About Rare Diseases
There are 729 companies in the Rare Diseases sector across the world, with 518 funded companies having collectively raised $53B in venture capital money and private equity. Overall 355 of these are Series A+ funded and the sector has seen the creation of 3 Unicorns.
Rare Diseases sector has seen 129 acquisitions and 99 IPOs. This represents almost 31.3% of all such companies - which is higher than the 4.0% exit rate of tech companies in general.
This sector includes companies engaged in R&D for developing medical devices, diagnostics tests, and pharmaceuticals for the diagnosis and treatment of Rare Diseases.
Rare Diseases sector is also known as Rare Diseases Treatment or Orphan Diseases.
Companies included in Rare Diseases sector
Medical devices, diagnostics tests, and pharmaceuticals for the diagnosis and treatment of rare diseasesCompanies excluded in Rare Diseases sector
Healthcare Service Providers, Digital Health Solutions (software, apps, online platforms)Market Map of Rare Diseases
The market map of Rare Diseases gives a visual overview of the sector, showing key business models, their interactions in the value chain, and industry evolution.
Reports related to Rare Diseases
Here are some reports related to Rare Diseases sector:
Free
Rare Diseases - Sector Report
Edition: September, 2026 (67 Pages)
Premium
Rare Diseases Investor Landscape
Edition: May, 2025 (12 Pages)
Premium
Rare Diseases Exit Landscape
Edition: August, 2026 (15 Pages)
View all reports related to Rare Diseases. Sign up today!Top Companies in Rare Diseases
There are more than 729 companies in Rare Diseases. BridgeBio is the top Rare Diseases company globally, ranked using Tracxn Score (what's this?).
Here are 5 of the top Rare Diseases companies globally:
Company | Description | Location | Founded | Current Stage | Private Funding Raised |
|---|---|---|---|---|---|
Developer of therapeutics for the treatment of genetic diseases | Palo Alto (United States) | 2015 | Public | $434M | |
Developer of RNA interference-based therapeutics for multiple disorders | Cambridge (United States) | 2002 | Public | $17M | |
Developer of small molecule drugs for the treatment of rare diseases | South Plainfield (United States) | 1998 | Public | $248M | |
Developer of small molecule drugs for the treatment of cancer | Boston (United States) | 2014 | Public | $58.2M | |
Developer of gene therapy for rare diseases | Philadelphia (United States) | 2013 | Acquired | $82.8M |
Companies founded year-on-year in the Rare Diseases sector
10 new startups have been founded so far in 2026 in the Rare Diseases sector. 52 Rare Diseases startups were founded in 2017 - this is the most number of startups founded in any year in the last 10 years.
- Total Number of Companies
Year | No. of companies |
|---|---|
2026 | 10 |
2025 | 3 |
2024 | 9 |
2023 | 10 |
2022 | 19 |
2021 | 37 |
2020 | 37 |
2019 | 44 |
2018 | 42 |
2017 | 52 |
Companies founded across countries in the Rare Diseases sector
United States has 416 Rare Diseases startups, the most by any country, followed by United Kingdom which has 38 startups and South Korea which has 26.
- Total Number of Companies
Country | No. of companies |
|---|---|
United States | 416 |
United Kingdom | 38 |
South Korea | 26 |
France | 25 |
Canada | 23 |
Switzerland | 23 |
China | 20 |
Israel | 17 |
Sweden | 17 |
Netherlands | 17 |
Stage-wise distribution of the Rare Diseases sector
Out of the total 729 Rare Diseases companies, 518 have secured funding. Among them, 355 have reached Series A or higher, and 301 to Series B or beyond.
Founded - 729
Funded - 518
Series A+ - 355
Series B+ - 301
Series C+ - 256
Company Stage | No. of companies |
|---|---|
Founded | 729 |
Funded | 518 |
Series A+ | 355 |
Series B+ | 301 |
Series C+ | 256 |
Want to know more about Rare Diseases ? Signup and explore Tracxn for free
- 725+ Total Companies
- $53B+ Total Funding
- 515+ Funded Companies
- 95+ IPOs
- 125+ Acquisitions
- 20+ Reports
Funding Trends in the Rare Diseases sector
Total funding year-on-year in the Rare Diseases sector
The Rare Diseases sector saw a total funding of more than $36B in the last 10 years. The most amount of funding was in 2018 at more than $7.25B. There has been a total funding of $1.69B in 2026 till date.
- Total Funding
Year | Total Funding |
|---|---|
2026 | $1.69B |
2025 | $1.38B |
2024 | $1.4B |
2023 | $1.2B |
2022 | $3.69B |
2021 | $4.54B |
2020 | $6.87B |
2019 | $4.89B |
2018 | $7.25B |
2017 | $3.07B |
Total funding across countries in the Rare Diseases sector
In the last 10 years, Rare Diseases startups in United States have recieved the highest funding among all countries - $21.1B. Switzerland follows with $5.19B and China with $1.89B.
- Total Funding
Country | Total Funding |
|---|---|
United States | $21.1B |
Switzerland | $5.19B |
China | $1.89B |
United Kingdom | $1.68B |
Sweden | $768M |
Canada | $625M |
Netherlands | $607M |
France | $556M |
South Korea | $155M |
Japan | $107M |
Total funding amount raised by stage in the Rare Diseases sector
Over the last 10 years, the Rare Diseases sector have seen $228M into Seed Stage funding rounds, $10B into Early Stage funding rounds and $5.08B go into Late Stage funding rounds.
