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.
All entries for: Investment
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
Boston, MA
51-200 employees
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 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 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 10, 2025
Dewpoint Therapeutics
Investment
Dewpoint Therapeutics raised $59.12 million to advance DPTX3186 into clinical trials for gastric cancer and accelerate development of another drug targeting MYC. The funding supports ongoing research and operations through early 2027, enabling the company to bring new therapies for rare and difficult-to-treat cancers closer to patients.
Disease Area: Rare Diseases
September 5, 2025
SciTech Development
Investment
SciTech Development raised $5.5 million to advance ST-001 nanoFenretinide, a nanoparticle therapy for rare cancers including T-cell lymphoma. Its delivery platform improves drug effectiveness and safety, and the therapy has received FDA Orphan Drug Designation.
Disease Area: Rare Diseases
August 27, 2025
Wugen
Investment
Wugen raised $115 million to advance WU-CART-007, an allogeneic CAR-T therapy for rare T-cell cancers including T-cell acute lymphoblastic leukemia (T-ALL) and T-cell lymphoblastic lymphoma (T-LBL). The therapy uses gene editing to improve safety and scalability, enabling broader patient access to potentially life-saving treatment.
Disease Area: Rare Diseases
August 26, 2025
Superluminal Medicines
Investment
Superluminal Medicines raised $5 million to develop next-generation small-molecule GPCR-targeted therapies for endocrine and cardiometabolic diseases
Disease Area: Rare Diseases
August 12, 2025
MyOme
Investment
MyOme received an undisclosed amount of funding to expand its rare disease testing, allowing it to identify more genetic conditions through more advanced genome analysis.
Disease Area: Rare Diseases