NCLS

M6P Therapeutics to Present at the 17th Annual International Congress on Neuronal Ceroid Lipofuscinosis

Retrieved on: 
Friday, October 8, 2021
Health, Clinical Trials, Research, Pharmaceutical, Science, Biotechnology, Linda, Person, Epilepsy, Endocytosis, N, Nervous system, Therapy, LSD, Dementia, CLN3, Plasmid, Endoglycosidase H, Batten disease, A-DNA, Neuronal ceroid lipofuscinosis, CLN2, PNGase F, Glycogen storage disease, Drug development, Lysosome, Mannose, Neuron, Neurodegeneration, Washington University in St. Louis, Eric, M6P, Prevalence, Severe cognitive impairment, NCLS, Enzyme, Communication, International, NCL, Autosome, International Congress on Tuberculosis, Disease, Patient, Enzyme replacement therapy, TPP1, Research, Technology, Phosphorylation, Pharmaceutical industry, Fine chemical

Neuronal ceroid lipofuscinosis type 2 (CLN2) is a neurodegenerative disorder caused by autosomal recessive mutations in the TPP1 gene, leading to the deficiency of TPP1, a lysosomal enzyme.

Key Points: 
  • Neuronal ceroid lipofuscinosis type 2 (CLN2) is a neurodegenerative disorder caused by autosomal recessive mutations in the TPP1 gene, leading to the deficiency of TPP1, a lysosomal enzyme.
  • Batten disease originally referred specifically to the juvenile and most common form of neuronal ceroid lipofuscinosis (NCL), now known as CLN3 .
  • Most forms are inherited in an autosomal recessive manner; however, autosomal dominant inheritance has been reported in one adult-onset form (neuronal ceroid lipofuscinosis 4B).
  • M6P Therapeutics mission is to translate advanced science into best-in-class therapies that address unmet needs within the LSD community.

Neurogene Announces FDA Clearance of IND for NGN-101 Gene Therapy to Treat CLN5 Batten Disease

Retrieved on: 
Tuesday, September 14, 2021
Biotechnology, Genetics, Health, IVT, Neurology, Food, ICV, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, FDA, NCLS, CEO, University, Batten disease, Orphan, Genetic testing, Caregiver, Seizure, Neurodegeneration, Patient, Gene, Death, Frederick, University of Rochester, IND, Disease, HIV disease progression rates, Severe cognitive impairment, Safety, Child, Inc., CLN5, Finnish heritage disease, Organization, Lists of diseases, Diagnosis, AAV, Research, Pharmaceutical industry, NGN-101, CLN5, a subtype of Batten disease, Neurogene, NGN-101, CLN5, A SUBTYPE OF BATTEN DISEASE, NEUROGENE

NGN-101, Neurogenes lead product candidate in its Batten disease franchise, is an investigational adeno-associated virus (AAV) gene therapy for the treatment of CLN5 Batten disease, a rare and fatal neurodegenerative disorder with no approved disease-modifying therapies available.

Key Points: 
  • NGN-101, Neurogenes lead product candidate in its Batten disease franchise, is an investigational adeno-associated virus (AAV) gene therapy for the treatment of CLN5 Batten disease, a rare and fatal neurodegenerative disorder with no approved disease-modifying therapies available.
  • NGN-101 is the first investigational gene therapy developed to address the devastating consequences of both neurodegeneration and vision loss in patients living with Batten disease.
  • IND clearance enables Neurogene to initiate a Phase 1/2 trial to assess the safety, tolerability, and efficacy of NGN-101 in patients with CLN5 Batten disease.
  • Neurogene is also developing novel gene therapy technologies to advance treatments for complex neurological diseases that conventional gene therapy cannot successfully address.