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.

Date
Date
Disease Area
Announcement Type
June 15, 2026

Sernova Biotherapeutics

Investment: Designated

prevention of diabetes due to total pancreatectomy

Disease Area: Rare Diseases
June 11, 2026

Shanghai Simnova Biotechnology Co. Ltd.

Investment: Designated

treatment of small cell lung cancer

Disease Area: Rare Diseases
June 11, 2026

Alkermes Inc.

Investment: Designated

treatment of idiopathic hypersomnia

Disease Area: Rare Diseases
June 11, 2026

GRT Therapeutics Inc.

Investment: Designated

treatment of Duchenne muscular dystrophy

Disease Area: Rare Diseases
June 10, 2026

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.

Disease Area: Rare Diseases
June 9, 2026

Ratio Therapeutics Inc.

Investment: Designated

treatment of soft tissue sarcoma

Disease Area: Rare Diseases
June 8, 2026

Langxin Qisheng (Suzhou) Biopharmaceutical Co. Ltd.

Investment: Designated

treatment of PROM1-associated retinal dystrophy

Disease Area: Rare Diseases
June 8, 2026

Kalevala Therapeutics Inc.

Investment: Designated

treatment of alternating hemiplegia

Disease Area: Rare Diseases
June 8, 2026

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.

Disease Area: Rare Diseases
June 8, 2026

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.

Disease Area: Rare Diseases
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