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Cambridge, MA, United States

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Patent
Sarepta Therapeutics, The United States Of America and University of Maryland College Park | Date: 2016-03-29

Provided are methods of treatment in subjects having progeroid diseases and related conditions which rely upon LMNA-targeted antisense oligonucleotides for reducing expression of one or more aberrantly spliced LMNA mRNA isoforms that encode progerin.


Patent
Sarepta Therapeutics | Date: 2017-02-22

The present invention relates to an oligonucleotide which specifically hybridizes to a target region in an exon of the human dystrophin gene inducing exon skipping. The present invention further relates to a pharmaceutical composition comprising an oligonucleotide which specifically hybridizes to a target region in an exon of the human dystrophin gene inducing exon skipping.


Patent
Sarepta Therapeutics | Date: 2016-01-26

Functionally-modified oligonucleotide analogues comprising modified intersubunit linkages and/or modified 3 and/or 5-end groups are provided. The disclosed compounds are useful for the treatment of diseases where inhibition of protein expression or correction of aberrant mRNA splice products produces beneficial therapeutic effects.


Patent
Sarepta Therapeutics | Date: 2016-08-23

Inhibitory oligonucleotide having the general formula: X_(1)CCN_(1)N_(2)N_(3)X_(2)N_(4)N_(5)GGGN_(6)X_(3)N_(7)(I) are disclosed which can be used in pharmaceutical compositions, whereby in formula (I)


Patent
Sarepta Therapeutics | Date: 2016-06-08

A method and compound for treating skeletal muscle mass deficiency in a human subject are disclosed. The composition is an oligomer of morpholino subunits and phosphorus-containing intersubunit linkages joining a morpholino nitrogen of one subunit to a 5 exocyclic carbon of an adjacent subunit, contains between 10-40 nucleotide bases, has a base sequence effective to hybridize to an expression-sensitive region of processed or preprocessed human myostatin RNA transcript, identified, in its processed form, by SEQ ID NO:6, and is capable of uptake by target muscle cells in the subject. In practicing the method, the compound is administered in an amount and at a dosage schedule to produce an overall reduction in the level of serum myostatin measured in the patient, and preferably to bring the myostatin level within the a range determined for normal, healthy individuals.


Patent
Sarepta Therapeutics | Date: 2016-05-25

Provided are antisense oligonucleotides and other agents that target and modulate IL-17 and/or IL-23 signaling activity in a cell, compositions that comprise the same, and methods of use thereof. Also provided are animal models for identifying agents that modulate 17 and/or IL-23 signaling activity.


Patent
Sarepta Therapeutics | Date: 2015-12-21

A method for enhancing, by at least 10 fold, the antibacterial activity of an antisense oligonucleotide composed of morpholino subunits linked by phosphorus-containing intersubunit linkages. The method includes one or both of: conjugating an arginine-rich carrier to a 3 or 5 end of the oligonucleotide and modifying the oligonucleotide to contain 20%-50% intersubunit linkages that are positively charged at physiological pH. Also disclosed is an antisense oligonucleotide having enhanced antibacterial activity by virtue of one or both modifications.


The invention relates to TGF- beta blocking agent-treated HSC compositions and methods comprising the same. The TGF- beta blocking agent-treated stem cells are viable for an extended time in culture without replication or differentiation and upon transfer to appropriate conditions are capable of long term hematopoietic reconstitution.


Patent
Sarepta Therapeutics | Date: 2015-09-11

Provided are antisense molecules capable of binding to a selected target site in the human dystrophin gene to induce exon skipping, and methods of use thereof to treat muscular dystrophy.


Patent
Sarepta Therapeutics | Date: 2015-09-11

Provided are antisense molecules capable of binding to a selected target site in the human dystrophin gene to induce exon skipping, and methods of use thereof to treat muscular dystrophy.

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