Clinical Trials

Date: 2017-08-08

Type of information: Initiation of the trial

phase: 1

Announcement: initiation of the trial

Company: RegenXBio (USA - MD)

Product: RGX-111 (adeno-associated virus vector serotype 9 expressing human a-L-iduronidase)

Action mechanism:

gene therapy. RGX-111 uses the AAV9 vector to deliver the human ?-l-iduronidase (IDUA) gene to the central nervous system (CNS). Mucopolysaccharidosis Type I (MPS I) is caused by deficiency of IDUA, an enzyme required for the breakdown of polysaccharides heparan sulfate and dermatan sulfate in the lysosomes of cells. Many patients develop symptoms related to glycosaminoglycan storage in the CNS, which can include excessive accumulation of fluid in the brain (hydrocephalus), spinal cord compression and cognitive impairment. Delivery of the enzyme within cells in the CNS could provide a permanent source of secreted IDUA beyond the blood-brain barrier, allowing for long-term cross correction of cells throughout the CNS. This strategy could also provide rapid IDUA delivery to the brain, potentially preventing the progression of cognitive deficits that otherwise occur in MPS I patients following bone marrow transplant.
RGX-111 is being developed under a collaboration with world-renowned gene therapy expert James M. Wilson, director of the Orphan Disease Center and Gene Therapy Program in the Perelman School of Medicine at the University of Pennsylvania.
RGX-111 has received orphan drug designation as well as rare pediatric disease designation from the FDA.

Disease: mucopolysaccharidosis type I

Therapeutic area: Rare diseases - Genetic diseases

Country:

Trial details:

Latest news:

• On August 8, 2017, RegenXbio announced the Investigational New Drug application (IND) is active for the planned multi-center, open-label, multiple-cohort, dose-escalation Phase I clinical trial of RGX-111 for the treatment of children and adult subjects with mucopolysaccharidosis type I (MPS I). Leading international gene therapy and lysosomal storage disease centers are expected to participate in the Phase I trial of RGX-111.