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NGN-101 · 1 trial · 1 indication
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| Arm | Type | Description |
|---|---|---|
| Cohort 1 | EXPERIMENTAL | The study treatment is a recombinant serotype 9 adeno-associated virus encoding a codon-optimized human CLN5 transgene (hCLN5opt). |
| Cohort 2 | EXPERIMENTAL | The study treatment is a higher dose of recombinant serotype 9 adeno-associated virus encoding a codon-optimized human CLN5 transgene (hCLN5opt). |
| Cohort 3 | EXPERIMENTAL | The study treatment is a higher dose of recombinant serotype 9 adeno-associated virus encoding a codon- optimized human CLN5 transgene (hCLN5opt). |
| Name | Type | Description |
|---|---|---|
| NGN-101 | GENETIC | Participants with confirmed mutations in the CLN5 gene who meet all the inclusion and none of the exclusion criteria will be treated with a single intracerebroventricular (ICV) dose and a single intravitreal (IVT) dose of the study treatment. |
Inclusion Criteria * Age from 3 to 9 years (Child) * Molecular genetic diagnosis of the CLN5 gene * Confirmed clinical diagnosis of CLN5 disease * Impaired motor and/or language function and/or impaired visual acuity * Written informed consent from parent or legal guardian and assent from study par...
NGN-101 is an investigational gene therapy being developed for Neuronal Ceroid Lipofuscinosis CLN5, a rare genetic disorder also known as CLN5 Batten disease. It is intended to treat children with this condition, which affects the nervous system and causes progressive loss of motor and cognitive function.
NGN-101 targets the CLN5 gene. It is designed to deliver a functional copy of the CLN5 gene to address the underlying genetic cause of CLN5 Batten disease. The therapy aims to restore the missing or defective CLN5 protein function in patients.
NGN-101 is being developed by Neurogene Inc., a biopharmaceutical company focused on gene therapies for rare neurological diseases. Neurogene is publicly traded on the Nasdaq under the ticker symbol NGNE.
NGN-101 is currently in Phase 1 clinical development. It is an investigational drug and has not been approved by the FDA. The ongoing Phase 1 trial is active but not recruiting participants, indicating that enrollment has been completed.
NGN-101 is being studied in a Phase 1 clinical trial with the identifier NCT05228145, titled "Gene Therapy Study for Children With CLN5 Batten Disease." The trial is active but not recruiting, with an enrollment of 6 participants. It is being conducted in the United States and United Kingdom.
NGN-101 is a gene therapy designed to treat CLN5 Batten disease by targeting the CLN5 gene. It is specifically developed for Neuronal Ceroid Lipofuscinosis CLN5, and the ongoing clinical trial is referred to as a gene therapy study for this condition. No other alternative names for NGN-101 have been disclosed.