Trusted Resources: News & Meetings
Latest announcements and gatherings
EMA Grants Orphan Drug Designation to JR-171 for the Treatment of Mucopolysaccaridosis Type I (MPS I)
JCR Pharmaceuticals Co., Ltd. announced today that European Medicines Agency (EMA) has granted orphan drug designation to JR-171, an investigational drug for the treatment of mucoplysaccharidosis type I (MPS I, or Hurler, Hurler-Scheie and Scheie syndrome). JR-171 is a blood-brain-barrier (BBB)-penetrating form recombinant α-L-iduronidase that was developed using JCR’s proprietary J-Brain Cargo® BBB technology.
JR-171 received the orphan drug designation from the US Food Drug Administration (FDA) in February 2021. Currently JR-171 is undergoing a global Phase 1/2 clinical trial and the first patient in Japan was dosed in October, 2020 and in Brazil in March, 2021. The trial is also scheduled for enrolling patients in the US. The summary of this study is registered on clinicaltrials.gov.
BookmarkRelated Content
-
Newborn Screening of Mucopolysaccharidosis Type IMucopolysaccharidosis type I (MPS I), a ...
-
Therapy-Type Related Long-Term Outcomes in Mucopolysaccaridosis Type II (Hunter Syndrome) – Case SeriesMucopolysaccharidosis type II (MPS II, H...
-
Boston Children’s – Lysosomal Storage Disease Program (BoLD)The Boston Children's Lysosomal Storage ...
-
Patricia Dickson, MDDr. Dickson is Division Chief of Genetic...
-
Case Report: Cerebral Revascularization in a Child With Mucopolysaccharidosis Type IMucopolysaccharidosis (MPS) type I is a ...
-
Mucopolysaccharidoses (MPS) and Gene TherapyMucopolysaccharidoses (MPS) are a group ...
-
Intracerebral Gene Therapy in Four Children With Sanfilippo B Syndrome: 5.5-Year Follow-Up ResultsWe report the safety (primary endpoint) ...
Powered by
