Biomarin Pharmaceutical Inc.

Sean M Bell

Emil D Kakkis

Daniel J Wendt

Augustus O Okhamafe

Paul Andrew Fitzpatrick

Michel Claude Vellard

Mubarack Muthalif

G Nick Zecherle

Kris Antonsen

Yanhong Zhang

Kieu Ly Tran

Mika Aoyagi-Scharber

Dong Wei

Joshua R Bliesath

Michel C Vellard

Paul A Fitzpatrick

Kieu Y Ly

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 provides variants of C-type natriuretic peptide (CNP) comprising one or more deletions; additions of and/or substitutions with natural amino acids, unnatural amino acids and/or peptidomimetics (including peptide bond isosteres); amino acid extensions; and/or other chemical moieties such as, e.g., poly(ethylene glycol) and hydrophobic acids. 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, e.g., skeletal dysplasias and achondroplasia, and vascular smooth muscle disorders such as, e.g., restenosis and arteriosclerosis.

Variants of C-type natriuretic peptides

The present invention is directed to 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. The invention provides compositions of prokaryotic PAL and biologically active fragments, mutants, variants or analogs thereof, as well as methods for the production, purification, and use of such compositions for therapeutic purposes, including the treatment of cancer.

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

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Novato, CA, United States of America

Sean M Bell