AcuraStem received $7.5 million in grant funding to advance AS-241, an antisense oligonucleotide therapy targeting TDP-43 dysfunction, toward first-in-human clinical trials for the treatment of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD), both rare and fatal neurodegenerative diseases.
All entries for: Investment: Grant
January 6, 2026
Amphix Bio
Investment: Grant
Amphix Bio received $0.25 million in grant funding to advance AMFX-200, an FDA-designated orphan drug peptide therapeutic for the treatment of acute spinal cord injury, a rare and devastating condition affecting approximately 18,000 patients annually in the United States.
Disease Area: Rare Diseases
September 23, 2025
Trethera
Investment: Grant
Trethera received $1.8 million in grant funding to advance TRE-515, an FDA-designated orphan drug for the treatment of acute disseminated encephalomyelitis (ADEM), a rare and severe autoimmune disease of the central nervous system that primarily affects children.
Disease Area: Rare Diseases
September 22, 2025
iVeena
Investment: Grant
iVeena received $2 million in grant funding to advance IVMED-80, an FDA-designated orphan drug eye drop for the treatment of keratoconus, a rare degenerative corneal disorder.
Disease Area: Rare Diseases