SAREPTA THERAPEUTICS, INC. Patent applications |
Patent application number | Title | Published |
20160040162 | EXON SKIPPING COMPOSITIONS FOR TREATING MUSCULAR DYSTROPHY - Antisense molecules capable of binding to a selected target site in the human dystrophin gene to induce exon 53 skipping are described. | 02-11-2016 |
20150344884 | INHIBITORY OLIGONUCLEOTIDES AND THEIR USE IN THERAPY - Inhibitory oligonucleotide having the general formula: | 12-03-2015 |
20150152415 | MULTIPLE EXON SKIPPING COMPOSITIONS FOR DMD - 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. | 06-04-2015 |
20150080311 | COMPOUND AND METHOD FOR TREATING MYOTONIC DYSTROPHY - An antisense compound for use in treating myotonic dystrophy DM1 or DM2, a method of enhancing antisense targeting to heart and quadricep muscles, and a method for treating DM1 or DM2 in a mammalian subject are disclosed. The oligonucleotide has 8-30 bases, with at least 8 contiguous bases being complementary to the polyCUG or polyCCUG repeats in the 3′UTR region of dystrophia myotonica protein kinase (DMPK) mRNA in DM1 or DM2, respectively. Conjugated to the oligonucleotide is a cell-penetrating peptide having the sequence (RXRR(B/X)R) | 03-19-2015 |
20150073140 | OLIGONUCLEOTIDE ANALOGUES HAVING MODIFIED INTERSUBUNIT LINKAGES AND/OR TERMINAL GROUPS - 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. | 03-12-2015 |
20150038462 | OLIGONUCLEOTIDE ANALOG AND METHOD FOR TREATING FLAVIVIRUS INFECTIONS - A method of inhibiting replication of a flavivirus in animal cells, and an oligonucleotide compound for use in the method are disclosed. The oligonucleotide analog (i) has a nuclease-resistant backbone, (ii) is capable of uptake by the cells, (iii) contains between 8-40 nucleotide bases, and (iv) has a sequence of at least 8 bases complementary to a region of the virus' positive strand RNA genome that includes at least a portion of SEQ ID NOS:1-4. Exposure of cells infected with a flavivirus to the analog is effective to form within the cells, a heteroduplex structure composed of the virus ssRNA and the oligonucleotide, characterized by a T | 02-05-2015 |
20140330006 | FUNCTIONALLY-MODIFIED OLIGONUCLEOTIDES AND SUBUNITS THEREOF - 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. | 11-06-2014 |
20140329881 | EXON SKIPPING COMPOSITIONS FOR TREATING MUSCULAR DYSTROPHY - Antisense molecules capable of binding to a selected target site in the human dystrophin gene to induce exon 44 skipping are described. | 11-06-2014 |
20140329772 | INDUCED EXON INCLUSION IN SPINAL MUSCLE ATROPHY - The invention relates to the use of an antisense compound for inducing exon inclusion as a treatment for Spinal Muscle Atrophy (SMA). More particularly it relates to inducing inclusion of exon 7 to restore levels of Survival Motor Neuron (SMN) protein encoded by the Survival Motor Neuron (SMN) gene. | 11-06-2014 |
20140329762 | COMPOSITIONS FOR TREATING MUSCULAR DYSTROPHY - Improved compositions and methods for treating muscular dystrophy by administering antisense molecules capable of binding to a selected target site in the human dystrophin gene to induce exon skipping are described. | 11-06-2014 |
20140323544 | EXON SKIPPING COMPOSITIONS FOR TREATING MUSCULAR DYSTROPHY - Antisense molecules capable of binding to a selected target site in the human dystrophin gene to induce exon 44 skipping are described. | 10-30-2014 |
20140315977 | EXON SKIPPING COMPOSITIONS FOR TREATING MUSCULAR DYSTROPHY - Antisense molecules capable of binding to a selected target site in the human dystrophin gene to induce exon 53 skipping are described. | 10-23-2014 |
20140315862 | COMPOSITIONS FOR TREATING MUSCULAR DYSTROPHY - Improved compositions and methods for treating muscular dystrophy by administering antisense molecules capable of binding to a selected target site in the human dystrophin gene to induce exon skipping are described. | 10-23-2014 |
