Shire Human Genetic Therapies, Inc.

Other

University of Minnesota

University of Colorado

Takeda Pharmaceutical Company Limited

Dennis Keefe

Serene Josiah

Hans De Haard

Michael W Heartlein

Michael F Concino

Bettina Strack-Logue

Natalie De Jonge

Sofie Gabriels

Lawrence Charnas

Thomas M Luby

Atsushi Asakura

Steven Abman

Mayank Verma

Gregory Seedorf

The present invention provides, among other things, anti-Flt-1 antibodies and methods for treating muscular dystrophy, in particular, Duchenne muscular dystrophy (DMD). In some embodiments, a method according to the present invention includes administering to an individual who is suffering from or susceptible to DMD an effective amount of an anti-Flt-1 antibody or antigen-binding protein thereof such that at least one symptom or feature of DMD is reduced in intensity, severity, or frequency, or has delayed onset.

Anti-Flt-1 antibodies for treating Duchenne muscular dystrophy

Anti-Flt-1 antibodies for treating duchenne muscular dystrophy

The present invention provides, among other things, methods and compositions for treating chronic lung disorders, in particular, bronchopulmonary dysplasia (BPD). In some embodiments, a method according to the present invention includes administering to an individual who is suffering from or susceptible to BPD an effective amount of an anti-Flt-1 antibody, or antigen binding fragment thereof, such that at least one symptom or feature of BPD is reduced in intensity, severity, or frequency, or has delayed onset.

Anti-FLT-1 antibodies in treating bronchopulmonary dysplasia

The present invention provides, among other things, methods and compositions for treating muscular dystrophy, in particular, Duchenne muscular dystrophy (DMD). In some embodiments, a method according to the present invention includes administering to an individual who is suffering from or susceptible to DMD an effective amount of an anti-Flt-1 antibody, or antigen binding fragment thereof, such that at least one symptom or feature of DMD is reduced in intensity, severity, or frequency, or has delayed onset.

Anti-FLT-1 antibodies in treating duchenne muscular dystrophy

The present invention provides, among other things, compositions and methods for treatment of Friedrich's Ataxia based on effective targeting of a therapeutic moiety to mitochondria that can substitute for natural FXN protein activity or rescue one or more phenotypes or symptoms associated with frataxin-deficiency. In some embodiments, the present invention provides a targeted therapeutic comprising a therapeutic moiety, which is a polypeptide having an N-terminus and a C-terminus, a mitochondrial targeting sequence associated with the therapeutic moiety at the N-terminus, and a mitochondrial membrane-penetrating peptide associated with the therapeutic moiety at the C-terminus, wherein the therapeutic moiety is targeted to mitochondria upon cellular entry.

Mitochondrial targeting and therapeutic use thereof

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Lexington, MA, United States of America

Dennis Keefe