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Genzyme Corporation patents


Recent patent applications related to Genzyme Corporation. Genzyme Corporation is listed as an Agent/Assignee. Note: Genzyme Corporation may have other listings under different names/spellings. We're not affiliated with Genzyme Corporation, we're just tracking patents.

ARCHIVE: New 2018 2017 2016 2015 2014 2013 2012 2011 2010 2009 | Company Directory "G" | Genzyme Corporation-related inventors


 new patent  Gene therapy for amyotrophic lateral sclerosis and other spinal cord disorders

This disclosure provides methods and compositions for treating disorders or injuries that affect motor function and control in a subject. In one aspect, the invention a transgene product is delivered to a subject's spinal cord by administering a recombinant viral vector containing the transgene to the spinal cord. ... Genzyme Corporation

Scfv-fc dimers that bind transforming growth factor-beta1 with high affinity, avidity and specificity

An scfv-fc dimer binds and neutralizes tgfβ1 selectively and with high affinity and avidity. The scfv region may comprise the same vh and vl domains or cdr regions as metelimumab. ... Genzyme Corporation

Bone-targeting antibodies

Provided are recombinant and chemically-conjugated antibodies and fragments thereof modified with one or more poly-aspartate (poly-d) peptides (e.g., a d10 sequence) to improve localization of the antibodies or fragments to bone. Methods of making and using of these antibodies and fragments also are disclosed.. ... Genzyme Corporation

Analytical ultracentrifugation for characterization of recombinant viral particles

Provided herein are methods to characterize preparations of recombinant viral particles using analytical ultracentrifugation. Recombinant viral particles include recombinant adeno-associated viral particles, recombinant adenoviral particles, recombinant lentiviral particles and recombinant herpes simplex virus particles. ... Genzyme Corporation

Affinity-based analytical purification of biotherapeutics for bioprocess monitoring

The invention as disclosed herein provides a method for purifying a non-antibody protein from solution, comprising a chromatography step wherein the solution is passed over an affinity construct containing an affinity ligand-coupled solid support, wherein the affinity construct is associated with a bioprocess unit operation, and isolating the non-antibody protein from solution.. . ... Genzyme Corporation

Novel beta-actin and rps21 promoters and uses thereof

The invention relates to isolation of novel β-actin promoters and uses thereof. In particular, this invention features nucleotide sequences for rodent β-actin promoters including β-actin promoter.. ... Genzyme Corporation

Multimeric constructs

Multimeric fusion proteins of an ig-like domain of flt-1 are rendered functional by inclusion of a linker moiety. Vectors encoding the fusion proteins and host cells expressing the fusion proteins can be used therapeutically to block neovascularization in individuals with pathological conditions related to neovascularization. ... Genzyme Corporation

Microcarrier perfusion culturing methods and uses thereof

Provided herein are methods of perfusion culturing an adherent mammalian cell using a shake flask and a plurality of microcarriers, and various methods that utilize these culturing methods.. . ... Genzyme Corporation

Early post-transfection isolation of cells (epic) for biologics production

Provided herein are methods for selecting a population of cells expressing a target polypeptide. In some aspects, the disclosure provides methods for sorting and selecting populations of transfected host cells based on their early expression of a selectable polypeptide. ... Genzyme Corporation

Chemical process

The present invention relates to chemical processes for the manufacture of certain quinazoline derivatives, or pharmaceutically acceptable salts thereof. The invention also relates to processes for the manufacture of certain intermediates useful in the manufacture of the quinazoline derivatives and to processes for the manufacture of the quinazoline derivatives utilising said intermediates. ... Genzyme Corporation

Perfusive organ hemostasis

Disclosed are compositions, methods and kits to control bleeding through the use of an internal occluder based on polymeric solutions, including use of reverse thermosensitive polymers in nephron-sparing surgeries, which produces a completely bloodless surgical field, allowing speedy resection. In certain embodiments, after a certain amount of time, the flow gradually resumes, with no apparent adverse consequences to the kidney. ... Genzyme Corporation

