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peboctocogene camaparvovec
- Development stage
- Phase 2
- Lead developer
- Bayer
- Modality
- Viral Vectors → Gene Addition/Replacement → Gene Therapies
- Administration
- Intravenous
01
Overview
Peboctocogene camaparvovec is an investigational gene therapy designed to treat severe hemophilia A. It utilizes a recombinant, non-replicating adeno-associated virus serotype hu37 (AAVhu37) vector to deliver a codon-optimized, B-domain deleted SQ variant of the human factor VIII (F8) gene directly to liver cells. The therapy aims for functional gene replacement by enabling hepatocytes to produce and secrete functional factor VIII protein, thereby addressing the underlying deficiency in hemophilia A patients. The drug was originally developed by Dimension Therapeutics and is being further developed by Bayer, with Ultragenyx Pharmaceutical holding patent protection[2][4][5]. Clinical development has reached phase I/II trials for hemophilia A[2][5].
- Other names
- peboctocogene camaparvovec
02
Targets
F8 (Coagulation Factor VIIIa)
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