Funding Stage | Total Funding |
|---|---|
Post IPO | $12.1B |
Early Stage | $10B |
Debt | $5.79B |
Late Stage | $5.08B |
Grant | $646M |
PE | $335M |
Seed Stage | $228M |
Recent Funding Rounds in Rare Diseases
In the year 2026, till September 2026, Rare Diseases companies have raised $1.4B in equity funding across 23 rounds.
In the same period last year (i.e. till September 2025), Rare Diseases companies had raised $872M across 24 rounds.
So, 2026 has seen a 60.25% rise in funding in Rare Diseases companies as compared to 2025.
Here are some recent, important funding rounds in Rare Diseases:
Company | Round Raised | Amount Raised | Funding Date | Investors |
|---|---|---|---|---|
Series A | 7308 | Sep 01, 2026 | ||
Series B | 5777 | Aug 11, 2026 | and 7 more | |
Series C | 9410 | Aug 11, 2026 | ||
Series B | 7682 | Jul 29, 2026 | ||
Series C | 7345 | Jul 23, 2026 | - |
Top Investors in Rare Diseases
HHS is the top investor in Rare Diseases companies based on number of companies invested.
Here are some top investors in Rare Diseases:
Investor | Location | Top Portfolio Companies |
|---|---|---|
Washington, DC (United States) | ||
New York City (United States) | ||
Menlo Park (United States) | ||
Boston (United States) | ||
M&A activity in Rare Diseases
In the year 2026, till September 2026, 8 acquisitions happened in Rare Diseases sector. Last year, in 2025, a total of 7 acquisitions happened in Rare Diseases sector.
Here are some recent, important acquisitions in Rare Diseases:
Company | Acquisition Type | Acquisition Date | Acquirers |
|---|---|---|---|
Business Acquisition | Sep 22, 2026 | ||
Business Acquisition | Aug 18, 2026 | ||
Business Acquisition | Aug 10, 2026 | ||
Business Acquisition | May 18, 2026 | ||
Business Acquisition | May 07, 2026 |
Acquisition trends in Rare Diseases
Here are the acquisition trends in Rare Diseases:
- Total Number of Acquisitions
Year | No. of companies |
|---|---|
2026 | 8 |
2025 | 7 |
2024 | 5 |
2023 | 9 |
2022 | 16 |
2021 | 12 |
2020 | 9 |
2019 | 15 |
2018 | 7 |
2017 | 9 |
IPOs in Rare Diseases
In the year 2026, till August 2026, 3 IPOs happened in Rare Diseases sector.
Here are some recent, important IPOs in Rare Diseases:
Company | IPO Date | Exchange Symbol |
|---|---|---|
Aug 12, 2026 | National Association of Securities Dealers Automated Quotations (NASDAQ) | |
Jun 09, 2026 | National Association of Securities Dealers Automated Quotations (NASDAQ) | |
Feb 02, 2026 | National Association of Securities Dealers Automated Quotations (NASDAQ) | |
Dec 18, 2025 | Korea Exchange (KRX) | |
Dec 03, 2024 | National Association of Securities Dealers Automated Quotations (NASDAQ) |
Colleges which produce the most Rare Diseases companies
Companies founded by alumni from top colleges in the Rare Diseases sector
Most number of Rare Diseases startups have been founded by alumni from Massachusetts Institute of Technology, University of California (Berkeley) and Harvard University.
College | No. of companies |
|---|---|
Massachusetts Institute of Technology | 17 |
University of California (Berkeley) | 15 |
Harvard University | 14 |
Stanford University | 11 |
University of Pennsylvania | 9 |
University of Chicago | 6 |
Cornell University | 5 |
University of California (Los Angeles) | 4 |
University of Cambridge | 4 |
Wharton School | 4 |
Total funding in companies founded by alumni from top colleges in the the Rare Diseases sector
In the last 10 years, Massachusetts Institute of Technology graduates' Rare Diseases companies have raised the most funding among top colleges. Stanford University alumni are second, and Harvard University alumni are third.
College | Total Funding |
|---|---|
Massachusetts Institute of Technology | $4.3B |
Stanford University | $2.56B |
Harvard University | $2.27B |
University of Chicago | $1.87B |
University of Cambridge | $1.42B |
Stanford Business School | $1.35B |
University of California (Berkeley) | $1.33B |
Cornell University | $565M |
University of Pennsylvania | $532M |
University of Oxford | $383M |
Top News in Rare Diseases
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New Drug Offers Treatment Option for Rare Bone DiseaseProPakistani•Sep 28, 2026•Mirum Pharmaceuticals
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Sobi to Present Clinical Data on Gamifant® at Histiocyte Society 2026PR Newswire•Sep 24, 2026•Swedish Orphan Biovitrum
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aTyr Pharma Announces Alignment with FDA on Phase 3 Study of Efzofitimod in Pulmonary SarcoidosisGlobeNewswire•Sep 24, 2026•aTyr Pharma
