公开(公告)号：US20220243201A1

公开(公告)日：2022-08-04

申请号：US17576562

申请日：2022-01-14

Applicant: BIOVERATIV THERAPEUTICS INC.

Inventor： Ajay Maghodia ,  Philip Zakas

IPC: C12N15/113 ,  A61K31/713 ,  A61K31/7105 ,  A61P7/04 ,  C07K14/755 ,  C12N15/86 ,  C12N7/00

Abstract: The present disclosure provides nucleic acid molecules comprising a first inverted terminal repeat (ITR), a second ITR, and a genetic cassette encoding a target sequence. In some embodiments, the first ITR and/or the second ITR is an ITR of a non-adeno-associated virus (AAV). Also disclosed are methods of using the nucleic acid molecules in gene therapy applications.

公开(公告)号：US20230084036A1

公开(公告)日：2023-03-16

申请号：US17821359

申请日：2022-08-22

Applicant: BIOVERATIV THERAPEUTICS INC.

Inventor： Ajay Maghodia ,  Tongyao Liu ,  Philip Zakas

IPC: C07K14/755 ,  C12N15/86 ,  A61K48/00

Abstract: The present disclosure provides codon optimized Factor VIII sequences, vectors, and host cells comprising codon optimized Factor VIII sequences, polypeptides encoded by codon optimized Factor VIII sequences, and methods of producing such polypeptides.

公开(公告)号：US20220356490A1

公开(公告)日：2022-11-10

申请号：US17576561

申请日：2022-01-14

Applicant: BIOVERATIV THERAPEUTICS INC.

Inventor： Tongyao Liu ,  Ajay Maghodia ,  Philip Zakas

IPC: C12N15/866 ,  C12N15/82

Abstract: Provided herein is a baculovirus expression vector system for the production of a desired protein. In one aspect, the desired protein is a closed-ended DNA (ceDNA) molecule comprising wild-type and/or truncated inverted terminal repeats derived from a genome of a member of the viral family Parvoviridae.

公开(公告)号：US20240350671A1

公开(公告)日：2024-10-24

申请号：US18440712

申请日：2024-02-13

Applicant: BIOVERATIV THERAPEUTICS INC.

Inventor： Ajay Maghodia ,  Philip Zakas

IPC: A61K48/00 ,  C07K14/755 ,  C12N15/86

CPC classification number: A61K48/0058 ,  C07K14/755 ,  C12N15/86 ,  C12N2710/14043 ,  C12N2830/008 ,  C12N2830/42

Abstract: The present disclosure provides nucleic acid molecules comprising a first inverted terminal repeat (ITR), a second ITR, and a genetic cassette encoding a target sequence. In some embodiments, the first ITR and/or the second ITR is an ITR of a non-adeno-associated virus (AAV). Also disclosed are methods of using the nucleic acid molecules in gene therapy applications.

公开(公告)号：US20240294940A1

公开(公告)日：2024-09-05

申请号：US18440711

申请日：2024-02-13

Applicant: BIOVERATIV THERAPEUTICS INC.

Inventor： Tongyao Liu ,  Ajay Maghodia ,  Philip Zakas

IPC: C12N15/86 ,  C07K14/005

CPC classification number: C12N15/86 ,  C07K14/005 ,  C12N2710/14043 ,  C12N2710/14044 ,  C12N2710/14052 ,  C12N2750/14122 ,  C12N2750/14143 ,  C12N2750/14222 ,  C12N2750/14243 ,  C12N2750/14322 ,  C12N2750/14343 ,  C12N2800/30

Abstract: Provided herein is a baculovirus expression vector system for the production of a desired protein. In one aspect, the desired protein is a closed-ended DNA (ceDNA) molecule comprising wild-type and/or truncated inverted terminal repeats derived from a genome of a member of the viral family Parvoviridae.