MANUFACTURE OF ACTIVE HIGHLY PHOSPHORYLATED HUMAN N-ACETYLGALACTOSAMINE-6-SULFATASE AND USES THEREOF

• Main Authors: Michel Claude Vellard, Augustus O. Okhamafe, Vish Koppaka, Kidisti Araya
• Format: Patent
• Language: English; Spanish
• Published: 05.09.2016
• Subjects: BEER; BIOCHEMISTRY; CHEMISTRY; COMPOSITIONS THEREOF; CULTURE MEDIA; ENZYMOLOGY; HUMAN NECESSITIES; HYGIENE; MEDICAL OR VETERINARY SCIENCE; METALLURGY; MICROBIOLOGY; MICROORGANISMS OR ENZYMES; MUTATION OR GENETIC ENGINEERING; PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES; PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS; SPIRITS; VINEGAR; WINE

This invention provides compositions of active highly phosphorylated human N- acetylgalactosamine-6-sulfatase (GALNS), and pharmaceutical compositions and formulations thereof, methods of producing and purifying GALNS, and its use in the diagnosis, prophylaxis, or treatment of diseases and conditions, including particularly lysosomal storage diseases that are caused by, or associated with, a deficiency in the GALNS enzyme, e.g., Mucopolysaccharidosis IVa (MPS IVa or Morquio A syndrome). La presente invención se refiere a composiciones de Nacetilgalactosamina-6-sulfatasa humana, altamente fosforilada (GALNS), y composiciones y formulaciones farmacéuticas de la misma, métodos para producir y purificar GALNS, y su uso en el diagnóstico, profilaxis, o tratamiento de enfermedades y condiciones, incluyendo particularmente enfermedades de almacenamiento lisosomal que son causadas por, o están - asociadas con, una deficiencia en la enzima GALNS, por ejemplo, Mucopolisacaridosis IVa (MPS IVa o síndrome de Morquio A). Figura 11.