Biomarin Pharmaceutical Inc.

Novartis Ag

Xoma Technology Ltd.

Novartis Vaccines & Diagnostics, Gmbh

Augustus O Okhamafe

Daniel J Wendt

Michel Claude Vellard

Christopher P Price

Tianwei Chou

Stuart Bunting

Clarence Yap

Sherry Bullens

Sianna Castillo

Emil D Kakkis

Paul Andrew Fitzpatrick

Mika Aoyagi-Scharber

Shinong Long

Ahmad Hashash

Sean M Bell

Kangwen L Lin

Rampurna Gullapalli

Vish Koppaka

Thomas Lester

Piotr Henryk Karpinski

Paul Allen Sutton

Alejandro Dorenbaum

Eric M Loeser

Daniel I Oppenheimer

Eduardo Sy

Kieu Ly Tran

Yanhong Zhang

G Nick Zecherle

Bao-lu Chen

Timothy D Machajewski

Kidisti Araya

Mubarack Muthalif

Kris Antonsen

David M Ryckman

Eric Harwood

Shaopei Cai

Charles A O'Neill

Saeed Moshashaee

Sean Ritchie

Erik Damien Foehr

Shuguang Zhu

Peng Shen

Xiao Shang

Xiaofeng Lu

Joyce Chou

Paul J Kostel

Marc S Tesconi

Patricia Frech

Rampurma Gullapalli

David Ryckman

Paul A Fitzpatrick

Kieu Y Ly

Thomas W Lester

Michel C Vellard

Paul John Kostel

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

Formulations comprising recombinant human tripeptidyl peptidase-1 (rhTPP1) for intrathecal, intracerebroventricular, or intraocular administration, and kits comprising the same, are disclosed. Methods of using rhTPP1 in the prevention and treatment of symptoms of Neuronal Ceroid Lipofuscinosis (CLN2) disease are also disclosed. The formulations and methods are effective in halting the progression of CLN2 disease and may be used to treat subjects having CLN2 or a family history of CLN2.

TPP-1 formulations and methods for treating CLN2 disease

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

This invention provides compositions of active highly phosphorylated human N-acetylgalactosamine-6-sulfatase (GALNS), and pharmaceutical compositions and formulations thereof, methods of producing and purifying GALNS, and its use in the diagnosis, prophylaxis, or treatment of diseases and conditions, including particularly lysosomal storage diseases that are caused by, or associated with, a deficiency in the GALNS enzyme, e.g., Mucopolysaccharidosis IVa (MPS IVa or Morquio A syndrome).

Manufacture of active highly phosphorylated human N-acetylgalactosamine-6-sulfatase and uses thereof

Dry blend powder formulations comprising a pharmaceutical formulation containing tetrahydrobiopterin, and methods of making and using the same, are disclosed herein.

Dry blend formulation of tetrahydrobiopterin

Stable liquid pharmaceutical compositions comprising an antagonist anti-CD40 antibody as a therapeutically or prophylactically active component and methods useful in their preparation are provided. These compositions comprise the antagonist anti-CD40 antibody, a buffering agent to maintain the pH of the composition between about pH 5.0 and about pH 7.0, and an amount of arginine-HCl sufficient to render the liquid composition near isotonic. The stable liquid antagonist anti-CD40 antibody-containing pharmaceutical compositions of the invention find use in methods for treating proliferative diseases and diseases having an autoimmune and/or inflammatory component.

Antagonist anti-CD40 antibody pharmaceutical compositions

Growing community of inventors

Concord, CA, United States of America

Augustus O Okhamafe