Horizon Orphan LLC

Raptor Pharmaceuticals Inc.

Raptor Pharmaceutical Corp

Bioasis Advanced Technologies Inc.

Mercaptor Discoveries, Inc.

Bioasis Technologies, Inc.

Todd C Zankel

Christopher M Starr

Sara Louise Isbell

Lorna Duffy

Benoit Gourdet

John Francis Unitt

Timothy Phillips

Amanda Anne Ko

Reinhard Gabathuler

Loma Duffy

The present disclosure relates, in general, to use of small diffusible thiols in the treatment of neurodegenerative diseases associated with glutamate excitotoxicity, protein aggregation and oxidative stress in the central nervous system, particularly in the brain.

Use of thiol compounds to treat neurological disease

The present disclosure is directed to methods for treating diseases for which cysteamine is indicated and compounds useful in such methods.

ADO-resistant cysteamine analogs and uses thereof

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 disclosure relates, in general, to compounds useful as inhibitors of fucosidase enzymes, and to methods and compositions for the treatment of tumors or cancers, such as liver disorders and liver tumors (e.g., hepatocellular carcinoma), with a compound as disclosed herein.

Fucosidase inhibitors

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

Growing community of inventors

San Francisco, CA, United States of America

Todd C Zankel