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.
All entries for: Investment
October 29, 2025
Rarity (Biotechnology)
Investment
Rarity Therapeutics raised $4.6 million to advance RDP-101, an ex vivo autologous hematopoietic stem cell gene therapy designed to restore immune function in patients with rare genetic diseases such as ADA-SCID.
Disease Area: Rare Diseases
October 23, 2025
ReCode Therapeutics
Investment
ReCode Therapeutics raised $31 million to advance its pipeline of therapies, including RCT1100, an inhaled mRNA treatment for Primary Ciliary Dyskinesia (PCD), a rare genetic disorder that causes chronic respiratory infections and progressive lung function decline.
Disease Area: Rare Diseases
October 15, 2025
Electra Therapeutics
Investment
Electra Therapeutics raised $183 million to advance its pipeline of SIRP-targeted therapies, including ELA026 for the rare hyperinflammatory disease secondary hemophagocytic lymphohistiocytosis (sHLH), supporting pivotal clinical trials and further development of its novel immune-cell–depleting treatments.
Disease Area: Rare Diseases
October 9, 2025
PharmassetX
Investment
PharmassetX raised $0.05 million to develop PSX-514, a novel small-molecule drug and an FDA-designated orphan therapy for pouchitis, aimed at addressing a significant unmet need in the treatment of chronic inflammatory diseases.
Disease Area: Rare Diseases