Regeneron Pharmaceuticals, Inc.

Andrew Baik

Katherine Cygnar

Christopher Schoenherr

Christos Kyratsous

Andrew J Murphy

Cheng Fang Wang

Cheng Wang

Maria Praggastis

Compositions and methods for delivering a therapeutic protein to the central nervous system (CNS), in order to treat diseases and disorders that impair the CNS, such as treating lysosomal storage diseases are disclosed. Therapeutic proteins delivered via a therapeutically effective amount of a nucleotide composition encoding the therapeutic protein conjugated to a cell surface receptor-binding protein that crosses the blood brain barrier (BBB) are provided.

Methods and compositions for therapeutic protein delivery

Antibodies, portions, and fusion proteins thereof to CD63 are provided. Also provided are nucleic acid sequences encoding same. Also provided are compositions comprising and methods of using same, e.g., for treating a patient in need thereof.

Anti-CD63 antibodies, conjugates, and uses thereof

Compositions and methods for treating enzyme-deficiency diseases are disclosed. Multidomain therapeutic proteins containing an internalization effector binding domain and a lysosomal replacement enzyme activity are disclosed. The multidomain therapeutic proteins are capable of entering cells, segregating to the lysosome, and delivering the replacement enzyme activity to the lysosome.

Compositions and methods for internalizing enzymes

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Bronx, NY, United States of America

Andrew Baik