July 29, 2026 —
Shape Therapeutics announced that it has received a research grant from The Michael J. Fox Foundation for Parkinson’s Research to advance SHP-201, its lead gene therapy candidate for Parkinson’s disease, through IND-enabling studies.
The award was granted through MJFF’s Parkinson’s Disease Therapeutics Pipeline Program, which supports preclinical and translational research evaluating promising therapeutic approaches for Parkinson’s disease. The funding will support studies characterizing the pharmacology, pharmacokinetics, biodistribution, and safety of SHP-201 ahead of a planned Investigational New Drug submission.
SHP-201 is designed as a single-dose, intravenously delivered genetic medicine intended to reduce alpha-synuclein, or aSyn, in the brain regions most affected by Parkinson’s disease. Alpha-synuclein, encoded by the SNCA gene, is widely recognized as a key driver of Parkinson’s disease pathology.
The therapy combines Shape’s engineered blood-brain barrier-crossing AAV capsid with an RNA-editing payload based on the company’s RNAfix® technology. The payload is designed to target and edit the SNCA transcript, reducing production of alpha-synuclein without requiring repeat invasive administration.
Parkinson’s disease remains a progressive neurological disorder with no approved disease-modifying therapy. Existing therapeutic approaches have been limited by the difficulty of delivering treatments durably and precisely to deep brain regions, including the substantia nigra, which is central to Parkinson’s disease progression.
Shape said preclinical models showed that the SHP-201 payload achieved robust and highly specific alpha-synuclein knockdown. In a pilot non-clinical study, intravenous delivery achieved on-target activity throughout the brain, with the highest activity observed in deep subcortical regions relevant to Parkinson’s disease.
With MJFF support, Shape will conduct IND-enabling studies across a range of doses to support clinical dose selection. These studies will assess pharmacology, pharmacokinetics, biodistribution, and toxicology to help prepare the program for potential clinical testing.
The program highlights a growing direction in CNS gene therapy: combining BBB-crossing AAV delivery with RNA-based regulatory technologies to target disease-driving transcripts in difficult-to-reach brain regions.
If successfully advanced, SHP-201 could represent a disease-modifying approach for Parkinson’s disease by durably reducing alpha-synuclein production after a single intravenous administration.
