Shape Therapeutics to Present Novel AAV5 Capsid and Alpha-Synuclein Targeting Therapy for Parkinson's Disease at ESGCT Congress
核心洞察
Shape Therapeutics (搜索) will present data on their engineered AAV5 (搜索) capsid SHP-DB1 (搜索) that efficiently targets the brain after intravenous injection and transduces over 95% of neurons in the substantia nigra.
The company will also present findings on targeted alpha-synuclein (搜索) knockdown supporting the development of SHP-201, an RNA-targeting disease-modifying gene therapy for Parkinson's disease (搜索).
Both presentations will occur at the European Society of Gene and Cell Therapy 32nd Annual Congress in Seville, Spain from October 7-10, 2025.
Shape Therapeutics (搜索) announced the acceptance of two oral presentations at the European Society of Gene and Cell Therapy (ESGCT) 32nd congress, showcasing breakthrough developments in their RNA-based gene therapy platform for Parkinson's disease (搜索) treatment. The presentations will take place October 7-10, 2025 in Seville, Spain.
Novel BBB-Penetrant AAV5 Capsid Shows Remarkable Brain Targeting
The first presentation, delivered by Dr. Adrian Briggs on October 7, will detail the company's engineered AAV5 (搜索) capsid SHP-DB1 (搜索), which demonstrates exceptional brain targeting capabilities. According to the presentation title, the capsid "efficiently targets the NHP brain after intravenous injection and transduces >95% of neurons in the Parkinson's disease (搜索)-critical substantia nigra."
This achievement represents a significant advancement in gene therapy delivery, as the substantia nigra is the brain region most affected by Parkinson's disease (搜索). The ability to achieve such high transduction rates in this critical area through intravenous administration could potentially transform treatment approaches for the neurodegenerative condition.
Alpha-Synuclein Targeting Therapy Development
The second presentation, scheduled for October 10 and presented by Dr. Leah Helton, will focus on "Targeted Knockdown of Alpha Synuclein in the Brain Supports the Therapeutic Development of SHP-201 for Parkinson's Disease (搜索)." This presentation will be part of the Gene Editing III: Technology & applications session.
SHP-201 represents Shape Therapeutics (搜索)' RNA-targeting disease-modifying gene therapy approach for Parkinson's disease (搜索). Alpha-synuclein (搜索) is a key protein implicated in Parkinson's disease pathology, and targeted knockdown of this protein could offer a disease-modifying therapeutic strategy.
AI-Driven Gene Therapy Platform
Shape Therapeutics (搜索) positions itself as a leader in RNA-based gene therapy, leveraging artificial intelligence to develop new payload, delivery and manufacturing technologies for the gene therapy industry. The company's platform includes AAV capsids with enhanced tropism and penetration profiles, enabling delivery of genetic medicines to previously inaccessible tissues.
The Seattle-based company's approach combines their proprietary RNA-targeting gene therapy portfolio with advanced delivery systems designed to overcome traditional barriers in gene therapy, particularly the challenge of delivering therapeutic agents across the blood-brain barrier to treat neurological conditions.