City Therapeutics raised $99.5 million to advance CITY-FXI, a next-generation RNAi therapy in Phase 1 clinical development for the prevention of thromboembolic diseases including stroke and deep vein thrombosis, as well as CITY-RBP4, an RNAi candidate for Stargardt disease, a rare inherited form of juvenile macular degeneration.
All entries for: Investment: Early Stage VC
May 19, 2026
Oorja Bio
Investment: Early Stage VC
Oorja Bio raised $29.19 million to advance ORJ-001, a first-in-class peptide therapeutic entering Phase 2 clinical trials for the treatment of idiopathic pulmonary fibrosis, a rare and progressive lung disease with no currently available disease-modifying therapies.
Disease Area: Rare Diseases
April 21, 2026
Serif (Biotechnology)
Investment: Early Stage VC
Serif Biomedicines raised $50 million to build a Modified DNA therapeutic platform with initial programs focused on genetically defined rare diseases and immune reprogramming, representing a first-in-class approach designed to combine the durability of gene therapy with the programmability and scalability of mRNA medicines.
Disease Area: Rare Diseases
November 6, 2025
Gut-Brain Axis Therapeutics
Investment: Early Stage VC
Gut-Brain Axis Therapeutics raised $1.97 million to advance its microbiota transplant therapy (MTT), an FDA-designated orphan drug in clinical development for the treatment of Pitt-Hopkins Syndrome, a rare and severe neurodevelopmental disorder.
Disease Area: Rare Diseases
November 5, 2025
Braveheart Bio
Investment: Early Stage VC
Braveheart Bio raised $185 million to advance BHB-1893, a selective cardiac myosin inhibitor in late-stage clinical development for the treatment of obstructive hypertrophic cardiomyopathy, a rare genetic heart condition.
Disease Area: Rare Diseases
September 9, 2025
Braveheart Bio
Investment: Early Stage VC
Braveheart Bio raised an undisclosed amount to advance BHB-1893, a selective cardiac myosin inhibitor in late-stage clinical development for the treatment of obstructive hypertrophic cardiomyopathy, a rare genetic heart condition.
Disease Area: Rare Diseases
July 9, 2025
Actithera
Investment: Early Stage VC
Actithera raised $75.5 million to advance its platform for treating Familial Adenomatous Polyposis, a rare disease.
Disease Area: Rare Diseases