Top list · Worldwide
Rare diseases startups worldwide
83 rare diseases startups worldwide have raised money in rounds we track. These are the 50 that have raised the most, $5.4B between them, with the United States the most common home.
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Ultragenyx PharmaceuticalUltragenyx Pharmaceutical focuses on the development of various products for the treatment of rare and ultra-rare diseases.Ultragenyx Pharmaceutical focuses on the development of various products for the treatment of rare and ultra-rare diseases.
Founded 2010Novato, United States$535Mlast round Nov 2025 - 2
OMass TherapeuticsOMass Therapeutics is a biotechnology company located in Oxford, UK, focused on discovering and developing novel small molecule medicines for rare diseases and OMass Therapeutics is a biotechnology company located in Oxford, UK, focused on discovering and developing novel small molecule medicines for rare diseases and
Founded 2016Oxford, United Kingdom$442Mlast round Oct 2026 - 3
SpringWorks TherapeuticsSpringWorks Therapeutics is a biopharmaceutical company that develops medicines for patients with severe rare diseases and cancer.SpringWorks Therapeutics is a biopharmaceutical company that develops medicines for patients with severe rare diseases and cancer.
Founded 2017Stamford, United States$400Mlast round Dec 2023 - 4
Star TherapeuticsStar Therapeutics is a clinical-stage biotechnology company based in South San Francisco, California, founded in 2018.Star Therapeutics is a clinical-stage biotechnology company based in South San Francisco, California, founded in 2018.
Founded 2018South San Francisco, United States$325Mlast round Oct 2026 - 5
Fulcrum TherapeuticsFulcrum Therapeutics is a biotechnology company that focuses on identifying and treating rare genetic diseases at their root cause.Fulcrum Therapeutics is a biotechnology company that focuses on identifying and treating rare genetic diseases at their root cause.
Founded 2016Cambridge, United States$310Mlast round Dec 2025 - 6
GondolaBioGondolaBio is a clinical-stage biopharmaceutical company based in Palo Alto, California.GondolaBio is a clinical-stage biopharmaceutical company based in Palo Alto, California.
Founded 2024Palo Alto, United States$300Mlast round Aug 2024 - 7
Disc MedicineDisc Medicine is a biopharmaceutical company that develops therapies for hematologic diseases and rare blood disorders.Disc Medicine is a biopharmaceutical company that develops therapies for hematologic diseases and rare blood disorders.
Founded 2017Watertown, United States$275Mlast round Oct 2025 - 8
Aeglea BioTherapeuticsAeglea BioTherapeutics is a clinical-stage biotechnology company that develops enzyme therapies for rare metabolic diseases.Aeglea BioTherapeutics is a clinical-stage biotechnology company that develops enzyme therapies for rare metabolic diseases.
Founded 2015Austin, United States$266Mlast round Jun 2023 - 9
GlycomineGlycomine is an early-stage biotech working on replacement therapies for rare diseases.Glycomine is an early-stage biotech working on replacement therapies for rare diseases.
Founded 2013San Carlos, United States$160Mlast round Apr 2025 - 10
Homology MedicinesHomology Medicines is a genetic medicines company dedicated to transforming the lives of patients suffering from rare genetic diseases.Homology Medicines is a genetic medicines company dedicated to transforming the lives of patients suffering from rare genetic diseases.
Founded 2015United States$144Mlast round Nov 2020 - 11Gemini TherapeuticsGemini Therapeutics focuses on genetically-defined dry age-related macular degeneration (AMD) and associated rare genetic diseases.
Gemini Therapeutics focuses on genetically-defined dry age-related macular degeneration (AMD) and associated rare genetic diseases.
Founded 2015United States$138Mlast round Feb 2021 - 12
Sidewinder TherapeuticsSidewinder Therapeutics is a biotechnology company that offers life-saving therapies for cancer and rare diseases.Sidewinder Therapeutics is a biotechnology company that offers life-saving therapies for cancer and rare diseases.
Founded 2023San Diego, United States$137Mlast round Apr 2026 - 13
Amryt PharmaAmryt Pharma creates and delivers innovative treatments to help patients with rare and orphan diseases live better lives.Amryt Pharma creates and delivers innovative treatments to help patients with rare and orphan diseases live better lives.
Founded -Dublin, Ireland$125Mlast round Feb 2022 - 14
VectivBioVectivBio is a biotechnology company committed to making innovative treatments for patients suffering from serious rare diseases.VectivBio is a biotechnology company committed to making innovative treatments for patients suffering from serious rare diseases.
Founded 2019Basel, Switzerland$125Mlast round Oct 2022 - 15
DynacureDynacure is a clinical-stage drug development company focused on improving the lives of patients with rare and orphan diseases.Dynacure is a clinical-stage drug development company focused on improving the lives of patients with rare and orphan diseases.
Founded 2016France$109Mlast round Apr 2020 - 16MyoKardiaMyoKardia is a clinical-stage biopharmaceutical company that develops targeted therapies for the treatment of rare cardiovascular diseases.
