# Rare Disease Market Map United States 2026: 36 Startups in 3 Segments > 36 rare disease startups in the United States, mapped in 3 segments: other rare disease therapies, small molecules and biologics and gene therapies and more. Source: https://millionrounds.com/market-maps/rare-disease-united-states-market-map (Millionrounds; every figure on the page links to its filing or article) ## Other rare disease therapies 25 - [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) - [Homology Medicines](https://millionrounds.com/company/homology-medicines) - [*GT* Gemini Therapeutics](https://millionrounds.com/company/gemini-therapeutics) - [Rezolute](https://millionrounds.com/company/rezolute) - [Mendra](https://millionrounds.com/company/mendra) - [Protara Therapeutics](https://millionrounds.com/company/protara-therapeutics) - [Inozyme](https://millionrounds.com/company/inozyme) - [Spruce Biosciences](https://millionrounds.com/company/spruce-biosciences) - [Suvoda](https://millionrounds.com/company/suvoda) - [Modis Therapeutics](https://millionrounds.com/company/modis-therapeutics) - [NFlection Therapeutics](https://millionrounds.com/company/nflection-therapeutics) - [Lumos Pharma](https://millionrounds.com/company/lumos-pharma) - [Synspira](https://millionrounds.com/company/synspira) - [Cellevolve](https://millionrounds.com/company/cellevolve) - [Nome Bio](https://millionrounds.com/company/nome-bio) - [Patent Lee](https://millionrounds.com/company/patent-lee) - [NovelMed Therapeutics](https://millionrounds.com/company/novelmed-therapeutics) - [Cerium Pharmaceuticals](https://millionrounds.com/company/cerium-pharmaceuticals) +1 more ## Small molecules and biologics 8 - [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) - [Pierrepont Therapeutics](https://millionrounds.com/company/pierrepont-therapeutics) ## Gene therapies 3 - [Avrobio](https://millionrounds.com/company/avrobio) - [GEMMABio](https://millionrounds.com/company/gemmabio) - [Aurora Therapeutics](https://millionrounds.com/company/aurora-therapeutics) ## Segment by segment ### Other rare disease therapies 25 startups · $2.9B 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 - [**Homology Medicines**](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. United States $144M ### Small molecules and biologics 8 startups · $756M raised - [**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 - [**Pierrepont Therapeutics**](https://millionrounds.com/company/pierrepont-therapeutics) Pierrepont Therapeutics, Inc. United States $2.4M ### Gene therapies 3 startups · $135M raised - [**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 **How this map is made.** Every startup here has raised money in rounds we track, and is based in United States. 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) [Market map **Rare disease market map** 70 startups · 5 segments · $5.0B](https://millionrounds.com/market-maps/rare-disease-market-map)[Investors map **Who invests in rare disease** 37 investors · 3 segments](https://millionrounds.com/market-maps/rare-disease-investors-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 · United States **Defense tech market map: United States** 83 startups · 5 segments · $34B](https://millionrounds.com/market-maps/defense-tech-us-market-map)[Ecosystem map · United States **United States startup ecosystem map** 24,816 startups · 12 sectors · $1.6T](https://millionrounds.com/market-maps/united-states-startup-ecosystem-map)[Market map · United States **Marketing tech market map: United States** 1,041 startups · 7 segments · $30B](https://millionrounds.com/market-maps/marketing-tech-united-states-market-map) ## Who backs rare disease? Open any company for its rounds and investors, or search the investors behind these startups in the app, with filters, lists and exports. [Find their investors](https://millionrounds.com/login?join=1&next=/app/investors)[Top lists](https://millionrounds.com/top-lists)