# Top 50 Rare Diseases Startups Worldwide by Funding (2026)

> The 50 best-funded rare diseases startups worldwide, ranked by total funding raised: $5.4B combined. With founding year, headquarters and each company's rounds.

Source: https://millionrounds.com/top-lists/rare-diseases-startups (Millionrounds; every figure on the page links to its filing or article)

- 1 [**Ultragenyx Pharmaceutical** Ultragenyx Pharmaceutical focuses on the development of various products for the treatment of rare and ultra-rare diseases.](https://millionrounds.com/company/ultragenyx-pharmaceutical) Ultragenyx Pharmaceutical focuses on the development of various products for the treatment of rare and ultra-rare diseases. *Founded* 2010 Novato, United States **$535M** last round Nov 2025
- 2 [**OMass Therapeutics** OMass Therapeutics is a biotechnology company located in Oxford, UK, focused on discovering and developing novel small molecule medicines for rare diseases and](https://millionrounds.com/company/omass-therapeutics) OMass Therapeutics is a biotechnology company located in Oxford, UK, focused on discovering and developing novel small molecule medicines for rare diseases and *Founded* 2016 Oxford, United Kingdom **$442M** last round Oct 2026
- 3 [**SpringWorks Therapeutics** SpringWorks Therapeutics is a biopharmaceutical company that develops medicines for patients with severe rare diseases and cancer.](https://millionrounds.com/company/springworks-therapeutics) SpringWorks Therapeutics is a biopharmaceutical company that develops medicines for patients with severe rare diseases and cancer. *Founded* 2017 Stamford, United States **$400M** last round Dec 2023
- 4 [**Star Therapeutics** Star Therapeutics is a clinical-stage biotechnology company based in South San Francisco, California, founded in 2018.](https://millionrounds.com/company/star-therapeutics) Star Therapeutics is a clinical-stage biotechnology company based in South San Francisco, California, founded in 2018. *Founded* 2018 South San Francisco, United States **$325M** last round Oct 2026
- 5 [**Fulcrum Therapeutics** Fulcrum Therapeutics is a biotechnology company that focuses on identifying and treating rare genetic diseases at their root cause.](https://millionrounds.com/company/fulcrum-therapeutics) Fulcrum Therapeutics is a biotechnology company that focuses on identifying and treating rare genetic diseases at their root cause. *Founded* 2016 Cambridge, United States **$310M** last round Dec 2025
- 6 [**GondolaBio** GondolaBio is a clinical-stage biopharmaceutical company based in Palo Alto, California.](https://millionrounds.com/company/gondolabio) GondolaBio is a clinical-stage biopharmaceutical company based in Palo Alto, California. *Founded* 2024 Palo Alto, United States **$300M** last round Aug 2024
- 7 [**Disc Medicine** Disc Medicine is a biopharmaceutical company that develops therapies for hematologic diseases and rare blood disorders.](https://millionrounds.com/company/disc-medicine) Disc Medicine is a biopharmaceutical company that develops therapies for hematologic diseases and rare blood disorders. *Founded* 2017 Watertown, United States **$275M** last round Oct 2025
- 8 [**Aeglea BioTherapeutics** Aeglea BioTherapeutics is a clinical-stage biotechnology company that develops enzyme therapies for rare metabolic diseases.](https://millionrounds.com/company/aeglea-biotherapeutics) Aeglea BioTherapeutics is a clinical-stage biotechnology company that develops enzyme therapies for rare metabolic diseases. *Founded* 2015 Austin, United States **$266M** last round Jun 2023
- 9 [**Glycomine** Glycomine is an early-stage biotech working on replacement therapies for rare diseases.](https://millionrounds.com/company/glycomine) Glycomine is an early-stage biotech working on replacement therapies for rare diseases. *Founded* 2013 San Carlos, United States **$160M** last round Apr 2025
- 10 [**Homology Medicines** Homology Medicines is a genetic medicines company dedicated to transforming the lives of patients suffering from rare genetic diseases.](https://millionrounds.com/company/homology-medicines) Homology Medicines is a genetic medicines company dedicated to transforming the lives of patients suffering from rare genetic diseases. *Founded* 2015 United States **$144M** last round Nov 2020
- 11 [**Gemini Therapeutics** Gemini Therapeutics focuses on genetically-defined dry age-related macular degeneration (AMD) and associated rare genetic diseases.](https://millionrounds.com/company/gemini-therapeutics) Gemini Therapeutics focuses on genetically-defined dry age-related macular degeneration (AMD) and associated rare genetic diseases. *Founded* 2015 United States **$138M** last round Feb 2021
