46 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
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
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
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
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
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