Dataset Information

Design and Rationale of the Global Phase 3 NEURO-TTRansform Study of Antisense Oligonucleotide AKCEA-TTR-L_Rx (ION-682884-CS3) in Hereditary Transthyretin-Mediated Amyloid Polyneuropathy.

ABSTRACT:

Introduction

AKCEA-TTR-L_Rx is a ligand-conjugated antisense (LICA) drug in development for the treatment of hereditary transthyretin amyloidosis (hATTR), a fatal disease caused by mutations in the transthyretin (TTR) gene. AKCEA-TTR-L_Rx shares the same nucleotide sequence as inotersen, an antisense medicine approved for use in hATTR polyneuropathy (hATTR-PN). Unlike inotersen, AKCEA-TTR-L_Rx is conjugated to a triantennary N-acetylgalactosamine moiety that supports receptor-mediated uptake by hepatocytes, the primary source of circulating TTR. This advanced design increases drug potency to allow for lower and less frequent dosing. The NEURO-TTRansform study will investigate whether AKCEA-TTR-L_Rx is safe and efficacious, with the aim of imp

SUBMITTER: Coelho T

PROVIDER: S-EPMC8140170 | biostudies-literature | 2021 Jun

REPOSITORIES: biostudies-literature

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Dataset Information

Design and Rationale of the Global Phase 3 NEURO-TTRansform Study of Antisense Oligonucleotide AKCEA-TTR-L_Rx (ION-682884-CS3) in Hereditary Transthyretin-Mediated Amyloid Polyneuropathy.

Introduction

OmicsDI is part of the ELIXIR infrastructure

Tweets

Dataset Information

Design and Rationale of the Global Phase 3 NEURO-TTRansform Study of Antisense Oligonucleotide AKCEA-TTR-LRx (ION-682884-CS3) in Hereditary Transthyretin-Mediated Amyloid Polyneuropathy.

Introduction

OmicsDI is part of the ELIXIR infrastructure

Tweets

Design and Rationale of the Global Phase 3 NEURO-TTRansform Study of Antisense Oligonucleotide AKCEA-TTR-L_Rx (ION-682884-CS3) in Hereditary Transthyretin-Mediated Amyloid Polyneuropathy.