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The US Food and Drug Administration (FDA) designated Neurogenicgene therapy, which aims to provide a working copy of the human CLN5 gene, an orphan drug to potentially treat CLN5, a form of Batten disease in late children.

Orphan drug This status helps encourage therapies for rare and serious diseases, through benefits such as seven years of market exclusivity and exemption from FDA filing fees.

“Receiving orphan drug designation from the FDA is an important regulatory step, and we look forward to advancing our gene therapy program in the clinic,” said Rachel McMinn, PhD, Founder and CEO of Neurogene, in a statement. Press release.

Batten’s disease, also called neuronal ceroid lipofuscinosis (NCL) is an umbrella term for rare inherited neurological diseases caused by mutations that impair the ability of cells to get rid of wastes.

This leads to the build-up of toxic deposits made up of fats and proteins, called lipofuscins, throughout the body, especially in nerve cells in the brain and cells in the eye.

Neurogene’s gene therapy uses a adeno-associated virus (AAV), a harmless virus that is not known to cause disease and effective as a delivery vehicle because it easily enters cells. Advances in the design of AAV vectors have shown that these vehicles can enable large-scale gene delivery to the brain and spinal cord, making them particularly useful for neurological diseases.

The company’s CLN5 gene therapy is currently on a new investigational drug enabling phase, which means it goes through the multiple studies required for regulatory clearance to begin clinical trials in patients.

CLN5, along with other types of late childhood Batten disease, is characterized by seizures, progressive deterioration of motor and cognitive abilities, vision loss, and premature death.

While the exact function of the CLN5 protein is unclear, previous preclinical studies have suggested its role in fat metabolism and in the maintenance of myelin, the fat-rich substance that envelops nerve fibers (axons), as well. than in the transport of proteins.

More recent preclinical evidence has shown that the loss of the CLN5 protein – due to mutations in the CLN5 gene – impairs the production of new, fully functional neurons. It also increased neuronal inflammation in mouse models of the disease.

Neurogene focuses on CLN5 and CLN7, another rare, late infantile and rapidly progressive form of Batten caused by defects in the CLN7/MFSD8 uncomfortable.

To better understand the evolution of CLN5 and CLN7 diseases, Neurogene sponsored a natural history study (NCT03822650) which is carried out as part of a collaboration between the company and UT Southwestern Medical Center.

Its objective is to study the clinical signs and the natural progression of symptoms in the two late forms of Batten disease. As an observational study, no medication is administered.

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