Investor & Industry Resources to De-Risk Parkinson’s Research

This program seeks to advance therapeutic development through pre-clinical and/or clinical testing of approaches addressing unmet needs of people with Parkinson’s disease (PD). The program is set up to benefit therapeutics with clear potential to prevent, stop, or delay disease progression or to reduce the burden of daily symptoms.

Proposals are accepted year-round on a rolling basis. 

The 2025 Parkinson's Biomarker report provides an overview of the Parkinson’s Disease (PD) biomarkers landscape, with a focus on biochemical and molecular based assays that MJFF staff is monitoring. It is intended to reflect biomarkers at various stages of development and validation for the indicated intended uses (e.g., diagnosis, monitoring, etc.)

This report tracks important therapies currently in clinical development for PD. It includes brief rationale and progress in key pipelines of therapeutic interest along with the development status of specific therapies. Where relevant, the report flags programs that have received financial or other significant enabling support from MJFF.

The Targets to Therapies Initiative aims to expand the number of promising, druggable biological targets within the Parkinson’s disease (PD) translational pipeline. We are currently collecting information on potential partners, and we look forward to leveraging the full expertise of our scientific community. Complete a short survey to express your interest in working with us on a specific target or targets. 

The LRRK2 Investigative Therapeutics Exchange (LITE) program aims to support new approaches to targeting LRRK2 with therapies, while also supporting the development of LRRK2-relevant clinical biomarkers to measure progress.

In 2010, The Michael J. Fox Foundation and a core group of academic scientists and industry partners launched the Parkinson’s Progression Markers Initiative (PPMI) toward critically needed biological markers of Parkinson’s onset and progression. PPMI has since engaged thousands of partners — more sites; scientists and clinicians; industry experts; philanthropic partners; and, most importantly, study volunteers — to build a cornerstone of Parkinson’s research. Analysis from its open-access data set and available biosample library has deepened understanding of disease and informed the design of dozens of therapeutic trials.

In light of the breakthrough biomarker test that was validated in 2023, the alpha-synuclein seeding amplification assay (aSyn SAA), MJFF is investing heavily in validating quantitative biomarkers for PD. 

Parkinson's disease and dementia with Lewy bodies are currently defined by their clinical features, with α-synuclein pathology as the gold standard to establish the definitive diagnosis. We propose that, given biomarker advances enabling accurate detection of pathological α-synuclein (ie, misfolded and aggregated) in CSF using the seed amplification assay, it is time to redefine Parkinson's disease and dementia with Lewy bodies as neuronal α-synuclein disease rather than as clinical syndromes. This major shift from a clinical to a biological definition of Parkinson's disease and dementia with Lewy bodies takes advantage of the availability of tools to assess the gold standard for diagnosis of neuronal α-synuclein (n-αsyn) in human beings during life. 

The FDA issued a “Letter of Support” in late Summer 2024, following multi-stakeholder collaboration amongst The Michael J. Fox Foundation for Parkinson’s Research (MJFF) and the Critical Path Institute (C-Path), with both organizations supporting use of the biomarker for clinical trials and advancing new drugs for Parkinson’s disease (PD).  

In the letter, the FDA emphasizes that their support stems from significant evidence collected as part of MJFF’s flagship Parkinson’s Progression Markers Initiative (PPMI), which first validated the αSyn-SAA biomarker in April 2023.  This is only the second Letter of Support issued for PD markers. 

The Path to Prevention (P2P) platform trial is designed to catalyze clinical development for prodromal PD. P2P capitalizes on PPMI study infrastructure, expertise, and ongoing clinical and biomarker data collection to de-risk industry investments in pioneering therapies for individuals at risk of PD diagnosis.

Join hundreds of scientists for the 3rd annual GBA1 Meeting, in May 2026, a golden opportunity to collaborate and advance GBA1 research and treatment.

Hosted by McGill University and the Michael J. Fox Foundation, this event is exclusively tailored for health professionals and stakeholders from both academia and industry engaging/specializing in GBA1 research.

Topics of discussion will include all aspects of GBA1 research, from basic science to clinical trials. The meeting will serve as a unique platform to collectively advance the understanding and treatment of GBA1-associated neurodegenerative diseases. 

Registration for this meeting is now open. 

The Michael J. Fox Foundation's annual Parkinson's Disease Therapeutics Conference (PDTC) is the only scientific conference around the world focused exclusively on Parkinson's disease drug development. The 18th annual PDTC is taking place October 14-15, 2026, in New York City.

Each year, PDTC brings together 300 research and business development professionals from both academia and industry and showcases the most exciting and innovative research from MJFF's research portfolio.

Registration for this meeting will open in spring 2026. 

The Parkinson's Disease Research Exchange (PDRx) is a global network of scientists from academic labs, pharma/biotech, CROs and investors focused on assessing the current state of Parkinson's disease research, identifying key challenges, and how to translate findings into improved therapies. In Fall 2024, MJFF launched the PDRx online platform to enhance networking and facilitate open discussion.