公开(公告)号：US20230296587A1

公开(公告)日：2023-09-21

申请号：US18172707

申请日：2023-02-22

Applicant: HOFFMANN-LA ROCHE INC.

Inventor： Veronica COSTA ,  Maj HEDTJÄRN ,  Marius HOENER ,  Ravi JAGASIA ,  Mads Aaboe JENSEN ,  Christoph PATSCH ,  Lykke PEDERSEN ,  Søren Vestergaard RASMUSSEN

IPC: G01N33/50 ,  C12N5/0797 ,  C12N15/11 ,  C12N15/113 ,  C12N5/0793 ,  C12N5/00 ,  C12N5/074

CPC classification number: G01N33/5014 ,  G01N33/5058 ,  C12N5/0623 ,  C12N15/111 ,  C12N15/113 ,  C12N5/0619 ,  C12N15/1137 ,  C12N5/0018 ,  C12N5/0696 ,  C12N15/11 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/3231 ,  C12N2310/341 ,  C12N2501/11 ,  C12N2501/119 ,  C12N2501/13 ,  C12N2501/41 ,  C12N2503/02 ,  C12N2310/20 ,  C12N2310/3341 ,  C12N2310/346 ,  C12N2320/34 ,  C12N2500/38 ,  C12N2500/44 ,  C12N2501/01 ,  C12N2501/115 ,  C12N2501/60 ,  C12N2501/727 ,  C12N2501/999 ,  C12N2506/45 ,  C12N2533/52 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/33 ,  C12N2310/351

Abstract: The present invention relates to oligonucleotides that are capable of inducing expression of ubiquitin-protein ligase E3A (UBE3A) from the paternal allele in animal or human neurons. The oligonucleotides target the suppressor of the UBE3A paternal allele by hybridization to SNHG14 long non-coding RNA downstream of SNORD1091B. The present invention further relates to pharmaceutical compositions and methods for treatment of Angelman syndrome.

公开(公告)号：US20240085402A1

公开(公告)日：2024-03-14

申请号：US18491513

申请日：2023-10-20

Applicant: Hoffmann-La Roche Inc.

Inventor： Veronica COSTA ,  Maj HEDTJÄRN ,  Marius HOENER ,  Ravi JAGASIA ,  Mads Aaboe JENSEN ,  Christoph PATSCH ,  Lykke PEDERSEN ,  Søren Vestergaard RASMUSSEN

IPC: G01N33/50 ,  C12N5/00 ,  C12N5/074 ,  C12N5/0793 ,  C12N5/0797 ,  C12N15/11 ,  C12N15/113

CPC classification number: G01N33/5014 ,  C12N5/0018 ,  C12N5/0619 ,  C12N5/0623 ,  C12N5/0696 ,  C12N15/11 ,  C12N15/111 ,  C12N15/113 ,  C12N15/1137 ,  G01N33/5058 ,  C12N2310/11 ,  C12N2310/20 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/3231 ,  C12N2310/33 ,  C12N2310/3341 ,  C12N2310/341 ,  C12N2310/346 ,  C12N2310/351 ,  C12N2320/34 ,  C12N2500/38 ,  C12N2500/44 ,  C12N2501/01 ,  C12N2501/11 ,  C12N2501/115 ,  C12N2501/119 ,  C12N2501/13 ,  C12N2501/41 ,  C12N2501/60 ,  C12N2501/727 ,  C12N2501/999 ,  C12N2503/02 ,  C12N2506/45 ,  C12N2533/52

Abstract: The present invention relates to oligonucleotides that are capable of inducing expression of ubiquitin-protein ligase E3A (UBE3A) from the paternal allele in animal or human neurons. The oligonucleotides target the suppressor of the UBE3A paternal allele by hybridization to SNHG14 long non-coding RNA downstream of SNORD109B. The present invention further relates to pharmaceutical compositions and methods for treatment of Angelman syndrome.

公开(公告)号：US20190144824A1

公开(公告)日：2019-05-16

申请号：US15977895

申请日：2018-05-11

Applicant: Hoffmann-La Roche Inc.

Inventor： Carlo CUSULIN ,  Veronica COSTA ,  Marius HOENER ,  Christoph PATSCH ,  Eva Christina THOMA

IPC: C12N5/0793 ,  C12N5/074 ,  C12N5/00 ,  C12N15/11

Abstract: This patent application relates to a method to generate standardized and robust neuronal cell culture assays from primate species, for example suitable for high-throughput screening of drug candidates. The method includes differentiating human and/or non-human primate neuronal precursor cells (NPCs) to neuronal cells (NCs) and producing uniform NC cultures based on a dissociation and reseeding step performed on the differentiated NCs resulting in robust cultures suitable for high-throughput drug screening assays, in particular to screen antisense oligonucleotides.

公开(公告)号：US20210261961A1

公开(公告)日：2021-08-26

申请号：US17245443

申请日：2021-04-30

Applicant: Hoffmann-La Roche Inc.

Inventor： Veronica COSTA ,  Heidi Rye HUDLEBUSCH ,  Ravi JAGASIA ,  Dheeraj MALHOTRA ,  Lykke PEDERSEN

IPC: C12N15/113

Abstract: The present invention relates to antisense oligonucleotides (oligomers) complementary to nucleic acids encoding mammalian T cell-restricted intracellular antigen-1 (TIA1), in particular antisense oligonucleotides targeting TIA1 pre-mRNA sequences, which are capable of inhibiting the expression of TIA1. Inhibition of TIA1expression is beneficial for a range of medical disorders including neurodegenerative diseases, such as amyotrophic lateral sclerosis (ALS) or Frontotemporal Dementia.

公开(公告)号：US20190154664A1

公开(公告)日：2019-05-23

申请号：US15977970

申请日：2018-05-11

Applicant: Hoffmann-La Roche Inc.

Inventor： Carlo CUSULIN ,  Veronica COSTA ,  Marius HOENER ,  Christoph PATSCH ,  Eva Christina THOMA

IPC: G01N33/50 ,  C12N5/0793 ,  C12N5/074

Abstract: This patent application relates to a method for determining the in vitro efficacy profile of a drug candidate using standardized cell cultures of uniformly distributed differentiated neural cells (NCs) from at least two primate species, wherein the differentiated NC cultures are qualified for high throughput screening based on a dissociation and reseeding step performed on the differentiated NCs. The method includes differentiating human and/or non-human primate neuronal precursor cells (NPCs) to neuronal cells (NCs) followed by dissociating the differentiated NCs from its support and reseeding the differentiated NCs in a high-throughput cell culture format resulting in robust cultures suitable for high-throughput drug screening assays, in particular to screen antisense oligonucleotides.