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The United States Food and Drug Administration (FDA) granted orphan drug designation to a gene therapy developed by Neurogenic for the treatment of CLN7 Batten disease.

This is the second neurogenic gene therapy for Batten disease to receive orphan drug designation from the FDA this summer. Gene therapy for CLN5 Batten disease was designated an orphan drug in July.

“Receiving the orphan drug designation for CLN7, our second designation for our Batten disease pipeline, demonstrates our commitment to treating patients with this devastating neurodegenerative disease,” said Rachel McMinn, PhD, Founder and CEO of Neurogene, in a press release. Press release.

The two CLN7 and CLN5 are forms of late childhood Batten disease, which commonly affects children aged 2 to 4 years and is characterized by seizures, loss of motor and cognitive skills, and reduced life expectancy. Both types are caused by mutations in their respective genes, which lead to alteration of the CLN7 or CLN5 protein. which in turn, cause the malfunction of the molecular machinery that cells use to remove waste, ultimately leading to toxicity.

“CLN7, like CLN5, has a significant unmet need and no FDA approved treatment options, and we are working with all stakeholders to advance effective gene therapy treatment into the clinic,” McMinn said.

The goal of gene therapy is to deliver an unmutated version of the mutated gene to cells in the body, which would allow them to make a functional version of the protein.

Neurogene’s CLN7 and CLN5 gene therapies achieve this by using an attenuated viral vector, in particular adeno-associated virus (AAV). This virus is commonly used in gene therapy because it is relatively easy to insert therapeutic genes into the virus. Because AAV itself is not known to cause serious human illness, the health risks from its use as therapy are considered relatively minimal.

The newly granted orphan drug designation is granted by the FDA to therapies that may improve the care of patients with rare diseases, defined as conditions that affect fewer than 200,000 people in the United States.

The designation confers certain benefits on the developer (s) of the therapy, including assistance in the drug development process, tax credits, not having to pay certain fees and seven years of exclusivity. marketing. The aim is to encourage the development of therapies for rare diseases which may prove to be less cost-effective than those used to treat more common diseases.

Currently, no cure exists for Batten disease. Available treatments, including anti-epileptic drugs, can help control symptoms of the disease.

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