# Rare Disease Market Map 2026: 70 Startups in 5 Segments > Market map of 70 rare disease startups worldwide in 5 segments, from other rare disease therapies, small molecules and biologics and gene therapies on. Source: https://millionrounds.com/market-maps/rare-disease-market-map (Millionrounds; every figure on the page links to its filing or article) ## Other rare disease therapies 40 - [Ultragenyx Pharmaceutical](https://millionrounds.com/company/ultragenyx-pharmaceutical) - [SpringWorks Therapeutics](https://millionrounds.com/company/springworks-therapeutics) - [Fulcrum Therapeutics](https://millionrounds.com/company/fulcrum-therapeutics) - [GondolaBio](https://millionrounds.com/company/gondolabio) - [Palvella Therapeutics](https://millionrounds.com/company/palvella-therapeutics) - [Beren Therapeutics](https://millionrounds.com/company/beren-therapeutics) - [Glycomine](https://millionrounds.com/company/glycomine) - [Quercis Pharma](https://millionrounds.com/company/quercis-pharma) - [Homology Medicines](https://millionrounds.com/company/homology-medicines) - [*GT* Gemini Therapeutics](https://millionrounds.com/company/gemini-therapeutics) - [VectivBio](https://millionrounds.com/company/vectivbio) - [Amryt Pharma](https://millionrounds.com/company/amryt-pharma) - [Dynacure](https://millionrounds.com/company/dynacure) - [Rezolute](https://millionrounds.com/company/rezolute) - [Mendra](https://millionrounds.com/company/mendra) - [Protara Therapeutics](https://millionrounds.com/company/protara-therapeutics) - [Spruce Biosciences](https://millionrounds.com/company/spruce-biosciences) - [Inozyme](https://millionrounds.com/company/inozyme) - [*R2* River 2 Renal](https://millionrounds.com/company/river-2-renal) - [Suvoda](https://millionrounds.com/company/suvoda) - [Scenic Biotech](https://millionrounds.com/company/scenic-biotech) - [Modis Therapeutics](https://millionrounds.com/company/modis-therapeutics) - [NFlection Therapeutics](https://millionrounds.com/company/nflection-therapeutics) - [Lumos Pharma](https://millionrounds.com/company/lumos-pharma) +16 more ## Small molecules and biologics 11 - [OMass Therapeutics](https://millionrounds.com/company/omass-therapeutics) - [Star Therapeutics](https://millionrounds.com/company/star-therapeutics) - [Sidewinder Therapeutics](https://millionrounds.com/company/sidewinder-therapeutics) - [Aardvark Therapeutics](https://millionrounds.com/company/aardvark-therapeutics) - [Atavistik Bio](https://millionrounds.com/company/atavistik-bio) - [Aeovian Pharmaceuticals](https://millionrounds.com/company/aeovian-pharmaceuticals) - [Allena Pharmaceuticals](https://millionrounds.com/company/allena-pharmaceuticals) - [Immusoft](https://millionrounds.com/company/immusoft) - [J2H Biotech](https://millionrounds.com/company/j2h-biotech) - [Astrogen, inc.](https://millionrounds.com/company/astrogen-inc) - [Pierrepont Therapeutics](https://millionrounds.com/company/pierrepont-therapeutics) ## Gene therapies 8 - [Vivet Therapeutics](https://millionrounds.com/company/vivet-therapeutics) - [Avrobio](https://millionrounds.com/company/avrobio) - [GEMMABio](https://millionrounds.com/company/gemmabio) - [Aurora Therapeutics](https://millionrounds.com/company/aurora-therapeutics) - [*A* AAVLife](https://millionrounds.com/company/aavlife) - [Arctos Medical](https://millionrounds.com/company/arctos-medical) - [Alia Therapeutics](https://millionrounds.com/company/alia-therapeutics) - [BlackfinBio](https://millionrounds.com/company/blackfinbio) ## Diagnosis and genomics 8 - [GenomeUp](https://millionrounds.com/company/genomeup) - [SeqOne](https://millionrounds.com/company/seqone) - [Affinergy](https://millionrounds.com/company/affinergy) - [Saventic Health](https://millionrounds.com/company/saventic-health) - [Mendelian](https://millionrounds.com/company/mendelian) - [gMendel](https://millionrounds.com/company/gmendel) - [RareSum](https://millionrounds.com/company/raresum) - [Constantiam Biosciences](https://millionrounds.com/company/constantiam-biosciences) ## RNA and oligonucleotides 3 - [*P* PhaseRx](https://millionrounds.com/company/phaserx) - [Vico Therapeutics](https://millionrounds.com/company/vico-therapeutics) - [Amylon Therapeutics](https://millionrounds.com/company/amylon-therapeutics) ## Segment