MyoGene Bio raised $0.4 million to advance the development of their gene editing therapy for Duchenne Muscular Dystrophy (DMD).
All entries for: Investment
October 30, 2025
Arbor Biotechnologies
Investment
Arbor Biotechnologies raised $73.9 million to advance its pipeline, including several gene therapy treatments for ALS.
Disease Area: Rare Diseases
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 21, 2025
GEn1E Lifesciences
Investment
GEn1E Life Sciences raised $11.5 million to accelerate first in class precision therapies for inflammatory & rare diseases.
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 10, 2025
Rare Disease Data Trust
Investment
Rare Disease Data Trust raised an undisclosed amount to provide rare disease diagnostic services that help patients receive fast, accurate genetic diagnoses.
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
October 7, 2025
Beren Therapeutics
Investment
Beren Therapeutics raised an undisclosed amount for the development of Adrabetadex, a treatment for Niemann-Pick disease type C (NPC).
Disease Area: Rare Diseases
September 29, 2025
ReCode Therapeutics
Investment
ReCode Therapeutics raised undisclosed funding to develop lipid nanoparticle platforms for precise delivery of genetic therapies to specific organs and tissues, offering new options for patients with life-limiting rare diseases.
Disease Area: Rare Diseases