Targets to Therapies: Endolysosomal Target Validation

Targets to Therapies: Endolysosomal Target Validation is sponsored by The Michael J. Fox Foundation for Parkinson's Research (MJFF). This program seeks to build a robust evidence base for therapeutic targets related to endolysosomal pathways in Parkinson's disease.

Targets to Therapies Initiative | Parkinson's Disease Search for topics, articles, videos, research, etc... Hit enter to search or ESC to close Targets to Therapies Initiative Key Initiatives and Findings Targets to Therapies Initiative The Targets to Therapies Initiative systematically identifies and investigates promising but understudied targets for Parkinson’s disease (PD) treatments.

It builds on the collected expertise of PD researchers worldwide to accelerate discoveries leading to critically needed therapies. Researchers: Access Interest & Nomination Forms for Targets The Targets to Therapies Initiative builds evidence for promising but understudied therapeutic targets.

It focuses on determining and validating whether specific proteins or genes within biological mechanisms involved in PD could serve as effective targets for PD drugs. Well-known examples of these targets that have therapies already in clinical testing for PD include alpha-synuclein, GBA1 and LRRK2.

But many more mechanisms impact the disease and could potentially function as therapeutic targets — if there was sufficient data and clarity to trigger more robust attention from drug developers. MJFF’s Targets to Therapies Initiative is designed to generate that data and clarity.

It employs a groundbreaking collaborative approach that unites fieldwide experts in gathering input on understudied targets and developing a shortlist of those with high potential. Then it funds research teams to mechanistically validate the selected targets for possible future drug development. To further drive progress, all learnings and tools are made freely available through a shared knowledgebase.

An evolution of MJFF’s longstanding focus on translating and validating biology for PD drug development, the initiative was launched in early 2024 by surveying the scientific community to gather input on which targets offer greatest potential for new PD treatments.

The Targets to Therapies Nomination Process More than 290 targets were evaluated, and 21 were nominated based on feasibility of addressing bottlenecks over the next two to three years. The Foundation then began convening expert validation teams to study these targets in-depth.

Key to success of the initiative is to leverage the full expertise of our scientific community to inform our efforts related to these targets, whether as a grantee, advisor or collaborator on a target validation team. Furthermore, while we have started with a shortlist of targets, we continue to gather input on additional targets from experts working on them. Nominate a new target for future prioritization .

Partnership Opportunities 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 Targets to Therapies Validation Engine Three main scientific cores power the Targets to Therapies Initiative: a prioritization and selection core, a validation and toolkit development core, and a public knowledgebase.

Prioritization and Selection Targets to Therapies assembled the Prioritization and Selection Core — a team of representatives from biotech, pharmaceutical companies, investment firms, and academic and nonprofit research organizations — to develop a framework for prioritizing PD targets and supporting ongoing evaluation of emerging targets. The initial round focused on selecting targets with potential to slow or modify PD progression.

Future efforts will focus on targets that could improve the range of symptoms that occur with PD and impact quality of life. Targets to Therapies aims to generate robust target data profiles and tools to increase confidence in drug development decisions. Preliminary validation plans for the top 21 prioritized targets provide clear starting points for each target’s validation.

Efforts are underway to consolidate information on preclinical tools, companies, and key opinion leaders (KOLs) for each target. The Validation Core — a team of PD biology experts, target validation scientists, and central nervous system drug development specialists — is developing comprehensive data profiles to accelerate target validation.

These efforts will address critical gaps, define experimental approaches, and evaluate the feasibility and impact of filling these gaps within a two- to three-year timeframe. In parallel, the Target Toolkit Development Core made up of experts in tool, assay and model development will create preclinical toolkits to support the needs of each target.

These targets stem from efforts across the field, including rich inputs from MJFF research program datasets and collaboration with the Aligning Science Across Parkinson’s (ASAP) initiative-supported programs including the Collaborative Research Network (CRN), the Parkinson’s Progression Markers Initiative (PPMI) and the Global Parkinson’s Genetics Program (GP2).

The Targets to Therapies Initiative launched a public knowledgebase to make findings on these targets easy to access for researchers and drug developers considering their therapeutic development. The knowledgebase incorporates high-quality, thoughtfully curated data from target prioritization and validation activities with active involvement from the initiative’s core team of therapeutic and target experts.

Funded Studies: De-risking Therapeutic Development for NOD2 Funded Studies: De-risking Therapeutic Development for OGA Scientific Advisory Committee The Governance Committee, comprised of internal and external stakeholders, provides strategic guidance and feedback, as well as insights to core groups.

The committee is designed to ensure target validation efforts align with industry needs and maximize the potential for advancing impactful therapies.

Targets to Therapies Scientific Advisory Committee Stacie Weninger Industry & Venture Capitalist Chief Mission Officer John Dunlop Industry & Venture Capitalist Coalition for Aligning Science Program Partner Brian Fiske Chief Scientific Officer Adam Knight Industry & Venture Capitalist SVP, Research Operations Additional advisors are regularly brought in to guide different aspects of the program through committees.

Prioritization and Selection Breckenfield Consulting Limited Amanda C.

