Shape Therapeutics Secures MJFF Grant to Advance SHP-201, a One-Time RNA-Editing Gene Therapy for Parkinson's Disease
核心洞察
Shape Therapeutics (搜索) received a research grant from The Michael J. Fox Foundation to fund IND-enabling studies for SHP-201, its lead gene therapy candidate for Parkinson's disease (搜索).
SHP-201 combines a blood-brain barrier-crossing AAV capsid with an RNA-editing payload that targets the SNCA (搜索) transcript to reduce alpha-synuclein (搜索) production via a single intravenous infusion.
The grant will support pharmacology, pharmacokinetic, biodistribution, and toxicology studies across a range of doses to support clinical dose selection ahead of a planned IND submission.
Shape Therapeutics (搜索), a Seattle-based leader in RNA editing-based gene therapy, announced on July 29, 2026 that it has been awarded a research grant from The Michael J. Fox Foundation for Parkinson's Research (MJFF) to advance IND-enabling studies of SHP-201, the company's lead gene therapy candidate for Parkinson's disease (搜索) (PD). The award will fund critical preclinical work characterizing the pharmacology, pharmacokinetics, and safety of SHP-201, paving the way for a future Investigational New Drug (IND) submission and subsequent clinical trials.
The grant was awarded through MJFF's Parkinson's Disease (搜索) Therapeutics Pipeline Program, which supports preclinical and translational research aimed at evaluating promising therapeutic approaches for Parkinson's disease.
The Unmet Need in Parkinson's Disease (搜索)
Parkinson's disease (搜索) is a progressive neurological disorder for which no disease-modifying therapy has received regulatory approval, despite decades of research. The pathological accumulation of the alpha-synuclein (搜索) protein (aSyn), encoded by the SNCA (搜索) gene, is widely understood to be a key driver of the neurodegeneration underlying both the motor and non-motor symptoms of PD. While reducing aSyn in the brain represents a promising path to a disease-modifying treatment, existing approaches have been limited by the difficulty of delivering therapy durably and precisely to the deep subcortical regions most affected in PD, such as the substantia nigra.
SHP-201: A Novel RNA-Editing Approach
SHP-201 is designed to overcome these longstanding delivery challenges. The therapeutic approach pairs Shape's engineered, blood-brain barrier (BBB)-crossing AAV capsid with an RNA-editing payload based on the company's RNAfix® technology. This payload targets and edits the SNCA (搜索) transcript to reduce production of aSyn. Delivered as a single intravenous infusion, SHP-201 is intended to provide durable, brain-wide aSyn reduction, including in the substantia nigra, without the need for repeat invasive administration.
In preclinical models, the SHP-201 payload has demonstrated robust, highly specific knockdown of aSyn. Furthermore, in a pilot non-clinical study, intravenous delivery achieved on-target activity throughout the brain, with the highest activity observed in the deep subcortical regions critical to Parkinson's disease (搜索) pathology.
IND-Enabling Studies and Next Steps
With MJFF's support, Shape Therapeutics (搜索) will conduct IND-enabling pharmacology, pharmacokinetic, biodistribution, and toxicology studies of SHP-201 across a range of doses. These studies are designed to support clinical dose selection ahead of a planned IND submission.
"We are delighted to receive this support from MJFF, which will significantly help us advance SHP-201 and contribute to the development of new therapeutic strategies for Parkinson's disease (搜索)," said Adrian Briggs, Chief Technology Officer of Shape Therapeutics (搜索).
Expert Perspective on Alpha-Synuclein (搜索) Targeting
"Alpha-synuclein (搜索) remains one of the most important therapeutic targets in Parkinson's disease (搜索), but significant challenges remain in delivering potential therapies broadly and effectively throughout the brain," said Shalini Padmanabhan, Ph.D., Senior Vice President and Head of Translational Research at The Michael J. Fox Foundation for Parkinson's Research. "The studies supported through this award will generate important translational data that may help evaluate this approach and inform the broader field of alpha-synuclein-targeted therapeutic development."
Shape Therapeutics (搜索) leverages AI to develop new payload, delivery, and manufacturing technologies for the gene therapy industry. Beyond its RNA-targeting gene therapy portfolio, the company's platform includes AAV capsids with enhanced tropism and penetration profiles, enabling delivery of genetic medicines to previously inaccessible tissues.