61 patents
Utility
Bile Acid Recycling Inhibitors for Treatment of Hypercholemia and Cholestatic Liver Disease
6 Jul 23
Provided herein are methods of treating or ameliorating hypercholemia or a cholestatic liver disease by administering to an individual in need thereof a therapeutically effective amount of an Apical Sodium-dependent Bile Acid Transporter Inhibitor (ASBTI) or a pharmaceutically acceptable salt thereof.
Bronislava GEDULIN, Michael GREY, Niall O'DONNELL
Filed: 23 Sep 22
Utility
Adeno-associated Virus Vectors Based Gene Therapy for Hereditary Angioedema
9 Feb 23
The present disclosure provides, among other things, a recombinant adeno-associated virus (rAAV) vector comprising an AAV8 capsid and a codon-optimized SERPING1 sequence encoding a human C1-esterase inhibitor.
Matthias Klugmann, Franziska Horling, Johannes Lengler, Patrice Douillard, Friedrich Scheiflinger, Hanspeter Rottensteiner, Bagirath Gangadharan
Filed: 23 Oct 20
Utility
Adeno Associated Viral Vector Delivery of Antibodies for the Treatment of Disease Mediated by Dysregulated Plasma Kallikrein
9 Feb 23
The present disclosure provides, among other things, a recombinant adeno-associated viral (rAAV) vector encoding an agent that inhibits the proteolytic activity of plasma kallikrein.
Jon Kenniston, Florie Borel, Madhusudan Natarajan, Vivian Choi, Dan Sexton, Alexey Seregin
Filed: 11 Jun 20
Utility
Methods and Compositions for CNS Delivery of IDURONATE-2-SULFATASE
26 Jan 23
The present invention provides, among other things, compositions and methods for CNS delivery of lysosomal enzymes for effective treatment of lysosomal storage diseases.
Gaozhong Zhu, Kris Lowe, Zahra Shahrokh, James Christian, Richard Fahrner, Jing Pan, Teresa Leah Wright, Pericles Calias
Filed: 18 Jan 22
Utility
Recombinant Heme Oxygenase-1 (HO-1) for the Treatment of Sickle Cell Disease
19 Jan 23
The present invention provides, among other things, methods and compositions for making and using recombinant heme oxygenase for treating sickle cell disease.
Solomon Fiifi Ofori-Acquah, Samit Ghosh, Joseph Sypek, Bohong Zhang, Xiuxia Sun, Clark Q. Pan, Daniel Minard Lajole, Chuan Shen
Filed: 24 Jul 20
Utility
Compositions comprising iduronate-2-sulfatase
20 Dec 22
The present invention provides, among other things, improved methods for purifying I2S protein produced recombinantly for enzyme replacement therapy.
Dave Nichols
Filed: 6 Nov 20
Utility
CNS delivery of therapeutic agents
18 Oct 22
The present invention provides an effective and less invasive approach for direct delivery of therapeutic agents to the central nervous system (CNS).
Pericles Calias, Jing Pan, Jan Powell, Lawrence Charnas, Thomas McCauley, Teresa Leah Wright, Richard Pfeifer, Zahra Shahrokh
Filed: 17 Sep 19
Utility
Bile Acid Recycling Inhibitors for Treatment of Pediatric Cholestatic Liver Diseases
25 Aug 22
Provided herein are methods of treating or ameliorating a pediatric cholestatic liver disease by non-systemically administering to an individual in need thereof a therapeutically effective amount of a pediatric formulation comprising an Apical Sodium-dependent Bile Acid Transporter Inhibitor (ASBTI) or a pharmaceutically acceptable salt thereof.
Bronislava GEDULIN, Michael Grey, Niall O'DONNELL
Filed: 27 Apr 22
Utility
Bile acid recycling inhibitors for treatment of pediatric cholestatic liver diseases
5 Jul 22
Provided herein are methods of treating or ameliorating a pediatric cholestatic liver disease by non-systemically administering to an individual in need thereof a therapeutically effective amount of a pediatric formulation comprising an Apical Sodium-dependent Bile Acid Transporter Inhibitor (ASBTI) or a pharmaceutically acceptable salt thereof.
Bronislava Gedulin, Michael Grey, Niall O'Donnell
Filed: 11 Oct 21
Utility
Methods and Compositions for CNS Delivery of Heparan N-sulfatase
5 May 22
The present invention provides, among other things, compositions and methods for CNS delivery of lysosomal enzymes for effective treatment of lysosomal storage diseases.
