Patents by Inventor Alfica Sehgal

Alfica Sehgal has filed for patents to protect the following inventions. This listing includes patent applications that are pending as well as patents that have already been granted by the United States Patent and Trademark Office (USPTO).

  • SERPINA1 iRNA COMPOSITIONS AND METHODS OF USE THEREOF
    Publication number: 20250043279
    Abstract: The invention relates to RNAi agents, e.g., double stranded RNAi agents, targeting the Serpina1 gene, and methods of using such RNAi agents to inhibit expression of Serpina1 and methods of treating subjects having a Serpina1 associated disease, such as a liver disorder.
    Type: Application
    Filed: April 24, 2024
    Publication date: February 6, 2025
    Inventors: Mark K. Schlegel, Maja Janas, Vasant R. Jadhav, Donald Foster, Muthiah Manoharan, Kallanthottathil G. Rajeev, Alexander V. Kel'in, Klaus Charisse, Jayaprakash K. Nair, Martin A. Maier, Shigeo Matsuda, Muthusamy Jayaraman, Alfica Sehgal, Christopher Brown, Kevin Fitzgerald, Stuart Milstein
  • PHENOTHIAZINE DERIVATIVES AND USES THEREOF
    Publication number: 20250034193
    Abstract: The present invention provides phenothiazine compounds, processes for their preparation, pharmaceutical compositions comprising the compounds, and the use of the compounds or the compositions in the treatment of various diseases or conditions, for example ribosomal disorders and ribosomopathies, e.g. Diamond Blackfan anemia (DBA).
    Type: Application
    Filed: October 7, 2024
    Publication date: January 30, 2025
    Inventors: David A. Bumcrot, Alfica Sehgal, Donald L. Hertzog
  • Phenothiazine derivatives and uses thereof
    Patent number: 12145963
    Abstract: The present invention provides phenothiazine compounds, processes for their preparation, pharmaceutical compositions comprising the compounds, and the use of the compounds or the compositions in the treatment of various diseases or conditions, for example ribosomal disorders and ribosomopathies, e.g. Diamond Blackfan anemia (DBA).
    Type: Grant
    Filed: November 16, 2022
    Date of Patent: November 19, 2024
    Assignee: Camp4 Therapeutics Corporation
    Inventors: David A. Bumcrot, Alfica Sehgal, Donald L. Hertzog
  • MODULATION OF GENE TRANSCRIPTION USING ANTISENSE OLIGONUCLEOTIDES TARGETING REGULATORY RNAS
    Publication number: 20240360451
    Abstract: Described herein are methods of modulating OTC gene transcription using antisense oligonucleotides (ASOs) targeting regulatory RNAs, such as promoter-associated RNAs and enhancer RNAs. These methods are useful for increasing expression of Ornithine transcarbamylase (OTC), thereby treating diseases associated with aberrant gene expression.
    Type: Application
    Filed: September 2, 2022
    Publication date: October 31, 2024
    Inventors: Alfica Sehgal, Bryan J. Matthews, Cynthia M. Smith, David A. Bumcrot, Justin A. Caravella, Mario Esteban Contreras Gamboa, Rachana S. Kelkar, Yun Joon Jung, Yuting Liu, Subhadeep Roy, Brynn Nicole-Yoshiko Akerberg, Rutuja Sudhakar Pai
  • MODULATION OF GENE TRANSCRIPTION USING ANTISENSE OLIGONUCLEOTIDES TARGETING REGULATORY RNAS
    Publication number: 20240336924
    Abstract: Described herein are methods of modulating gene transcription using antisense oligonucleotides (ASOs) targeting regulatory RNAs, such as promoter-associated RNAs and enhancer RNAs. These methods are useful for modulating the levels of gene products, for example, increasing expression of Carbamoyl-Phosphatase Synthetase 1 (CPS1), thereby treating diseases associated with aberrant gene expression.
    Type: Application
    Filed: June 24, 2024
    Publication date: October 10, 2024
    Inventors: Alfica Sehgal, Bryan J. Matthews, David A. Bumcrot, Justin A. Caravella, Mario Esteban Contreras Gamboa, Rachana S. Kelkar, Yun Joon Jung, Yuting Liu, Rutuja Sudhakar Pai, Henry Munir Moukheiber, Subhadeep Roy, Yuchun Guo
  • SERPINA1 iRNA compositions and methods of use thereof
    Patent number: 12071619
    Abstract: The invention relates to RNAi agents, e.g., double-stranded RNAi agents, targeting the Serpina1 gene, and methods of using such RNAi agents to inhibit expression of Serpina1 and methods of treating subjects having a Serpina1 associated disease, such as a liver disorder.
    Type: Grant
    Filed: January 5, 2021
    Date of Patent: August 27, 2024
    Assignee: Alnylam Pharmaceuticals, Inc.
