Biomarin Pharmaceutical Inc.

Stuart Bunting

Augustus O Okhamafe

Daniel J Wendt

Tianwei Chou

Sherry Bullens

Clarence Yap

Christopher P Price

Peter Cameron Colosi

Erno Pungor

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

The invention provides adeno-associated virus (AAV) Factor VIII (FVIII)-encoding/expressing vectors and virus, including AAV FVIII vectors with high expression activity and AAV FVIII vectors that express full-length or truncated functional FVIII protein. The invention also relates to methods of making the herein described AAV FVIII vectors, recombinant AAV FVIII virus particles comprising or expressing such vectors, associated pharmaceutical formulations comprising the same and therapeutic uses thereof.

Adeno-associated virus Factor VIII vectors, associated viral particles and therapeutic formulations comprising the same

Adeno-associated virus factor VIII vectors, associated viral particles and therapeutic formulations comprising the same

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Novato, CA, United States of America

Stuart Bunting