Report Highlights
Here are the key highlights from the report:
- Feed Scope: Companies engaged in R&D for developing medical devices, diagnostics tests, and pharmaceuticals for the diagnosis and treatment of Rare Diseases.
Companies included in the report
Companies engaged in R&D for developing medical devices, diagnostics tests, and pharmaceuticals for the diagnosis and treatment of Rare Diseases.Top Rare Diseases Companies
The top 3 companies in Rare Diseases are BridgeBio, Alnylam and PTC Therapeutics.Recent Investments
Recent noteworthy Investments in Rare Diseases sector include:- $177M, Series C round in Epicrispr Bio on Aug 11, 2026
- $155M, Series B round in AIRNA on Apr 01, 2025
- $152M, Series B round in Vaderis on Aug 11, 2026
Most Active Investors
- Seed: HHS
- Series A/B: HHS
- Series C+: BVF Partners
Top Acquisitions
- Notable acquisitions in the Rare Diseases sector include Alesta Therapeutics being acquired by BioMarin Pharmaceutical on Aug 18, 2026, Actio Biosciences acquired by Jazz Pharmaceuticals on Aug 10, 2026 and OrphAI Therapeutics acquired by Quince Therapeutics on May 18, 2026.
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