Drug intelligence / Profile preview

CNS10-NPC-GDNF

Development stage
Phase 1
Lead developer
Cedars-Sinai
Modality
Cell Therapies, Gene Therapies
Administration
Intracerebral
01

Overview

CNS10-NPC-GDNF is a combined cell and gene therapy that involves transplanting neural progenitor cells genetically engineered to produce glial cell line-derived neurotrophic factor (GDNF) into the motor cortex of patients with amyotrophic lateral sclerosis (ALS). GDNF is a protein that promotes the survival of neural cells. The therapy aims to protect motor neurons and improve the quality of life for ALS patients.

Other names
Human Neural Progenitor Cells Secreting Glial Cell Line-Derived Neurotrophic Factor
02

Targets

GFRA1 (Glial cell line-derived neurotrophic factor family receptor alpha 1)

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