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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 designed to address the underlying genetic cause of this condition, which primarily affects the nervous system in children.
NGN-101 is being developed by Neurogene Inc., a biopharmaceutical company focused on 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 therapy, meaning it has not been approved by regulatory authorities and is still being studied for safety and efficacy in clinical trials.
NGN-101 is being evaluated 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, and is being conducted in the United States and United Kingdom.
NGN-101 is a gene therapy designed to deliver a functional copy of the CLN5 gene to cells. This approach aims to correct the genetic deficiency that causes CLN5 Batten disease, potentially slowing or halting disease progression. The therapy is administered to children aged 3 years and older.