by segment ### Other rare disease therapies 40 startups · $3.5B raised - [**Ultragenyx Pharmaceutical**](https://millionrounds.com/company/ultragenyx-pharmaceutical) Ultragenyx Pharmaceutical focuses on the development of various products for the treatment of rare and ultra-rare diseases. United States $535M - [**SpringWorks Therapeutics**](https://millionrounds.com/company/springworks-therapeutics) SpringWorks Therapeutics is a biopharmaceutical company that develops medicines for patients with severe rare diseases and cancer. United States $400M - [**Fulcrum Therapeutics**](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. United States $310M - [**GondolaBio**](https://millionrounds.com/company/gondolabio) GondolaBio is a clinical-stage biopharmaceutical company based in Palo Alto, California. United States $300M - [**Palvella Therapeutics**](https://millionrounds.com/company/palvella-therapeutics) Palvella Therapeutics is a clinical-stage biopharmaceutical company based in Wayne, Pennsylvania. United States $210M - [**Beren Therapeutics**](https://millionrounds.com/company/beren-therapeutics) Beren Therapeutics P.B.C. United States $165M - [**Glycomine**](https://millionrounds.com/company/glycomine) Glycomine is an early-stage biotech working on replacement therapies for rare diseases. United States $160M - [**Quercis Pharma**](https://millionrounds.com/company/quercis-pharma) Quercis Pharma is a biopharmaceutical company that brings innovative and safe medicine to patients with orphan diseases. Switzerland $150M ### Small molecules and biologics 11 startups · $1.2B raised - [**OMass Therapeutics**](https://millionrounds.com/company/omass-therapeutics) OMass Therapeutics is a biotechnology company located in Oxford, UK, focused on discovering and developing novel small molecule me United Kingdom $442M - [**Star Therapeutics**](https://millionrounds.com/company/star-therapeutics) Star Therapeutics is a clinical-stage biotechnology company based in South San Francisco, California, founded in 2018. United States $325M - [**Sidewinder Therapeutics**](https://millionrounds.com/company/sidewinder-therapeutics) Sidewinder Therapeutics is a biotechnology company that offers life-saving therapies for cancer and rare diseases. United States $137M - [**Aardvark Therapeutics**](https://millionrounds.com/company/aardvark-therapeutics) Aardvark Therapeutics specializes in small molecule therapeutics for obesity and rare genetic metabolic disorders. United States $85M - [**Atavistik Bio**](https://millionrounds.com/company/atavistik-bio) Atavistik Bio is a biotechnology company based in Cambridge, Massachusetts, founded in 2021. United States $80M - [**Aeovian Pharmaceuticals**](https://millionrounds.com/company/aeovian-pharmaceuticals) Aeovian Pharmaceuticals, Inc. United States $55M - [**Allena Pharmaceuticals**](https://millionrounds.com/company/allena-pharmaceuticals) Allena Pharmaceuticals develops and commercializes non-systemic protein therapeutics to treat metabolic and orphan diseases. United States $43M - [**Immusoft**](https://millionrounds.com/company/immusoft) Immusoft is a biotechnology company that develops novel cell therapies for therapeutic protein delivery for rare diseases. United States $29M ### Gene therapies 8 startups · $259M raised - [**Vivet Therapeutics**](https://millionrounds.com/company/vivet-therapeutics) Vivet Therapeutics is a biotechnology company developing gene therapy treatments for orphan diseases. France $96M - [**Avrobio**](https://millionrounds.com/company/avrobio) Avrobio is a clinical stage company developing step-change cell and gene therapies targeting cancer and rare disease. United States $85M - [**GEMMABio**](https://millionrounds.com/company/gemmabio) GEMMABio is a therapeutics company that develops gene therapies for individuals living with rare diseases. United States $34M - [**Aurora Therapeutics**](https://millionrounds.com/company/aurora-therapeutics) Aurora Therapeutics is a biotechnology company based in Cambridge, Massachusetts, specializing in personalized gene editing for ra United States $16M - [*A* **AAVLife**](https://millionrounds.com/company/aavlife) Wildlife engages in developing gene therapy for the treatment of