Thermo-sensitive bone growth compositions

A non-invasive injectable composition that contains type i collagen, an osteogenic growth factor (osf), such as a bone morphogenetic protein and a reverse thermo-sensitive biodegradable polymer such as poloxamer 407 in an aqueous vehicle. The formulation can be administered non-invasively, e.g., by injection, thus circumventing limitations of many currently marketed bone-inducing products. ... Genzyme Corporation

Fc containing polypeptides with altered glycosylation and reduced effector function

Provided are binding polypeptides (e.g., antibodies), and drug conjugates thereof, comprising an fc domain with an altered glycosylation profile and reduced effector function. In particular embodiment, the fc domain comprises: an asparagine residue at amino acid position 298, according to eu numbering; and a serine or threonine residue at amino acid position 300, according to eu numbering. ... Genzyme Corporation

Quinazoline derivatives as vegf inhibitors

The invention relates to quinazoline derivatives of formula (i), wherein m is an integer from 1 to 3; r1 represents halogeno or c1-3alkyl; x1 represents —o—; r2 is selected from one of the following three groups: 1) c1-5alkylr3 (wherein r3 is piperidin-4-yl which may bear one or two substituents selected from hydroxy, halogeno, c1-4 alkyl, c1-4hydroxyalkyl and c1-4alkoxy; 2) c2-5alkenylr3 (wherein r3 is as defined hereinbefore); 3) c2-5alkynylr3 (wherein r3 is as defined hereinbefore); and wherein any alkyl, alkenyl or alkynyl group may bear one or more substituents selected from hydroxy, halogeno and amino; and salts thereof; processes for their preparation, pharmaceutical compositions containing a compound of formula (i) or a pharmaceutically acceptable salt thereof as active ingredient. The compounds of formula (i) and the pharmaceutically acceptable salts thereof inhibit the effects of vegf, a property of value in the treatment of a number of disease states including cancer and rheumatoid arthritis.. ... Genzyme Corporation

03/29/18 / #20180088140

Assays for detecting the presence or amount of an anti-drug antibody

. . Methods and kits for detecting antibodies (e.g., anti-drug antibodies). Such methods and kits permit the detection of, for example, anti-drug antibodies in human body fluids, such as blood, plasma and serum.. ... Genzyme Corporation

03/29/18 / #20180086838

Anti-cxcr3 antibodies and methods of use thereof

The present disclosure provides anti-cxcr3 antibodies and methods of using the antibodies to diagnose and/or treat cxcr3-associated disorders such as diabetes mellitus type i (t1d), particularly new-onset t1d. In certain embodiments, disclosed herein are cxcr3 neutralizing antibodies.. ... Genzyme Corporation

03/29/18 / #20180085436

Methods for introducing mannose 6-phosphate and other oligosaccharides onto glycoproteins and applications thereof

Methods to introduce highly phosphorylated mannopyranosyl oligosaccharide derivatives containing mannose-6-phosphate (m6p), or other oligosaccharides bearing other terminal hexoses, to carbonyl groups on oxidized glycans of glycoproteins while retaining their biological activity are described. The methods are useful for modifying glycoproteins, including those produced by recombinant protein expression systems, to increase uptake by cell surface receptor-mediated mechanisms, thus improving their therapeutic efficacy in a variety of applications.. ... Genzyme Corporation

03/08/18 / #20180065957

Glucosylceramide synthase inhibitors

The invention relates to inhibitors of glucosylceramide synthase (gcs) useful for the treatment of metabolic diseases, such as lysosomal storage diseases, either alone or in combination with enzyme replacement therapy, cystic disease and for the treatment of cancer.. . ... Genzyme Corporation

03/01/18 / #20180055939

Excipients for use in adeno-associated virus pharmaceutical formulations and pharmaceutical formulations made therewith

Stable pharmaceutical compositions comprising recombinant adeno-associated virus (aav) virions are described. The compositions provide protection against loss of recombinant aav vector genomes and transduceability under conditions such as exposure to cycles of freezing and thawing and storage in glass or polypropylene vials. ... Genzyme Corporation

03/01/18 / #20180055916

Dose escalation enzyme replacement therapy for treating acid sphingomyelinase deficiency

