Shire Human Genetic Therapies, Inc.

Andrea Iskenderian

Clark Qun Pan

Michael F Concino

Muthuraman Meiyappan

Angela Norton

Bettina Strack-Logue

Bohong Zhang

Jing Pan

Kevin Holmes

Dianna Lundberg

Alla Romashko

Pericles Calias

Paolo Martini

Richard Pfeifer

Huang Yan

Mary Alessandrini

Hicham Naimy

Lieh Yoon Low

Jianwen Feng

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

The present invention provides, among other things, methods and compositions for treating muscular dystrophy, in particular, Duchenne muscular dystrophy (DMD). In some embodiments, a method according to the present invention includes administering to an individual who is suffering from or susceptible to DMD an effective amount of a recombinant PLGF protein such that at least one symptom or feature of DMD is reduced in intensity, severity, or frequency, or has delayed onset. The present invention also provides exemplary recombinant PLGF proteins including monomeric, dimeric and single-chain PLGF proteins.

Recombinant placenta growth factor for treating Duchenne muscular dystrophy

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

The invention provides compositions and methods for effective lysosomal targeting mediated by SORT1. In particular, the compositions and methods provided by the invention may be used to treat lysosomal storage diseases such as Sanfilippo syndrome type B.

Lysosomal targeting and uses thereof

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

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Lexington, MA, United States of America

Andrea Iskenderian