- 12 [**Sidewinder Therapeutics** Sidewinder Therapeutics is a biotechnology company that offers life-saving therapies for cancer and rare diseases.](https://millionrounds.com/company/sidewinder-therapeutics) Sidewinder Therapeutics is a biotechnology company that offers life-saving therapies for cancer and rare diseases. *Founded* 2023 San Diego, United States **$137M** last round Apr 2026
- 13 [**Amryt Pharma** Amryt Pharma creates and delivers innovative treatments to help patients with rare and orphan diseases live better lives.](https://millionrounds.com/company/amryt-pharma) Amryt Pharma creates and delivers innovative treatments to help patients with rare and orphan diseases live better lives. *Founded* - Dublin, Ireland **$125M** last round Feb 2022
- 14 [**VectivBio** VectivBio is a biotechnology company committed to making innovative treatments for patients suffering from serious rare diseases.](https://millionrounds.com/company/vectivbio) VectivBio is a biotechnology company committed to making innovative treatments for patients suffering from serious rare diseases. *Founded* 2019 Basel, Switzerland **$125M** last round Oct 2022
- 15 [**Dynacure** Dynacure is a clinical-stage drug development company focused on improving the lives of patients with rare and orphan diseases.](https://millionrounds.com/company/dynacure) Dynacure is a clinical-stage drug development company focused on improving the lives of patients with rare and orphan diseases. *Founded* 2016 France **$109M** last round Apr 2020
- 16 [**MyoKardia** MyoKardia is a clinical-stage biopharmaceutical company that develops targeted therapies for the treatment of rare cardiovascular diseases.](https://millionrounds.com/company/myokardia) MyoKardia is a clinical-stage biopharmaceutical company that develops targeted therapies for the treatment of rare cardiovascular diseases. *Founded* 2012 Brisbane, United States **$98M** last round Apr 2015
- 17 [**Therachon** Therachon is a global biotechnology company focused on developing medicines for rare, genetic diseases](https://millionrounds.com/company/therachon) Therachon is a global biotechnology company focused on developing medicines for rare, genetic diseases *Founded* 2014 France **$95M** last round Aug 2018
- 18 [**Lassen Therapeutics** Lassen Therapeutics is a clinical-stage biopharmaceutical company based in San Diego, California.](https://millionrounds.com/company/lassen-therapeutics) Lassen Therapeutics is a clinical-stage biopharmaceutical company based in San Diego, California. *Founded* 2019 San Diego, United States **$85M** last round Dec 2023
- 19 [**Edison Pharmaceuticals** Edison Pharmaceuticals develops medicines for the treatments of children and adults diagnosed with rare and neglected diseases.](https://millionrounds.com/company/edison-pharmaceuticals) Edison Pharmaceuticals develops medicines for the treatments of children and adults diagnosed with rare and neglected diseases. *Founded* 2005 United States **$84M** last round Mar 2014
- 20 [**Mendra** Mendra is an AI-native biopharmaceutical company based in San Francisco, founded in 2025.](https://millionrounds.com/company/mendra) Mendra is an AI-native biopharmaceutical company based in San Francisco, founded in 2025. *Founded* - San Francisco, United States **$82M** last round Jan 2026
- 21 [**Atavistik Bio** Atavistik Bio is a biotechnology company based in Cambridge, Massachusetts, founded in 2021.](https://millionrounds.com/company/atavistik-bio) Atavistik Bio is a biotechnology company based in Cambridge, Massachusetts, founded in 2021. *Founded* 2021 Cambridge, United States **$80M** last round Mar 2026
- 22 [**PellePharm** PellePharm is a biotechnology company dedicated to developing treatments for rare dermatologic diseases, with a particular focus on Gorlin syndrome and basal ce](https://millionrounds.com/company/pellepharm) PellePharm is a biotechnology company dedicated to developing treatments for rare dermatologic diseases, with a particular focus on Gorlin syndrome and basal ce *Founded* 2016 San Francisco, United States **$78M** last round Nov 2018
- 23 [**Protara Therapeutics** Protara Therapeutics develops transformative therapies for people with cancer and rare diseases with limited treatment options.](https://millionrounds.com/company/protara-therapeutics) Protara Therapeutics develops transformative therapies for people with cancer and rare diseases with limited treatment options. *Founded* - New York, United States **$75M** last round Dec 2025
- 24 [**Lumena Pharmaceuticals** Lumena Pharmaceuticals develops oral therapeutics for rare liver diseases to improve liver function and relieve disease symptoms.](https://millionrounds.com/company/lumena-pharmaceuticals) Lumena Pharmaceuticals develops oral therapeutics for rare liver diseases to improve liver function and relieve disease symptoms. *Founded* 2011 San Diego, United States **$71M** last round Mar 2014