The invention relates to dose escalation enzyme replacement therapy using acid sphingomyelinase (asm) for the treatment of human subjects having acid sphingomyelinase deficiency (asmd), and, in particular, patients with non-neurological manifestations of niemann-pick disease (npd), and in certain embodiments, npd type b.. . ... Genzyme Corporation

02/22/18 / #20180051054

Methods of processing a fluid including a recombinant therapeutic protein and use thereof

Provided herein are methods of processing a fluid that include the use of one or both of a circuit system including a tangential flow filtration (tff) unit and a circuit system including a tangential flow virus filtration (tfvf) unit. Also provided are integrated and continuous processes for manufacturing a therapeutic protein drug substance that include a step of processing a fluid including the recombinant therapeutic protein using any of the methods provided herein.. ... Genzyme Corporation

02/15/18 / #20180044412

Modified-igg antibodies that bind transforming growth factor-beta1 with high affinity, avidity and specificity

A modified igg antibody binds and neutralizes tgfβ1 selectively and with high affinity and avidity. The modified igg antibody comprises four polypeptide chains and may comprise modifications to the elbow regions of the polypeptide chains. ... Genzyme Corporation

02/08/18 / #20180036295

Methods for treating proteinopathies

This disclosure relates to a method of treating a proteinopathy in a subject, the method comprising administering to the subject an effective amount of a quinuclidine compound. The disclosure also relates to a method of reducing, reversing or preventing the accumulation of protein aggregates in tissue of a subject diagnosed as having a proteinopathy, or being at risk of developing a proteinopathy, the method comprising administering to the subject an effective amount of a quinuclidine compound. ... Genzyme Corporation

02/01/18 / #20180030134

Inhibitors of t-cell activation

The present invention provides a bispecific biologic comprising a ligand specific for ctla-4 and a ligand specific for a pmhc complex.. . ... Genzyme Corporation

01/25/18 / #20180023082

Variant rnai

Provided herein are rnai molecules including a first strand containing a guide sequence and a second strand comprising a non-guide sequence where the non-guide sequence contains a bulge opposite the seed region of the guide sequences; e.g., opposite the cleavage sequence. In some aspects, the invention provides rnai for treating huntington's disease. ... Genzyme Corporation

01/04/18 / #20180002365

Oligosaccharide-protein conjugates

Provided herein are conjugates comprising a protein and an oligosaccharide of one of formulae i-vi. Also provided herein are pharmaceutical compositions comprising such conjugates. ... Genzyme Corporation

01/04/18 / #20180000728

Pharmaceutical formulations of tropomyosin related kinase (trk) inhibitors

Pharmaceutical formulations with a tropomyosin-related kinase inhibitor (“trk inhibitor”) are disclosed. The pharmaceutical formulations comprise 3-(3-methoxy-4-((4-methoxybenzyl)oxy)benzyl)-6-(1-methyl-1h-pyrazol-4-yl)-3h-imidazo[4,5-b]pyridin-2-amine in microcrystalline suspension formulations in its monohydrate form, which shows improved characteristics over the anhydrate form, and in extended release formulations. ... Genzyme Corporation

12/28/17 / #20170369584

Hyperglycosylated binding polypeptides

Provided are binding polypeptides (e.g., antibodies), and effector moiety conjugates thereof, comprising a ch1 domain (e.g., a human igg1 ch1 domain), wherein the ch1 domain has an engineered n-linked glycosylation site at amino acid position 114, according to kabat numbering. Also provided are nucleic acids encoding the antigen-binding polypeptides, recombinant expression vectors and host cells for making such antigen-binding polypeptides. ... Genzyme Corporation

12/28/17 / #20170369583

Methods and compositions for treating lupus

The invention provides methods of treating lupus in a patient with an anti-cd52 antibody. Also includes are methods of increasing infiltration of regulatory t cells to affected sides of the patient's body, methods of reducing urine protein and/or albumin levels and methods of depleting lymphocytes to alleviate lupus symptoms.. ... Genzyme Corporation

12/28/17 / #20170369541

Methods to produce rod-derived cone viability factor (rdcvf)

The invention is related to methods of producing rod-derived cone viability factor (rdcvf). This invention also relates to the treatment of an ocular disease in a mammal using rdcvf. ... Genzyme Corporation

