Shire Human Genetic Therapies, Inc.

Takeda Pharmaceutical Company Limited

Patrick Anthony John Haslett

Albert Barnes Seymour

Madhusudan Natarajan

Ann Barbier

Paolo G V Martini

Hicham Naimy

Yongchang Qiu

Paolo GV Martini

The present invention provides, among other things, bi-specific molecules including, but not limited to, antibodies, fynomers, aptamers, fusion proteins, and protein binding domains that bind both CCL2 and LOXL2 and uses thereof, in particular, for treatment of scleroderma and related fibrotic and/or inflammatory diseases, disorders and conditions. In some embodiments, the present invention further provides methods and compositions for treatment of scleroderma and related fibrotic and/or inflammatory diseases, disorders and conditions based on the combination of mono-specific anti-CCL2 and anti-LOXL2 molecules.

Anti-CCL2 and anti-LOXL2 combination therapy for treatment of scleroderma

The present invention provides, among other things, effective treatment for Sanfilippo Syndrome Type A (MPS IIIA) based on intrathecal delivery of recombinant heparan N-sulfatase (HNS) enzyme. The present invention also includes methods of treating Sanfilippo Syndrome Type A (MPS IIIA) Syndrome by intrathecal administration of a recombinant HNS enzyme at a therapeutically effective dose and an administration interval for a period sufficient to decrease glycosaminoglycan (GAG) heparan sulfate level in the cerebrospinal fluid (CSF) relative to baseline (e.g., prior to treatment) as well as to improve, stabilize, or reduce decline of cognitive function, disability, behavior, quality of life and/or auditory brainstem response relative to baseline (e.g., prior to treatment).

Treatment of cognitive impairment of mucopolysaccharidosis type IIIA by intrathecal delivery of heparan N-sulfatase

Detecting glycosaminoglycans (GAGs) and/or determining the level of one or more glycosaminoglycans can be useful, e.g., in identifying or monitoring various medical conditions, the status of patients having various medical conditions, and/or the response to treatment of individuals having various medical conditions. The present invention provides methods for detecting glycosaminoglycans and/or determining the level of glycosaminoglycans through the use of, e.g., mass spectrometry.

Determination of glycosaminoglycan levels by mass spectrometry

Growing community of inventors

Somerville, MA, United States of America

Patrick Anthony John Haslett