News Updates
Tracking Rare Disease Drug Development Challenges Amid a Shifting Policy Environment
Rare diseases are defined as those which affect fewer than 200,000 people in the United States. Despite advancements in rare disease research and development due to incentives created in the Orphan Drug Act more than 40 years ago, many rare diseases still lack effective treatments due to the complex science and limited market incentives involved in their development. Of the 10,000+ known rare diseases, just 5% have an FDA-approved treatment. Incubate's Rare Disease Investment Tracker measures the impacts of a challenging policy environment on rare disease R&D and notes where passage of policies like the ORPHAN Cures Act have had a positive impact.
Sernova Biotherapeutics
Investment: Designated
prevention of diabetes due to total pancreatectomy
Shanghai Simnova Biotechnology Co. Ltd.
Investment: Designated
treatment of small cell lung cancer
Alkermes Inc.
Investment: Designated
treatment of idiopathic hypersomnia
GRT Therapeutics Inc.
Investment: Designated
treatment of Duchenne muscular dystrophy
Beren Therapeutics
Investment: Later Stage VC
Beren Therapeutics raised $300 million to support the potential commercial launch of adrabetadex, an FDA Breakthrough Therapy-designated cyclodextrin therapy under Priority Review for the treatment of infantile-onset Niemann-Pick disease type C, a rare and rapidly fatal pediatric neurodegenerative disorder.
Ratio Therapeutics Inc.
Investment: Designated
treatment of soft tissue sarcoma
Langxin Qisheng (Suzhou) Biopharmaceutical Co. Ltd.
Investment: Designated
treatment of PROM1-associated retinal dystrophy
Kalevala Therapeutics Inc.
Investment: Designated
treatment of alternating hemiplegia
Infinant Health
Investment: Later Stage VC
Infinant Health raised $39.25 million to advance INF108, an FDA-designated orphan drug and rare pediatric disease-designated live biotherapeutic for the prevention of necrotizing enterocolitis, a rare and life-threatening intestinal condition primarily affecting preterm infants.
City Therapeutics
Investment: Early Stage VC
City Therapeutics raised $99.5 million to advance CITY-FXI, a next-generation RNAi therapy in Phase 1 clinical development for the prevention of thromboembolic diseases including stroke and deep vein thrombosis, as well as CITY-RBP4, an RNAi candidate for Stargardt disease, a rare inherited form of juvenile macular degeneration.