12/07/17 / #20170349665

Anti-cd52 antibodies

Anti-human cd52 antibodies and antigen-binding fragments thereof are provided. Also provided are isolated nucleic acids, recombinant vectors and host cells for making the antibodies and fragments. ... Genzyme Corporation

11/30/17 / #20170342144

Engineered anti-tgf-beta antibodies and antigen-binding fragments

Antibodies or antigen-binding fragments thereof are engineered to bind transforming growth factor-β (tgfβ). Tgfβ-isoform selective antibodies or antigen-binding fragments thereof may selectively bind human tgfβ1, compared to human tgfβ2 and human tgfβ3, or may selectively bind human tgfβ3, compared to human tgfβ1 and human tgfβ2. ... Genzyme Corporation

11/30/17 / #20170342127

Fusion proteins comprising pdgf and vegf binding portions and methods of using thereof

The present provides fusion proteins comprising pdgf and vegf binding portions, and recombinant viral particles encoding the fusion proteins. Compositions comprising the fusion proteins and viral particles as well as methods of using the same are also provided.. ... Genzyme Corporation

11/23/17 / #20170334903

Method of preparing glucosylceramide synthase inhibitors

The invention relates to a method of preparing inhibitors of glucosylceramide synthase (gcs) useful for the treatment metabolic diseases, such as lysosomal storage diseases, either alone or in combination with enzyme replacement therapy, and for the treatment of cancer.. . ... Genzyme Corporation

11/23/17 / #20170334888

Synthesis of udp-glucose: n-acylsphingosine glucosyltransferase inhibitors

Disclosed is a novel enantiomeric synthesis ceramide-like inhibitors of udp-glucose: n-acylsphingosine glucosyltransferase. Also disclosed are novel intermediates formed during the synthesis.. ... Genzyme Corporation

11/16/17 / #20170326203

Methods of treating autoimmune diseases

Novel methods for treating patients with autoimmune diseases are disclosed. The methods of the invention include first depleting circulating lymphocytes in the mammal, e.g., by administering anti-thymocyte antibody, and then, during the course of repopulation, administering to the mammal a therapeutically effective amount of latent tgf-β and/or another agent that promotes expansion of regulatory t cells. ... Genzyme Corporation

11/16/17 / #20170326172

Sachet formulation for amine polymers

A powder formulation comprises a pharmaceutically acceptable anionic stabilizer and an aliphatic amine polymer or a pharmaceutically acceptable salt thereof mixed with the anionic stabilizer. The powder formulation is conveniently packaged in a container, such as a sachet. ... Genzyme Corporation

11/09/17 / #20170319691

Combination therapy for treating breast cancer

The invention provides compositions and methods for treating breast cancer. Specifically, the invention relates to administering a transforming growth factor beta (tgfβ) antagonist in combination with capecitabine and ixabepilone to treat breast cancer.. ... Genzyme Corporation

10/26/17 / #20170307629

Method of identifying risk for thyroid disorder

A method for identifying a patient that is at risk for developing a thyroid disorder that occurs subsequent to treatment with a regimen that depletes lymphocytes, comprising determining whether antibodies directed against thyroid peroxidase or thyroid microsomes are present in the patient, wherein if the antibodies are present in the patient then the patient is at increased risk for developing a thyroid disorder. A particular embodiment is a method for identifying a patient with multiple sclerosis that is at risk for developing a thyroid disorder that occurs subsequent to treatment with a regimen that depletes cd52-positive cells, comprising determining whether antibodies directed against thyroid peroxidase or thyroid microsomes are present in the patient, wherein if the antibodies are present in the patient then the patient is at risk for developing the thyroid disorder.. ... Genzyme Corporation

10/26/17 / #20170304465

Adeno-associated viral vectors for treating myocilin (myoc) glaucoma

Provided herein are methods for treating myocilin (myoc) glaucoma using adeno-associated viral (aav) vectors. In some aspects, the aav vectors encode r-spondin 1 (rspo1), r-spondin 2 (rspo2), r-spondin 3 (rspo3) or r-spondin 4 (rspo4) and/or rnai that targets myocilin (myoc). ... Genzyme Corporation

