75 patents
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Utility
Adeno-Associated Virus Factor VIII Vectors
27 Oct 22
The invention provides improved adeno-associated virus (AAV) Factor VIII (FVIII) vectors, including AAV FVIII vectors that produce a functional Factor VIII polypeptide and AAV FVIII vectors with high expression activity.
Peter Cameron Colosi, Amit Nathwani, Jenny McIntosh, Edward Tuddenham, Andrew Davidoff
Filed: 30 Jun 22
Utility
Characterization of Gene Therapy Viral Particles Using Size Exclusion Chromatography and Multi-angle Light Scattering Technologies
29 Sep 22
This disclosure relates to the use of size exclusion chromatography and/or size exclusion chromatography with multi-angle light scattering technology to characterize viral particles such as adeno-associated virus and lentivirus particles.
Vikas BHAT, Geoffrey Yehuda BERGUIG, Nicole Louise MCINTOSH
Filed: 25 Sep 20
Utility
Hydrophobic Peptide Salts for Extended Release Compositions
8 Sep 22
The present disclosure, relates, in general, to hydrophobic salts of hydrophilic peptides that form low solubility materials in aqueous solutions and are capable of extended or sustained release of the peptide component when administered to a subject.
Jake Ang, Sunil Kumar Battu, Joshua Laber, Karol Estrada, Jonathan LeBowitz, Joyce Chou
Filed: 12 Aug 20
Utility
Methods for Treating CLN2 Disease in Pediatric Subjects
1 Sep 22
Provided herein are methods of treating Neuronal Ceroid Lipofuscinosis (CLN2) disease in a subject less than 3 years old.
David Jacoby, Joshua Henshaw
Filed: 31 Aug 20
Utility
Targeted Therapeutic Lysosomal Enzyme Fusion Proteins, Associated Formulations and Uses Thereof
11 Aug 22
The present disclosure relates in general to therapeutic lysosomal enzyme fusion proteins useful for treating lyso-somal storage diseases, liquid formulations comprising such fusion proteins and associated methods useful for treating lysosomal storage diseases in mammals.
SAEED MOSHASHAEE, JASON K. PINKSTAFF, ADAM SHAYWITZ, NATALIE CIACCIO
Filed: 25 Apr 22
Utility
Adeno-associated virus factor VIII vectors
9 Aug 22
The present disclosure provides methods of generating multiplexed genetically modified animals, for example, porcine endogenous retrovirus (PERV)-inactivated pigs.
Peter Cameron Colosi, Amit Nathwani, Jenny McIntosh, Edward Tuddenham, Andrew Davidoff
Filed: 30 Sep 19
Utility
Glycolate oxidase inhibitors for the treatment of disease
12 Jul 22
Described herein are compounds, methods of making such compounds, pharmaceutical compositions and medicaments containing such compounds, and methods of using such compounds to treat or prevent diseases or disorders associated with the enzyme glycolate oxidase (GO).
Bing Wang, Qi Chao
Filed: 28 Dec 18
Utility
N-(1-hydroxy-3-(pyrrolidinyl)propan-2-yl)pyrrolidine-3-carboxamide derivatives as glucosylceramide synthase inhibitors
14 Jun 22
Described herein are compounds of Formula I, methods of making such compounds, pharmaceutical compositions and medicaments containing such compounds, and methods of using such compounds to treat or prevent diseases or conditions associated with the enzyme glucosylceramide synthase (GCS).
Bing Wang
Filed: 12 Feb 19
Utility
Lysosomal targeting peptides and uses thereof
7 Jun 22
The present invention provides further improved compositions and methods for efficient lysosomal targeting based on the GILT technology.
Jonathan LeBowitz, John Maga
Filed: 24 Jul 17
Utility
Targeted therapeutic lysosomal enzyme fusion proteins, associated formulations and uses thereof
31 May 22
The present disclosure relates in general to therapeutic lysosomal enzyme fusion proteins useful for treating lysosomal storage diseases, liquid formulations comprising such fusion proteins and associated methods useful for treating lysosomal storage diseases in mammals.
Saeed Moshashaee, Jason K. Pinkstaff, Adam Shaywitz, Natalie Ciaccio
Filed: 24 Feb 17
Utility
Use of C-type Natriuretic Peptide Variants to Treat Osteoarthritis
26 May 22
The disclosure relates to the use of variants of C-type natriuretic peptide (CNP) to treat osteoarthritis, to ameliorate one or more symptoms of osteoarthritis, and to treat disorders having an osteoarthritis component.
