158 patents
Utility
Methods of treating Fabry disease in patients having a mutation in the GLA gene
5 Dec 23
Provided are methods of treating a patient diagnosed with Fabry disease and methods of enhancing α-galactosidase A in a patient diagnosed with or suspected of having Fabry disease.
Elfrida Benjamin, Xiaoyang Wu
Filed: 7 Aug 20
Utility
Methods Of Treating Fabry Patients Having Renal Impairment
30 Nov 23
Provided are methods for treatment of Fabry disease in patients having HEK assay amenable mutations in α-galactosidase A.
Jeff Castelli, Elfrida Benjamin
Filed: 31 May 23
Utility
Methods Of Treating Fabry Patients Having Renal Impairment
30 Nov 23
Provided are methods for treatment of Fabry disease in patients having HEK assay amenable mutations in α-galactosidase A.
Jeff Castelli, Elfrida Benjamin
Filed: 31 May 23
Utility
Methods of treating Fabry patients having renal impairment
28 Nov 23
Provided are methods for treatment of Fabry disease in patients having HEK assay amenable mutations in α-galactosidase A.
Jeff Castelli, Elfrida Benjamin
Filed: 22 Oct 20
Utility
Dosing Regimens for the Treatment of Lysosomal Storage Diseases Using Pharmacological Chaperones
16 Nov 23
The present invention provides dosing regimens for administering pharmacological chaperones to a subject in need thereof.
Jeff Castelli, David J. Lockhart
Filed: 18 May 23
Utility
Methods of treating Fabry patients having renal impairment
14 Nov 23
Provided are methods for treatment of Fabry disease in patients having HEK assay amenable mutations in α-galactosidase A.
Jeff Castelli, Elfrida Benjamin
Filed: 21 Dec 22
Utility
Highly Purified Batches Of Pharmaceutical Grade Migalastat And Methods Of Producing The Same
2 Nov 23
Provided are methods of producing Active Pharmaceutical Ingredient (API) grade migalastat hydrochloride, and for purifying intermediate grade migalastat hydrochloride.
Kamlesh Sheth, Sergey Tesler, James Cartwright, Clive King, Wendy Cross
Filed: 26 Jun 23
Utility
Methods of treating Fabry patients having renal impairment
17 Oct 23
Provided are methods for treatment of Fabry disease in patients having HEK assay amenable mutations in α-galactosidase A.
Jeff Castelli, Elfrida Benjamin
Filed: 21 Dec 22
Utility
Treatment Of Fabry Disease In ERT-Naïve And ERT-Experienced Patients
12 Oct 23
Provided are dosing regimens for the treatment of Fabry disease in a patient.
Jeff Castelli
Filed: 18 May 23
Utility
Highly potent acid alpha-glucosidase with enhanced carbohydrates
12 Sep 23
Recombinant human alpha glucosidase (rhGAA) composition derived from CHO cells that contains a more optimized glycan composition consisting of a higher amount of rhGAA containing N-glycans carrying mannose-6-phosphate (M6P) or bis-M6P than conventional rhGAAs, along with low amount of non-phosphorylated high mannose glycans, and low amount of terminal galactose on complex oligosaccharides.
Russell Gotschall, Hung V. Do
Filed: 23 Feb 21
Utility
Gene Therapy Constructs and Methods of Use
27 Jul 23
Provided herein are improved gene therapy vectors and methods of use, in some embodiments, comprising sequences for improved expression and cellular targeting of a therapeutic protein.
Hung V. Do, Steven Tuske, Russell Gotschall, Ce Feng Liu
Filed: 7 Nov 22
Utility
Methods Of Treating Fabry Patients Having Renal Impairment
13 Jul 23
Provided are methods for treatment of Fabry disease in patients having HEK assay amenable mutations in α-galactosidase A.
Jeff Castelli, Elfrida Benjamin
Filed: 21 Dec 22
Utility
Method For Capturing And Purification Of Biologics
13 Jul 23
Methods for the continuous production, capturing and purification of biologics such as recombinant proteins are described.
Kumar Dhanasekharan, Christine Caroselli, Paul Langevin
Filed: 4 Sep 20
Utility
Methods Of Treating Fabry Patients Having Renal Impairment
13 Jul 23
Provided are methods for treatment of Fabry disease in patients having HEK assay amenable mutations in α-galactosidase A.
Jeff Castelli, Elfrida Benjamin
Filed: 21 Dec 22
Utility
Highly Potent Acid Alpha-Glucosidase With Enhanced Carbohydrates
29 Jun 23
Recombinant human alpha glucosidase (rhGAA) composition derived from CHO cells that contains a more optimized glycan composition consisting of a higher amount of rhGAA containing N-glycans carrying mannose-6-phosphate (M6P) or bis-M6P than conventional rhGAAs, along with low amount of non-phosphorylated high mannose glycans, and low amount of terminal galactose on complex oligosaccharides.
Russell Gotschall, Hung V. Do
Filed: 17 Feb 23
Utility
Methods Of Treating Fabry Patients Having Renal Impairment
29 Jun 23
Provided are methods for treatment of Fabry disease in patients having HEK assay amenable mutations in α-galactosidase A.
Jeff Castelli, Elfrida Benjamin
Filed: 21 Dec 22
Utility
Highly purified batches of pharmaceutical grade 1-deoxygalactonojirimycin compounds
27 Jun 23
Provided are methods of producing Active Pharmaceutical Ingredient (API) grade migalastat hydrochloride, and for purifying intermediate grade migalastat hydrochloride.
Kamlesh Sheth, Sergey Tesler, James Cartwright, Clive King, Wendy Cross
Filed: 15 Dec 21
Utility
Recombinant Alpha-Galactosidase A For Treatment Of Fabry Disease
25 May 23
Described are compositions comprising α-galactosidase A enzymes with unique carbohydrate profiles, as well as methods for manufacturing and purifying such enzymes.
Russell Gotschall
Filed: 13 Jun 22
Utility
Methods of treating Fabry patients having renal impairment
9 May 23
Provided are methods for treatment of Fabry disease in patients having HEK assay amenable mutations in α-galactosidase A.
Jeff Castelli, Elfrida Benjamin
Filed: 22 Oct 20
Utility
Methods Of Treating Fabry Disease In Patients Having A Mutation In The GLA Gene
4 May 23
Provided are methods of treating a patient diagnosed with Fabry disease and methods of enhancing α-galactosidase A in a patient diagnosed with or suspected of having Fabry disease.
Elfrida Benjamin, Xiaoyang Wu
Filed: 5 Mar 21