10/12/17 / #20170290948

Temporary embolization using inverse thermosensitive polymers

Once aspect of the present invention relates to methods of embolizing a vascular site in a mammal comprising introducing into the vasculature of a mammal a composition comprising an inverse thermosensitive polymer, wherein said inverse thermosensitive polymer gels in said vasculature, which composition may be injected through a small catheter, and which compsitions gel at or below body temperature. In certain embodiments of the methods of embolization, said composition further comprises a marker molecule, such as a dye, radiopaque, or an mri-visible compound.. ... Genzyme Corporation

10/05/17 / #20170281641

Cyclic urea compounds as tropomyosin-related kinase (trk) inhibitors

Tropomyosin-related kinase inhibitors (trk inhibitors) are small molecule compounds useful in the treatment of disease. Trk inhibitors can be used as pharmaceutical agents and in pharmaceutical compositions. ... Genzyme Corporation

10/05/17 / #20170281593

Methods of treating fatty liver disease

The disclosure provides methods for treating fatty liver disease and associated conditions by inhibiting the synthesis of glucosphingolipids, as exemplified by the use of glucosylceramide synthase substrate analogs.. . ... Genzyme Corporation

09/21/17 / #20170267774

Site-specific antibody-drug conjugation through glycoengineering

The current disclosure provides binding polypeptides (e.g., antibodies), and effector moiety conjugates thereof (e.g., antibody-drug conjugates or adcs), comprising a site-specifically engineered drug-glycan linkage within native or engineered glycans of the binding polypetpide. The current disclosure also provides nucleic acids encoding the antigen-binding polypeptides, recombinant expression vectors and host cells for making such antigen-binding polypeptides. ... Genzyme Corporation

09/14/17 / #20170260545

Methods for producing preparations of recombinant aav virions substantially free of empty capsids

. . Methods for separating aav empty capsids from mixtures of aav vector particles and aav empty capsids are described. The methods use column chromatography techniques and provide for commercially viable levels of recombinant aav virions.. ... Genzyme Corporation

08/31/17 / #20170247664

Compositions and methods to prevent aav vector aggregation

Compositions and methods are provided for preparation of concentrated stock solutions of aav virions without aggregation. Formulations for aav preparation and storage are high ionic strength solutions (e.g. ... Genzyme Corporation

08/31/17 / #20170246114

Aliphatic amine polymer salts for tableting

The tablets, compositions and methods of the present invention, comprising a carbonate salt of an aliphatic amine polymer and s monovalent anion can prevent or ameliorate acidosis, in particular acidosis in patients with renal disease. The tablets and compositions of the present invention maintain a disintegration time of no greater than 30 minutes at 37° c. ... Genzyme Corporation

08/24/17 / #20170238938

Methods for preventing retropulsion of concretions and fragments during lithotripsy

One aspect of the present invention provides a method for the treatment of lithiasis, which mitigates the risk of damage to surrounding body tissue when removing a calculi (e.g., biological concretions, such as urinary, biliary, and pancreatic stones) that obstructs or may otherwise be present within a body's anatomical lumen. In one embodiment, the instant invention provides a method of using a polymer plug to occlude a lumen distal to a calculi, whereby calculi fragments resulting from lithotripsy are prevented from traveling up the lumen. ... Genzyme Corporation

08/17/17 / #20170233465

Treatment of myocardial infarction using tgf beta antagonists

Disclosed herein is a method of treating a patient suffering a myocardial infarction, particularly an acute myocardial infarction, or of reducing an adverse consequence of a myocardial infarction in a patient comprising administering an antagonist of tgf-β to the patient during the acute stage of the myocardial infarction.. . ... Genzyme Corporation

08/17/17 / #20170232117

Aav virions with decreased immunoreactivity and uses therefor

Methods of making and using recombinant aav virions with decreased immunoreactivity are described. The recombinant aav virions include mutated capsid proteins or are derived from non-primate mammalian aav serotypes and isolates that display decreased immunoreactivity relative to aav-2.. ... Genzyme Corporation

