feeding tubes. The company also made some organizational changes. Greg Martini was promoted to CFO, and Tammi Gaskins to Chief Commercial Officer. At the same time, the workforce was cut by close to 50 percent.

Sector: Biotechnology and pharmaceuticals, with a focus on neurology

HQ: 225 Binney St, Cambridge, MA

Year Founded: 1978

Key Leaders: Chris Viehbacher, President and CEO

Number of Employees: 7,500+

Stage: Public

Financial Snapshot: $21.89 billion market cap

Notable Investors: The Vanguard Group, Primcap Management, BlackRock

Key Products: Biogen markets Spinraza, Tysabri, Tecfidera, and Qalsody for neurological diseases like multiple sclerosis and ALS. The company is also advancing new therapies for Alzheimer’s, Parkinson’s, and rare neurodegenerative conditions.

Recent Highlights: In October of this year, Biogen acquired global rights to a preclinical program for an immune disorder from Vanqua Bio for up to $1.06 billion. The company also made regulatory progress with its Alzheimer's therapy Leqembi, developed with Eisai, which received full EU approval for early-stage Alzheimer's in March. Regarding leadership changes, Robin C. Kramer was appointed Executive Vice President and CFO following the retirement of Michael R. McDonnell.

Sector: Biotech, particularly mRNA therapeutics and vaccines

HQ: 325 Binney St, Cambridge, MA

Year Founded: 2010

Key Leaders: Stéphane Bancel, CEO

Number of Employees: Fewer than 5,000

Stage: Public

Financial Snapshot: $9.78 billion market cap

Notable Investors: The Vanguard Group, BlackRock, Baillie Gifford & Co.

Key Products: Moderna markets Spikevax, its FDA-approved mRNA COVID-19 vaccine. It is also developing a range of mRNA vaccines for RSV, influenza, and cancer, as well as mRNA-based therapeutics for rare genetic diseases.

Recent Highlights: In March of 2025, Moderna closed a $750 million investment round from Blackstone Life Sciences to support its expanded flu vaccine and mRNA pipeline, highlighting its shift following COVID. The company also partnered with IBM to integrate generative AI and quantum computing into its mRNA development platform. Additionally, the new Moderna Innovation and Technology Centre launched in the UK, further solidifying its global presence in mRNA manufacturing.

Sector: Biopharmaceuticals, particularly RNAi therapeutics

HQ: 675 West Kendall St, Cambridge, MA

Year Founded: 2002

Key Leaders: Yvonne Greenstreet, MD, CEO

Number of Employees: More than 2,100

Stage: Public

Financial Snapshot: $62.62 billion market cap

Notable Investors: The Vanguard Group, BlackRock, Capital Research Global Investors

Key Products: Alnylam markets Onpattro, Givlaari, Oxlumo, and Amvuttra, all FDA-approved RNAi therapies for rare genetic and metabolic diseases. The company is advancing next-generation RNAi medicines, including Zilebesiran for hypertension.

Recent Highlights: Alnylam hit a big regulatory milestone in March 2025 when the FDA approved its RNAi drug Amvuttra (vutrisiran) for ATTR-CM, making it the first drug approved for both heart and nerve symptoms. The company also posted strong 2024 revenue growth of 30–33% and has a broad pipeline of RNAi therapies. Alnylam’s strength comes from its RNA interference technology and multiple approved RNAi drugs, helping it lead in rare disease innovation.

Sector: Oncology and rare diseases

HQ: 45 Sidney St, Cambridge, MA

Year Founded: 2011

Key Leaders: Kate Haviland, President and CEO

Number of Employees: 650–800

Stage: Acquired in 2025 (by Sanofi)

Financial Snapshot: $8.37 billion market cap

Notable Investors: Third Rock Ventures, FMR LLC, The Vanguard Health Care Fund

Key Products: Blueprint markets Ayvakit for systemic mastocytosis and Gavreto for RET-altered cancers. The company is also developing next-generation kinase inhibitors for fibrotic diseases and oncology indications.