    Inventors: Alfica Sehgal, Klaus Charisse, Brian Bettencourt, Martin A. Maier, Kallanthottathil G. Rajeev, Gregory Hinkle, Muthiah Manoharan
  • SERPINA1 SIRNAS: COMPOSITIONS OF MATTER AND METHODS OF TREATMENT
    Publication number: 20240255880
    Abstract: The technology described herein relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the Serpina1 gene, and methods of using such dsRNA compositions to inhibit expression of Serpina1.
    Type: Application
    Filed: August 29, 2023
    Publication date: August 1, 2024
    Inventors: Alfica Sehgal, David Bumcrot, Brian Bettencourt
  • SERPINC1 iRNA COMPOSITIONS AND METHODS OF USE THEREOF
    Publication number: 20230365965
    Abstract: 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.
    Type: Application
    Filed: January 17, 2023
    Publication date: November 16, 2023
    Applicant: Genzyme Corporation
    Inventors: Akin Akinc, Alfica Sehgal, Ivanka Toudjarska, Donald Foster, Stuart Milstein, Brian Bettencourt, Martin A. Maier, Klaus Charisse, Satyanarayana Kuchimanchi, Kallanthottathil G. Rajeev, Muthiah Manoharan
  • PHENOTHIAZINE DERIVATIVES AND USES THEREOF
    Publication number: 20230257410
    Abstract: The present invention provides phenothiazine compounds, processes for their preparation, pharmaceutical compositions comprising the compounds, and the use of the compounds or the compositions in the treatment of various diseases or conditions, for example ribosomal disorders and ribosomopathies, e.g. Diamond Blackfan anemia (DBA).
    Type: Application
    Filed: November 16, 2022
    Publication date: August 17, 2023
    Inventors: David A. Bumcrot, Alfica Sehgal, Donald L. Hertzog
  • MODIFIED RNA AGENTS WITH REDUCED OFF-TARGET EFFECT
    Publication number: 20230256001
    Abstract: One aspect of the present invention relates to double-stranded RNA (dsRNA) agent capable of inhibiting the expression of a target gene. The antisense strand of the dsRNA molecule comprises at least one thermally destabilizing nucleotide occurring at a seed region; the dsRNA comprises at least four 2?-fluoro modifications, and the sense strand of the dsRNA molecule comprises ligand, wherein the ligand is an ASGPR ligand. Other aspects of the invention relate to pharmaceutical compositions comprising these dsRNA molecules suitable for therapeutic use, and methods of inhibiting the expression of a target gene by administering these dsRNA molecules, e.g., for the treatment of various disease conditions.
    Type: Application
    Filed: September 30, 2022
    Publication date: August 17, 2023
    Applicant: ALNYLAM PHARMACEUTICALS, INC.
    Inventors: Mark K. SCHLEGEL, Maja JANAS, Vasant R. JADHAV, Muthiah MANOHARAN, Kallanthottathil G. RAJEEV, Muthusamy JAYARAMAN, Alexander V. KEL'IN, Shigeo MATSUDA, Klaus CHARISSE, Jayaprakash K. NAIR, Martin A. MAIER, Alfica SEHGAL, Christopher BROWN, Christopher THEILE, Stuart MILSTEIN
  • Serpina1 iRNA compositions and methods of use thereof
    Patent number: 11725207
    Abstract: The invention relates to RNAi agents, e.g., double stranded RNAi agents, targeting the Serpina1 gene, and methods of using such RNAi agents to inhibit expression of Serpina1 and methods of treating subjects having a Serpina1 associated disease, such as a liver disorder.
    Type: Grant
    Filed: March 8, 2021
    Date of Patent: August 15, 2023
    Assignee: Alnylam Pharmaceuticals, Inc.
    Inventors: Mark K. Schlegel, Maja Janas, Vasant R. Jadhav, Donald Foster, Muthiah Manoharan, Kallanthottathil G. Rajeev, Alexander V. Kel'in, Klaus Charisse, Jayaprakash K. Nair, Martin A. Maier, Shigeo Matsuda, Muthusamy Jayaraman, Alfica Sehgal, Christopher Brown, Kevin Fitzgerald, Stuart Milstein
  • Phenothiazine derivatives and uses thereof
    Patent number: 11542290
    Abstract: The present invention provides phenothiazine compounds, processes for their preparation, pharmaceutical compositions comprising the compounds, and the use of the compounds or the compositions in the treatment of various diseases or conditions, for example ribosomal disorders and ribosomopathies, e.g. Diamond Blackfan anemia (DBA).
    Type: Grant
    Filed: April 5, 2019
    Date of Patent: January 3, 2023
    Assignee: CAMP4 THERAPEUTICS CORPORATION
    Inventors: David A. Bumcrot, Alfica Sehgal, Donald L. Hertzog
  • Modified RNA agents with reduced off-target effect
    Patent number: 11504391
    Abstract: One aspect of the present invention relates to double-stranded RNA (dsRNA) agent capable of inhibiting the expression of a target gene. The antisense strand of the dsRNA molecule comprises at least one thermally destabilizing nucleotide occurring at a seed region; the dsRNA comprises at least four 2?-fluoro modifications, and the sense strand of the dsRNA molecule comprises ligand, wherein the ligand is an ASGPR ligand. Other aspects of the invention relates to pharmaceutical compositions comprising these dsRNA molecules suitable for therapeutic use, and methods of inhibiting the expression of a target gene by administering these dsRNA molecules, e.g., for the treatment of various disease conditions.
