Shire Human Genetic Therapies, Inc.

Synageva Biopharma Corporation

Alla Romashko

Michael F Concino

Muthuraman Meiyappan

Pericles Calias

Paolo Martini

Angela Norton

Bettina Strack-Logue

Bohong Zhang

Andrea Iskenderian

Dianna Lundberg

Jing Pan

Richard Pfeifer

Kevin Holmes

Mary Alessandrini

Huang Yan

Michael W Heartlein

Lawrence Charnas

Jan Powell

Lieh Yoon Low

Hicham Naimy

Brian Felice

Brian Pescatore

Nancy Savioli

Angela W Norton

Yan Huang

Among other things, the present invention provides methods and compositions of treating Sanfilippo syndrome type B (Sanfilippo B) by, e.g., intrathecal (IT) administration of a Naglu protein. A suitable Naglu protein can be a recombinant, gene-activated or natural protein. In some embodiments, a suitable Naglu protein is a recombinant Naglu protein. In some embodiments, a recombinant Naglu protein is a fusion protein containing a Naglu domain and a lysosomal targeting moiety. In some embodiments, the lysosomal targeting domain is an IGF-II moiety.

Therapeutic fusion protein comprising an alpha-n-acetylglucosaminidase and a lysosomal targeting moiety

A targeted therapeutic including a lysosomal enzyme and a lysosomal targeting moiety that is a peptide containing at least one N-linked glycosylation site. Methods of producing the targeted therapeutic may include nucleotide acids encoding the same and host cells co-expressing GNPT. Pharmaceutical compositions comprising the targeted therapeutic and methods of using the same to treat a lysosomal storage disease.

Mannose-6-phosphate bearing peptides fused to lysosomal enzymes

Treatment of Sanfilippo syndrome type B by intrathecal administration of alpha-n-acetylglucosaminidase

The present invention provides compositions and methods for effective treatment of a lysosomal acid lipase deficiency (LALD) disease, in particular, Wolman's disease and Cholesteryl Ester Storage Disease (CESD). Among other things, the present invention provides a method of treating developmental impairment or malnutrition in an individual suffering from a lysosomal acid lipase deficiency (LALD) disease, comprising administering to the individual a therapeutic effective amount of a lysosomal acid lipase.

Methods for treating lysosomal acid lipase deficiency

Growing community of inventors

Lexington, MA, United States of America

Alla Romashko