Neurogene said investors included Samsara BioCapital, EcoR1 Capital, Cormorant Asset Management, Redmile Group and an undisclosed healthcare investment fund.
The proceeds will support a product pipeline with various Adeno-Associated Virus (AAV)-based gene therapies with disease-modifying potential and creation of a company-owned AAV manufacturing plant.
The funding will be used by Neurogene in advancing several gene therapy programs into IND-enabling studies and clinical trials for patients with rare neurological diseases, invest in new technologies for indications that have not been addressed by traditional gene therapy approaches and establish a viral vector manufacturing facility.
The company claims to have partnered with academic researchers, patient advocacy organizations and caregivers to bring therapies that can address underlying genetic cause in a broad spectrum of neurological diseases where presently there exist no effective treatments.
Neurogene founder and CEO Rachel McMinn said: “Neurogene is reimagining the future for patients with rare neurological diseases and their families. Through partnerships and our own internal expertise, we are advancing our gene therapy programs for rare neurological disorders. The vast majority of rare diseases remain unaddressed, and our goal is to enable a better future for patients with these diseases.”
The company stated that its programs use AAV-based gene therapy technology to deliver a normal gene to patients with a dysfunctional gene.
It is working on several diseases including Aspartylglucosaminuria (AGU), a rare, neurodegenerative, lysosomal storage disorder, caused by a deficiency of the aspartylglucosaminidase (AGA) enzyme, which leads to toxic accumulation of N-acetylglucosamines and cellular dysfunction.
Neurogene is also working on Charcot-Marie-Tooth (CMT4J) disease, a rare form of CMT caused by mutations in the FIG4 gene. Patients with this form of CMT usually develop symptoms of leg weakness in early childhood and early onset disease is associated with a rapidly progressive course, ultimately leading to loss of ambulation, quadriplegia, respiratory compromise and early death.