Recent Highlights: Blueprint Medicines' biggest financial milestone came in July of this year, when Sanofi acquired it for approximately $9.1 to $9.5 billion. The acquisition brought Blueprint's approved therapy, Ayvakit/Ayvakyt, and an advanced KIT-driven immunology pipeline into Sanofi's holdings. Another major milestone was the FDA approval of their flagship drug, AYVAKIT, for the treatment of systemic mastocytosis. This is especially noteworthy because it is the first and only medicine of its kind to receive FDA approval.

Sector: Genetic medicines for rare diseases

HQ: 215 First St, Cambridge, MA

Year Founded: 1980

Key Leaders: Douglas S. Ingram, President and CEO and a member of the board

Number of Employees: 1,300+

Stage: Public

Financial Snapshot: $2.49 billion market cap

Notable Investors: BlackRock, The Vanguard Group, D. E. Shaw & Co.

Key Products: Sarepta markets Elevidys, a gene therapy for Duchenne muscular dystrophy (DMD). It also offers Exondys 51, Vyondys 53, and Amondys 45, which are RNA-based exon-skipping therapies for DMD subtypes. Additionally, Sarepta is developing next-generation gene therapies for neuromuscular and rare diseases.

Recent Highlights: In November of last year, Sarepta signed a global licensing and collaboration deal with Arrowhead Pharmaceuticals, Inc., worth $825 million. Broken down, $500 million went toward an upfront payment, and the remaining $325 million was an equity investment in Arrowhead stock. Sarepta now has access to experimental treatments from Arrowhead's RNAi platform for diseases, and can suggest up to 6 new targets over the next 5 years using Arrowhead's RNAi platform.

Sector: RNA medicines

HQ: 733 Concord Ave, Cambridge, MA

Year Founded: 2012

Key Leaders: Paul Bolno, MD, President and CEO

Number of Employees: 250–300

Stage: Public

Financial Snapshot: $1.52 billion market cap

Notable Investors: RA Capital Management, Adage Capital Partners, BlackRock

Key Products: While Wave Life Sciences does not yet have a marketed product, it is developing WVE-003 for Huntington’s disease and WVE-N531 for Duchenne muscular dystrophy. The company is also working on other stereopure RNA therapies for neurological and rare genetic disorders.

Recent Highlights: Wave Life Sciences continues to advance its RNA medicines platform, reporting positive data in March 2025 from its Phase 2 FORWARD-53 trial of WVE-N531 in Duchenne muscular dystrophy. This trial demonstrated significant functional benefits and reversal of muscle damage. Meanwhile, the company announced that WVE-007, a GalNAc-siRNA targeting INHBE for obesity, has completed its initial dosing and is moving towards biomarker and weight-loss milestones. As for leadership shakeups, Dr. Christopher Wright was appointed as Chief Medical Officer in May 2025.

Sector: Gene editing

HQ: 11 Hurley St, Cambridge, MA

Year Founded: 2013

Key Leaders: Gilmore O'Neill, President and CEO

Number of Employees: Around 250

Stage: Public

Financial Snapshot: $284.15 million market cap

Notable Investors: The Vanguard Group, BlackRock, as well as early-stage investors that include Google Ventures and Bill Gates

Key Products: Editas Medicine’s lead program, EDIT-101, is a CRISPR gene-editing therapy for Leber congenital amaurosis (LCA10). Additional CRISPR programs for sickle cell disease, beta-thalassemia, and oncology are also in development.

Recent Highlights: In 2025, Editas Medicine made progress in its preclinical programs. Lab and primate studies showed its lipid nanoparticle platform can efficiently edit HBG1/2 promoters, supporting plans for gene editing in sickle cell and beta-thalassemia. In September of this year, the company named EDIT-401, targeting LDLR, as its lead program. Editas also hit a milestone with Bristol-Myers Squibb when its CD19 alloCAR-T IND/CTA was accepted, extending its cash runway into 2027.