08/10/17 / #20170227551

Biomarkers of polycystic kidney disease and uses thereof

Provided herein are methods for determining the efficacy of treatment for polycystic kidney disease (pkd) in a patient, diagnosing pkd in a patient, staging pkd in a patient, and monitoring pkd in a patient. These methods include determining a single or multiple levels of one or more markers selected from the group of proliferating cell nuclear antigen (pcna), cyclin d1, cyclin d3, mapkerk kinase 1 (mek), ribosomal protein s6 (s6), phosphorylated ribosomal protein s6 (ps6), extracellular signal-regulated kinase (erk), phosphorylated extracellular signal-regulated kinase (perk), protein kinase b (akt), phosphorylated protein kinase b (pakt), caspase-2, total s6, and retinoblastoma binding protein (rbbp). ... Genzyme Corporation

08/10/17 / #20170227441

Flare (flow cytometry attenuated reporter expression) technology for rapid bulk sorting

Provided herein are methods and compositions for batch production of producer cells using fluorescence activated cell sorting (facs). In some aspects, the disclosure provides a drug-selection-free method for batch production of producer cells using facs. ... Genzyme Corporation

08/10/17 / #20170224704

Tropomyosin-related kinase (trk) inhibitors

Tropomyosin-related kinase inhibitors (trk inhibitors) are small molecule compounds useful in the treatment of disease. Trk inhibitors can be used as pharmaceutical agents and in pharmaceutical compositions. ... Genzyme Corporation

08/03/17 / #20170218012

Integrated continuous manufacturing of therapeutic protein drug substances

Provided herein are integrated continuous biomanufacturing processes for producing a therapeutic protein drug substance. Also provided are systems that are capable of continuously producing a therapeutic protein drug substance.. ... Genzyme Corporation

07/27/17 / #20170209482

Chemically modified dendrimers

Dendrimers comprising n-acyl urea terminal moieties are described herein. The dendrimers can be used, for example, in the treatment of arthritis.. ... Genzyme Corporation

07/20/17 / #20170202872

Direct compression polymer tablet core

The present invention provides a tablet core which comprises at least about 95% by weight of an aliphatic amine polymer. The invention also provides a method of producing a tablet core comprising at least about 95% by weight of an aliphatic amine polymer resin the method comprises the step of compressing the aliphatic amine polymer to form the tablet core. ... Genzyme Corporation

07/13/17 / #20170198263

Recombinant aav vectors expressing osteoprotective genes, including has2 and lubricin, useful in the treatment of osteoarthritis and related joint conditions in mammals

The present disclosure relates to recombinant viral vectors, to pharmaceutical compositions comprising such recombinant vectors, and to methods for prevention and treatment of osteoarthritis in mammals. In particular, this disclosure provides adeno-associated virus (aav) vectors capable of expressing, in a host, osteoprotective/chondroprotective bioactive proteins, including hyaluronan synthase 2 (has2) and lubricin (prg4). ... Genzyme Corporation

06/29/17 / #20170182189

Inhibiting or downregulating glycogen synthase by creating premature stop codons using antisense oligonucleotides

The present disclosure relates to antisense oligonucleotides (aons) for modulating the expression of glycogen synthase. Aons of the present disclosure may be useful in treating diseases associated with the modulation of the expression of the enzyme glycogen synthase, such as pompe disease. ... Genzyme Corporation

06/29/17 / #20170182171

Multiple oligonucleotide moieties on peptide carrier

The present disclosure relates to antisense oligonucleotides (aons), such as phosphorodiamidate morpholino oligonucleotides (pmos). The present disclosure further relates to the conjugation of multiple pmos to cationic cell penetrating peptides (cpps) to enhance the uptake of pmos into skeletal and cardiac muscle cells.. ... Genzyme Corporation

06/29/17 / #20170182087

Main chain polyamines

Main chain polyamines comprise amine and ammonium groups along the polymer backbone. Main chain polyamines can be used as pharmaceutical agents and in pharmaceutical compositions. ... Genzyme Corporation

06/22/17 / #20170173183

Gene therapy for retinitis pigmentosa

Provided herein are methods for treating retinitis pigmentosa using an aav particles encoding mir-708. In one aspect, viral particles are administered to the eye of a human subject; for example, by subretinal injection. ... Genzyme Corporation

