Drugs / NGN-101
NGN-101
Trials 1
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/2 | NCT05228145 | Jan 2022 → Nov 2028 expected | neuronal ceroid lipofuscinosis 5 | Neurogene Inc. | Active not recruiting | No outcome recorded |
Evidence & citations 4 cited values
Every value below carries the sentence it was read from. 1 source stands behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | NGN-101 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT05228145 ↗ |
| Modality | Gene therapy (AAV / viral vector) | “adeno-associated viral vector serotype 9 (AAV9) carrying the gene encoding human ceroid-lipofuscinosis neuronal protein 5 (CLN5)” NCT05228145 ↗ |
| Route | Other | “single intracerebroventricular (ICV) dose” NCT05228145 ↗ |
| Target | CLN5 | “encoding a codon-optimized human CLN5 transgene (hCLN5opt)” NCT05228145 ↗ |