Shire Human Genetic Therapies, Inc.

University of Minnesota

University of Kentucky Research Foundation

Mitotix, Inc.

Serene Josiah

Dennis Keefe

Michael W Heartlein

Michael F Concino

Bettina Strack-Logue

Stefan Stamm

Manli Shen

Lawrence Charnas

Thomas M Luby

Atsushi Asakura

Mayank Verma

Michael Boisclair

The present invention provides, among other things, oligonucleotide modulators of human 5'-HT2C receptor (HTR2C) and improved methods and composition for treating HTR2C-related diseases, disorders or conditions based on such modulators. In particular, oligonucleotides modulators according to the invention target specific regions in the Exon V/Intron V junction of the human HTR2C pre-mRNA and drive expression of HTR2C Vb splice isoform, leading to increased generation of non-edited strong HTR2C receptor and enhanced serotonin receptor activity.

Antisense oligonucleotide modulators of serotonin receptor 2C and uses thereof

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

A high throughput enzyme screen has been developed which relies on metal chelate interaction for capture of the product of the enzymatic reaction. In the present assay system, a detectable moiety is attached to a substrate having a chelating capturable moiety, which can be captured by an immobilized metal. Detection is effected due to the presence of a detectable label on the reaction product immobilized on the solid phase. Only signal associated with tagged protein bound to the solid phase is detected. The present assay can reliably measure enzyme activity, and has high reproducibility, which benefits high throughput screening.

High-throughput screening assays utilizing metal-chelate capture

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Cambridge, MA, United States of America

Serene Josiah