NexEos Bio received a $3 million investment to advance NTX-1024, an ophthalmic solution for the treatment of the rare disease vernal keratoconjunctivitis (VKC)
All entries for: Investment
January 21, 2026
Mendra
Investment
Mendra raised $82 million to apply artificial intelligence to the development of rare-disease drugs, accelerating discovery and commercialization.
Disease Area: Rare Disease
January 12, 2026
Juvena Therapeutics
Investment
Juvena Therapeutics raised $79.5 million to advance protein-based therapeutics that restore cellular function and promote tissue repair for chronic and rare diseases.
Disease Area: 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
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
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
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
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
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
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