PUBLISHER: DelveInsight | PRODUCT CODE: 1226585
PUBLISHER: DelveInsight | PRODUCT CODE: 1226585
"AMT-130 Emerging Drug Insight and Market Forecast - 2032" report provides comprehensive insights about AMT-130 for Huntington's disease in the 7MM. A detailed picture of the AMT-130 for Huntington's disease in the 7MM, i.e., the United States, EU4 (Germany, France, Italy, and Spain) and the UK, and Japan, for the study period 2019-2032 is provided in this report along with a detailed description of the AMT-130 for Huntington's disease. The report provides insight about mechanism of action, dosage and administration, as well as research and development activity including regulatory milestones, along with other developmental activities. Further, it also consists of future market assessments inclusive of the AMT-130 market forecast, analysis for Huntington's disease in the 7MM, descriptive analysis such as SWOT, analyst views, comprehensive overview of market competitors, and brief about other emerging therapies in Huntington's disease.
AMT-130 is a gene therapy for Huntington's disease; rare, fatal, neurodegenerative genetic disorder that affects motor function and leads to behavioral symptoms, and cognitive decline in young adults, resulting in total physical and mental deterioration. AMT-130 consists of an AAV5-vector carrying an artificial microRNA specifically tailored to silence the huntingtin gene, leveraging the proprietary miQURE silencing technology. The therapeutic goal is to inhibit the production of the mutant protein (mHTT). Using AAV vectors to deliver microRNAs (miRNAs) directly to the brain for non-selective knockdown of the huntingtin gene represents a highly innovative and promising approach to treating Huntington's disease.
AMT-130, a gene therapy candidate for Huntington's disease, is differentiated in that:
The drug has received orphan drug designation and fast track designation from the US FDA for the treatment of Huntington's disease and is currently in Phase I/II clinical development (NCT05243017, NCT04120493).The studies were designed to investigate the safety, tolerability, and efficacy signals of multiple ascending doses of striatally administered AMT-130 total Huntingtin gene (HTT) lowering therapy in patients with early manifest Huntington's disease; a randomized, double-blind, sham control study was carried out to investigate the safety, tolerability, and efficacy signals of multiple ascending doses of striatally administered AMT-130 total Huntingtin gene (Both studies are recruiting trial subjects, and their primary completion dates are March 2027 and April 2029, respectively.
The report is built using data and information sourced primarily from internal databases, primary and secondary research and in-house analysis by DelveInsight's team of industry experts. Information and data from the secondary sources have been obtained from various printable and nonprintable sources like search engines, news websites, global regulatory authorities websites, trade journals, white papers, magazines, books, trade associations, industry associations, industry portals and access to available databases.
This report provides a detailed market assessment of AMT-130 in Huntington's disease in the 7MM, i.e., the United States, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan. This segment of the report provides forecasted sales data from 2024 to 2032.
The report provides the clinical trials information of AMT-130 in Huntington's disease covering trial interventions, trial conditions, trial status, start and completion dates.