Patents by Inventor Sergio Pablo Sardi
Sergio Pablo Sardi 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).
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Patent number: 11781137Abstract: 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. In some aspects, the invention provides RNAi for treating Huntington's disease. Further provided herein are expression cassettes, vectors (e.g., rAAV, recombinant adenoviral, recombinant lentiviral, and recombinant HSV vectors), cells, viral particles, and pharmaceutical compositions containing the RNAi. Yet further provided herein are methods and kits related to the use of the RNAi, for example, to treat Huntington's disease.Type: GrantFiled: July 28, 2020Date of Patent: October 10, 2023Assignee: Genzyme CorporationInventors: Lisa M. Stanek, Adam Palermo, Brenda Richards, Sergio Pablo Sardi, Catherine O′Riordan, Antonius Song
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Publication number: 20210047641Abstract: 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. In some aspects, the invention provides RNAi for treating Huntington's disease. Further provided herein are expression cassettes, vectors (e.g., rAAV, recombinant adenoviral, recombinant lentiviral, and recombinant HSV vectors), cells, viral particles, and pharmaceutical compositions containing the RNAi. Yet further provided herein are methods and kits related to the use of the RNAi, for example, to treat Huntington's disease.Type: ApplicationFiled: July 28, 2020Publication date: February 18, 2021Inventors: Lisa M. STANEK, Adam PALERMO, Brenda RICHARDS, Sergio Pablo SARDI, Catherine O'RIORDAN, Antonius SONG
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Patent number: 10760079Abstract: 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. In some aspects, the invention provides RNAi for treating Huntington's disease. Further provided herein are expression cassettes, vectors (e.g., rAAV, recombinant adenoviral, recombinant lentiviral, and recombinant HSV vectors), cells, viral particles, and pharmaceutical compositions containing the RNAi. Yet further provided herein are methods and kits related to the use of the RNAi, for example, to treat Huntington's disease.Type: GrantFiled: September 10, 2019Date of Patent: September 1, 2020Assignee: Genzyme CorporationInventors: Lisa M. Stanek, Adam Palermo, Brenda Richards, Sergio Pablo Sardi, Catherine O'Riordan, Antonius Song
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Publication number: 20200197374Abstract: This disclosure relates to a method of treating a proteinopathy in a subject, the method comprising administering to the subject an effective amount of a quinuclidine compound. The disclosure also relates to a method of reducing, reversing or preventing the accumulation of protein aggregates in tissue of a subject diagnosed as having a proteinopathy, or being at risk of developing a proteinopathy, the method comprising administering to the subject an effective amount of a quinuclidine compound. Also disclosed is a pharmaceutical composition comprising a quinuclidine compound for use in said methods. The proteinopathy may be a synucleinopathy or a tauopathy, such as Parkinson's disease, Alzheimer's disease or dementia with Lewy bodies.Type: ApplicationFiled: March 2, 2020Publication date: June 25, 2020Inventors: Seng H. CHENG, Lamya SHIHABUDDIN, Sergio Pablo SARDI
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Publication number: 20200124624Abstract: Provided herein are methods of determining the efficacy of a treatment for a proteopathy, diagnosing a proteopathy in a subject, determining a subject's risk of developing a proteopathy, determining the stage of a proteopathy in a subject, monitoring a proteopathy in a subject, selecting a treatment for a proteopathy for a subject, and selecting a subject for a clinical trial that include determining a level of at least one sphingolipid in a sample including a biological fluid from the subject.Type: ApplicationFiled: March 24, 2017Publication date: April 23, 2020Inventors: Sergio Pablo Sardi, Clemens Scherzer
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Publication number: 20200109401Abstract: 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. In some aspects, the invention provides RNAi for treating Huntington's disease. Further provided herein are expression cassettes, vectors (e.g., rAAV, recombinant adenoviral, recombinant lentiviral, and recombinant HSV vectors), cells, viral particles, and pharmaceutical compositions containing the RNAi. Yet further provided herein are methods and kits related to the use of the RNAi, for example, to treat Huntington's disease.Type: ApplicationFiled: September 10, 2019Publication date: April 9, 2020Inventors: Lisa M. STANEK, Adam PALERMO, Brenda RICHARDS, Sergio Pablo SARDI, Catherine O'RIORDAN, Antonius SONG
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Patent number: 10450563Abstract: 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. In some aspects, the invention provides RNAi for treating Huntington's disease. Further provided herein are expression cassettes, vectors (e.g., rAAV, recombinant adenoviral, recombinant lentiviral, and recombinant HSV vectors), cells, viral particles, and pharmaceutical compositions containing the RNAi. Yet further provided herein are methods and kits related to the use of the RNAi, for example, to treat Huntington's disease.Type: GrantFiled: February 9, 2016Date of Patent: October 22, 2019Assignee: Genzyme CorporationInventors: Lisa M. Stanek, Adam Palermo, Brenda Richards, Sergio Pablo Sardi, Catherine O'Riordan, Antonius Song
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Publication number: 20180036295Abstract: This disclosure relates to a method of treating a proteinopathy in a subject, the method comprising administering to the subject an effective amount of a quinuclidine compound. The disclosure also relates to a method of reducing, reversing or preventing the accumulation of protein aggregates in tissue of a subject diagnosed as having a proteinopathy, or being at risk of developing a proteinopathy, the method comprising administering to the subject an effective amount of a quinuclidine compound. Also disclosed is a pharmaceutical composition comprising a quinuclidine compound for use in said methods. The proteinopathy may be a synucleinopathy or a tauopathy, such as Parkinson's disease, Alzheimer's disease or dementia with Lewy bodies.Type: ApplicationFiled: March 9, 2016Publication date: February 8, 2018Inventors: Seng H. CHENG, Lamya SHIHABUDDIN, Sergio Pablo SARDI
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Publication number: 20180023082Abstract: 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. In some aspects, the invention provides RNAi for treating Huntington's disease. Further provided herein are expression cassettes, vectors (e.g., rAAV, recombinant adenoviral, recombinant lentiviral, and recombinant HSV vectors), cells, viral particles, and pharmaceutical compositions containing the RNAi. Yet further provided herein are methods and kits related to the use of the RNAi, for example, to treat Huntington's disease.Type: ApplicationFiled: February 9, 2016Publication date: January 25, 2018Inventors: Lisa M. STANEK, Adam PALERMO, Brenda RICHARDS, Sergio Pablo SARDI, Catherine O'RIORDAN, Antonius SONG
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Publication number: 20150284472Abstract: This disclosure relates to methods for improving neural function in a mammal with a proteinopathy comprising administering a therapeutically effective amount of an agent that increases glucocerebrosidase activity in the mammal. Also disclosed are methods for reducing toxic lipids, reducing ?-synuclein, and/or inhibiting the accumulation of protein aggregates in a mammal with a proteinopathy comprising administering a therapeutically effective amount of an agent that increases glucocerebrosidase activity.Type: ApplicationFiled: November 4, 2013Publication date: October 8, 2015Inventors: Sergio Pablo Sardi, Lamya Shihabuddin, Seng Cheng