Genome Plasmid
Details
| Contract Title: | Genome Plasmid |
| Published Date: | Apr 1, 2026 |
| Notice Type: | Award (ContractAwardNotice) |
| Value Banding: | High Value |
| Delivery Point: | United Kingdom |
Description:
We are planning a clinical trial to treat a rare genetic disease that causes progressive dementia in children. Our therapy is based on the following: Autologous haematopoietic stem progenitor cells (HSPCs) are modified ex vivo with a lentiviral vector (LV) to express supraphysiologic levels of a secreted enzyme in myeloid progeny, in essence using gene-modified monocytes/macrophages as a durable and effective vehicle for central nervous system (CNS) enzyme delivery. In order to progress this t...