20140303238 | OLIGONUCLEOTIDES FOR TREATING EXPANDED REPEAT DISEASES - The invention provides for a method for selectively reducing the expression of a mutant mRNA and/or protein having an expanded nucleotide repeat relative to a wild-type mRNA, comprising contacting a cell with an antisense oligonucleotide of sufficient length and complementarity to the expanded nucleotide repeat. More particularly it relates to selectively reducing the expression of mutant Huntington protein associated with Huntington's disease. The antisense oligonucleotide comprising either a nucleotide or a repeated three nucleotide sequence as defined in the claims. | 10-09-2014 |
20140303073 | ANTISENSE ANTIVIRAL COMPOUND AND METHOD FOR TREATING INFLUENZA VIRAL INFECTION - 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. | 10-09-2014 |
20140296321 | METHODS AND COMPOSITIONS FOR MANIPULATING TRANSLATION OF PROTEIN ISOFORMS FROM ALTERNATIVE INITIATION OF START SITES - Provided herein are antisense oligonucleotides, compositions comprising antisense oligonucleotides, and methods for the use of antisense oligonucleotides in manipulating translation. Expression of isoforms of proteins expressed from different start codons of the same transcript are inhibited by antisense oligonucleotides, which may also enhance expression of non-target isoforms. | 10-02-2014 |
20140287983 | ANTISENSE COMPOSITIONS AND METHODS FOR MODULATING CONTACT HYPERSENSITIVITY OR CONTACT DERMATITIS - Provided are methods and compositions, including topical compositions, for inducing tolerance to a sensitizing agent known to provoke contact hypersensitivity in a subject. Included are methods of topically applying to the subject an effective amount of an antisense composition targeting the start site or splice site of a CFLAR mRNA. | 09-25-2014 |
20140213737 | ANTIBACTERIAL ANTISENSE OLIGONUCLEOTIDE AND METHOD - 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. | 07-31-2014 |
20140194612 | IMMUNOSUPPRESSION COMPOUND AND TREATMENT METHOD - A method and compound for suppressing an immune response in a mammalian subject, for the treatment or prevention of an autoimmune condition or transplantation rejection are disclosed. The compound is an antisense oligonucleotide analog compound having a targeting sequence complementary to a preprocessed CTLA-4 mRNA region identified by SEQ ID NO: 22 in SEQ ID NO: 1, spanning the splice junction between intron 1 and exon 2 of the preprocessed mRNA of the subject. The compound is effective, when administered to a subject, to form within host cells, a heteroduplex structure (i) composed of the preprocessed CTLA-4 mRNA and the oligonucleotide compound, (ii) characterized by a Tm of dissociation of at least 45° C., and (iii) resulting in an increased ratio of processed mRNA encoding ligand-independent CTLA-4 to processed mRNA encoding full-length CTLA-4. | 07-10-2014 |
20140107013 | COMPOUND AND METHOD FOR TREATING MYOTONIC DYSTROPHY - Provided are 9-base morpholino antisense compounds targeted to polyCUG repeats in the 3′UTR region of dystrophia myotonica protein kinase (DMPK) mRNA, and related methods for treating myotonic dystrophy DM1. | 04-17-2014 |
20140094500 | MULTIPLE EXON SKIPPING COMPOSITIONS FOR DMD - 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. | 04-03-2014 |
20140045916 | SPLICE-REGION ANTISENSE COMPOSITION AND METHOD - 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. | 02-13-2014 |
20140024821 | OLIGONUCLEOTIDE ANALOGUES TARGETING HUMAN LMNA - Provided are LMNA-targeted antisense oligonucleotides for reducing expression of one or more aberrantly spliced LMNA mRNA isoforms that encode progerin. | 01-23-2014 |
20140024698 | METHODS FOR TREATING PROGEROID LAMINOPATHIES USING OLIGONUCLEOTIDE ANALOGUES TARGETING HUMAN LMNA - 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. | 01-23-2014 |
20130288369 | dsRNA MOLECULES COMPRISING OLIGONUCLEOTIDE ANALOGS HAVING MODIFIED INTERSUBUNIT LINKAGES AND/OR TERMINAL GROUPS - Morpholino oligomers containing modified intersubunit linkages and/or terminal groups are provided for use within dsRNA molecules. The oligomers are oligonucleotide analogs containing predetermined sequences of base-pairing moieties. Also provided are such oligomers conjugated to peptide transporter moieties, where the transporters are preferably composed of arginine subunits, or arginine dimers, alternating with neutral amino acid subunits. | 10-31-2013 |
20130190390 | MULTIPLE EXON SKIPPING COMPOSITIONS FOR DMD - 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. | 07-25-2013 |