    Type: Grant
    Filed: November 22, 2017
    Date of Patent: November 22, 2022
    Assignee: Alnylam Pharmaceuticals, Inc.
    Inventors: Mark K. Schlegel, Maja Janas, Vasant R. Jadhav, Donald Foster, Muthiah Manoharan, Kallanthottathil G. Rajeev, Muthusamy Jayaraman, Alexander V. Kel'in, Shigeo Matsuda, Klaus Charisse, Jayaprakash K. Nair, Martin A. Maier, Alfica Sehgal, Christopher Brown, Christopher Theile, Stuart Milstein
  • ASSAYS AND METHODS FOR DETERMINING ACTIVITY OF A THERAPEUTIC AGENT IN A SUBJECT
    Publication number: 20220307083
    Abstract: The invention relates to methods and assays for determining the activity of a composition comprising a therapeutic gene administered to a subject.
    Type: Application
    Filed: February 14, 2022
    Publication date: September 29, 2022
    Inventors: Dinah Sah, David Bumcrot, Alfica Sehgal
  • COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF THE LECT2 GENE
    Publication number: 20220213473
    Abstract: The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the LECT2 gene, and methods of using such dsRNA compositions to alter (e.g., inhibit) expression of LECT2.
    Type: Application
    Filed: August 2, 2021
    Publication date: July 7, 2022
    Inventors: Brian Bettencourt, Kevin Fitzgerald, Gregory Hinkle, Alfica Sehgal
  • METHODS AND COMPOSITIONS FOR TREATING UREA CYCLE DISORDERS
    Publication number: 20220168316
    Abstract: Provided are methods and compositions for the treating a patient with a urea cycle disorder. Methods and compositions are also provided for modulating genes encoding enzymes that participate in the urea cycle by altering gene signaling networks.
    Type: Application
    Filed: January 27, 2022
    Publication date: June 2, 2022
    Inventors: Alfica Sehgal, Alla A. Sigova, Igor Zlobine, Brian E. Schwartz, David A. Bumcrot, Vaishnavi Rajagopal, Yun Joon Jung, Yuichi Nishi
  • SERPINA1 SIRNAS: COMPOSITIONS OF MATTER AND METHODS OF TREATMENT
    Publication number: 20220155716
    Abstract: The technology described herein relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the Serpina1 gene, and methods of using such dsRNA compositions to inhibit expression of Serpina1.
    Type: Application
    Filed: October 5, 2021
    Publication date: May 19, 2022
    Applicant: ALNYLAM PHARMACEUTICALS, INC.
    Inventors: Alfica SEHGAL, David BUMCROT, Brian BETTENCOURT
  • METHODS OF TREATING LIVER DISEASES
    Publication number: 20220107328
    Abstract: Provided herein are methods and compositions for the treating a patient with one or more conditions associated with PNPLA3, such as nonalcoholic fatty liver disease (NAFLD), nonalcoholic steatohepatitis (NASH), and/or alcoholic liver disease (ALD). Methods and compositions are also provided for modulating the expression of the PNPLA3 gene in a cell by altering gene signaling networks. Companion diagnostic methods, compositions and kits are also provided.
    Type: Application
    Filed: August 14, 2019
    Publication date: April 7, 2022
    Inventors: David A. Bumcrot, Alfica Sehgal, Alla A. Sigova, Brian Elliott Schwartz, Gavin Whissell, Iris Grossman, Vaishnavi Rajagopal, Cynthia Marie Smith, Mario Esteban Contreras Gamboa
  • Methods and compositions for treating urea cycle disorders
    Patent number: 11266655
    Abstract: Provided are methods and compositions for the treating a patient with a urea cycle disorder. Methods and compositions are also provided for modulating genes encoding enzymes that participate in the urea cycle by altering gene signaling networks.
    Type: Grant
    Filed: December 14, 2020
    Date of Patent: March 8, 2022
    Assignee: CAMP4 THERAPEUTICS CORPORATION
    Inventors: Alfica Sehgal, Alla A. Sigova, Igor Zlobine, Brian E. Schwartz, David A. Bumcrot, Vaishnavi Rajagopal, Yun Joon Jung, Yuichi Nishi
  • SERPINA1 iRNA COMPOSITIONS AND METHODS OF USE THEREOF
    Publication number: 20210317452
    Abstract: The invention relates to RNAi agents, e.g., double-stranded RNAi agents, targeting the Serpina1 gene, and methods of using such RNAi agents to inhibit expression of Serpina1 and methods of treating subjects having a Serpina1 associated disease, such as a liver disorder.
    Type: Application
    Filed: January 5, 2021
    Publication date: October 14, 2021
    Inventors: Alfica Sehgal, Klaus Charisse, Brian Bettencourt, Martin A. Maier, Kallanthottathil G. Rajeev, Gregory Hinkle, Muthiah Manoharan