Mitchell, PhD Valo Health Andy Singleton, PhD Global Parkinson’s Genetics Program Benjamin Logsdon, PhD Cajal Therapeutics, Inc. Bruce Leuchter, MD Neurvati Neurosciences Cornelis Blauwendraat, PhD Coalition for Aligning Science, Aligning Science Across Parkinson’s, Global Parkinson’s Genetics Program Darryle Schoepp, PhD Independent David Stone, PhD ProFound Therapeutics Elisa Tinelli, PhD Golgi Neurosciences Srl Fiona Ducotterd, PhD Alzheimer’s Research UK UCL Drug Discovery Institute Jan Stoehr, PhD Abbvie, Neuroscience Discovery Jessica Sadick, PhD Valo Health Jonas Hannestad, MD Independent Jonathan Behr, PhD Dementia Discovery Fund, SV Health Investors Julie Miller, PhD Depts.

of Neuroscience and Speech, Language and Hearing Sciences, University of Arizona Lee Rubin, PhD Dept of Stem Cell and Regenerative Biology, Harvard University Martin Citron, PhD UCB Biopharma Matthew Nelson, PhD Genscience LLC Mina Ryten, PhD UK Dementia Research Institute and Department of Clinical Neurosciences, School of Clinical Medicine, University of Cambridge Robin Kleiman, PhD Myrobalan Therapeutics Sarah Silvergleid Schrodinger Steve Wood, PhD Neuron23, Inc. Tina Schwabe, PhD Tom Otis, PhD Lario Therapeutics Victoria Dardov, PhD Technome Virginie Buggia-Prevot, PhD Valo Health Alexandra Nelson, MD, PhD UC San Francisco Anatol C.

Kreitzer, PhD MapLight Therapeutics Andy Singleton, PhD Global Parkinson’s Genetics Program Carina Oehrn, MD, PhD University of California, Davis Cornelis Blauwendraat, PhD Coalition for Aligning Science, Aligning Science Across Parkinson’s, Global Parkinson’s Genetics Program Darryle Schoepp, PhD Independent consultant David G.

Standaert, MD, PhD University of Alabama at Birmingham, Heersink School of Medicine David Stone, PhD ProFound Therapeutics Julie Miller, PhD University of Arizona Lee Dawson, PhD Cerevance Ltd. Mark Forman, MD, PhD Ventyx Biosciences Milton Biagioni, MD UCB Biopharma Rebekah Evans, MD Georgetown University Medical Center Robin Kleiman, PhD Myrobalan Therapeutics Sarah Almond, PhD Independent Sarah Silvergleid Schrodinger Shelia Fleming, PhD Northeast Ohio Medical University Stephanie M McTighe McTighe BioScience LLC Thomas Wichmann, MD Emory University Tom Otis, PhD Advisors for the Validation Target Team include: Irit Rappley, PhD Partner, Other Stuff Strategies Aggregation Targets* Michael Ahlijanian, PhD Ani Consulting, LLC Aggregation Targets* Abijal Mariga, PhD Neuroscience Discovery, Bristol Myers Squibb Aggregation Targets* Alastair Reith, PhD Breckenfield Consulting Ltd. Endolysosomal Targets* Anastasia Henry, PhD Denali Therapeutics Endolysosomal Targets* Leon Murphy, PhD Casma Therapeutics Endolysosomal Targets* Magdalene Moran, PhD Topo Therapeutics Endolysosomal Targets* Radha Desai, PhD Taravera Mitochondrial Targets* Ken Nakamura, PhD Independent Mitochondrial Targets* Tom Otis, PhD Lario Therapeutics Mitochondrial Targets* Benjamin Logsdon, PhD Cajal Therapeutics, Inc. NOD2 David Rickard, PhD Independent (formerly GSK) NOD2 Gabriel Nunez, MD University of Michigan NOD2 Ivo Gomperts Boneca, PhD Institut Pasteur & INSERM NOD2 Jia Wolfe, PhD Orchard Therapeutics NOD2 John Nuss, PhD Ventyx Biosciences, Inc. NOD2 Jonathan Behr, PhD Dementia Discovery Fund, SV Health Investors NOD2* Lee Dawson, PhD Cerevance Ltd. NOD2 Rebecca Senter, PhD Vertero Therapeutics NOD2* Stewart Campbell, PhD Vertero Therapeutics NOD2 Žiga Jakopin University of Ljubljana NOD2 Irit Rappley, PhD Partner, Other Stuff Strategies OGA* Kalpana Merchant, PhD TransThera Consulting Co.

and Northwestern University OGA* Ryan Schubert, MD Asceneuron OGA Ernest McEachern, PhD Alectos Therapeutics OGA Hilal Lashuel, PhD Ecole Polytechnique Fédérale de Lausanne OGA Paul W Thompson, PhD Lelantos Bio Ltd. STING1* & TREM* Rita Cowell, PhD University of Alabama at Birmingham STING1* & TREM* Christian Mirescu, PhD Independent (formerly GSK) TLR2* Rebecca Senter, PhD Vertero Therapeutics TLR2* *Co-Chair The network for the Target to Therapies Initiative includes: In-depth Description of the Targets to Therapies Initiative in npj Parkinson's Disease Targets to Therapies: A New Target De-Risking Initiative at MJFF MJFF Target Prioritization Initiative: Latest Updates and Spring 2024 Survey Outcomes Express interest in a target on our list: Researchers with an interest in working with us on a specific target can complete a short Target Interest Form .

MJFF research staff will evaluate submitted interest forms and, when there is a fit, reach out for additional information. Nominate another target: If you have a target that we should know about, please complete this form . Ask a question about the program: If you have a question about the Targets to Therapies Initiative, please send an email to [email protected] .

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