Orchard Therapeutics announced data from an ongoing proof-of-concept clinical trial evaluating the safety and efficacy of OTL-203, a gene therapy for the treatment of mucopolysaccharidosis
type I developed at the San Raffaele-Telethon Institute for Gene Therapy.
Daix (France), September 2, 2019 - Inventiva (Euronext: IVA), a clinical-stage biopharmaceutical company developing oral small molecule therapies for the treatment of diseases in the areas of fibrosis, lysosomal storage disorders and oncology, today announced the recruitment of the first patients in a new biomarker study in children and adults with mucopolysaccharidosis
type VI (MPS VI)
The oral presentations will highlight data from the company's neurometabolic portfolio, including investigational candidates OTL-203 for mucopolysaccharidosis
type I (MPS-I) and OTL-200 for metachromatic leukodystrophy (MLD).
Dr Hayes will lead the company's preclinical research and spearhead the near-term advancement of ISP-001 for the treatment of Mucopolysaccharidosis
type I (MPS I) and additional Immune System Programming (ISP) candidates into clinical development.
A clinical study of 77 patients with mucopolysaccharidosis
(MPS) is composed of a group of rare inherited lysosomal storage disorders of the mucopolyssacharide, or glycosaminoglycan (GAGs), metabolism.
Clinicopathological diagnosis of mucopolysaccharidosis
type IIIB (MPS IIIB; Sanfilippo syndrome B), an inherited autosomal recessive lysosomal storage disease, as a cause of losses in a commercial emu flock and screening breeders using a mutation-specific DNA test are described.
Type IVA (MPS IVA or Morquio A disease; Online Mendelian Inheritance in Man 253000) was first described in 1929 by a pediatrician, L.
type II," in GeneReviews, R.
Hunter syndrome (mucopolysaccharidosis
type II (MPS II)) is a lysosomal disease characterized by deficiency of the lysosomal enzyme iduronate-2-sulfatase (I2S).
Food and Drug Administration (FDA) has approved MEPSEVII[TM] (vestronidase alfa), the first medicine approved for the treatment of children and adults with Mucopolysaccharidosis
VII (MPS VII, Sly syndrome).