75 patents
Utility
Method of Treating Duchenne Muscular Dystrophy
28 Dec 23
Provided herein are methods of treating or delaying the onset of Duchenne muscular dystrophy using modified antisense oligonucleotides.
Charles A. O'Neill, Shripad S. Bhagwat, David Riley Neil, David Reid Jacoby
Filed: 3 May 23
Utility
Process for Enriching Adeno-associated Virus
21 Dec 23
The present invention provides process for enriching adeno-associated virus particles using anion exchange chromatography and zonal ultracentrifugation.
John MAGA, Harmit VORA, Rahul SHETH, Daniel GOLD, Anant RISHI, Yanhong ZHANG, Kieu TRAN
Filed: 2 Nov 21
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
Dystrophin Exon Skipping Oligonucleotides
2 Nov 23
Provided herein are antisense oligonucleotides (AONs) that induce skipping of exon 51 of human dystrophin pre-mRNA, and pharmaceutically acceptable derivatives thereof.
Steven Michael Froelich, Josh Christopher Woloszynek, Jenna-Marie Magat, Judith Christina Theodora van Deutekom, Peter Christian de Visser, Nicole Anne Datson
Filed: 29 Mar 23
Utility
Treatment of Hereditary Angioedema with Liver-specific Gene Therapy Vectors
26 Oct 23
Provided herein are compositions and methods for treating a Cl esterase inhibitor deficiency by normalizing levels of the Cl esterase inhibitor protein in a subject having HAE.
Peter COLOSI
Filed: 13 Nov 20
Utility
Novel Adeno-associated Virus Capsid Proteins
12 Oct 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: 8 Dec 22
Utility
Lysosomal Targeting Peptides and Uses Thereof
3 Aug 23
The present invention provides further improved compositions and methods for efficient lysosomal targeting based on the GILT technology.
Jonathan LeBowitz, John Maga
Filed: 27 Jul 22
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
C-Type Natriuretic Peptide Variants to Treat Skeletal Dysplasia in Children
4 May 23
The present disclosure provides for use of variants of C-type natriuretic peptide (CNP), for the treatment of skeletal dysplasias in children, and improvement in one or more symptoms of skeletal dysplasias, such as long bone growth or growth velocity, and use in other disorders having a skeletal dysplasia and/or CNP-associated symptom or component.
Jonathan Day, Elena Fisheleva
Filed: 11 Jul 22
Utility
Glycolate Oxidase Inhibitors for the Treatment of Disease
23 Mar 23
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: 23 May 22
Utility
Glycolate Oxidase Inhibitors for the Treatment of Disease
23 Mar 23
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 a defect in glyoxylate metabolism, for example a disease or disorder associated with the enzyme glycolate oxidase (GO) or alterations in oxalate metabolism.
Bing WANG, Brett E. CRAWFORD, Shripad BHAGWAT, Xiaomei BAI
Filed: 18 Jun 20
Utility
Glycolate Oxidase Inhibitors for the Treatment of Disease
16 Mar 23
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: 23 May 22
Utility
Virus-free Cell Cultures
9 Mar 23
The invention is directed to methods of removing adventitious virus from cells and cell lines and virus-free cells and cell lines obtainable by such methods.
Michael LOCHRIE, Peter COLOSI, Barrie CARTER, Tomas CINEK
Filed: 9 Feb 21
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
Novel Adeno-associated Virus Capsid Proteins
22 Dec 22
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
Secondary Container with Flow-through Apertures for Freezing, Thawing, and Shipping Products, and Associated Methods
24 Nov 22
Containers are provided herein for facilitating freezing, thawing, and/or storage of products that may be perishable, such as pharmaceuticals.
Richard WISNIEWSKI, Saeed MOSHASHAEE, Pooja SANE
Filed: 13 Nov 20
Utility
Compositions of Prokaryotic Phenylalanine Ammonia-lyase and Methods of Treating Adolescent Subjects
24 Nov 22
Phenylalanine ammonia-lyase (PAL) variants with a greater phenylalanine-converting activity and/or a reduced immunogenicity as compared to a wild-type PAL for therapeutic uses, including the treatment of adolescent subjects having PKU.
Debra LOUNSBURY
Filed: 18 May 22
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
Lysosomal Targeting Peptides and Uses Thereof
10 Nov 22
The present invention provides further improved compositions and methods for efficient lysosomal targeting based on the GILT technology.
Jonathan LeBowitz, John Maga
Filed: 1 Jun 22