Beren Therapeutics raised $300 million to support the potential commercial launch of adrabetadex, an FDA Breakthrough Therapy-designated cyclodextrin therapy under Priority Review for the treatment of infantile-onset Niemann-Pick disease type C, a rare and rapidly fatal pediatric neurodegenerative disorder.
All entries for: Investment: Later Stage VC
June 8, 2026
Infinant Health
Investment: Later Stage VC
Infinant Health raised $39.25 million to advance INF108, an FDA-designated orphan drug and rare pediatric disease-designated live biotherapeutic for the prevention of necrotizing enterocolitis, a rare and life-threatening intestinal condition primarily affecting preterm infants.
Disease Area: Rare Diseases
May 27, 2026
Secretome Therapeutics
Investment: Later Stage VC
Secretome Therapeutics raised $30 million to advance STM-01, a neonatal cardiac progenitor cell therapy in clinical development for the treatment of Duchenne muscular dystrophy-associated cardiomyopathy, a rare and fatal genetic disease in which cardiac complications are among the leading causes of death.
Disease Area: Rare Diseases
May 7, 2026
Illexcor Therapeutics
Investment: Later Stage VC
Illexcor Therapeutics raised $13 million to advance ILX002, a first-in-class oral hemoglobin S polymerization inhibitor approaching first-in-human clinical trials for the treatment of sickle cell disease, a rare and debilitating hereditary blood disorder.
Disease Area: Rare Diseases
April 21, 2026
Ray Therapeutics
Investment: Later Stage VC
Ray Therapeutics raised $125 million to advance RTx-015, a first-in-class optogenetic gene therapy granted FDA Regenerative Medicine Advanced Therapy designation for the treatment of retinitis pigmentosa, a rare inherited retinal disease causing progressive vision loss and blindness.
Disease Area: Rare Diseases
April 13, 2026
Quiver Bioscience
Investment: Later Stage VC
Quiver Bioscience received an undisclosed investment to advance its UBE3A antisense oligonucleotide program toward a development candidate for the treatment of chromosome 15q duplication syndrome, a rare neurodevelopmental disorder.
Disease Area: Rare Diseases
February 5, 2026
ImmunoMet
Investment: Later Stage VC
ImmunoMet raised $0.84 million to advance Lixumistat, an FDA-designated orphan drug in clinical trials for the treatment of pancreatic cancer and glioblastoma multiforme, two rare and difficult-to-treat malignancies with significant unmet need.
Disease Area: Rare Diseases
January 6, 2026
Mediar Therapeutics
Investment: Later Stage VC
Mediar Therapeutics raised $75.7 million to advance MTX-463, an FDA-designated orphan drug antibody for the treatment of idiopathic pulmonary fibrosis, a rare and progressive scarring disease of the lungs.
Disease Area: Rare Diseases
December 4, 2025
Aminex Therapeutics
Investment: Later Stage VC
Aminex Therapeutics raised $15.08 million to advance AMXT 1501 in combination with DFMO, an FDA-designated orphan drug regimen in clinical trials for the treatment of neuroblastoma, a rare and aggressive pediatric cancer of the nervous system.
Disease Area: Rare Diseases
September 5, 2025
SciTech Development
Investment: Later Stage VC
SciTech Development raised $5.5 million to advance ST-001 nanoFenretinide, an FDA-designated orphan drug in clinical trials for the treatment of T-cell non-Hodgkin lymphoma, a rare and aggressive blood cancer.
Disease Area: Rare Diseases