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San Rocco Therapeutics
- Ownership
- Private
- Development stage
- Phase 1
Overview
San Rocco Therapeutics has developed a gene therapy for Sickle Cell Disease and Beta Thalassemia using a lentiviral vector carrying the natural wild-type beta globin gene. The company claims superior safety compared to competitors due to lack of clonal dominance or leukemia in treated patients. The therapy aims for affordability and broad access, with plans for further clinical trials in the U.S. and Europe by late 2025[3][4][6].
- Therapeutic areas
- Hematology
- Modalities
- Gene therapy
- Industry
- Biotech
Drug pipeline
9 assets8 more assets in the full pipeline.
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