Biomarin Pharmaceutical Inc.

Daniel J Wendt

Augustus O Okhamafe

Mika Aoyagi-Scharber

Shinong Long

Jonathan LeBowitz

Michel Claude Vellard

Melita Dvorak-Ewell

Christopher P Price

Daniel Solomon Gold

Teresa Margaret Christianson

Emil D Kakkis

Stuart Bunting

Tianwei Chou

Sherry Bullens

Paul Andrew Fitzpatrick

Sean M Bell

Clarence Yap

Sianna Castillo

Mubarack Muthalif

Kieu Ly Tran

Kris Antonsen

G Nick Zecherle

Yanhong Zhang

Paul A Fitzpatrick

Dong Wei

Joshua R Bliesath

Kieu Y Ly

Michel C Vellard

Use of C-Type Natriuretic Peptide Variants to Treat Skeletal Dysplasia

The present disclosure provides for use of variants of C-type natriuretic peptide (CNP), and novel pharmaceutical compositions and formulations comprising CNP variant peptides for the treatment of skeletal dysplasias, one or more symptoms of skeletal dysplasias, such as long bone growth or growth velocity, and other disorders having a skeletal dysplasia and/or CNP-associated symptom or component.

Use of C-type natriuretic peptide variants to treat skeletal dysplasia

Provided herein are phenylalanine ammonia-lyase (PAL) variants produced by prokaryotes, wherein such prokaryotic PAL variant has a greater phenylalanine-converting activity and/or a reduced immunogenicity as compared to a wild-type PAL. Further provided are compositions of prokaryotic PAL and biologically active fragments, mutants, variants or analogs thereof, as well as methods for the production, purification, formulation, and use of such compositions for industrial and therapeutic purposes, e.g., treating hyperphenylalaninemia, including phenylketonuria, and other disorders, including cancer.

Compositions of prokaryotic phenylalanine ammonia-lyase variants and methods of using compositions thereof

The present invention relates in general to therapeutic fusion proteins useful to treat lysosomal storage diseases and methods for treating such diseases. Exemplary therapeutic fusion proteins comprise a lysosomal enzyme, a lysosomal targeting moiety, e.g., an IGF-II peptide, and a spacer peptide. Also provided are compositions and methods for treating Mucopolysaccharidosis Type IIIB (Sanfilippo B Syndrome), comprising a targeted therapeutic fusion protein comprising alpha-N-acetylglucosaminidase (Naglu), a lysosomal targeting moiety, e.g., an IGF-II peptide, and a spacer peptide.

Targeted therapeutic lysosomal enzyme fusion proteins and uses thereof

The present disclosure provides variants of C-type natriuretic peptide (CNP), pharmaceutical compositions comprising CNP variants, and methods of making CNP variants. The CNP variants are useful as therapeutic agents for the treatment of diseases responsive to CNP, including but not limited to bone-related disorders, such as skeletal dysplasias (e.g., achondroplasia), and vascular smooth muscle disorders (e.g., restenosis and arteriosclerosis).

Variants of C-type natriuretic peptide

Nucleic acids encoding targeted therapeutic lysosomal enzyme fusion proteins

Growing community of inventors

Walnut Creek, CA, United States of America

Daniel J Wendt