06/01/17 / #20170152524

Gene therapy for neurometabolic disorders

The disclosure pertains to methods and compositions for treating disorders affecting the central nervous system (cns). These disorders include neurometabolic disorders such as lysosomal storage diseases that affect the central nervous system, e.g., niemann-pick a disease. ... Genzyme Corporation

06/01/17 / #20170152347

Amide dendrimer compositions

Amide compounds, amide polymers, compositions including amide compounds and amide polymers may be used to bind target ions, such as phosphorous-containing compounds in the gastrointestinal tract of animals. In some cases, amide compounds and amide polymers may include a core derived from an amide polyol and an organic polyacid or ester.. ... Genzyme Corporation

05/04/17 / #20170121734

Early post-transfection isolation of cells (epic) for biologics production

Provided herein are methods for selecting a population of cells expressing a target polypeptide. In some aspects, the disclosure provides methods for sorting and selecting populations of transfected host cells based on their early expression of a selectable polypeptide. ... Genzyme Corporation

04/20/17 / #20170107231

Nampt inhibitors and methods

The invention features compounds of formula (i), formula (i)-a and formula (i)-b as disclosed herein, as well as methods of synthesis, therapy, diagnostics, and assays.. . ... Genzyme Corporation

04/06/17 / #20170096683

Aav vectors for retinal and cns gene therapy

Provided herein are improved raav (e.g., raav2, raavrh8r, etc.) for enhanced gene therapy of ocular disorders or cns disorders wherein the raav comprise one or more substitutions of amino acids that interact with heparan sulfate proteoglycan. The invention provides methods for improved transduction of retinal cells and methods for treating ocular diseases with improved compositions of raav particles. ... Genzyme Corporation

03/30/17 / #20170087212

Gene therapy for neurodegenerative disorders

Compositions and methods for treating disorders affecting motor function, such as motor function affected by disease or injury to the brain and/or spinal cord, are disclosed.. . ... Genzyme Corporation

03/30/17 / #20170086849

Confinement of kidney stone fragments during lithotripsy

The present invention improves significantly the success rate of lithotripsy and reduces the risk of tissue damage, by injecting a temporary plug in front, and optionally behind a concretion (for extracorporeal lithotripsy) or behind a concretion (for intracorporeal lithotripsy). One aspect of the present invention relates to injecting an inverse thermosensitive polymer solution into a lumen, thereby preventing the migration of a concretion, or its fragments, upon extracorporeal or intracorporeal lithotripsy.. ... Genzyme Corporation

03/09/17 / #20170065725

Thyroid stimulating hormone compositions

Described herein are compositions of thyroid stimulating hormone (tsh), wherein at least one polyalkylene glycol polymer is attached to a carbohydrate site of the tsh. Also described are compositions of mutated thyroid stimulating hormone (tsh) and at least one polyalkylene glycol polymer, wherein the mutated tsh comprises a tsh in which one or more amino acid residues has been substituted with cysteine residue, and the polyalkylene glycol polymer is attached to the mutated tsh at the site of the substituted cysteine residue. ... Genzyme Corporation

02/16/17 / #20170042979

Targeting of glycoprotein therapeutics

. . Methods of making ligand-decorated polymer conjugates of therapeutic glycoproteins are described. Improved targeting of glycoproteins to specific tissues is achieved by masking the natural carbohydrate and other surface determinants with high molecular weight polymers, such as, e.g., peg, polysialic acid, etc., which in turn are decorated with target-specific ligands. ... Genzyme Corporation

01/19/17 / #20170014520

Oligosaccharides comprising an aminooxy group and conjugates thereof

The invention provides methods for the synthesis of oligosaccharides comprising an aminooxy group. The invention further provides oligosaccharides comprising an aminooxy group, methods for coupling oligosaccharides comprising an aminooxy group to glycoproteins, and oligosaccharide-protein conjugates. ... Genzyme Corporation

01/12/17 / #20170007719

Compositions and methods for treating and preventing macular degeneration

Compositions and methods for treating macular degeneration are disclosed. The methods utilize gene delivery to human eyes of soluble flt-1 receptors, as well fusion proteins including a soluble flt-1 receptor.. ... Genzyme Corporation








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