RGX-111 is being developed as a novel, one-time, direct-to-central nervous system (CNS) treatment for mucopolysaccharidosis type I (MPS I) that includes the NAV AAV9 vector encoding a gene for human iduronidase (IDUA).
Delivery of the enzyme could provide a permanent source of secreted IDUA, which is otherwise untreated in intravenous-only therapy due to the blood-brain barrier. This strategy could also provide rapid IDUA delivery to the brain, potentially preventing the progression of cognitive deficits that otherwise occurs in MPS I patients.
MPS I is a rare autosomal recessive genetic disease caused by deficiency of IDUA, an enzyme required for the breakdown of the polysaccharides in lysosomes.
These polysaccharides, called glycosaminoglycans, accumulate in tissues of MPS I patients, resulting in characteristic storage lesions and diverse clinical signs and symptoms including in the CNS.
Regenxbio is seeking to improve lives through the curative potential of gene therapy with the NAV technology platform, a proprietary adeno-associated virus gene delivery platform, consists of exclusive rights to more than 100 novel AAV vectors.
GC Biopharma's GC1130A receives EMA Orphan Drug Designation
Guangzhou Fermion Technology's FZ008-145 IND application receives Chinese regulatory approval
Newron enrols 290 patients in schizophrenia study
Vistagen granted European patent for AV-101 to treat neuropathic pain
Alora Pharmaceuticals announces Relexxii commercial launch
IGC Pharma granted patent for Alzheimer's drug formulation
NRx Pharmaceuticals signs data agreement with Columbia University for IV Ketamine trials
Cambridge Cognition launches AQUA for automated quality assurance in CNS clinical trials
Shanghai Zhimeng Biopharma's CB03 receives US FDA orphan drug designation
Genentech's Alecensa demonstrates 76% reduction in disease recurrence risk
NRx Pharmaceuticals announces presentation at 8th Annual Dawson James Conference