CALABASAS, Calif., Feb. 7, 2017 /PRNewswire/ -- ArmaGen, Inc., a privately held biotechnology company focused on developing groundbreaking therapies to treat severe neurological disorders, announced today that Roberto Giugliani, M.D., Ph.D., of Hospital de Clínicas in Porto Alegre, Brazil, will present initial data from ArmaGen's ongoing Phase 2 proof-of-concept (POC) study of AGT-181 at the 13th Annual WORLDSymposium on Thursday, February 16, 2017 in San Diego, California. AGT-181 is an investigational therapy for the treatment of Hurler or Hurler-Scheie syndrome, also known as mucopolysaccharidosis type I, or MPS I.
- Date: February 16, 2017
- Time: 11:15 a.m. PST
- Location: Manchester Grant Hyatt, San Diego, CA
About Mucopolysaccharidosis I (MPS I)
MPS I is a rare, hereditary, lysosomal storage disease that arises from a deficiency or absence of the enzyme iduronidase (IDUA), which is needed to break down complex sugars produced by the body. MPS I affects approximately 3,000 patients worldwide, with approximately 6.7 percent of affected patients in the U.S. The most severe form of MPS I, Hurler syndrome affects the brain and spinal cord in children, resulting in medical and cognitive challenges that can include developmental delay, progressive mental decline, loss of physical function, impaired language development, airway obstruction, corneal and retinal damage, carpal tunnel syndrome, and restricted joint movement. Attenuated or less severe forms of MPS I include Hurler-Scheie and Scheie syndromes. Patients with Hurler-Scheie syndrome may suffer from mild cognitive impairment or problems with attention. Patients with Scheie syndrome generally have a later onset, milder symptoms, and a slower disease progression, although they can develop significant morbidity.
AGT‑181 is a novel, investigational enzyme replacement therapy for the treatment of both somatic and cognitive symptoms in patients with MPS I. ArmaGen developed AGT-181 by re-engineering the enzyme iduronidase (IDUA) as fusion protein with an immunoglobulin G (IgG) antibody targeting the insulin receptor. Utilizing ArmaGen's proprietary "Trojan Horse" technology, AGT-181 takes advantage of the body's natural system for transporting proteins and other large molecules non-invasively across the blood-brain barrier (BBB), in this case by binding the same receptor that transports insulin across the BBB into the brain.
ArmaGen, Inc., is a privately held biotechnology company focused on developing groundbreaking therapies for severe neurological disorders. The company is developing a robust pipeline of innovative therapies for the treatment of lysosomal storage disorders including neurological symptoms such as Hurler syndrome (MPS I), Hunter syndrome (MPS II), metachromatic leukodystrophy, Sanfilippo A and B syndromes, as well as other diseases with severe CNS manifestations. ArmaGen's pipeline is based on decades of scientific leadership in engineering therapies to cross the blood-brain barrier and a dominant intellectual property portfolio. The company is advancing its pipeline through licensing and collaboration agreements, in-house development programs, and other partnering opportunities. For more information, visit www.armagen.com.
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SOURCE ArmaGen, Inc.