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Children with CLN5, a type of late childhood Batten disease, are currently being recruited for a first-in-man clinical trial testing NGN-101, Neurogene’s experimental gene therapy for the rare neurological disease.

The trial will be conducted at the University of Rochester Medical Center (URMC) in upstate New York, the company said.

This type of disease is caused by mutations in a gene called CLN5. Also known as CLN5, the disorder is characterized by symptoms such as seizures, motor dysfunction, cognitive difficulties, and visual impairment. There is currently no approved treatment for CLN5.

NGN-101 is designed to provide a working version of the CLN5 gene to a patient’s brain cells, thereby correcting the underlying cause of the disease. The therapy works by using a modified viral vector, called an adeno-associated virus (AAV), to deliver its genetic payload to cells in the body. AAV is commonly used in gene therapy because it is easy to handle in the laboratory and does not cause disease in humans.

The Phase 1/2 clinical trial, called CLN5-200 (NCT05228145), seeks to enroll approximately three children, ages 3 to 8, who have a genetically confirmed diagnosis of CLN5. To be eligible for participation, children must have some visual acuity loss, as well as impaired motor and/or language development. Participants must also be able to walk, with or without assistance.

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During the trial, all study participants will be treated with NGN-101. The therapy is delivered via two injections, one in the brain and another in an eye, which are administered during the same procedure.

Participants will be followed for safety and efficacy results for five years after treatment with NGN-101. For the first six months after treatment, participants should reside close to the clinical trial site to facilitate follow-up and care. After that, participants will check in regularly by phone and with increasingly infrequent in-person visits to the trial site.

The study will monitor a battery of motor, language and visual function assessments, as well as safety-related outcomes.

Neurogene, which is sponsoring the trial, has a comprehensive travel and expense policy in place, whereby it will pay for trial-related fees and expenses. Although the study site is in the United States, the trial is open to children worldwide; a translator can be made available for participants who do not speak English.

More information about the trial can be obtained by contacting Neurogene by phone at 1-877-237-5020. Interested families can also contact the company by email at [email protected]while healthcare providers can contact the company at [email protected].

Plans for the trial were announced late last year, shortly after Neurogene obtained approval to begin testing from the US Food and Drug Administration (FDA).

Neurogene is also enrolling patients with CLN5 or CLN7 (another type of late infantile batten) in a natural history study (NCT03822650) that assesses how these diseases tend to progress in the absence of treatment. More information about this study can be found here.