PATENTSCOPE: EN_ALL:nmr AND PA:shire

CONJUGATED C1 ESTERASE INHIBITOR AND USES THEREOF (Fri, 13 Oct 2017)
The present invention provides, among other things, a conjugated Cl-INH for improved treatment of complement-mediated disorders, including hereditary angioedema (HAE). In some embodiments, a conjugated Cl-INH provided by the present invention is a PEGylated Cl-INH. In some embodiments, a conjugated Cl-INH provided by the present invention is a polysialic acid (PSA) conjugated Cl-INH.
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LYSOSOMAL TARGETING AND USES THEREOF (Fri, 03 Jun 2016)
The invention provides compositions and methods for effective lysosomal targeting mediated by PCSK9. In particular, the compositions and methods provided by the invention may be used to treat lysosomal storage diseases such as Pompe Disease and Sanfilippo Syndrome Type B, and they may be used for targeting lysosomal enzymes to the various muscles of the human body.
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STEREOCHEMICALLY ENRICHED COMPOSITIONS FOR DELIVERY OF NUCLEIC ACIDS (Thu, 31 Dec 2015)
Provided, in part, is a composition comprising one or more chemical entities of formula I, each of which is a compound of formula (I):a pharmaceutically acceptable salt thereof, a solvate thereof, or a solvate of a pharmaceutically acceptable salt thereof, the composition characterized in that greater than a first threshold amount of the total amount of chemical entities of formula I in the composition: are chemical entities of formula I.a, wherein the first threshold amount is 50%; or are chemical entities of formula I.b.1, wherein the first threshold amount is 25%; or are chemical entities of formula I.b.2, wherein the first threshold amount is 25%, wherein the chemical entities of formula I.a, I.b.1, and I.b.2, are described herein, and methods of using such compositions, for example, for the delivery of a polynucleotide in vivo.
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QUANTITATIVE ASSESSMENT FOR CAP EFFICIENCY OF MESSENGER RNA (Fri, 26 Sep 2014)
The present invention provides, among other things, methods of quantitating mRNA capping efficiency, particularly for mRNA synthesized in vitro. In some embodiments, the methods comprise chromatographic methods of quantifying capping efficiency and methylation status of the caps.
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RIBONUCLEIC ACIDs WITH 4'-THIO-MODIFIED NUCLEOTIDES AND RELATED METHODS (Fri, 26 Sep 2014)
Disclosed are messenger RNA molecules and related compositions incorporating a 4'-thio modification in the furanose ring of at least one nucleotide residue, and methods of using these mRNAs to produce an encoded therapeutic protein in vivo and to treat or prevent diseases or disorders. In certain embodiments, the 4'-thio modified mRNA provides for enhanced stability and/or reduced immunogenicity in in vivo therapies.
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METHODS FOR PURIFICATION OF ARYLSULFATASE A (Fri, 18 Jul 2014)
The present invention provides, among other things, improved methods for purifying arylsulfatase A (ASA) protein produced recombinantly for enzyme replacement therapy. The present invention is, in part, based on the surprising discovery that recombinant ASA protein can be purified from unprocessed biological materials, such as, ASA-containing cell culture medium, using a process involving as few as four chromatography columns and only one step of post-chromatographic ultrafiltration/diafiltration.
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AMPHETAMINE PRODRUGS (Sat, 04 Jan 2014)
The present invention relates to amphetamine prodrugs which provide colonic release of amphetamine.
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PURIFICATION OF IDURONATE-2-SULFATASE (Sat, 04 Jan 2014)
The present invention provides, among other things, improved methods for purifying 12 S protein produced recombinantly for enzyme replacement therapy. The present invention is, in part, based on the surprising discovery that recombinant I2S protein can be purified from unprocessed biological materials, such as, I2S-containing cell culture medium, using a process involving as few as four chromatography columns.
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IONIZABLE CATIONIC LIPIDS (Fri, 04 Oct 2013)
Disclosed herein are novel compounds, pharmaceutical compositions comprising such compounds and related methods of their use. The compounds described herein are useful, e.g., as liposomal delivery vehicles to facilitate the delivery of encapsulated polynucleotides to target cells and subsequent transfection of said target cells, and in certain embodiments are characterized as having one or more properties that afford such compounds advantages relative to other similarly classified lipids.
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CLEAVABLE LIPIDS (Fri, 14 Dec 2012)
Disclosed herein are novel compounds, pharmaceutical compositions comprising such compounds and related methods of their use. The compounds described herein are useful, e.g., as liposomal delivery vehicles to facilitate the delivery of encapsulated polynucleotides to target cells and subsequent transfection of said target cells, and in certain embodiments are characterized as having one or more properties that afford such compounds advantages relative to other similarly classified lipids.
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MEXILETINE PRODRUGS (Fri, 29 Jun 2012)
The present invention concerns prodrugs of mexiletine (and mexiletine's active metabolite) pharmaceutical compositions containing such prodrugs. Methods for treating myotonic conditions, while reducing the inherent adverse G1 side effects associated with mexiletine, increasing the bioavailability of mexiletine, and improving the pharmacokinetic reproducibility of mexiletine with the aforementioned prodrugs are also provided.
