A biotechnology company, Neurogene Inc., has raised $115 million in a second-round financing to be used to accelerate clinical trials. A range of treatments for inherited neurological diseases are building a valuable pipeline, now leading with a novel therapy for Batten disease. The financing round was led by EcoR1 Capital, including the participation from existing investors.
Batten disease is a common name for a group of disorders based on neuronal ceroid lipofuscinoses (NCL). At least 13 genes, primarily autosomal recessive, have been reported in association with the disease, and the most prevalent form of Batten disease has been linked to mutations in the CLN3 gene on chromosome 16, encoded by battenin, a transmembrane protein first described in 1903. The worldwide prevalence of Batten disease is ~1 in 100,000 live births and until the past few years there is no effective treatment for this condition. Retinal disease starts with macular involvement (red-cherry spot) and later expands to peripheral retina. The protein encoded by CLN3 is found in the lysosomes and in synapses and this can build up in nervous tissue, causing seizures, visual impairment, mobility loss and early death. The diagnosis of Batten disease is based on a combination of clinical signs and symptoms, ophthalmological evaluations, EEG and brain MRI and is subsequently confirmed with genetic and biochemical tests. In 2017, the FDA approved an enzyme replacement therapy (ERT) for the first treatment to delay the progression of CLN2 (Brineura; BioMarin Pharmaceutical).
Following the achievement of the investment, Rachel McMinn, Ph.D., Founder and Chief Executive Officer stated that, “gene therapy has generated tremendous hope for the many families and patients with severe genetic disorders. 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,” said Rachel McMinn, Ph.D., Founder and Chief Executive Officer. “Proceeds from this round of financing will help us execute on our mission to develop safe and effective genetic therapies for patients and families living with neurological diseases.”