Biomarin Pharmaceutical Inc.

The United States of America, As Represented by the Secretary, Department of Health and Human Services

Ucl Business Plc

St. Jude Children's Research Hospital

Other

University College London

Peter Cameron Colosi

Amit Nathwani

Jenny McIntosh

Edward Tuddenham

Andrew Davidoff

Erno Pungor

Stuart Bunting

Paul Albert Sieving

Ronald Avery Bush

Yong Zeng

William I Wood

David W Leung

Hassibullah Akeefe

R Glenn Hammonds, Jr

Steven A Spencer

Michael Lochrie

Robert Ng

Rajeev Mahimkar

Geoffrey Berguig

Silvia Ramirez

The invention provides improved adeno-associated virus (AAV) Factor VIII (FVIII) vectors, including AAV FVIII vectors that produce a functional Factor VIII polypeptide and AAV FVIII vectors with high expression activity.

Adeno-associated virus factor VIII vectors

The invention relates to novel adeno-associated virus (AAV) capsid proteins, AAV particles comprising a novel capsid protein, polynucleotides encoding these capsid proteins and AAV vectors expressing these capsid proteins. The invention also relates to methods of making the herein described AAV vectors expressing the novel capsid proteins of the invention and associated therapeutic uses of thereof.

Liver targeting adeno-associated viral vectors

Provided herein are methods of treating phenylketonuria by normalizing levels of amino acids, neurotransmitters, and neurotransmitter metabolites in a subject having phenylketonuria.

Methods of treating phenylketonuria

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 capsid proteins

The present disclosure provides methods of generating multiplexed genetically modified animals, for example, porcine endogenous retrovirus (PERV)-inactivated pigs. The disclosure also provides methods of improving the birth rate of multiplexed genetically modified animals. In some embodiments, the present closure is concerned with the generation and utilization of porcine cells in which porcine endogenous retroviral (PERV) elements have been inactivated. In sonic embodiments, the PERV-free or PERV-reduced porcine cells are cloned to produce porcine embryos. In some embodiments, the PERV-free or PERV-reduced embryos may be grown into adult swine from which organs and/or tissues may be extracted and used for such purposes as xenotransplantation into non-porcine animals such as humans.

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

Expression vectors and therapeutic methods of using such vectors in the treatment of diseases of the eye resulting from failure to produce a specific protein in the eye, or the production of a non-functional protein in the eye.

Methods and compositions for treating genetically linked diseases of the eye

Growth hormone receptor and growth hormone binding protein are purified enabling amino acid sequence and DNA isolates coding for growth hormone receptor and growth hormone binding protein and methods of obtaining such DNA are provided, together with expression systems for recombinant production of growth hormone receptor and growth hormone binding protein. Therapeutically useful forms of the growth hormone receptor and growth hormone binding protein and anti-receptor antibodies are described.

Compositions and methods for the synthesis of growth hormone receptor

Growing community of inventors

Novato, CA, United States of America

Peter Cameron Colosi