Market map · United States

Rare disease market map: United States

36 rare disease startups based in the United States that have raised money in rounds we track, placed in 3 segments by what each says it does. Together they have raised $3.8B; the biggest segment is other rare disease therapies.

36startups on the map
3segments
$3.8Braised between them
Other rare disease therapiesmost funded segment

Other rare disease therapies

25

+1 more

Small molecules and biologics

8

Gene therapies

3

Segment by segment

Other rare disease therapies

25 startups · $2.9B raised

  1. Ultragenyx PharmaceuticalUltragenyx Pharmaceutical focuses on the development of various products for the treatment of rare and ultra-rare diseases.United States$535M
  2. SpringWorks TherapeuticsSpringWorks Therapeutics is a biopharmaceutical company that develops medicines for patients with severe rare diseases and cancer.United States$400M
  3. Fulcrum TherapeuticsFulcrum Therapeutics is a biotechnology company that focuses on identifying and treating rare genetic diseases at their root cause.United States$310M
  4. GondolaBioGondolaBio is a clinical-stage biopharmaceutical company based in Palo Alto, California.United States$300M
  5. Palvella TherapeuticsPalvella Therapeutics is a clinical-stage biopharmaceutical company based in Wayne, Pennsylvania.United States$210M
  6. Beren TherapeuticsBeren Therapeutics P.B.C.United States$165M
  7. GlycomineGlycomine is an early-stage biotech working on replacement therapies for rare diseases.United States$160M
  8. Homology MedicinesHomology 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

  1. Star TherapeuticsStar Therapeutics is a clinical-stage biotechnology company based in South San Francisco, California, founded in 2018.United States$325M
  2. Sidewinder TherapeuticsSidewinder Therapeutics is a biotechnology company that offers life-saving therapies for cancer and rare diseases.United States$137M
  3. Aardvark TherapeuticsAardvark Therapeutics specializes in small molecule therapeutics for obesity and rare genetic metabolic disorders.United States$85M
  4. Atavistik BioAtavistik Bio is a biotechnology company based in Cambridge, Massachusetts, founded in 2021.United States$80M
  5. Aeovian PharmaceuticalsAeovian Pharmaceuticals, Inc.United States$55M
  6. Allena PharmaceuticalsAllena Pharmaceuticals develops and commercializes non-systemic protein therapeutics to treat metabolic and orphan diseases.United States$43M
  7. ImmusoftImmusoft is a biotechnology company that develops novel cell therapies for therapeutic protein delivery for rare diseases.United States$29M
  8. Pierrepont TherapeuticsPierrepont Therapeutics, Inc.United States$2.4M

Gene therapies

3 startups · $135M raised

  1. AvrobioAvrobio is a clinical stage company developing step-change cell and gene therapies targeting cancer and rare disease.United States$85M
  2. GEMMABioGEMMABio is a therapeutics company that develops gene therapies for individuals living with rare diseases.United States$34M
  3. Aurora TherapeuticsAurora Therapeutics is a biotechnology company based in Cambridge, Massachusetts, specializing in personalized gene editing for raUnited 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.

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