- 25 [**Apertura Gene Therapy** Apertura Gene Therapy is a biotechnology company focused on developing advanced gene therapies for rare and genetic diseases.](https://millionrounds.com/company/apertura-gene-therapy) Apertura Gene Therapy is a biotechnology company focused on developing advanced gene therapies for rare and genetic diseases. *Founded* 2021 New York, United Kingdom **$67M** last round Apr 2022
- 26 [**Actio Biosciences** Actio Biosciences is a research center that develops drugs for rare diseases.](https://millionrounds.com/company/actio-biosciences) Actio Biosciences is a research center that develops drugs for rare diseases. *Founded* 2021 San Diego, United States **$66M** last round Jun 2025
- 27 [**MBX Biosciences** MBX Biosciences is a preclinical-stage biotech company that creates therapies to treat rare endocrine diseases.](https://millionrounds.com/company/mbx-biosciences) MBX Biosciences is a preclinical-stage biotech company that creates therapies to treat rare endocrine diseases. *Founded* 2019 Carmel, United States **$64M** last round Aug 2024
- 28 [**Inozyme** Inozyme is a biopharmaceutical company developing therapeutics for rare diseases, with a focus on bone health.](https://millionrounds.com/company/inozyme) Inozyme is a biopharmaceutical company developing therapeutics for rare diseases, with a focus on bone health. *Founded* 2016 Boston, United States **$60M** last round Jul 2023
- 29 [**Spruce Biosciences** Spruce Biosciences is a biopharmaceutical company developing therapies for rare diseases that target the endocrine system.](https://millionrounds.com/company/spruce-biosciences) Spruce Biosciences is a biopharmaceutical company developing therapies for rare diseases that target the endocrine system. *Founded* 2016 South San Francisco, United States **$60M** last round Apr 2026
- 30 [**Aeovian Pharmaceuticals** Aeovian Pharmaceuticals, Inc.](https://millionrounds.com/company/aeovian-pharmaceuticals) Aeovian Pharmaceuticals, Inc. *Founded* 2012 Berkeley, United States **$55M** last round Dec 2025
- 31 [**Medopad** Medopad, now known as Huma, is a global health technology company founded in 2011 and based in London, with additional offices in the US and China.](https://millionrounds.com/company/medopad) Medopad, now known as Huma, is a global health technology company founded in 2011 and based in London, with additional offices in the US and China. *Founded* 2011 London, United Kingdom **$54M** last round Nov 2019
- 32 [**Oak Hill Bio** Oak Hill Bio is a clinical-stage biotechnology company dedicated to developing drugs for rare diseases that have been overlooked by larger pharmaceutical firms.](https://millionrounds.com/company/oak-hill-bio) Oak Hill Bio is a clinical-stage biotechnology company dedicated to developing drugs for rare diseases that have been overlooked by larger pharmaceutical firms. *Founded* 2021 Altrincham, United Kingdom **$40M** last round Jun 2026
- 33 [**Apic Bio** Apic Bio, Inc.](https://millionrounds.com/company/apic-bio) Apic Bio, Inc. *Founded* 2017 Cambridge, United States **$40M** last round Jan 2019
- 34 [**Suvoda** Suvoda is a global clinical trial technology company specializing in complex, life-sustaining studies in therapeutic areas like oncology, central nervous system](https://millionrounds.com/company/suvoda) Suvoda is a global clinical trial technology company specializing in complex, life-sustaining studies in therapeutic areas like oncology, central nervous system *Founded* 2013 Conshohocken, United States **$40M** last round Dec 2019
- 35 [**GEMMABio** GEMMABio is a therapeutics company that develops gene therapies for individuals living with rare diseases.](https://millionrounds.com/company/gemmabio) GEMMABio is a therapeutics company that develops gene therapies for individuals living with rare diseases. *Founded* - Philadelphia, United States **$34M** last round Dec 2024
- 36 [**Coave Therapeutics** Horama develops gene therapies for rare inherited ocular and CNS (central nervous system) diseases.](https://millionrounds.com/company/coave-therapeutics) Horama develops gene therapies for rare inherited ocular and CNS (central nervous system) diseases. *Founded* 2014 Paris, France **$33M** last round Jan 2025
- 37 [**Scenic Biotech** Scenic Biotech is a biopharmaceutical company based in Amsterdam, Netherlands, founded in 2017.](https://millionrounds.com/company/scenic-biotech) Scenic Biotech is a biopharmaceutical company based in Amsterdam, Netherlands, founded in 2017. *Founded* 2017 Amsterdam, Netherlands **$31M** last round Mar 2022
- 38 [**Modis Therapeutics** Modis Therapeutics is bringing disease-modifying therapies to patients with rare genetic diseases.](https://millionrounds.com/company/modis-therapeutics) Modis Therapeutics is bringing disease-modifying therapies to patients with rare genetic diseases. *Founded* 2016 United States **$30M** last round Oct 2018
