Objective:
The Targets to Therapies Initiative aims to expand the number of promising, druggable biological targets within the Parkinson’s disease (PD) translational pipeline. Leveraging The Michael J. Fox Foundation’s (MJFF) many years of experience supporting target validation and advancement, the initiative takes a fresh approach combining community-sourced target nomination with expert panel-guided prioritization and validation roadmaps to deliver targets that are ripe for Parkinson’s therapeutic development.
Program Details: Genetic and biological studies have linked many potential cellular pathways and targets to PD pathogenesis. Some of these targets have advanced far into the PD drug development pipeline, including targets such as alpha-synuclein and LRRK2. However, many additional promising targets lack sufficient data and clarity to trigger robust therapeutic attention.
To serve this need, MJFF’s Target to Therapies Initiative will expand and mechanistically validate promising, druggable targets in the field of Parkinson’s disease by identifying a shortlist of priority targets for dedicated de-risking focus over the next three -to -four years.
Launched in early 2024 as a next-step evolution of MJFF’s long-standing target validation programs, this new initiative sourced target nominations from the PD research and drug development community to establish an initial list of over 300 promising biological targets. Using a team of industry, academic and investor experts who worked with MJFF to develop rigorous selection criteria, MJFF narrowed this list to roughly 20 targets with the highest therapeutic potential. The initiative will develop de-risking roadmaps for a subset of these prioritized targets, identifying key expertise and critical resource needs to support their advancement. These roadmaps may leverage a variety of approaches, including community funding calls, tool and resource development and/or coordinated, MJFF-led activities.
The initiative will also deliver target-relevant information through a knowledgebase platform currently in development.
If successful, this initiative will reduce the risk of biotech and pharmaceutical investment in exploring new treatment opportunities in the field of Parkinson’s disease.
To Learn More: Targets to Therapies: A New Target De-Risking Initiative at MJFF
MJFF Target Prioritization Initiative: Latest Updates and Spring 2024 Survey Outcomes
If you have questions about the program or are interested in nominating a target or contributing to the initiative, please email: targets2therapies@michaeljfox.org