Delving into the Latest Updates on Visirna Therapeutics HK Limited with Synapse

Overview

Basic Info

Introduction

Visirna Therapeutics HK Limited is an emerging small nucleic acid therapeutics firm with a foundation in China and a vision that extends internationally. Its mission is to develop into a fully-integrated biopharmaceutical company excelling in research, manufacturing, and commercialization. Established in 2022, it has formed a sustained strategic alliance with Arrowhead Pharmaceuticals (NASDAQ: ARWR), a global frontrunner in the field of small nucleic acid medicines.

Tags

Endocrinology and Metabolic Disease

Other Diseases

Congenital Disorders

siRNA

Disease domain score

A glimpse into the focused therapeutic areas

Technology Platform

Most used technologies in drug development

Targets

Most frequently developed targets

Disease Domain	Count

Endocrinology and Metabolic Disease	2

Top 5 Drug Type	Count

siRNA	3
Chemical drugs	1

Top 5 Target	Count

ANGPTL3(Angiopoietin like 3)	1
17β-HSD13(17-beta-hydroxysteroid dehydrogenase 13)	1
APOC3(Apolipoprotein C-III)	1

Human genetic studies have shown that loss of function (LOF) mutations in HSD17β13 gene have a protective effect on the progression of alcohol-related and non-alcohol-related liver diseases, such as NASH, without significant adverse phenotypes.
VSA006 is a siRNA drug targeting HSD17β13 mRNA in the liver and reduce the protein level of HSD17β13. Based on phase 1 study results in healthy volunteers and NASH/suspected NASH patients, this phase 2 study is designed to evaluate the efficacy, safety, PK profiles and immunogenicity of VSA006 in Chinese NASH patients.

Start Date01 Apr 2024

Sponsor / Collaborator

Visirna Therapeutics HK Limited

CTR20233245 / RecruitingPhase 2

一项在中国成年非酒精性脂肪性肝炎（NASH）患者中评价VSA006注射液的疗效和安全性的多中心、随机双盲、安慰剂对照的II期临床试验

[Translation] A multicenter, randomized, double-blind, placebo-controlled Phase II clinical trial to evaluate the efficacy and safety of VSA006 injection in Chinese adult patients with non-alcoholic steatohepatitis (NASH)

评价VSA006注射液治疗NASH患者的疗效。评价VSA006注射液在NASH患者中的安全性和耐受性。评价VSA006注射液在NASH患者中的药代动力学（PK）特征。探索VSA006注射液的免疫原性特征。

[Translation]

Evaluate the efficacy of VSA006 injection in the treatment of NASH patients. Evaluate the safety and tolerability of VSA006 injection in NASH patients. Evaluate the pharmacokinetic (PK) characteristics of VSA006 injection in NASH patients. Explore the immunogenicity characteristics of VSA006 injection.

Start Date08 Mar 2024

Sponsor / Collaborator

Visirna Biotechnology (Shanghai) Co., Ltd.

CTR20231418 / Active, not recruitingPhase 3

一项在中国家族性乳糜微粒血症综合征成人患者中评价VSA001注射液有效性和安全性的III期临床研究

[Translation] A Phase III clinical study evaluating the efficacy and safety of VSA001 injection in Chinese adult patients with familial chylomicronemia syndrome

该研究的主要目的是在中国家族性乳糜微粒血症综合征（FCS）成人患者中评价VSA001的有效性和安全性。

[Translation]

The primary objective of the study is to evaluate the efficacy and safety of VSA001 in Chinese adult patients with familial chylomicronemia syndrome (FCS).

Start Date10 Jul 2023

Sponsor / Collaborator

Visirna Therapeutics HK Limited

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100 Clinical Results associated with Visirna Therapeutics HK Limited

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0 Patents (Medical) associated with Visirna Therapeutics HK Limited

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100 Deals associated with Visirna Therapeutics HK Limited

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100 Translational Medicine associated with Visirna Therapeutics HK Limited

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Pipeline

Pipeline Snapshot as of 07 Nov 2024

The statistics for drugs in the Pipeline is the current organization and its subsidiaries are counted as organizations,Early Phase 1 is incorporated into Phase 1, Phase 1/2 is incorporated into phase 2, and phase 2/3 is incorporated into phase 3

IND Application

1

2

Phase 2

Phase 3

1

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Current Projects

Drug(Targets)	Indications	Global Highest Phase

Plozasiran ( APOC3 )	Familial chylomicronaemia syndrome More	Phase 3
VSA-003 ( ANGPTL3 )	Homozygous familial hypercholesterolemia More	Phase 2
VSA-006 ( 17β-HSD13 )	Nonalcoholic Steatohepatitis More	Phase 2