Raptor Pharmaceuticals Inc.

Glyko, Inc.

Biomarin Pharmaceutical Inc.

Raptor Pharmaceutical Corp

Bioasis Advanced Technologies Inc.

Horizon Orphan LLC

Bioasis Technologies, Inc.

Christopher M Starr

Todd C Zankel

Dan J Wendt

Paul Andrew Fitzpatrick

Lin Chen

Minmin Qin

Gary N Zecherle

Wai-Pan Chan

John C Klock

John M Henstrand

Emil D Kakkis

Reinhard Gabathuler

Harvey I Miller

John C Klock, Jr

Peter Edridge

Stuart Sweidler

Steven K Striepeke

John M Hendstrand

Paul A Fitzpatrick

Stuart Swiedler

The present invention provides for compositions and methods for treating, ameliorating or preventing a lysosomal storage disease by administering to a patient suffering front a lysosomal storage disease a P97 conjugated with an enzyme which is capable of transportation into the lysosomes of cells on either sides of the blood brain barrier.

Use of P97 as an enzyme delivery system for the delivery of therapeutic lysosomal enzymes

The present invention relates generally to receptor-selective variants of the low-density lipoprotein receptor-associated protein (RAP) and compositions thereof, methods of generating such variants and methods of using such receptor-selective RAP variant compositions for therapeutic purposes.

Compositions comprising receptor-associated protein (RAP) variants specific for CR-containing proteins and uses thereof

The present invention relates generally to receptor-selective variants of the low-density lipoprotein receptor-associated protein (RAP) and compositions thereof, methods of generating such variants and methods of using such receptor-selective RAP variant compositions for therapeutic purposes. The invention also relates to antibodies that bind to one or a small subset of CR-containing proteins.

Compositions comprising receptor-associated protein (RAP) variants specific for LRP2 and uses thereof

The present invention relates to the use of receptor-associate protein (RAP) and fragments and variants thereof to improve delivery of therapeutic compounds to the liver and provides methods to treat liver disorders and conditions, such as hepatic carcinoma, by administering RAP or RAP variants conjugated to active agents.

Treatment of liver disorders by administration of RAP conjugates

The present invention is directed to a methods and compositions for receptor mediated drug delivery, particularly across the blood-brain barrier.

Megalin-based delivery of therapeutic compounds to the brain and other tissues

The present invention provides for compositions and methods for treating, ameliorating or preventing a lysosomal storage disease by administering to a patient suffering from a lysosomal storage disease a P97 conjugated with an enzyme which is capable of transportation into the lysosomes of cells on either sides of the blood brain barrier.

Use of p97 as an enzyme delivery system for the delivery of therapeutic lysosomal enzymes

Cyclic receptor-associated protein (RAP) peptides

Receptor associated protein (RAP) conjugates

Methods of increasing delivery of active agents to brain comprising administering receptor associated protein (RAP) fragments conjugated to active agents

This invention provides compounds of conjugates of therapeutic or active agents with RAP or a RAP polypeptide, their pharmaceutical compositions and methods for using the such compounds and compositions in the diagnosis, prophylaxis, or treatment of diseases and conditions, including particularly diseases of the central nervous system or lysosomal storage diseases.

The present invention provides a highly purified recombinant human precursor N-acetylgalactosamine-4-sulfatase and biologically active mutants, fragments and analogs thereof as well as pharmaceutical formulations comprising highly purified recombinant human precursor N-acetylgalactosamine-4-sulfatase. The invention also provides methods for treating diseases caused all or in part by deficiencies in human N-acetylgalactosamine-4-sulfatase including MPS VI and methods for producing and purifying the recombinant precursor enzyme to a highly purified form.

Precursor of N-acetylgalactosamine-4-sulfatase, methods of treatment using said enzyme and methods for producing and purifying said enzyme

Precursor N-acetylgalactosamine-4-sulfatase, methods of treatment using said enzyme and methods for producing and purifying said enzyme

The present invention provides a recombinant human &agr;-L-iduronidase and biologically active fragments and mutants thereof, large scale methods to produce and purify commercial grade recombinant human &agr;-L-iduronidase enzyme as well as methods to treat certain genetic disorders including &agr;-L-iduronidase deficiency and mucopolysaccharidosis I (MPS 1).

Recombinant &agr;-L-iduronidase, methods for producing and purifying the same and methods for treating diseases caused by deficiencies thereof

The present invention relates to an electrophoresis media comprising a gel and a compound capable of restricting the diffusion of water within the gel. Diffusion restricting compounds useful in the present invention include but are not limited to polyols, polymeric alcohols, polysaccharides, polyoxyethylene ethers, polyamines, polypeptides, gums, zwiterionic detergents and mixtures thereof. The present method relates to any electrophoresis media and includes but is not limited to polyacrylamide, agarose, starch, cellulose acetate and sepharose. A further embodiment of the present invention relates to a stacking gel wherein a diffusion restricting compound is used to prevent diffusion of water between the stack gel and resolving gel. The addition of a diffusion restricting compound to the stacking gel has the particular advantage of causing the stacking gel to become opaque, thereby facilitating the addition of samples to the gel. The present invention also relates to enclosing electrophoresis media within a vacuum sealed container. In yet a further embodiment of the present invention, the media enclosed in the vacuum sealed container further comprises a diffusion restricting compound.

Utilization of additives and defined storage systems to increase the

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Sonoma, CA, United States of America

Christopher M Starr