rare diseases. France $12M - [**Arctos Medical**](https://millionrounds.com/company/arctos-medical) Arctos Medical is a developed gene therapy that does not correct rare genetic eye diseases but much more common cases of blindness. - $8.2M - [**Alia Therapeutics**](https://millionrounds.com/company/alia-therapeutics) Alia Therapeutics is an innovative biotechnology company based in Trento, Italy, founded in October 2018. Italy $4.8M - [**BlackfinBio**](https://millionrounds.com/company/blackfinbio) BlackfinBio is a biotechnology company based in the UK and a spinout from the University of Sheffield. United Kingdom $3.5M ### Diagnosis and genomics 8 startups · $18M raised - [**GenomeUp**](https://millionrounds.com/company/genomeup) Digital biotech startup offers an ecosystem of AI-powered solutions for genetic analysis and clinical decision support for rare di Italy $4.9M - [**SeqOne**](https://millionrounds.com/company/seqone) SeqOne is a biotechnology company based in Montpellier, France, founded in 2017. France $3.4M - [**Affinergy**](https://millionrounds.com/company/affinergy) Affinergy is a biotechnology company based in Research Triangle Park, North Carolina, founded in 2003. United States $3.0M - [**Saventic Health**](https://millionrounds.com/company/saventic-health) Saventic Health is a med-tech company based in Toruń, Poland, founded in 2019 by Szymon Piątkowski and Professor Grzegorz Basak. Poland $2.1M - [**Mendelian**](https://millionrounds.com/company/mendelian) Mendelian is helping doctors diagnose rare genetic diseases faster. United Kingdom $1.7M - [**gMendel**](https://millionrounds.com/company/gmendel) gMendel is a medical technology company based in Denmark, founded in 2020. Denmark $1.4M - [**RareSum**](https://millionrounds.com/company/raresum) RareSum is an Istanbul-based company that specializes in AI-powered decision support platforms for diagnosing and managing rare diseases. Türkiye $670K - [**Constantiam Biosciences**](https://millionrounds.com/company/constantiam-biosciences) Preclinical biotech with deep expertise in functional genomics to map genetic variants and advance a rare disease pipeline led by CB-001. United States $485K ### RNA and oligonucleotides 3 startups · $40M raised - [*P* **PhaseRx**](https://millionrounds.com/company/phaserx) PhaseRx is a startup company focused on the delivery of siRNA and other macromolecular therapeutics for the treatment of orphan diseases. United States $27M - [**Vico Therapeutics**](https://millionrounds.com/company/vico-therapeutics) Vico Therapeutics is a clinical-stage genetic medicines company based in Leiden, the Netherlands. Netherlands $12M - [**Amylon Therapeutics**](https://millionrounds.com/company/amylon-therapeutics) Amylon Therapeutics is a biotechnology company based in the Netherlands, with a presence in Cambridge, Massachusetts. Netherlands $349K **How this map is made.** Every startup here has raised money in rounds we track. It joins the map when its sectors, read from its rounds, or the opening of its own description say it works in rare disease, and goes to the first segment whose words it carries. Each segment shows its best-funded first. Rebuilt nightly; updated 15 November 2026. ## More market maps [All maps](https://millionrounds.com/market-maps) [Investors map **Who invests in rare disease** 37 investors · 3 segments](https://millionrounds.com/market-maps/rare-disease-investors-map)[Market map · United States **Rare disease market map: United States** 36 startups · 3 segments · $3.8B](https://millionrounds.com/market-maps/rare-disease-united-states-market-map)[Market map · Europe **Rare disease market map: Europe** 19 startups · 3 segments · $695M](https://millionrounds.com/market-maps/rare-disease-europe-market-map)[Market map **Marketing tech market map** 1,963 startups · 7 segments · $41B](https://millionrounds.com/market-maps/marketing-tech-market-map)[Market map **Fintech market map** 1,633 startups · 8 segments · $139B](https://millionrounds.com/market-maps/fintech-market-map)[Market map **Crypto and Web3 market map** 1,297 startups · 8 segments · $33B](https://millionrounds.com/market-maps/crypto-and-web3-market-map) ## Who backs rare disease? 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