Atavistik Bio raised $120 million to to advance its selective oral AKT1 inhibitor for hereditary hemorrhagic telangiectasia (HHT), a severe rare bleeding disorder.
All entries for: Investment
December 17, 2025
Addition Therapeutics
Investment
Addition Therapeutics raised $106.5 million to advance its all-RNA PRINT™ platform and expand genomic medicine approaches for both chronic and rare diseases.
Disease Area: Rare Disease
December 16, 2025
Ambros Therapeutics
Investment
Ambros Therapeutics raised $154 million to further develop its lead investigational program for complex regional pain syndrome type 1 (CRPS-1), a rare and debilitating metabolic bone disease.
Disease Area: Rare Disease
December 1, 2025
Endios Bio
Investment
Baton Rouge, LA
1-50 employees
Endios Bio raised an undisclosed amount of funding to advance its first-in-class PEG10-targeted therapeutics for amyotrophic lateral sclerosis (ALS) and related neurodevelopmental disorders.
Disease Area: Rare Diseases
December 1, 2025
Protego Biopharma
Investment
Protego Biopharma raised $130 million to advance PROT-001 into a pivotal clinical trial for AL amyloidosis, a rare and often fatal protein misfolding disorder that leads to progressive organ damage, particularly in the heart.
Disease Area: Rare Disease
December 1, 2025
Mantle Therapeutics
Investment
Mantle Bio raised an undisclosed amount of funding to develop therapies for rare, fatal diseases lacking curative options.
Disease Area: Rare Disease
November 24, 2025
Neela Therapeutics
Investment
Neela Therapeutics raised $12.01 million to develop a pediatric central nervous system therapy designed to address rare neuro-genetic diseases.
Disease Area: Rare Diseases
November 21, 2025
Phoenics
Investment
Phoenics raised $13.81 million to develop therapies for patients living with rare diseases.
Disease Area: Rare Diseases
November 13, 2025
Elixirgen Therapeutics
Investment
Elixirgen Therapeutics raised $8.2 million toward developing therapies using ZSCAN4 to extend telomeres in stem cells for patients with Telomere Biology Disorders, a rare disease, and is also advancing its Bobcat mRNA technology to deliver large therapeutic genes, including full-length dystrophin for Duchenne muscular dystrophy.
Disease Area: Rare Diseases
November 10, 2025
Arbor Biotechnologies
Investment
Arbor Biotechnologies raised $73.9 million to advance its pipeline, including several gene therapy treatments for ALS.
Disease Area: Rare Diseases