75 patents
Page 4 of 4
Utility
Use of C-type natriuretic peptide variants to treat skeletal dysplasia
11 May 20
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: 24 Jan 18
Utility
Adeno-associated Virus Factor VIII Vectors
1 Apr 20
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 Mclntosh, Edward Tuddenham
Filed: 29 Sep 19
Utility
Histone Deacetylase Inhibitors
18 Mar 20
Provided herein are compounds and methods for inhibiting histone deacetylase (“HDAC”) enzymes (e.g., HDAC1, HDAC2, and HDAC3).
Gregory Luedtke, Shripad Bhagwat
Filed: 21 Dec 17
Utility
Stable Expression of Aav Vectors In Juvenile Subjects
4 Mar 20
The invention relates to the use of adeno-associated virus (AAV) vectors to achieve long term expression of a transgene in the liver of a juvenile subject.
Stuart Bunting
Filed: 13 May 19
Utility
Adeno-associated Virus Factor VIII Vectors, Associated Viral Particles and Therapeutic Formulations Comprising the Same
26 Feb 20
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: 5 Nov 19
Utility
Manufacture of active highly phosphorylated human lysosomal sulfatase enzymes and uses thereof
17 Feb 20
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
Filed: 11 Jan 18
Utility
Histone deacetylase inhibitors
6 Jan 20
This invention relates to generally inhibiting histone deacetylase (“HDAC”) enzymes (e.g., HDAC1, HDAC2, and HDAC3).
Vincent Jacques, James R. Rusche, Norton P. Peet, Jasbir Singh
Filed: 2 Jan 18
Utility
Adeno-associated virus factor VIII vectors, associated viral particles and therapeutic formulations comprising the same
23 Dec 19
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: 22 Sep 16
Utility
Hdac Inhibitors
18 Dec 19
Vincent Jacques, James R. Rusche
Filed: 21 Aug 19
Utility
Histone Deacetylase Inhibitors
18 Dec 19
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
Methods of Treating Phenylketonuria
11 Dec 19
Provided herein are methods of treating phenylketonuria by normalizing levels of amino acids, neurotransmitters, and neurotransmitter metabolites in a subject having phenylketonuria.
Geoffrey Berguig, Rajeev Mahimkar, Hassib Akeefe, Peter Colosi
Filed: 7 May 19
Utility
Compositions of Prokaryotic Phenylalanine Ammonia-lyase Variants and Methods of Using Compositions Thereof
13 Nov 19
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: 10 Jan 19
Utility
Adeno-associated virus factor VIII vectors
4 Nov 19
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
Filed: 13 Oct 16
Utility
Ceramide Galactosyltransferase Inhibitors for the Treatment of Disease
23 Oct 19
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 ceramide galactosyltransferase (CGT), such as, for example, lysosomal storage diseases.
Bing WANG
Filed: 18 Dec 17
Utility
HDAC inhibitors
30 Sep 19
Vincent Jacques, James R. Rusche
Filed: 14 Jun 18