27 patents
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
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 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
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
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
Utility
Therapeutic fusion protein comprising an alpha-n-acetylglucosaminidase and a lysosomal targeting moiety
20 Jul 21
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: 26 Sep 17
Utility
Methods and compositions for CNS delivery of heparan n-sulfatase
20 Jul 21
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: 16 Jul 18
Utility
Anti-CCL2 and anti-LOXL2 combination therapy for treatment of scleroderma
13 Jul 21
The present invention provides, among other things, bi-specific molecules including, but not limited to, antibodies, fynomers, aptamers, fusion proteins, and protein binding domains that bind both CCL2 and LOXL2 and uses thereof, in particular, for treatment of scleroderma and related fibrotic and/or inflammatory diseases, disorders and conditions.
Paolo G. V. Martini, Madhusudan Natarajan, Patrick Anthony John Haslett, Albert Barnes Seymour
Filed: 23 May 14
Utility
Modular vial adapter
15 Jun 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: 13 Dec 17
Utility
Methods and compositions for CNS delivery of arylsulfatase A
1 Jun 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: 17 Feb 17
Utility
Small unit dosage plunger rod stops
25 May 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: 6 Nov 17
Utility
Glucocerebrosidase gene therapy for Parkinson's disease
6 Apr 21
The present invention is directed, in part, to the treatment of a subject having a neurodegenerative disorder, such as Parkinson's disease (PD), by providing glucocerebrosidase enzyme.
Yung Hee Park, Ole Isacson
Filed: 6 May 16
Utility
Drug monitoring tool
19 Jan 21
Embodiments of the present disclosure relate to a drug monitoring tool.
Gerald Spotts, Roman Pichler, Michael Nelson
Filed: 22 Jan 18
Utility
Anti-Flt-1 antibodies for treating duchenne muscular dystrophy
28 Sep 20
The present invention provides, among other things, anti-Flt-1 antibodies and methods for treating muscular dystrophy, in particular, Duchenne muscular dystrophy (DMD).
Dennis Keefe, Hans De Haard, Natalie De Jonge, Sofie Gabriels
Filed: 6 Apr 16
Utility
Enzymatic activity assays for glucocerebrosidase
31 Aug 20
The present invention provides, among other things, methods and compositions for determining enzyme kinetic parameters (e.g., Vmax, Km, and specific activity, etc.) indicative of clinically relevant properties of glucocerebrosidase using a physiologically relevant substrate, in particular, a substrate that is representative of substrates that typically accumulate in patients suffering from Gaucher disease such as glucosylceramide.
Peter Bernhardt, Chen-Chung Willy Yen, Vijay Chhajlani
Filed: 15 Dec 15
Utility
Anti-FLT-1 antibodies in treating bronchopulmonary dysplasia
10 Aug 20
The present invention provides, among other things, methods and compositions for treating chronic lung disorders, in particular, bronchopulmonary dysplasia (BPD).
Dennis Keefe, Steven Abman, Gregory Seedorf
Filed: 6 Apr 16
Utility
Inhibitors of plasma kallikrein and uses thereof
3 Aug 20
The present invention provides compounds and compositions thereof which are useful as inhibitors of plasma kallikrein and which exhibit desirable characteristics for the same.
Nikolaos Papaioannou, Sarah Jocelyn Fink, Thomas Allen Miller, Gerald Wayne Shipps, Jr., Jeremy Mark Travins, David Edward Ehmann, Alastair Rae, John Mark Ellard
Filed: 11 Mar 19
Utility
Recombinant placenta growth factor for treating Duchenne muscular dystrophy
3 Aug 20
The present invention provides, among other things, methods and compositions for treating muscular dystrophy, in particular, Duchenne muscular dystrophy (DMD).
Angela Norton, Michael F. Concino, Muthuraman Meiyappan, Andrea Iskenderian
Filed: 27 Jan 14
Utility
Mannose-6-phosphate bearing peptides fused to lysosomal enzymes
27 Jul 20
A targeted therapeutic including a lysosomal enzyme and a lysosomal targeting moiety that is a peptide containing at least one N-linked glycosylation site.
Michael F. Concino, Bettina Strack-Logue, Muthuraman Meiyappan, Angela W. Norton, Bohong Zhang, Andrea Iskenderian, Lieh Yoon Low, Dianna Lundberg, Alla Romashko, Hicham Naimy
Filed: 10 Aug 15