SEATTLE, Washington — October 10, 2025 — Leads & Copy — Shape Therapeutics presented new preclinical data at the European Society of Gene and Cell Therapy (ESGCT) 32nd congress, highlighting the therapeutic potential of its lead product (SHP-201), a disease-modifying gene therapy for Parkinson’s Disease (PD). The company also presented data showcasing its novel BBB-penetrant, engineered AAV5 capsid (SHP-DB1) that efficiently targets the substantia nigra in the NHP brain.
Adrian Briggs, CTO and interim CEO of Shape Therapeutics, said the company is delighted to share key breakthroughs at ESGCT, including compelling animal data in its gene therapy program for Parkinson’s disease. Briggs added that by combining their protein-free RNA-editing payload and engineered AAV delivery technologies, Shape has built a unique drug paradigm: one-and-done gene editing that leaves the genome intact.
Key findings include that SHP-DB1, a next-generation AAV5-based gene therapy capsid, is engineered to address two major barriers in neurological gene therapy: poor blood-brain barrier (BBB) penetration and widespread pre-existing immunity to AAV9. Following intravenous administration in NHPs, SHP-DB1 demonstrates broad and efficient neuronal transduction across the brain, including 18–38% in cortical regions and 45-96% in deep brain structures. Most strikingly, SHP-DB1 achieves 96% neuronal transduction in the substantia nigra, a critical region for Parkinson’s Disease therapy.
Additionally, no approved disease slowing or modifying treatments exist for PD, despite decades of understanding that the pathological accumulation of the alpha-synuclein protein (aSyn) is a key driver of PD’s pathophysiology. SHP-201’s AAV-delivered payload, based on Shape’s RNAfix® technology, encodes a custom small RNA that targets and edits the SNCA transcript to prevent aSyn production.
Shape Therapeutics is leveraging AI to develop new payload, delivery and manufacturing technologies for the gene therapy industry. The company is headquartered in Seattle, Washington.
Contact: info@shapetx.com
Source: Shape Therapeutics
