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.
All entries for: Investment
September 23, 2025
Kinea Bio
Investment
Kinea Bio raised $1.1 million to advance next-generation AAV-based gene therapies for neuromuscular diseases, leveraging engineered tissue modeling and splicing strategies to treat rare genetic disorders.
Disease Area: Rare Diseases
September 22, 2025
Star Therapeutics
Investment
Star Therapeutics raised $125 million to develop antibody-based therapeutics for bleeding disorders and other underserved conditions, using proprietary platforms to provide targeted, patient-centered treatments.
Disease Area: Rare Diseases
September 22, 2025
Ensoma
Investment
Ensoma raised $53 million to develop in vivo genomic medicines that deliver gene modification technologies directly to blood and immune cells, offering potentially curative treatments in a single injection for rare diseases.
Disease Area: Rare Diseases
September 19, 2025
SignaBlok
Investment
SignaBlok raised $0.15 million to develop targeted peptide therapeutics and integrated delivery systems that block cell receptor signaling, enabling innovative treatments for serious diseases with unmet medical needs, including scleroderma.
Disease Area: Rare Diseases
September 16, 2025
Hopewell Therapeutics
Investment
Hopewell Therapeutics raised $2.5 million to develop tissue-targeted lipid nanoparticles (ttLNPs) for delivering genomic medicines. Their technology aims to precisely target cells throughout the body, enabling potential treatments for rare genetic diseases, lung disorders, oncology, and neurological conditions.
Disease Area: Rare Diseases
September 16, 2025
AllRock Bio
Investment
AllRock Bio raised $50 million to advance ROC-101, an oral therapy for pulmonary hypertension (PH) and related lung diseases. The drug works by relaxing blood vessels, reducing inflammation, and preventing tissue scarring, offering a potential new option for patients with these serious rare conditions.
Disease Area: Rare Diseases
September 13, 2025
Nacuity Pharmaceuticals
Investment
Nacuity Pharmaceuticals raised $4.5 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 Diseases
September 12, 2025
Nephrogen
Investment
Nephrogen raised $0.22 million to engineer AI-driven gene delivery vectors for kidney and pancreatic diseases, enabling researchers and clinicians to advance genomic therapies and improve patient outcomes.
Disease Area: Rare Diseases
September 10, 2025
SignaBlok
Investment
SignaBlok raised $0.5 million to develop targeted peptide therapeutics and integrated delivery systems that block cell receptor signaling, enabling innovative treatments for serious diseases with unmet medical needs, including scleroderma.
Disease Area: Rare Diseases
