61 patents
Page 3 of 4
Utility
Treatment of Cognitive Impairment of Hunter Syndrome by Intrathecal Delivery of IDURONATE-2-SULFATASE
2 Dec 20
The present invention, provides a method of treating cognitive impairment of Hunter syndrome.
Ann Barbier, Thomas McCauley, Charles W. Richard, III
Filed: 14 Jan 20
Utility
MANNOSE-6-PHOSPHATE Bearing Peptides Fused to Lysosomal Enzymes
2 Dec 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: 17 Jun 20
Utility
ANTI-FLT-1 Antibodies In Treating Bronchopulmonary Dysplasia
18 Nov 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: 4 Dec 19
Utility
Formulations Comprising Glucocerebrosidase and Isofagomine
7 Oct 20
The invention provides a composition of glucocerebrosidase, such as velaglucerase alfa, and isofagomine, in a molar ratio of at least about 1:2.5.
Yung Hee PARK, Nancy CHEN, Jun HU, Muthuraman MEIYAPPAN, Thomas Allen MILLER
Filed: 24 Oct 18
Utility
Inhibitors of Plasma Kallikrein and Uses Thereof
7 Oct 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: 21 Jun 20
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
Recombinant Follistatin-fc Fusion Proteins and Use In Treating Duchenne Muscular Dystrophy
5 Aug 20
The present invention provides, among other things, methods and compositions for treating muscular dystrophy, in particular, Duchenne muscular dystrophy (DMD).
Andrea Iskenderian, Angela W. Norton, Chuan Shen, Clark Pan
Filed: 2 Mar 17
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
Stable Formulations for CNS Delivery of Arylsulfatase a
29 Jul 20
The present invention provides, among other things, compositions and methods for CNS delivery of lysosomal enzymes (e.g., recombinant human arylsulfatase A (rhASA)) for effective treatment of lysosomal storage diseases (e.g., Metachromatic Leukodystrophy Disease).
Nazila Salamat-Miller, Katherine Taylor, Paul Campolieto, Zahra Shahrokh, Jing Pan, Lawrence Charnas, Teresa Leah Wright, Pericles Calias, Keethkumar Jain, Sujit Basu
Filed: 16 Apr 20
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
Utility
Follistatin in treating duchenne muscular dystrophy
27 Jul 20
The present invention provides, among other things, methods and compositions for treating muscular dystrophy, in particular, Duchenne muscular dystrophy (DMD).
Rochelle Mineau
Filed: 11 Apr 18
Utility
Anti-FLT-1 antibodies in treating bronchopulmonary dysplasia
13 Jul 20
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: 6 Apr 16
Utility
Collagen 7 compositions and methods of using the same
29 Jun 20
The present disclosure relates to compositions comprising collagen 7 and methods of using the same to treat DEB.
Igor Quinones-Garcia, Lin Guey, Kris Lowe, Vinh Nguyen, Bing He, Amey Bandekar, Sujit Basu
Filed: 20 Dec 16
Utility
Stable formulations for CNS delivery of arylsulfatase A
25 May 20
The present invention provides, among oilier things, compositions and methods for CNS delivery of lysosomal enzymes (e.g., recombinant human arylsulfatase A (rhASA)) for effective treatment of lysosomal storage diseases (e.g.
Nazila Salamat-Miller, Katherine Taylor, Paul Campolieto, Zahra Shahrokh, Jing Pan, Lawrence Charnas, Teresa Leah Wright, Pericles Calias, Keethkumar Jain, Sujit Basu
Filed: 4 Jun 18
Utility
Methods and compositions for CNS delivery of arylsulfatase A
11 May 20
The present invention provides, among other things, compositions and methods for CNS delivery of lysosomal enzymes for effective treatment of lysosomal storage diseases.
Nazila Salamat-Miller, Katherine Taylor, Paul Campolieto, Zahra Shahrokh, Jing Pan, Lawrence Charnas, Teresa Leah Wright, Pericles Calias
Filed: 22 Aug 17
Utility
Treatment of cognitive impairment of mucopolysaccharidosis type IIIA by intrathecal delivery of heparan N-sulfatase
4 May 20
The present invention provides, among other things, effective treatment for Sanfilippo Syndrome Type A (MPS IIIA) based on intrathecal delivery of recombinant heparan N-sulfatase (HNS) enzyme.
Patrick Anthony John Haslett
Filed: 19 Jul 16
Utility
CNS Delivery of Therapeutic Agents
15 Apr 20
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: 16 Sep 19