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PROCESS FOR THE PREPARATION OF ANAGRELIDE AND ANALOGUES THEREOF (Fri, 27 Apr 2012)
The present invention relates to a novel process for producing anagrelide, 6,7-dichloro-1,5- dihydroimidazo [2,1-b] quinazolin 2 (3H)-one, or certain analogues thereof. The process of the invention also provides improved processes for producing key intermediates required for the synthesis of anagrelide or certain analogues thereof.
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USE OF PRODRUGS TO AVOID GI MEDIATED ADVERSE EVENTS (Fri, 13 Apr 2012)
The present invention relates to prodrugs of a wide variety of drugs and pharmaceutical compositions containing such prodrugs. Methods for minimizing locally mediated (from within the gut lumen) adverse gastrointestinal events associated with the underivatised drug and increasing the sustainment of plasma drug levels with the aforementioned prodrugs are also provided. Thus, the present invention relates to the use of prodrugs of a wide diversity of drugs (other than opioids) to transiently inactivate them and so reduce directly, locally mediated adverse gastrointestinal (GI) side–effects normally evident after administration of the parent compound. Additionally, such prodrugs may confer improved pharmacokinetics.
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PRODRUGS OF GUANFACINE (Fri, 23 Mar 2012)
Prodrugs of guanfacine, pharmaceutical compositions containing such prodrugs and a method for providing therapeutic benefit in the treatment of ADHD/ODD (attention deficient hyperactivity disorder and oppositional defiance disorder) with guanfacine prodrugs are provided herein. Additionally, methods for improving the pharmacokinetics of guanfacine or minimizing or avoiding the adverse gastrointestinal side effects associated with guanfacine administration are provided herein.
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DESAZADESFERROTHIOCIN AND DESAZADESFERROTHIOCIN POLYETHER ANALOGUES AS METAL CHELATION AGENTS (Fri, 11 Nov 2011)
Disclosed herein are new compounds of desazadesferrothiocin polyether (DADFT- PE) analogues, as well as pharmaceutical compositions comprising them and their application as metal chelation agents for the treatment of disease. Methods of chelation of iron and other metals in a human or animal subject are also provided for the treatment of metal overload and toxicity.
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PRODRUGS OF OPIOIDS AND USES THEREOF (Fri, 15 Jul 2011)
The present invention concerns prodrugs of opioid analgesics and pharmaceutical compositions containing such prodrugs. Methods for providing more consistent pain relief by increasing the bioavailability of the opioid analgesic with the aforementioned prodrugs are provided. The invention also provides for decreasing the adverse GI side effects of opioid analgesics.
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DELIVERY OF MRNA FOR THE AUGMENTATION OF PROTEINS AND ENZYMES IN HUMAN GENETIC DISEASES (Fri, 10 Jun 2011)
Disclosed herein are compositions and methods of modulating the expression of gene or the production of a protein by transfecting target cells with nucleic acids. The compositions disclosed herein demonstrate a high transfection efficacy and are capable of ameliorating diseases associated with protein or enzyme deficiencies.
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[1,4]-BENZODIAZEPINES AS VASOPRESSIN V2 RECEPTOR ANTAGONISTS (Fri, 01 Apr 2011)
The invention relates to a novel class of [1,4]-benzodiazepine derivatives, processes for their preparation, intermediates usable in these processes, and pharmaceutical compositions containing the compounds. Other aspects of the invention are directed to the use of said [1,4]-benzodiazepine derivatives in therapy based on the capability of said compounds to interfere with the binding of the peptide hormone, vasopressin, to its receptors. In particular as vasopressin V2 receptor antagonists and therefore useful for treating involving increased vascular resistance, cardiac insufficiency, and water retention.
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PRODRUGS OF GUANFACINE (Fri, 25 Mar 2011)
Prodrugs of guanfacine with amino acids or short peptides,pharmaceutical compositions containing such prodrugs and a method for providing therapeutic benefit in the treatment of ADHD/ODD (attention deficient hyperactivity disorder and oppositional defiance disorder) with guanfacine prodrugs are provided herein. Additionally, methods for minimizing or avoiding the adverse gastrointestinal side effects associated with guanfacine administration, as well as improving the pharmacokinetics of guanfacine are provided herein.
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GALANTAMINE AMINO ACID AND PEPTIDE PRODRUGS AND USES THEREOF (Fri, 28 Jan 2011)
Prodrugs of galantamine or its 3-hydroxy metabolite with amino acids or short peptides, pharmaceutical compositions containing such prodrugs and methods for treating a memory or cognition disorder with the galantamine prodrugs are provided herein. Prodrugs having side chains of valine, phenylalanine, tyrosine or para amino benzoic acid and mono-, di- and tripeptides thereof are preferred. Additionally, methods for avoiding or minimizing the adverse gastrointestinal side effects associated with galantamine administration, as well as improving the pharmacokinetics of galantamine are provided herein.
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