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LT-HSC- based gene therapy(NGH-204)

  • 2022/04/13

NextGeM is also developing gene therapy technology to extract congenitally abnormal hematopoietic stem cells from the body after isolating LT-HSCs and amplifying outside of the body using our proprietary technology, and then introduce the necessary genes into LT-HSCs and transplant back into the patient.

The target disease is expected to be intractable rare diseases in pediatric patients.

In addition, more than 6,000 types of genetic diseases (many of which are intractable rare diseases) have been reported so far. We aim to expand the scope of this technology to a wider range of intractable rare diseases through the development of this technology in the future.