28 patents
Utility
Liver targeting adeno-associated viral vectors
21 Nov 23
The invention relates to novel adeno-associated virus (AAV) capsid proteins, AAV particles comprising a novel capsid protein, polynucleotides encoding these capsid proteins and AAV vectors expressing these capsid proteins.
Peter Cameron Colosi, Silvia Ramirez
Filed: 14 May 19
Utility
Adeno-associated virus Factor VIII vectors, associated viral particles and therapeutic formulations comprising the same
4 Jul 23
The invention provides adeno-associated virus (AAV) Factor VIII (FVIII)-encoding/expressing vectors and virus, including AAV FVIII vectors with high expression activity and AAV FVIII vectors that express full-length or truncated functional FVIII protein.
Stuart Bunting, Peter Cameron Colosi, Erno Pungor
Filed: 6 Nov 19
Utility
Use of C-type natriuretic peptide variants to treat skeletal dysplasia
28 Feb 23
The present disclosure provides for use of variants of C-type natriuretic peptide (CNP), and novel pharmaceutical compositions and formulations comprising CNP variant peptides for the treatment of skeletal dysplasias, one or more symptoms of skeletal dysplasias, such as long bone growth or growth velocity, and other disorders having a skeletal dysplasia and/or CNP-associated symptom or component.
Sherry Bullens, Stuart Bunting, Tianwei Chou, Augustus O. Okhamafe, Christopher P. Price, Daniel J. Wendt, Clarence Yap
Filed: 1 Apr 20
Utility
Adeno-associated virus capsid proteins
21 Feb 23
The invention relates to novel adeno-associated virus (AAV) capsid proteins, AAV particles comprising a novel capsid protein, polynucleotides encoding these capsid proteins and AAV vectors expressing these capsid proteins.
Peter Cameron Colosi, Michael Lochrie, Robert Ng
Filed: 25 Jul 17
Utility
Compositions of prokaryotic phenylalanine ammonia-lyase variants and methods of using compositions thereof
22 Nov 22
Provided herein are phenylalanine ammonia-lyase (PAL) variants produced by prokaryotes, wherein such prokaryotic PAL variant has a greater phenylalanine-converting activity and/or a reduced immunogenicity as compared to a wild-type PAL.
Augustus O. Okhamafe, Sean M. Bell, G. Nick Zecherle, Kris Antonsen, Yanhong Zhang, Kieu Ly Tran, Paul A. Fitzpatrick, Emil D. Kakkis, Michel Claude Vellard, Daniel J. Wendt, Mubarack Muthalif
Filed: 11 Jan 19
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
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
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
Manufacture of active highly phosphorylated human lysosomal sulfatase enzymes and uses thereof
2 Mar 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: 6 Feb 20
Utility
Glucosylceramide synthase inhibitors for the treatment of diseases
23 Feb 21
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, Daniel Chu, Alexander James Bridges
Filed: 31 Jan 19
Utility
Histone deacetylase inhibitors
30 Nov 20
Provided herein are compounds and methods for inhibiting histone deacetylase (“HDAC”) enzymes (e.g., HDAC1, HDAC2, and HDAC3).
Shripad Bhagwat, Gregory Luedtke, Alexander Bridges
Filed: 30 Jun 16
Utility
Quantification of non-reducing end glycan residual compounds for determining the presence, identity, or severity of a disease or condition
5 Oct 20
Provided herein are methods of diagnosing or monitoring the treatment of abnormal glycan accumulation or a disorder associated with abnormal glycan accumulation.
Brett E. Crawford, Jillian R. Brown, Charles A. Glass, Jim R. Beitel, Robin M. Jackman
Filed: 19 Feb 19
Utility
TPP-1 formulations and methods for treating CLN2 disease
31 Aug 20
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: 27 Mar 19