公开(公告)号：US20210077593A1

公开(公告)日：2021-03-18

申请号：US16883407

申请日：2020-05-26

Applicant: ICAHN SCHOOL OF MEDICINE AT MOUNT SINAI ,  GENZYME CORPORATION

Inventor： Edward H. Schuchman ,  Robert J. Desnick ,  Gerald F. Cox ,  Laura P. Andrews ,  James M. Murray

IPC: A61K38/46

Abstract: The invention relates to dose escalation enzyme replacement therapy using acid sphingomyelinase (ASM) for the treatment of human subjects having acid sphingomyelinase deficiency (ASMD), and, in particular, patients with non-neurological manifestations of Niemann-Pick Disease (NPD), and in certain embodiments, NPD type B.

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：US20210008227A1

公开(公告)日：2021-01-14

申请号：US16910834

申请日：2020-06-24

Applicant: Genzyme Corporation

Inventor： James DODGE ,  Lamya S. SHIHABUDDIN ,  Marco A. PASSINI ,  Seng H. CHENG ,  Catherine O'RIORDAN

IPC: A61K48/00 ,  C12N15/864 ,  C07K14/475

Abstract: This disclosure provides methods and compositions for treating disorders or injuries that affect motor function and control in a subject. In one aspect, the invention provides a method to deliver a transgene to a subject's spinal cord by administering a recombinant neurotropic viral vector containing the transgene. The viral vector delivers the transgene to a region of the deep cerebellar nuclei region of the brain. Also provided are compositions and methods to deliver a transgene to a subject's spinal cord by administering a recombinant neurotropic viral vector containing the transgene to the motor cortex region of the subject's brain.

公开(公告)号：US10821154B2

公开(公告)日：2020-11-03

申请号：US14888385

申请日：2013-05-01

Applicant: Genzyme Corporation

Inventor： Marco A. Passini ,  Lamya S. Shihabuddin ,  Catherine R. O'Riordan ,  Seng H. Cheng

IPC: A61K38/17 ,  A61K48/00 ,  A61K9/00

Abstract: 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. In one aspect, the viral particles are administered the spinal column or cisterna magna in a human subject; for example, a pediatric human subject. Viral particles comprising AAV9 capsids are contemplated.

公开(公告)号：US20200299402A1

公开(公告)日：2020-09-24

申请号：US16842864

申请日：2020-04-08

Applicant: GENZYME CORPORATION

Inventor： Kevin P. Brower ,  Chris Hwang ,  Rao Koduri ,  Konstantin B. Konstantinov ,  Veena Warikoo ,  Jin Yin ,  Marcella Yu

IPC: C07K16/28 ,  C07K16/00 ,  C07K1/113 ,  A61K39/395 ,  C07K16/22

Abstract: Disclosed herein are methods that have been developed to control the formation of disulfide bonds between polypeptides of a multimeric protein produced by a bioprocess. Also disclosed are protein solution parameters that allow for controlling the formation of disulfide bonds. In one example, the methods disclosed herein can be used to control the proportion of half antibody molecules in an antibody solution.

公开(公告)号：US10781252B2

公开(公告)日：2020-09-22

申请号：US16197802

申请日：2018-11-21

Applicant: Genzyme Corporation ,  Optein, Inc.

Inventor： Steven R. Ledbetter ,  Celia Patricia Hart ,  Robert G. Holgate ,  Lutz U. Jermutus ,  Catriona L. Buchanan ,  Alexander R. Duncan ,  Donna K. Finch

IPC: C07K16/22 ,  A61P13/12 ,  A61K39/00 ,  A61K39/395

Abstract: The present invention relates to antibody molecules, in particular antibody molecules that bind Transforming Growth Factor beta (TGFβ), and uses thereof. More particularly, the invention relates to antibody molecules that bind and preferably neutralise TGFβ1, TGFβ2 and TGFβ3, so-called “pan-specific” antibody molecules, and uses of such antibody molecules. Preferred embodiments within the present invention are antibody molecules, whether whole antibody (e.g. IgG, such as IgG1 or IgG4) or antibody fragments (e.g. scFv, Fab, dAb).

公开(公告)号：US20200255798A1

公开(公告)日：2020-08-13

申请号：US16807057

申请日：2020-03-02

Applicant: Genzyme Corporation

Inventor： Jianguo Yang ,  Yang Yang ,  Jennifer Tengtrakool ,  Weichang Zhou

IPC: C12N5/00

Abstract: Provided herein are methods of perfusion culturing an adherent mammalian cell using a shake flask and a plurality of microcarriers, and various methods that utilize these culturing methods.

公开(公告)号：US10730936B2

公开(公告)日：2020-08-04

申请号：US15670631

申请日：2017-08-07

Applicant: GENZYME CORPORATION

Inventor： Ronnie Wei ,  Aaron Moulin ,  Magali Mathieu ,  Clark Pan ,  Sunghae Park ,  Huawei Qiu

IPC: A61K39/00 ,  A61K39/395 ,  C07K16/22

Abstract: Antibodies or antigen-binding fragments thereof are engineered to bind Transforming Growth Factor-β (TGFβ). TGFβ-isoform selective antibodies or antigen-binding fragments thereof may selectively bind human TGFβ1, compared to human TGFβ2 and human TGFβ3, or may selectively bind human TGFβ3, compared to human TGFβ1 and human TGFβ2. The design of the antibodies or antigen-binding fragments thereof is facilitated by a co-crystal structure of a recombinant Fab fragment of GC1008 bound to TGFβ2 and by another co-crystal structure of the scFv version of GC1008 bound to TGFβ1.

公开(公告)号：US20200216844A1

公开(公告)日：2020-07-09

申请号：US16220157

申请日：2018-12-14

Applicant: Genzyme Corporation

Inventor： Akin Akinc ,  Alfica Sehgal ,  Ivanka Toudjarska ,  Donald Foster ,  Stuart Milstein ,  Brian Bettencourt ,  Martin A. Maier ,  Klaus Charisse ,  Satyanarayana Kuchimanchi ,  Kallanthottathil G. Rajeev ,  Muthiah Manoharan

IPC: C12N15/113 ,  A61K31/713

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.

公开(公告)号：US20200163987A1

公开(公告)日：2020-05-28

申请号：US16630044

申请日：2018-07-10

Applicant: Genzyme Corporation

Inventor： Akin AKINC

IPC: A61K31/7088 ,  A61P7/04

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 treat a bleeding event in a subject having a hemophilia (e.g., with or without inhibitors).