MJFF Initiates Funding for Emerging Therapeutic Targets in Parkinson's Disease

NEW YORK, Dec. 8, 2025 /PRNewswire/ -- The Michael J. Fox Foundation for Parkinson's Research (MJFF) today announced the initiation of its first target validation efforts through the Targets to Therapies ("T2T") Initiative, representing a major step forward in advancing new biological targets for Parkinson's disease treatments. The Targets to Therapies ("T2T") Initiative is a multi-year program developed in partnership with leading scientists across academia, industry, venture capital funds and global PD networks to reduce the risk and uncertainty that have historically slowed progress in Parkinson's therapeutic development.

Over the past two years, MJFF and a global network of scientific, clinical, and industry advisors have evaluated more than 280 nominated targets using a structured prioritization framework stemming from efforts across the field, including rich target inputs from MJFF research program datasets and collaboration with the Aligning Science Across Parkinson's (ASAP) initiative's supported programs including the Collaborative Research Network (CRN), the Parkinson's Progression Markers Initiative (PPMI) and the Global Parkinson's Genetics Program (GP2). This process yielded 21 priority targets and their pathways, which now guide MJFF's near-term validation efforts and form the foundation of the program's next phase. 

With the selection process complete, T2T is now shifting from target identification to active validation. MJFF has launched funding for the first three multidisciplinary validation teams, including work on top-priority targets such as NOD2, OGA, and key endolysosomal mechanisms like TRPML1, TMEM175 and ATP13A2, with additional target teams expected throughout 2026. Together, these targets address fundamental processes that contribute to Parkinson's disease including the clearance of toxic proteins, the maintenance of lysosomal and mitochondrial health, and the regulation of immune and inflammatory activity in the brain. This shift into hands-on validation responds directly to one of the field's most persistent barriers: many promising targets lack the early mechanistic evidence, tools and experimental models needed to advance toward therapeutic development.

To date, MJFF has awarded $7.5 million in early validation grants through T2T, with additional funding expected to be committed as part of this multi-year investment in high-potential targets.

"Our mission is to widen the path toward new treatments for people living with Parkinson's," said Gaia Skibinski, PhD (she/her), Director of Translational Therapeutics at MJFF. "By moving from identifying promising targets to actively validating them, we're beginning to build the evidence needed to expand the number of well-characterized, druggable opportunities in the PD pipeline. With the launch of target-directed funding, the release of target profiles, and the future sharing of funded project data through of our T2T Target Explorer platform, the field now has a transparent, shared view of where the most compelling therapeutic possibilities lie and where we can make the greatest impact together."

As part of this next phase, MJFF is also releasing T2T's first public set of target profiles, approximately 59 high-potential targets, that provide key evidence gaps and proposed validation approaches. These profiles will be available through the T2T Target Explorer platform, a digital open-science resource that consolidates curated evidence, expert assessments, and evolving validation plans into one accessible hub. These profiles summarize what is known and what remains to be learned about each target and represent the culmination of T2T's 2024–25 selection process. Researchers, companies and collaborators can explore the new target profiles, access the platform at https://mjff-t2t-target-explorer.streamlit.app/

The Target Explorer Platform will continue to expand as validation teams generate new evidence. In its current release, the platform includes target profiles, curated mechanistic data, and a public listing of the funded projects and grantee teams advancing the initial three priority targets. Researchers can use the platform to explore target biology, assess tool availability, and follow validation progress. This open-science approach helps the broader field make more confident decisions about where to invest time and resources.

"This kind of coordinated, field-wide effort is exactly what's needed to unlock the next generation of Parkinson's therapeutics," said Stacie Weninger, PhD, President of FBRI, Venture Partner at F-Prime and member of the T2T Steering Committee. "By bringing together experts across industry and academia and making data openly accessible, MJFF is helping de-risk early target biology so developers can pursue the most promising opportunities with confidence."

MJFF expects to complete contracting for numerous validation projects by the end of the year, reflecting strong interest from academic and industry teams eager to collaborate on high-priority targets. With additional validation teams launching in 2026, the Foundation's investment, one of the largest in its translational research portfolio, is poised to meaningfully expand the landscape of druggable biology in Parkinson's disease.

SOURCE The Michael J. Fox Foundation for Parkinson's Research