MyoKardia is a clinical-stage biopharmaceutical company that develops targeted therapies for the treatment of rare cardiovascular diseases.
Founded 2012Brisbane, United States$98Mlast round Apr 2015 - 17
TherachonTherachon is a global biotechnology company focused on developing medicines for rare, genetic diseasesTherachon is a global biotechnology company focused on developing medicines for rare, genetic diseases
Founded 2014France$95Mlast round Aug 2018 - 18
Lassen TherapeuticsLassen Therapeutics is a clinical-stage biopharmaceutical company based in San Diego, California.Lassen Therapeutics is a clinical-stage biopharmaceutical company based in San Diego, California.
Founded 2019San Diego, United States$85Mlast round Dec 2023 - 19Edison PharmaceuticalsEdison Pharmaceuticals develops medicines for the treatments of children and adults diagnosed with rare and neglected diseases.
Edison Pharmaceuticals develops medicines for the treatments of children and adults diagnosed with rare and neglected diseases.
Founded 2005United States$84Mlast round Mar 2014 - 20
MendraMendra is an AI-native biopharmaceutical company based in San Francisco, founded in 2025.Mendra is an AI-native biopharmaceutical company based in San Francisco, founded in 2025.
Founded -San Francisco, United States$82Mlast round Jan 2026 - 21
Atavistik BioAtavistik Bio is a biotechnology company based in Cambridge, Massachusetts, founded in 2021.Atavistik Bio is a biotechnology company based in Cambridge, Massachusetts, founded in 2021.
Founded 2021Cambridge, United States$80Mlast round Mar 2026 - 22
PellePharmPellePharm is a biotechnology company dedicated to developing treatments for rare dermatologic diseases, with a particular focus on Gorlin syndrome and basal cePellePharm is a biotechnology company dedicated to developing treatments for rare dermatologic diseases, with a particular focus on Gorlin syndrome and basal ce
Founded 2016San Francisco, United States$78Mlast round Nov 2018 - 23
Protara TherapeuticsProtara Therapeutics develops transformative therapies for people with cancer and rare diseases with limited treatment options.Protara Therapeutics develops transformative therapies for people with cancer and rare diseases with limited treatment options.
Founded -New York, United States$75Mlast round Dec 2025 - 24
Lumena PharmaceuticalsLumena Pharmaceuticals develops oral therapeutics for rare liver diseases to improve liver function and relieve disease symptoms.Lumena Pharmaceuticals develops oral therapeutics for rare liver diseases to improve liver function and relieve disease symptoms.
Founded 2011San Diego, United States$71Mlast round Mar 2014 - 25
Apertura Gene TherapyApertura Gene Therapy is a biotechnology company focused on developing advanced gene therapies for rare and genetic diseases.Apertura Gene Therapy is a biotechnology company focused on developing advanced gene therapies for rare and genetic diseases.
Founded 2021New York, United Kingdom$67Mlast round Apr 2022 - 26
Actio BiosciencesActio Biosciences is a research center that develops drugs for rare diseases.Actio Biosciences is a research center that develops drugs for rare diseases.
Founded 2021San Diego, United States$66Mlast round Jun 2025 - 27
MBX BiosciencesMBX Biosciences is a preclinical-stage biotech company that creates therapies to treat rare endocrine diseases.MBX Biosciences is a preclinical-stage biotech company that creates therapies to treat rare endocrine diseases.
Founded 2019Carmel, United States$64Mlast round Aug 2024 - 28
InozymeInozyme is a biopharmaceutical company developing therapeutics for rare diseases, with a focus on bone health.Inozyme is a biopharmaceutical company developing therapeutics for rare diseases, with a focus on bone health.
Founded 2016Boston, United States$60Mlast round Jul 2023 - 29
Spruce BiosciencesSpruce Biosciences is a biopharmaceutical company developing therapies for rare diseases that target the endocrine system.Spruce Biosciences is a biopharmaceutical company developing therapies for rare diseases that target the endocrine system.
Founded 2016South San Francisco, United States$60Mlast round Apr 2026 - 30
Aeovian PharmaceuticalsAeovian Pharmaceuticals, Inc.Aeovian Pharmaceuticals, Inc.
Founded 2012Berkeley, United States$55Mlast round Dec 2025 - 31
MedopadMedopad, 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.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 2011London, United Kingdom$54Mlast round Nov 2019 - 32
Oak Hill BioOak Hill Bio is a clinical-stage biotechnology company dedicated to developing drugs for rare diseases that have been overlooked by larger pharmaceutical firms.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 2021Altrincham, United Kingdom$40Mlast round Jun 2026 - 33
Apic BioApic Bio, Inc.Apic Bio, Inc.
Founded 2017Cambridge, United States$40Mlast round Jan 2019 - 34
SuvodaSuvoda is a global clinical trial technology company specializing in complex, life-sustaining studies in therapeutic areas like oncology, central nervous systemSuvoda is a global clinical trial technology company specializing in complex, life-sustaining studies in therapeutic areas like oncology, central nervous system
Founded 2013Conshohocken, United States$40Mlast round Dec 2019 - 35
GEMMABioGEMMABio is a therapeutics company that develops gene therapies for individuals living with rare diseases.GEMMABio is a therapeutics company that develops gene therapies for individuals living with rare diseases.
