Regeneron Pharmaceuticals, Inc.

Regeneran Pharmaceuticals, Inc.

Christopher Schoenherr

Katherine Cygnar

Andrew Baik

Aris N Economides

Christos Kyratsous

Sarah Jane Hatsell

Vincent J Idone

Andrew J Murphy

Cheng Fang Wang

Cheng Wang

Leah Sabin

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

A genetically modified rodent is provided that comprises a modified Acvr1 gene that comprises a conditional altered exon 7 encoding R258G in antisense orientation, flanked by site-specific recombinase recognition sites, wherein the altered exon is inverted to sense orientation upon action of a recombinase, resulting in ectopic bone formation.

Nucleic acids comprising a modified rodent activin a receptor type 1 (Acvr1) gene

A genetically modified rodent is provided that comprises a modified Acvr1 gene that comprises a conditional altered exon 7 encoding R258G in anti sense orientation, flanked by site-specific recombinase recognition sites, wherein the altered exon is inverted to sense orientation upon action of a recombinase, resulting in ectopic bone formation.

Genetically modified rodent with an inducible ACVR1 mutation in exon 7 that causes ectopic bone formation

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Piermont, NY, United States of America

Christopher Schoenherr