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▎WuXi AppTec's content team editor, uniQure, today announced that its investigational gene therapy AMT-130 for Huntington's disease has been dosed in the first two patients in a Phase 1b/2 clinical trial in Europe
.
Huntington's disease is a rare inherited neurodegenerative disorder that causes motor symptoms as well as abnormal behavior and cognitive decline
.
The disease is an autosomal dominant disorder in which CAG repeat expansion occurs in the exon of the HTT gene encoding huntingtin protein, resulting in the production and aggregation of abnormal proteins in the brain
.
Although the cause of Huntington's disease is clear, there are currently no approved treatments to slow the onset or progression of the disease
.
AMT-130 is uniQure's first gene therapy focused on the central nervous system (CNS).
The AAV5 vector carries a microRNA (microRNA) that specifically silences HTT gene expression.
Using the company's proprietary miQURE silencing technology, it inhibits mutant huntingtin (mHTT) ) is produced
.
The use of AAV vectors to deliver microRNAs directly to the brain for non-selective knockdown of the HTT gene represents a highly innovative approach to the treatment of Huntington's disease
.
The European Phase 1b/2 clinical trial will explore the safety, proof-of-concept and dosing of AMT-130 in patients with early-stage overt Huntington's disease, with an expected dose of 15 patients
.
Dr.
Ricardo Dolmetsch, President of R&D at uniQure, said: "We are very pleased to expand the clinical development of AMT-130
.
We expect to complete patient enrollment in this European study by the end of the year and provide safety and target-reach data for the full 10 low-dose patient cohort in the U.
S.
Phase 1/2 clinical trial in the second quarter of this year
.
"Reference: [1] uniQure Announces Dosing of First Patients in European Open-Label Clinical Trial of AMT-130 Gene Therapy in Huntington's Disease.
Retrieved February 7, 2022, from https://tools.
eurolandir.
com/tools/Pressreleases /GetPressRelease/?ID=4040264&lang=en-GB&companycode=nl-qure&v=Disclaimer: WuXi AppTec's content team focuses on introducing the progress of global biomedical health research
.
This article is for information exchange purposes only, and the opinions in this article do not represent WuXi AppTec's position.
Nor does it mean that WuXi AppTec supports or opposes the views in the article
.
This article is not a recommendation for a treatment plan
.
For guidance on treatment plans, please go to a regular hospital for treatment
.
.
Huntington's disease is a rare inherited neurodegenerative disorder that causes motor symptoms as well as abnormal behavior and cognitive decline
.
The disease is an autosomal dominant disorder in which CAG repeat expansion occurs in the exon of the HTT gene encoding huntingtin protein, resulting in the production and aggregation of abnormal proteins in the brain
.
Although the cause of Huntington's disease is clear, there are currently no approved treatments to slow the onset or progression of the disease
.
AMT-130 is uniQure's first gene therapy focused on the central nervous system (CNS).
The AAV5 vector carries a microRNA (microRNA) that specifically silences HTT gene expression.
Using the company's proprietary miQURE silencing technology, it inhibits mutant huntingtin (mHTT) ) is produced
.
The use of AAV vectors to deliver microRNAs directly to the brain for non-selective knockdown of the HTT gene represents a highly innovative approach to the treatment of Huntington's disease
.
The European Phase 1b/2 clinical trial will explore the safety, proof-of-concept and dosing of AMT-130 in patients with early-stage overt Huntington's disease, with an expected dose of 15 patients
.
Dr.
Ricardo Dolmetsch, President of R&D at uniQure, said: "We are very pleased to expand the clinical development of AMT-130
.
We expect to complete patient enrollment in this European study by the end of the year and provide safety and target-reach data for the full 10 low-dose patient cohort in the U.
S.
Phase 1/2 clinical trial in the second quarter of this year
.
"Reference: [1] uniQure Announces Dosing of First Patients in European Open-Label Clinical Trial of AMT-130 Gene Therapy in Huntington's Disease.
Retrieved February 7, 2022, from https://tools.
eurolandir.
com/tools/Pressreleases /GetPressRelease/?ID=4040264&lang=en-GB&companycode=nl-qure&v=Disclaimer: WuXi AppTec's content team focuses on introducing the progress of global biomedical health research
.
This article is for information exchange purposes only, and the opinions in this article do not represent WuXi AppTec's position.
Nor does it mean that WuXi AppTec supports or opposes the views in the article
.
This article is not a recommendation for a treatment plan
.
For guidance on treatment plans, please go to a regular hospital for treatment
.
