Neurogene discontinues research program for CLN7 Batten
But the company is still enrolling patients for its CLN5 program
Neurogene has decided to discontinue its research program focusing on CLN7, a form of late-infantile Batten disease, including the CLN7 portion of its natural history study.
The goal of this natural history study (NCT03822650), focused on patients with CLN7 and CLN5 forms of Batten disease, is to help understand the disease course better and identify clinically relevant outcomes for more clinical trials.
Neurogene remains committed to its CLN5 program, which is enrolling patients in a Phase 1/2 clinical trial (NCT05228145) evaluating the experimental gene therapy NGN-101.
“We recognize the deep disappointment the CLN7 community may feel upon hearing this update,” Rachel McMinn, PhD, Neurogene’s founder and CEO, wrote in a wrote in a letter addressed to the “CLN7 Batten disease community.”
“We want the community to know this decision was not made lightly; the decision was only made after rigorous discussion with, and at the direction of, the Board of Directors,” the letter stated.
With help from the CLN7 community, the natural history study, underway at the University of Rochester Medical Center in New York, has generated important CLN7 data so far.
Neurogene is collaborating with the University of Rochester to decide how the data will be analyzed to advance the understanding of CLN7 and find ways to help researchers gain access to these data. The study also has yielded information that will be used to help develop content for CLN7 educational materials.
Neurogene is actively seeking to identify potential industry and scientific partners to continue the work on CLN7.
“We are disappointed by this development and understand this news will be deeply upsetting for many,” the Global Batten Disease Organizations on CLN7 Research wrote in a statement. “We would like to acknowledge and thank the many families, patients, and researchers who contributed to the development of this program, and hope that Neurogene will continue to seek potential industry and scientific partners to continue this work into the future.”
Neurogene said it would continue to communicate with CLN7 patient advocacy organizations to address any questions that may arise.
In CLN7, mutations in the MFSD8 gene cause the progressive neurodegeneration seen in late-infantile Batten patients. The CLN7 protein encoded by this gene normally functions as a chloride channel that regulates the function of lysosomes, the compartment in cells that degrade cellular waste.
Such mutations impair the function of lysosomes, resulting in the buildup of waste products, especially in nerve cells. Symptoms, such as seizures, speech impairment, motor and mental issues, and vision loss, emerge between 2-5 years of age.
Although there is no cure for CLN7, several treatments are available or being developed to help ease symptoms.
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