CRISPR Will be Used to Develop Genetic Medicines for Sickle Cell
July 27, 2023| |
Researchers will develop in vivo therapies for sickle cell disease and other genomic diseases. The project team will use Scribe Therapeutics' CRISPR XE technologies and Sanofi's non-viral delivery technologies.
In vivo genome editing involves directly modifying genes inside the body, and it provides numerous advantages compared to existing treatment methods and late-stage investigational therapies. One of the benefits of the in vivo approach is that it may be used to streamline sickle cell disease's treatment process.
In vivo genome editing may also reduce complications related to investigational ex vivo autologous treatments, which includes long, complex cell manufacturing processes and conditioning regimen toxicities. The in vivo approach also aims to minimize the time and cost necessary for treatment, which will boost patients' access to the technology.
Read the press release of Scribe Therapeutics for more information.
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