公开(公告)号：US20210047641A1

公开(公告)日：2021-02-18

申请号：US16941303

申请日：2020-07-28

Applicant: Genzyme Corporation

Inventor： Lisa M. STANEK ,  Adam PALERMO ,  Brenda RICHARDS ,  Sergio Pablo SARDI ,  Catherine O'RIORDAN ,  Antonius SONG

IPC: C12N15/113

Abstract: 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.

公开(公告)号：US20240182896A1

公开(公告)日：2024-06-06

申请号：US18483474

申请日：2023-10-09

Applicant: Genzyme Corporation

Inventor： Lisa M. STANEK ,  Adam PALERMO ,  Brenda RICHARDS ,  Sergio Pablo SARDI ,  Catherine O'RIORDAN ,  Antonius SONG

IPC: C12N15/113

CPC classification number: C12N15/113 ,  C12N2310/14 ,  C12N2310/531 ,  C12N2310/533

Abstract: 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.

公开(公告)号：US20180023082A1

公开(公告)日：2018-01-25

申请号：US15549895

申请日：2016-02-09

Applicant: Genzyme Corporation

Inventor： Lisa M. STANEK ,  Adam PALERMO ,  Brenda RICHARDS ,  Sergio Pablo SARDI ,  Catherine O'RIORDAN ,  Antonius SONG

IPC: C12N15/113

Abstract: 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.

公开(公告)号：US20200109401A1

公开(公告)日：2020-04-09

申请号：US16566565

申请日：2019-09-10

Applicant: Genzyme Corporation

Inventor： Lisa M. STANEK ,  Adam PALERMO ,  Brenda RICHARDS ,  Sergio Pablo SARDI ,  Catherine O'RIORDAN ,  Antonius SONG

IPC: C12N15/113

Abstract: 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.

公开(公告)号：US20180036295A1

公开(公告)日：2018-02-08

申请号：US15556444

申请日：2016-03-09

Applicant: Genzyme Corporation

Inventor： Seng H. CHENG ,  Lamya SHIHABUDDIN ,  Sergio Pablo SARDI

IPC: A61K31/439 ,  A61K9/00 ,  A61K45/06

CPC classification number: A61K31/439 ,  A61K9/0053 ,  A61K31/00 ,  A61K45/06 ,  A61K2300/00

Abstract: 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.