Founded -Philadelphia, United States$34Mlast round Dec 2024 - 36
Coave TherapeuticsHorama develops gene therapies for rare inherited ocular and CNS (central nervous system) diseases.Horama develops gene therapies for rare inherited ocular and CNS (central nervous system) diseases.
Founded 2014Paris, France$33Mlast round Jan 2025 - 37
Scenic BiotechScenic Biotech is a biopharmaceutical company based in Amsterdam, Netherlands, founded in 2017.Scenic Biotech is a biopharmaceutical company based in Amsterdam, Netherlands, founded in 2017.
Founded 2017Amsterdam, Netherlands$31Mlast round Mar 2022 - 38
Modis TherapeuticsModis Therapeutics is bringing disease-modifying therapies to patients with rare genetic diseases.Modis Therapeutics is bringing disease-modifying therapies to patients with rare genetic diseases.
Founded 2016United States$30Mlast round Oct 2018 - 39
Citizen HealthCitient Health is an AI-powered health technology company founded in 2023, dedicated to supporting individuals and families dealing with rare and complex diseases.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 2023San Francisco, United States$30Mlast round Aug 2025 - 40
ImmusoftImmusoft is a biotechnology company that develops novel cell therapies for therapeutic protein delivery for rare diseases.Immusoft is a biotechnology company that develops novel cell therapies for therapeutic protein delivery for rare diseases.
Founded 2009Seattle, United States$29Mlast round Jan 2018 - 41
Kither BiotechKither Biotech is a biopharmaceutical company based in Turin, Italy, founded in November 2011 as a spin-off from the University of Turin.Kither Biotech is a biopharmaceutical company based in Turin, Italy, founded in November 2011 as a spin-off from the University of Turin.
Founded 2011Turin, Italy$27Mlast round Mar 2022 - 42
J2H BiotechJ2H Biotech is a bio-venture company focusing on the development of small-molecule drugs for the treatment of intractable rare diseases.J2H Biotech is a bio-venture company focusing on the development of small-molecule drugs for the treatment of intractable rare diseases.
Founded 2014Seongnam$26Mlast round Jul 2021 - 43
NFlection TherapeuticsNFlection Therapeutics is a biopharmaceutical company focusing on targeted treatments for rare diseases.NFlection Therapeutics is a biopharmaceutical company focusing on targeted treatments for rare diseases.
Founded 2014Boston, United States$20Mlast round Jun 2022 - 44
Lumos PharmaLumos Pharma is a clinical-stage biopharmaceutical company based in Austin, Texas.Lumos Pharma is a clinical-stage biopharmaceutical company based in Austin, Texas.
Founded 1999Austin, United States$20Mlast round May 2015 - 45
AdvicenneAdvicenne is a specialty pharmaceutical company dedicated to improving the lives of patients who suffer from rare kidney diseases.Advicenne is a specialty pharmaceutical company dedicated to improving the lives of patients who suffer from rare kidney diseases.
Founded 2007Paris, France$17Mlast round Mar 2017 - 46
Aurora TherapeuticsAurora Therapeutics is a biotechnology company based in Cambridge, Massachusetts, specializing in personalized gene editing for rare diseases.Aurora Therapeutics is a biotechnology company based in Cambridge, Massachusetts, specializing in personalized gene editing for rare diseases.
Founded -Cambridge, United States$16Mlast round Jan 2026 - 47
The MightyThe Mighty is a digital platform and community dedicated to health and wellness, connecting over 3.5 million registered users.The Mighty is a digital platform and community dedicated to health and wellness, connecting over 3.5 million registered users.
Founded 2014Los Angeles, United States$14Mlast round Jul 2019 - 48
Vico TherapeuticsVico Therapeutics is a clinical-stage genetic medicines company based in Leiden, the Netherlands.Vico Therapeutics is a clinical-stage genetic medicines company based in Leiden, the Netherlands.
Founded 2019Leiden, Netherlands$12Mlast round Jun 2024 - 49AAVLifeWildlife engages in developing gene therapy for the treatment of rare diseases.
Wildlife engages in developing gene therapy for the treatment of rare diseases.
Founded 2014Paris, France$12Mlast round Apr 2014 - 50
Paradox ImmunotherapeuticsParadox Immunotherapeutics is an pharmaceutical company developing immunotherapies for rare diseases using a proven-successful immunotherapy drug design platform.Paradox Immunotherapeutics is an pharmaceutical company developing immunotherapies for rare diseases using a proven-successful immunotherapy drug design platform.
Founded 2019Toronto, Canada$10Mlast round Feb 2025
Novato, United States
Oxford, United Kingdom
Dublin, Ireland
Basel, Switzerland
France
Amsterdam, Netherlands
Turin, Italy
Toronto, CanadaCompanies 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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