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Neurogene has raised $ 115 million in second-round funding that will accelerate its plans to launch clinical trials of a range of gene therapies for hereditary neurological diseases, initially focusing on one form of the disease. Batten.

Batten disease is a group of disorders caused by a protein deficiency that allows fatty substances to build up in nervous tissue, causing seizures, visual impairment, loss of mobility and premature death.

New York biotechnology, founded by former Wall Street analyst Rachel McMinn, Ph.D., two years ago,noted some of the money will be used to advance its main programs targeting Batten disease caused by the CLN5 and CLN7 mutations, two rare and rapidly progressive subtypes that occur later in childhood.

It will also fund the development of candidates for the lysosomal storage disorders of Charcot-Marie-Tooth disease (CMT) type 4J, caused by changes in the FIG4 gene, and aspartylglucosaminuria (AGU), which occurs when the AGA gene is mutated.

The funding will also be used to develop its gene therapy platform and increase its manufacturing capacity. It adds to the $ 68.5 million that biotechnology raised in its Series A last year.

The AGU and CMT4J The programs were due to reach the clinic this year, but Neurogene has made faster progress in the fight against Batten disease.

The most advanced at the moment is its CLN5 candidate, which is expected to begin clinical trials next year. Batten disease is an autosomal recessive disease, which means that it only develops if a person inherits two copies of a defective gene for their parents.

Neurogene intends to treat it by using an adeno-associated viral vector to deliver a replacement CLN5 gene, restoring the activity of the protein for which it codes. Although the function of this protein is not well understood, the hope is that restoring its activity will slow or stop the progression of the disease.

Neurogene is not the only group considering the potential of gene therapies to make a difference in the lives of patients with Batten disease.

Abeona Therapeutics has been in this field for a few years and earlier this year fired its candidate ABO-202 for CLN1 disease, also known as childhood Batten disease, at Taysha Gene Therapies for an initial fee of $ 7. million dollars and up to $ 56 million in stages. He is also developing ABO-201 for CLN3 disease.

Last year, Amicus Therapeutics presented the first results with its CLN6 gene therapy– acquired as part of its $ 100 million buyout of Celenex in 2018 – which seems to indicate a slowing of neurological decline.

Meanwhile, researchers at Cornell University conducted a phase 1 gene therapy trial in late childhood disease (CLN2) with similar positive results, and also began a phase 1/2 study in this group.

“Gene therapy has generated immense hope for the many families and patients with serious genetic disorders,” McMinn said.

“We believe that our focus on improved product design, innovative technology, advanced vector manufacturing and leading analyzes will help realize the potential of genetic treatments. “

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