- 39 [**Citizen Health** Citient Health is an AI-powered health technology company founded in 2023, dedicated to supporting individuals and families dealing with rare and complex diseases.](https://millionrounds.com/company/citizen-health) Citient Health is an AI-powered health technology company founded in 2023, dedicated to supporting individuals and families dealing with rare and complex diseases. *Founded* 2023 San Francisco, United States **$30M** last round Aug 2025
- 40 [**Immusoft** Immusoft is a biotechnology company that develops novel cell therapies for therapeutic protein delivery for rare diseases.](https://millionrounds.com/company/immusoft) Immusoft is a biotechnology company that develops novel cell therapies for therapeutic protein delivery for rare diseases. *Founded* 2009 Seattle, United States **$29M** last round Jan 2018
- 41 [**Kither Biotech** Kither Biotech is a biopharmaceutical company based in Turin, Italy, founded in November 2011 as a spin-off from the University of Turin.](https://millionrounds.com/company/kither-biotech) Kither Biotech is a biopharmaceutical company based in Turin, Italy, founded in November 2011 as a spin-off from the University of Turin. *Founded* 2011 Turin, Italy **$27M** last round Mar 2022
- 42 [**J2H Biotech** J2H Biotech is a bio-venture company focusing on the development of small-molecule drugs for the treatment of intractable rare diseases.](https://millionrounds.com/company/j2h-biotech) J2H Biotech is a bio-venture company focusing on the development of small-molecule drugs for the treatment of intractable rare diseases. *Founded* 2014 Seongnam **$26M** last round Jul 2021
- 43 [**NFlection Therapeutics** NFlection Therapeutics is a biopharmaceutical company focusing on targeted treatments for rare diseases.](https://millionrounds.com/company/nflection-therapeutics) NFlection Therapeutics is a biopharmaceutical company focusing on targeted treatments for rare diseases. *Founded* 2014 Boston, United States **$20M** last round Jun 2022
- 44 [**Lumos Pharma** Lumos Pharma is a clinical-stage biopharmaceutical company based in Austin, Texas.](https://millionrounds.com/company/lumos-pharma) Lumos Pharma is a clinical-stage biopharmaceutical company based in Austin, Texas. *Founded* 1999 Austin, United States **$20M** last round May 2015
- 45 [**Advicenne** Advicenne is a specialty pharmaceutical company dedicated to improving the lives of patients who suffer from rare kidney diseases.](https://millionrounds.com/company/advicenne) Advicenne is a specialty pharmaceutical company dedicated to improving the lives of patients who suffer from rare kidney diseases. *Founded* 2007 Paris, France **$17M** last round Mar 2017
- 46 [**Aurora Therapeutics** Aurora Therapeutics is a biotechnology company based in Cambridge, Massachusetts, specializing in personalized gene editing for rare diseases.](https://millionrounds.com/company/aurora-therapeutics) Aurora Therapeutics is a biotechnology company based in Cambridge, Massachusetts, specializing in personalized gene editing for rare diseases. *Founded* - Cambridge, United States **$16M** last round Jan 2026
- 47 [**The Mighty** The Mighty is a digital platform and community dedicated to health and wellness, connecting over 3.5 million registered users.](https://millionrounds.com/company/the-mighty) The Mighty is a digital platform and community dedicated to health and wellness, connecting over 3.5 million registered users. *Founded* 2014 Los Angeles, United States **$14M** last round Jul 2019
- 48 [**Vico Therapeutics** Vico Therapeutics is a clinical-stage genetic medicines company based in Leiden, the Netherlands.](https://millionrounds.com/company/vico-therapeutics) Vico Therapeutics is a clinical-stage genetic medicines company based in Leiden, the Netherlands. *Founded* 2019 Leiden, Netherlands **$12M** last round Jun 2024
- 49 [**AAVLife** Wildlife engages in developing gene therapy for the treatment of rare diseases.](https://millionrounds.com/company/aavlife) Wildlife engages in developing gene therapy for the treatment of rare diseases. *Founded* 2014 Paris, France **$12M** last round Apr 2014
- 50 [**Paradox Immunotherapeutics** Paradox Immunotherapeutics is an pharmaceutical company developing immunotherapies for rare diseases using a proven-successful immunotherapy drug design platform.](https://millionrounds.com/company/paradox-immunotherapeutics) Paradox Immunotherapeutics is an pharmaceutical company developing immunotherapies for rare diseases using a proven-successful immunotherapy drug design platform. *Founded* 2019 Toronto, Canada **$10M** last round Feb 2025

**Total funding** how the list is ranked

**15 November 2026** updated nightly

**Rounds we track** source: Millionrounds

Companies whose own description names this theme, ranked by the total they have raised in rounds we hold. Nobody pays to be on a list.

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## More lists worldwide

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