Blood disorders
15 public biotech companies we cover are developing treatments for sickle cell disease, thalassemia, hemophilia, PNH and other non-cancer blood conditions. 6 upcoming catalysts are listed below, with dates as the companies gave them.
Companies
- Actinium Pharmaceuticals (ATNM)Thesis Challenged
Sickle cell disease
- Agios Pharmaceuticals (AGIO)Thesis Challenged
Thalassemia, Sickle cell disease, Immune thrombocytopenia, Pyruvate kinase deficiency
- Akebia Therapeutics (AKBA)Thesis Intact
Anemia, Iron deficiency anemia
- Annexon (ANNX)Thesis Intact
Cold agglutinin disease
- Autolus Therapeutics (AUTL)Thesis Intact
Light-chain amyloidosis
- Climb Bio (CLYM)Thesis Intact
Immune thrombocytopenia
- CRISPR Therapeutics (CRSP)Thesis Intact
Beta thalassemia, Sickle cell disease, Immune thrombocytopenia, Warm autoimmune hemolytic anemia
- InflaRx (IFRX)Thesis Intact
Atypical hemolytic uremic syndrome
- Jasper Therapeutics (JSPR)Thesis Challenged
Paroxysmal nocturnal hemoglobinuria
- Metagenomi Therapeutics (MGX)Thesis Intact
Hemophilia A, Antithrombin iii deficiency
- Nuvectis Pharma (NVCT)Thesis Intact
Paroxysmal nocturnal hemoglobinuria
- Omeros (OMER)Thesis Challenged
Transplant-associated thrombotic microangiopathy
- Rocket Pharmaceuticals (RCKT)Thesis Intact
Fanconi anemia, Pyruvate kinase deficiency
- uniQure (QURE)Thesis Challenged
Hemophilia b
- vTv Therapeutics (VTVT)Thesis Intact
Sickle cell disease
Drug pages
Upcoming catalysts
Firm dates · a specific day set by the company or regulator
Next 30 days
- Nov 1in 7 daysMitapivat · FDA decision (supplemental application) · Sickle cell disease
Estimated timing · a month, quarter, half-year or year-end the company gave
Expected in Q4 2026
- Q4 2026Budoprutug · Phase 1b/2a results · Immune thrombocytopenia
- Q4 2026MGX-001 · IND submission · Hemophilia A
- 4Q 2026NXP100 · IND submission · Paroxysmal nocturnal hemoglobinuria
Expected by end of 2026
- Fall 2026ANX1502 · Enrollment complete · Cold agglutinin disease
- year-end 2026AUTO8 · Phase 1 interim results (ALARIC) · Light-chain amyloidosis