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In some embodiments, the present invention provides method of identifying compounds that bind to phosphoinositol 4-phosphate adaptor protein-2 (FAPP2), including the steps of computationally identifying a compound that binds to FAPP2 using the atomic coordinates of at least the amino acids which make up the substrate binding pocket of FAPP2. Also provided are methods of designing, selecting and/or optimizing a compound that binds to FAPP2.

A lipoprotein lipase (LPL) protein for treating and/or preventing HTG and its associated diseases, including but not limited to acute pancreatitis (AP), and in particular, acute pancreatitis secondary to or exacerbated by hypertriglyceridemia, and hypertriglyceridemia and its associated diseases in general, including cardiovascular and metabolic diseases, endocrine disorders, and fat embolism syndrome.

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.

Shire Inc | Date: 2017-09-06

The present invention provides, among other things, methods and compositions for treating complement mediated disease, in particular, chronic diseases requiring prophylactic and/or maintenance treatment. In one aspect, C1-INH fusion proteins having longer half-life than native plasma-derived C1-INH are provided. In some embodiments, a method according to the present invention includes administering to an individual who is suffering from or susceptible to a complement-mediated disease, an effective amount of a recombinant C1-INH fusion protein such that at least one symptom or feature of said complement-mediated disease is prevented and/or reduced in intensity, severity, or frequency.

A method of producing mannose-6-phosphate (M6P)-containing recombinant alpha-N-acetyl-glucosaminidase (Naglu), including the steps of providing a high mannose containing recombinant Naglu protein; and contacting the high mannose containing recombinant Naglu protein with N-acetyl-glucosamine-1-phosphotransferase (GNPT) under conditions that permit phosphorylation of one or more mannose residues on the recombinant Naglu protein, thereby generating M6P-containing recombinant Naglu. A mannose-6-phosphate (M6P)-containing recombinant alpha-N-acetyl-glucosaminidase (Naglu) protein produced by this method, a composition comprising such Naglu protein, and a method of treating Sanfilippo Syndrome Type B (MPS IIIB) including administering to a subject in need of treatment this composition.

A targeted therapeutic including a lysosomal enzyme and a lysosomal targeting moiety that is a peptide containing at least one N-linked glycosylation site. Methods of producing the targeted therapeutic may include nucleotide acids encoding the same and host cells co-expresing GNPT. Pharmaceutical compositions comprising the targeted therapeutic and methods of using the same to treat a lysosomal storage disease.

Shire Inc | Date: 2017-03-01

The present invention provides, among other things, methods of purifying messenger RNA (mRNA) including the steps of (a) precipitating mRNA from an impure preparation; (b) subjecting the impure preparation comprising precipitated mRNA to a purification process involving membrane filtration such that the precipitated mRNA is captured by a membrane; and (c) eluting the captured precipitated mRNA from the membrane by re-solubilizing the mRNA, thereby resulting in a purified mRNA solution. In some embodiments, a purification process involving membrane filtration suitable for the present invention is tangential flow filtration.

Shire Inc | Date: 2017-07-26

The invention provides compositions and methods for effective lysosomal targeting mediated by SORT1. In particular, the compositions and methods provided by the invention may be used to treat lysosomal storage diseases such as Sanfilippo syndrome type B.

Shire Inc | Date: 2017-02-01

The present invention provides, among other things, a method of ocular delivery of messenger RNA (mRNA), comprising administering into an eye of a subject in need of delivery a composition comprising an mRNA encoding a protein, such that the administration of the composition results in expression of the protein encoded by the mRNA in the eye.

Shire Inc | Date: 2017-05-10

The present invention provides an improved process for lipid nanoparticle formulation and mRNA encapsulation. In some embodiments, the present invention provides a process of encapsulating messenger RNA (mRNA) in lipid nanoparticles comprising a step of mixing a mRNA solution and a lipid solution, wherein the mRNA solution and/or the lipid solution are at a pre-determined temperature greater than ambient temperature.

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