103 patents
Utility
Pharmaceutical compositions comprising venglustat
2 Jan 24
The present disclosure relates to pharmaceutical compositions, including dosage forms, such as tablets or capsules, comprising venglustat, in free base, or pharmaceutically acceptable salt form, a diluent/filler and a lubricant, optionally in combination with one or more additional therapeutic agents, to processes for manufacture thereof, and to methods of use in the treatment or prevention of disease.
Audrey Jeanjean, Danielle Combessis, David Rigal, Chris Ho, Pankaj Taneja
Filed: 23 Jul 21
Utility
Sterile chromatography resin and use thereof in manufacturing processes
12 Dec 23
Provided herein are methods of reducing bioburden of (e.g., sterilizing) a chromatography resin that include exposing a container including a composition including a chromatography resin and at least one antioxidant agent and/or chelator to a dose of gamma-irradiation sufficient to reduce the bioburden of the container and the chromatography resin, where the at least one antioxidant agent and/or chelator are present in an amount sufficient to ameliorate the loss of binding capacity of the chromatography resin after/upon exposure to the dose of gamma-irradiation.
Rahul Godawat, Veena Warikoo, Rohan Patil, Konstantin Konstantinov, Venkat Kishore Ryakala
Filed: 14 Jan 21
Utility
Methods for purification of recombinant AAV vectors
12 Dec 23
Provided herein are methods for the purification of recombinant adeno-associated virus (rAAV) vectors that can be used for gene transfer and specifically for gene therapy or vaccination.
Paulene McLean Quigley Sheldon, Peter S. Gagnon, Gina Nichols, Barbara A. Thorne
Filed: 28 Sep 20
Utility
Modified-IgG antibodies that bind transforming growth factor-BETA1 with high affinity, avidity and specificity
5 Dec 23
A modified IgG antibody binds and neutralizes TGFβ1 selectively and with high affinity and avidity.
Huawei Qiu, Julie Bird
Filed: 11 Apr 22
Utility
Methods for absolute quantification of low-abundance polypeptides using mass spectrometry
5 Dec 23
The present invention provides methods for improved label-free absolute quantification of relatively low abundant polypeptides by liquid chromatography/mass spectrometry analysis of peptide products obtained from simple or complex polypeptide mixtures.
Martha Stapels, Michelle Busch
Filed: 17 Dec 20
Utility
Crystalline imidazo[4,5-b]pyridine compound, pharmaceutical compositions, and their use in treating medical conditions
14 Nov 23
The invention provides a crystalline imidazo[4,5-b]pyridine compound, pharmaceutical compositions, methods of inhibiting tropomyosin-related kinase and/or c-FMS, and methods of treating medical diseases and conditions, such as pain.
Benoit Robert
Filed: 11 Nov 22
Utility
Hyperglycosylated binding polypeptides
7 Nov 23
Provided are binding polypeptides (e.g., antibodies), and effector moiety conjugates thereof, comprising a CH1 domain (e.g., a human IgG1 CH1 domain), wherein the CH1 domain has an engineered N-linked glycosylation site at amino acid position 114, according to Kabat numbering.
Clark Pan, Huawei Qiu, Pradeep Dhal, Bo Chen, Diego Gianolio
Filed: 17 Oct 19
Utility
Inhibiting or downregulating glycogen synthase by creating premature stop codons using antisense oligonucleotides
31 Oct 23
The present disclosure relates to antisense oligonucleotides (AONs) for modulating the expression of glycogen synthase.
Carol A. Nelson, Bruce M. Wentworth, Ronald K. Scheule, Timothy E. Weeden, Nicholas P. Clayton
Filed: 5 May 20
Utility
Methods for quantifying drug concentration in a prodrug composition
24 Oct 23
Methods for quantifying the amount of drug present in a crosslinked hyaluronic acid-linker-peptide (xHA-L-P) prodrug composition are provided.
Kim Alving, Bing Wang
Filed: 6 May 20
Utility
Pharmaceutical formulations of tropomyosin related kinase (TRK) inhibitors
24 Oct 23
Pharmaceutical formulations with a tropomyosin-related kinase inhibitor (“Trk inhibitor”) are disclosed.
Harvey Lieberman, Donglai Yang, C. Michael Philbrook, Michael Santos, Chris Ho
Filed: 20 Aug 21
Utility
Variant RNAi
10 Oct 23
Provided herein are RNAi molecules including a first strand containing a guide sequence and a second strand comprising a non-guide sequence where the non-guide sequence contains a bulge opposite the seed region of the guide sequences; e.g., opposite the cleavage sequence.
Lisa M. Stanek, Adam Palermo, Brenda Richards, Sergio Pablo Sardi, Catherine O′Riordan, Antonius Song
Filed: 28 Jul 20
Utility
Methods for the quantitation of polypeptides
12 Sep 23
Provided are methods for quantitating an amount of a polypeptide that comprises a portion of an antibody present in a sample (e.g., a plasma or serum sample) wherein the antibody comprises a constant region (e.g., a heavy chain or light chain constant region) that comprises an engineered mutation.
Christopher Morgan, Xiaokui Zhang
Filed: 16 Jun 21
Utility
Site-specific glycoengineering of targeting moieties
11 Jul 23
The current disclosure provides binding polypeptides (e.g., antibodies), and targeting moiety conjugates thereof, comprising a site-specifically engineered glycan linkage within native or engineered glycans of the binding polypeptide.
Luis Avila, Clark Pan, Huawei Qiu, Qun Zhou
Filed: 29 Mar 21
Utility
Methods for detecting AAV
11 Jul 23
Provided herein are methods for determining the serotype of a virus particle and/or or determining the heterogeneity of a virus particle (e.g., an AAV particle).
Xiaoying Jin, Catherine O'Riordan, Lin Liu, Kate Zhang
Filed: 14 Aug 17
Utility
Early post-transfection isolation of cells (EPIC) for biologics production
27 Jun 23
Provided herein are methods for selecting a population of cells expressing a target polypeptide.
Victor R. Cairns, Christine DeMaria, Jason Vitko
Filed: 14 Nov 19
Utility
Analytical ultracentrifugation for characterization of recombinant viral particles
2 May 23
Provided herein are methods to characterize preparations of recombinant viral particles using analytical ultracentrifugation.
Catherine R. O'Riordan, Brenda Burnham
Filed: 21 Aug 19
Utility
FLARE (flow cytometry attenuated reporter expression) technology for rapid bulk sorting
25 Apr 23
Provided herein are methods and compositions for batch production of producer cells using fluorescence activated cell sorting (FACS).
Victor R. Cairns, Jose Ignacio Sancho Chavida, Christine DeMaria
Filed: 7 Oct 16
Utility
Variant RNAi
14 Mar 23
Provided herein are RNAi molecules for treating Huntington's disease.
Catherine R. O'Riordan, Adam Palermo, Brenda Richards, Lisa M. Stanek
Filed: 21 Sep 18
Utility
Gene therapy for amyotrophic lateral sclerosis and other spinal cord disorders
17 Jan 23
This disclosure provides methods and compositions for treating disorders or injuries that affect motor function and control in a subject.
James Dodge, Lamya Shihabuddin, Catherine O'Riordan
Filed: 20 Aug 19
Utility
Deuterated colony stimulating factor-1 receptor (CSF-1R) inhibitors
20 Dec 22
John L. Kane, Jr., Nellwyn A. Hagan, Maria A. Fitzgerald
Filed: 11 Feb 22