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.
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.
Sarepta Therapeutics | Date: 2016-07-08
Antisense compositions targeted against an mRNA sequence coding for a selected protein, at a region having its 5 end from 1 to about 25 base pairs downstream of a normal splice acceptor junction in the preprocessed mRNA, are disclosed. The antisense compound is RNase-inactive, and is preferably a phosphorodiamidate-linked morpholino oligonucleotide. Such targeting is effective to inhibit natural mRNA splice processing, produce splice variant mRNAs, and inhibit normal expression of the protein.
Sarepta Therapeutics | Date: 2016-08-25
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.
Sarepta Therapeutics | Date: 2017-08-02
The present invention relates to antisense antiviral compounds and methods of their use and production in inhibition of growth of viruses of the Orthomyxoviridae family and in the treatment of a viral infection. The compounds are particularly useful in the treatment of influenza virus infection in a mammal. Exemplary antisense antiviral compounds are substantially uncharged, or partially positively charged, morpholino oligonucleotides having 1) a nuclease resistant backbone, 2) 12-40 nucleotide bases, and 3) a targeting sequence of at least 12 bases in length that hybridizes to a target region selected from the following: a) the 5 or 3 terminal 25 bases of the negative sense viral RNA segment of Influenzavirus A, Influenzavirus B and Influenzavirus C; b) the terminal 30 bases of the 5 or 3 terminus of the positive sense vcRNA; c) the 45 bases surrounding the AUG start codon of an influenza viral mRNA and; d) 50 bases surrounding the splice donor or acceptor sites of influenza mRNAs subject to alternative splicing.
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.
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)
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.
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.
Sarepta Therapeutics | Date: 2016-05-18
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.