34 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
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
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
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
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
Modular Vial Adapter
16 Sep 21
The present disclosure is directed to a vial adapter for interconnecting a vial and a fluid delivery device, and, more particularly, to a vial adapter having a modular design consisting of separately constructed components cooperatively arranged and coupled to one another.
Scott Richard ARIAGNO, Daniel Edward ROUSH
Filed: 3 Jun 21
Utility
Small Unit Dosage Plunger Rod Stops
9 Sep 21
Plunger rod stops attach to existing syringes to limit the range of motion of the plunger, thereby to precisely control the amount of liquid that can be expelled from the syringe when the plunger rod stop is in place.
Steve BOWMAN, Chase FETZER, Richard BRAGA, Evi SHIAKOLAS
Filed: 13 May 21
Utility
ANTI-FLT-1 Antibodies In Treating Bronchopulmonary Dysplasia
26 Aug 21
The present invention provides, among other things, methods and compositions for treating chronic lung disorders, in particular, bronchopulmonary dysplasia (BPD).
Dennis Keefe, Jennifer Bosco, Steven Abman
Filed: 21 Jun 19
Utility
ANTI-FLT-1 Antibodies for Treating Duchenne Muscular Dystrophy
8 Jul 21
The present invention provides, among other things, anti-Flt-I antibodies and methods for treating muscular dystrophy, in particular, Duchenne muscular dystrophy (DMD).
Dennis KEEFE, Hans DE HAARD, Natalie DE JONGE, Sofie GABRIELS
Filed: 12 Aug 20
Utility
ANTI-FLT-1 Antibodies In Treating Duchenne Muscular Dystrophy
3 Jun 21
The present invention provides, among other things, methods and compositions for treating muscular dystrophy, in particular.
Serene Josiah, Thomas M. Luby, Atsushi Asakura, Dennis Keefe, Lawrence Charnas, Mayank Verma
Filed: 14 Jul 20
Utility
ANTI-FLT-1 Antibodies In Treating Bronchopulmonary Dysplasia
6 May 21
The present invention provides, among other things, methods and compositions for treating chronic lung disorders, in particular, bronchopulmonary dysplasia (BPD).
Dennis KEEFE, Hans DE HAARD
Filed: 5 Jun 20