CRISPR-CAS9 MODIFIED HUMAN CD34+ HEMATOPOIETIC STEM AND PROGENITOR CELLS AND THEIR USES
MX434316BActive Publication Date: 2026-05-19VERTEX PHARMACEUTICALS INC
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Patent Information
- Application Number
- MX2020005849
- Authority / Receiving Office
- MX · MX
- Patent Type
- Patents
- Current Assignee / Owner
- Priority Date
- 2018-09-21
- Filing Date
- 2020-07-13
- Publication Date
- 2026-05-19
- Estimated Expiration
- 2038-12-05
Abstract
In some modalities, methods and compositions are provided herein for the treatment of subjects with β-thalassemia and subjects with severe sickle cell disease by using autologous CRISPR-Cas9-modified human CD34+ hematopoietic stem and progenitor cells.
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