290 patents
Utility
Treatment of Hemophilia with Fitusiran
4 Jan 24
The present disclosure provides methods for using fitusiran to treat patients with hemophilia A or hemophilia B.
Shauna Andersson, Baisong Mei, Fadi Shammas, Christian Sussebach
Filed: 8 Jun 23
Utility
Compositions and Methods for Treating Spinal Muscular Atrophy
4 Jan 24
The present provides methods for treating spinal muscular atrophy using a self-complementary recombinant adeno-associated virus (rAAV) viral particle comprising a transgene expressing SMN.
Marco A. Passini, Lamya S. Shihabuddin, Catherine R. O'Riordan, Seng H. Cheng
Filed: 21 Apr 23
Utility
5-MEMBERED Heteroarylaminosulfonamides for Treating Conditions Mediated by Deficient CFTR Activity
4 Jan 24
The invention relates to heteroaryl compounds, pharmaceutically acceptable salts thereof, and pharmaceutical preparations thereof.
Junkai Liao, Mark Munson, Zhongli Gao, Gregory Hurlbut, Sylvie Baltzer, Bertrand Vivet, Brian Freed, Hans Peter Nestler, Helen Yeoman, Ingrid Mechin, Martin Smrcina, Nina Ma, Sylvain Lebreton, Ryan Hartung, William Wire, Sukanthini Thurairatnam
Filed: 11 May 22
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
Use of an IRAK4 Modulator for Gene Therapy
21 Dec 23
Provided herein are methods for enhancing gene therapy in an individual by administering an IRAK degrader with the gene therapy to suppress innate immunity to the gene therapy.
Sourav Roy CHOUDHURY, Mona MOTWANI, Christian MUELLER, John REED
Filed: 12 Apr 23
Utility
Use of IRAK4 Modulators for Gene Therapy
21 Dec 23
Provided herein are methods for enhancing gene therapy in an individual by administering an IRAK modulator (e.g., an IRAK-4 degrader) with the gene therapy to suppress innate immunity to the gene therapy.
Sourav Roy CHOUDHURY, Mona MOTWANI, Christian MUELLER, John REED
Filed: 12 Apr 23
Utility
Dendritic Cell Assay for Innate Immunogenicity to Gene Therapy Agents
21 Dec 23
Provided herein are methods for determining the innate immunogenicity of a gene therapy agent in an individual.
Sourav Roy CHOUDHURY, Mona MOTWANI
Filed: 12 Apr 23
Utility
Methods of Treating Metachromatic Leukodystrophy
14 Dec 23
Provided herein are expression cassettes for expressing a transgene in a liver cell, wherein the transgene encodes an ARSA polypeptide.
Shyam RAMACHANDRAN
Filed: 15 May 23
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 Binding Polypeptides for Optimized Drug Conjugation
7 Dec 23
Engineered antibodies that are stable and can be conjugated to a ligand or drug at a ligand/drug to antibody ratio of above 3, making these conjugates suitable for treatment of a variety of indications, are provided.
Qun Zhou, Sunghae Park, Huawei Qiu, Marie-Priscille Brun, Francis Duffieux
Filed: 19 Apr 23
Utility
VARIANT RNAi
7 Dec 23
Provided herein are RNAi molecules for treating Huntington's disease.
Catherine R. O'RIORDAN, Adam Palermo, Brenda Richards, Lisa M. Stanek
Filed: 15 Feb 23
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
Methods for Reducing Glycosphingolipid Concentration In Brain Tissue and Methods of Treatment of Neurodegenerative Diseases Involving the Same
23 Nov 23
Provided are methods for treating or preventing Parkinson's disease or dementia with Lewy Bodies in a human subject.
Tanya Zaremba FISCHER, Judith PETERSCHMITT
Filed: 30 Jul 21
Utility
Induction of Immune Tolerance by Using Methotrexate
23 Nov 23
The invention provides methods for reducing undesired immune responses, such as anti-drug antibody (ADA) responses and other T- and/or B-cell-mediated immune responses, in patients by using treatment with methotrexate.
Alexandra JOSEPH, Susan RICHARDS, Melanie RUZEK, Richard GARMAN
Filed: 28 Apr 23
Utility
Pharmaceutical Recombinant Human Acid Sphingomyelinase Compositions and Methods
16 Nov 23
Disclosed here are compositions comprising recombinant acid sphingomyelinase (rASM) having desired purity, specific activity, and/or rASM isoforms.
Thomas M. Wasylenko, Kevin Brower, Xiaoying Jin
Filed: 20 Mar 23
Utility
SERPINC1 iRNA COMPOSITIONS AND METHODS OF USE THEREOF
16 Nov 23
The invention relates to iRNA, e.g., double-stranded ribonucleic acid (dsRNA), compositions targeting the Serpinc1 gene, and methods of using such iRNA, e.g., dsRNA, compositions to inhibit expression of Serpinc1 and methods of treating subjects having a bleeding disorder, such as a hemophilia.
Akin Akinc, Alfica Sehgal, Ivanka Toudjarska, Donald Foster, Stuart Milstein, Brian Bettencourt, Martin A. Maier, Klaus Charisse, Satyanarayana Kuchimanchi, Kallanthottathil G. Rajeev, Muthiah Manoharan
Filed: 17 Jan 23
Utility
Targeted Gene Therapy for DM-1 Myotonic Dystrophy
16 Nov 23
Provided herein are RNAi molecules for treating myotonic dystrophy type 1 (DM1).
Seng Cheng, Sarah Melissa Jacobo, Takako Moriguchi, Catherine O'Riordan, Guoxiang Ruan
Filed: 5 Apr 23
Utility
Gene Therapy for Amyotrophic Lateral Sclerosis and Other Spinal Cord Disorders
16 Nov 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: 14 Dec 22