Neurogene adds another $115M to the pot for trials for its gene therapies

DNA helix forming inside a test tube
(Getty Images)

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

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

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

Sponsored By Metabolon

Five Translational Insights Key to a Successful First-in-Human (FIH) Study – Metabolite-Based Biomarker Discovery and Validation

Translational success rates from pre-clinical animal studies to human clinical trials remain frustratingly low. Learn how metabolomics helps you bridge between the theoretical & practical, between the function & actual activity of your drug molecule to get you closer to the phenotype, sooner.

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

The funding will also go toward developing its gene therapy platform and scaling up its manufacturing capacity. It adds to the $68.5 million the biotech raised in its series A round last year.

The AGU and CMT4J programs had been scheduled to reach the clinic this year, but Neurogene has been making quicker progress with the Batten disease drive.

Furthest along right now is its CLN5 candidate, which should start clinical trials next year. Batten disease is an autosomal recessive disorder, which means it only develops if a person inherits two copies of a faulty gene for their parents.

Neurogene intends to treat it by using an adeno-associated virus vector to deliver a replacement CLN5 gene, restoring the activity of the protein it codes for. While the function of that protein isn’t well understood, the hope is that restoring its activity will slow down or halt the progression of the disease.

Neurogene isn’t the only group eyeing the potential for gene therapies to make a difference to the lives of patients with Batten disease.

Abeona Therapeutics has been working in this area for a few years, and earlier this year licensed its ABO-202 candidate for CLN1 disease—also known as infantile Batten disease—to Taysha Gene Therapies for $7 million upfront and up to $56 million in milestones. It 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 takeover of Celenex in 2018—that seem to indicate a slowing of neurological decline.

Meanwhile, researchers at Cornell University have carried out a phase 1 gene therapy trial in late- infantile (CLN2) disease with similar positive results, and have also started a phase 1/2 study in this group.

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

“We believe our focus on improved product design, innovative technology, cutting-edge vector manufacturing and premier analytics will help fulfill the potential of genetic treatments.”