The patent badge is an abbreviated version of the USPTO patent document. The patent badge does contain a link to the full patent document.

The patent badge is an abbreviated version of the USPTO patent document. The patent badge covers the following: Patent number, Date patent was issued, Date patent was filed, Title of the patent, Applicant, Inventor, Assignee, Attorney firm, Primary examiner, Assistant examiner, CPCs, and Abstract. The patent badge does contain a link to the full patent document (in Adobe Acrobat format, aka pdf). To download or print any patent click here.

Date of Patent:
Jul. 20, 2021

Filed:

Sep. 26, 2017
Applicant:

Shire Human Genetic Therapies, Inc., Lexington, MA (US);

Inventors:

Michael F. Concino, Bolton, MA (US);

Pericles Calias, Melrose, MA (US);

Jing Pan, Boxborough, MA (US);

Kevin Holmes, Belmont, MA (US);

Paolo Martini, Boston, MA (US);

Alla Romashko, Lexington, MA (US);

Muthuraman Meiyappan, Jamaica Plain, MA (US);

Bohong Zhang, Newton, MA (US);

Andrea Iskenderian, Arlington, MA (US);

Dianna Lundberg, Brentwood, NH (US);

Angela Norton, Reading, MA (US);

Bettina Strack-Logue, Somerville, MA (US);

Huang Yan, Billerica, MA (US);

Mary Alessandrini, Clinton, MA (US);

Richard Pfeifer, North Granby, CT (US);

Assignee:
Attorneys:
Primary Examiner:
Int. Cl.
CPC ...
A61K 8/66 (2006.01); C12N 9/24 (2006.01); A61K 38/47 (2006.01); A61K 35/76 (2015.01); A61K 35/761 (2015.01); A61K 9/19 (2006.01); A61K 9/00 (2006.01); A61K 38/46 (2006.01); C07K 14/65 (2006.01); C12N 9/42 (2006.01); A61K 9/08 (2006.01); A61K 47/02 (2006.01); A61K 47/26 (2006.01);
U.S. Cl.
CPC ...
A61K 38/47 (2013.01); A61K 9/0019 (2013.01); A61K 9/0085 (2013.01); A61K 9/08 (2013.01); A61K 9/19 (2013.01); A61K 35/76 (2013.01); A61K 35/761 (2013.01); A61K 38/46 (2013.01); A61K 38/465 (2013.01); A61K 47/02 (2013.01); A61K 47/26 (2013.01); C07K 14/65 (2013.01); C12N 9/2402 (2013.01); C12N 9/2437 (2013.01); C12Y 301/06008 (2013.01); C12Y 301/06013 (2013.01); C12Y 302/0105 (2013.01); C12Y 302/01045 (2013.01); C12Y 302/01046 (2013.01); C12Y 310/01001 (2013.01);
Abstract

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.


Find Patent Forward Citations

Loading…