Methodology

All entries for: Rare Disease

January 9, 2026

Aurora Therapeutics

Investment

Aurora Therapeutics raised $16 million to develop next-generation CRISPR-based gene-editing therapies that correct rare genetic mutations using customizable guide RNA technology.

Disease Area: Rare Disease
Source
Source
January 9, 2026

Orca Bio

Investment

Orca Bio raised $250 million to advance the commercialization of Orca-T for the treatment of hematologic malignancies.

Disease Area: Rare Disease
Source
Source
January 8, 2026

Diagonal Therapeutics

Investment

Diagonal Therapeutics raised $125 million to advance DIAG723, designed to restore ALK1 signaling in hereditary hemorrhagic telangiectasia (HHT), addressing the root cause of abnormal blood vessel formation and chronic anemia.

Disease Area: Rare Disease
Source
Source
January 7, 2026

STRM.BIO

Investment

STRM.BIO raised $8 million to advance its non-viral, cell-derived megakaryocyte vesicle delivery platform for in vivo genetic medicines, targeting rare hematologic diseases such as Fanconi anemia and CAR-T–based therapies.

Disease Area: Rare Disease
Source
Source
December 29, 2025

Nacuity Pharmaceuticals

Investment

Nacuity Pharmaceuticals raised $12.89 million to advance drug candidates that target ocular oxidative stress in retinitis pigmentosa and other rare neurological disorders, enabling clinicians and patients to access improved therapeutic options and preserve vision.

Disease Area: Rare Disease
Source
Source
December 23, 2025

Myrtelle

Investment

Myrtelle raised $30.53 to advance their gene therapy targeting oligodendrocytes to treat Canavan disease, a rare and fatal pediatric neurodegenerative disorder.

Disease Area: Rare Disease
Source
Source
December 23, 2025

GEn1E Lifesciences

Investment

GEn1E Lifesciences raised $5.52 million to accelerate first in class precision therapies for inflammatory & rare diseases.

Disease Area: Rare Disease
Source
Source
December 18, 2025

Atavistik Bio

Investment

Atavistik Bio raised $120 million to to advance its selective oral AKT1 inhibitor for hereditary hemorrhagic telangiectasia (HHT), a severe rare bleeding disorder.

Disease Area: Rare Disease
Source
Source
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
Source
Source
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
Source
Source
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