Charles A. O'Neill, Todd M. Oppeneer, Jason K. Pinkstaff
Filed: 3 Dec 21
Utility
Targeted Therapeutic Lysosomal Enzyme Fusion Proteins and Uses Thereof
28 Apr 22
The present invention relates in general to therapeutic fusion proteins useful to treat lysosomal storage diseases and methods for treating such diseases.
Mika Aoyagi-Scharber, Teresa Margaret Christianson, Melita Dvorak-Ewell, Daniel J. Wendt, Shinong Long, Jonathan LeBowitz, Daniel Solomon Gold
Filed: 10 Jan 22
Utility
Cell-based assays for detection of antibodies or other factors that neutralize uptake of lysosomal enzymes
1 Mar 22
The present invention relates to cell-based methods for screening body fluids or tissues for factors that prevent cellular uptake of lysosomal enzymes, including neutralizing factors such as neutralizing antibodies, that arise as a result of lysosomal enzyme replacement therapy.
Andrew Melton, Stephen Zoog, Lynne Jesaitis
Filed: 7 Nov 16
Utility
Targeted therapeutic lysosomal enzyme fusion proteins and uses thereof
22 Feb 22
The present invention relates in general to therapeutic fusion proteins useful to treat lysosomal storage diseases and methods for treating such diseases.
Mika Aoyagi-Scharber, Teresa Margaret Christianson, Melita Dvorak-Ewell, Daniel J. Wendt, Shinong Long, Jonathan LeBowitz, Daniel Solomon Gold
Filed: 8 Apr 19
Utility
TPP-1 formulations and methods for treating CLN2 disease
25 Jan 22
Formulations comprising recombinant human tripeptidyl peptidase-1 (rhTPP1) for intrathecal, intracerebroventricular, or intraocular administration, and kits comprising the same, are disclosed.
Thomas Lester, Saeed Moshashaee, Augustus O. Okhamafe, Charles A. O'Neill
Filed: 28 Jul 20
Utility
Use of C-type natriuretic peptide variants to treat osteoarthritis
21 Dec 21
The disclosure relates to the use of variants of C-type natriuretic peptide (CNP) to treat osteoarthritis, to ameliorate one or more symptoms of osteoarthritis, and to treat disorders having an osteoarthritis component.
Charles A. O'Neill, Todd M. Oppeneer, Jason K. Pinkstaff
Filed: 8 Dec 16
Utility
Substances for targeting various selected organs or tissues
11 Nov 21
The invention provides conjugates, comprising an organ or tissue targeting moiety linked to a biologically active moiety or linked to a diagnostic moiety.
Peter Christian DE VISSER, Adriana Marie AARTSMA-RUS, Silvana Maria Gerarda JIRKA, Peter Abraham Christiaan 'T HOEN, Maria BegoƱa AGUILERA DIEZ
Filed: 15 Nov 17
Utility
Manufacture of Active Highly Phosphorylated Human Lysosomal Sulfatase Enzymes and Uses Thereof
24 Jun 21
This invention provides compositions of active highly phosphorylated lysosomal sulfatase enzymes, their pharmaceutical compositions, methods of producing and purifying such lysosomal sulfatase enzymes and compositions and their use in the diagnosis, prophylaxis, or treatment of diseases and conditions, including particularly lysosomal storage diseases that are caused by, or associated with, a deficiency in the lysosomal sulfatase enzyme.
Erno Pungor, Charles Hague, Zhi Chen, Melita Dvorak-Ewell, Michel Claude Vellard, Vish Koppaka
Filed: 1 Mar 21
Utility
Delivery of therapeutic compounds to the brain and other tissues
22 Jun 21
The present invention relates to the intrathecal (IT) administration of recombinant enzyme to treat lysosomal storage disorders.
Emil D. Kakkis
Filed: 6 Jan 17
Utility
Glycolate Oxidase Inhibitors for the Treatment of Disease
10 Jun 21
Described herein are compounds, methods of making such compounds, pharmaceutical compositions and medicaments containing such compounds, and methods of using such compounds to treat or prevent diseases or disorders associated with the enzyme glycolate oxidase (GO).
Bing Wang, Qi Chao
Filed: 28 Dec 18