Farah Natoli, Gaozhong Zhu, Jennifer Terew, Yuan Jiang, Jamie Tsung, Zahra Shahrokh, Brian Vernaglia, Jing Pan, Richard Pfeifer, Pericles Calias
Filed: 17 Jun 21
Utility
Treatment of Sanfilippo Syndrome Type B
5 May 22
Among other things, the present invention provides methods and compositions of treating Sanfilippo syndrome type B (Sanfilippo B) by, e.g., intrathecal (IT) administration of a Naglu protein.
Michael F. Concino, Pericles Calias, Jing Pan, Kevin Holmes, Paolo Martini, Alla Romashko, Muthuraman Meiyappan, Bohong Zhang, Andrea Iskenderian, Dianna Lundberg, Angela Norton, Bettina Strack-Logue, Huang Yan, Mary Alessandrini, Richard Pfeifer
Filed: 17 Jun 21
Utility
Methods and Compositions for Treating Chronic Lung Diseases
24 Mar 22
The present invention provides, among other things, methods and compositions for treating Chronic Lung Disease (CLD), comprising administering to a subject in need of treatment a composition comprising insulin-like growth factor-1 (IGF-1) or an agonist or an analog thereof.
Norman Barton, Alexandra Mangili
Filed: 11 May 21
Utility
HTT Repressors and Uses Thereof
3 Mar 22
Disclosed herein are HTT repressors and methods and compositions for use of these HTT repressors.
Galen Carey, Matthew Chiocco, Vivian Choi, Brian Felice, Steven Froelich, Debra Klatte, Jeffrey Miller, David Paschon, Edward Rebar, Bryan Zeitler, Lei Zhang, H. Steve Zhang
Filed: 15 Jan 20
Utility
Methods and compositions for CNS delivery of iduronate-2-sulfatase
1 Mar 22
The present invention provides, among other things, compositions and methods for CNS delivery of lysosomal enzymes for effective treatment of lysosomal storage diseases.
Gaozhong Zhu, Kris Lowe, Zahra Shahrokh, James Christian, Richard Fahrner, Jing Pan, Teresa Leah Wright, Pericles Calias
Filed: 18 Jul 19
Utility
Bile Acid Recycling Inhibitors for Treatment of Pediatric Cholestatic Liver Diseases
27 Jan 22
Provided herein are methods of treating or ameliorating a pediatric cholestatic liver disease by non-systemically administering to an individual in need thereof a therapeutically effective amount of a pediatric formulation comprising an Apical Sodium-dependent Bile Acid Transporter Inhibitor (ASBTI) or a pharmaceutically acceptable salt thereof.
Bronislava GEDULIN, Michael Grey, Niall O'DONNELL
Filed: 11 Oct 21
Utility
Bile acid recycling inhibitors for treatment of pediatric cholestatic liver diseases
25 Jan 22
Provided herein are methods of treating or ameliorating a pediatric cholestatic liver disease by non-systemically administering to an individual in need thereof a therapeutically effective amount of a pediatric formulation comprising an Apical Sodium-dependent Bile Acid Transporter Inhibitor (ASBTI) or a pharmaceutically acceptable salt thereof.
Bronislava Gedulin, Michael Grey, Niall O'Donnell
Filed: 11 Nov 19
Utility
Adeno Associated Virus Vectors for the Treatment of Hunter Disease
28 Oct 21
The present disclosure provides, among other things, a recombinant adeno-associated virus (rAAV) vector comprising an AAV8 or AAV9 capsid and a codon-optimized sequence encoding a human iduronate-2-sulfatase (I2S) enzyme.
Vivian Choi, Xing Li
Filed: 9 Dec 20
Utility
Methods and Compositions for CNS Delivery of Arylsulfatase a
14 Oct 21
Provided are methods of treating metachromatic leukodystrophy comprising administering to a subject in need of treatment a therapeutically effective amount of recombinant arylsulfatase A enzyme.
Margaret Wasilewski, Anna Wijatyk
Filed: 30 Apr 21
Utility
Methods and Compositions for Treatment of Hunter Syndrome
14 Oct 21
The present invention provides, among other things, improved methods for purifying I2S protein produced recombinantly for enzyme replacement therapy.
Dave Nichols
Filed: 6 Nov 20
Utility
Antibody-resin coupling apparatus and methods
21 Sep 21
An antibody-resin coupling apparatus quickly and efficiently activates resin beads and couples them to antibodies, while preventing breakdown and crosslinking of the beads, thereby improving downstream column purification processes, extending the usable life of the resin beads, and increasing molecule capture efficiency of the resultant resin-antibody complexes, to allow improved isolation and purification of factor VIII molecules or other drug compounds.
Robert Toso, Richard Spanggord, Mei Tan, Van Tairiol, Yekaterina Lin
Filed: 30 Jul 18