POLARIS · Progress in Leukodystrophies: Advance, Rate and Implement new treatment Strategies

Lines connect the coordinator with its partners. CORDIS does not always give exact coordinates for projects before 2014. These points are placed at city or country level.

Project objective

Leukodystrophies are severe genetic disorders of the brain white matter that cause profound disability and premature death, mostly in children, and lack effective treatments. POLARIS aims to accelerate therapy development by training the next generation of leukodystrophy scientists capable of collaborating across the full spectrum of discovery, development, evaluation and implementation of innovative and accessible therapies. Researchers will develop gene-therapy approaches (RO1), pharmacological strategies (RO2) and treatment-evaluation tools (RO3) to advance therapies for leukodystrophies and strengthen R&I frameworks for other rare diseases.A key barrier to progress is fragmentation: discovery, preclinical development, outcome definition and regulatory pathways rarely coexist in a single environment. POLARIS bridges this through 3 cross-cutting themes: (I) Affordable, Accessible and Timely—embedding cost, accessibility and real-world feasibility from the earliest stages; (II) Relevant Outcomes—focusing on what matters to patients, caregivers, clinicians, regulators and society; and (III) Transferable Knowledge & Tools—producing broadly applicable insights and platforms beyond individual diseases. The programme integrates all relevant stakeholders, including strong engagement of patient organisations, industry and public bodies.POLARIS generates major scientific advances through innovative gene-therapy and pharmacological platforms, validated biomarkers and harmonised outcome measures that directly support future clinical trials. Its end-to-end translational pipeline creates scalable technologies relevant to a wide range of rare neurological disorders. POLARIS enhances researchers’ career prospects, skills, employability and contributions to Europe’s innovation capacity. By reducing development barriers and enabling more effective, affordable treatments, POLARIS delivers substantial economic and societal benefits for patients, families and healthcare systems.

Original text from CORDIS.

Participants

• STICHTING AMSTERDAM UMC · AmsterdamCoordinatorNetherlands
• AGENTSCHAP COLLEGE TER BEOORDELING VAN GENEESMIDDELEN · UtrechtNetherlands
• Alex, The Leukodystrophy Charity · LondonCity levelUnited Kingdom
• Alliance MLC Foundation · Middletown, DECountry levelUnited States
• CONSIGLIO NAZIONALE DELLE RICERCHE · RomaItaly
• Data Powered Therapeutics GmbH · GöttingenCity levelGermany
• EBERHARD KARLS UNIVERSITAET TUEBINGEN · TuebingenGermany
• ELA International · LuxembourgCity levelLuxembourg
• ENAMINE GERMANY GMBH · Frankfurt am MainGermany
• FUNDACIO INSTITUT HOSPITAL DEL MAR D INVESTIGACIONS MEDIQUES · BarcelonaSpain
• Fundament Subsidieadvies · HilversumNetherlands
• INSTITUT DU CERVEAU ET DE LA MOELLE EPINIERE · ParisFrance
• ISTITUTO SUPERIORE DI SANITA · RomaItaly
• KYPRIAKO IDRYMA EREVNON GIA TI MYIKI DISTROFIA · AGIOS DOMETIOSCyprus
• OULUN YLIOPISTO · OuluFinland
• Orchard Therapeutics (Europe) Limited · LondonCity levelUnited Kingdom
• SORBONNE UNIVERSITE · ParisFrance
• STICHTING VU · AmsterdamNetherlands
• SYNAPTOLOGICS BV · AMSTERDAMNetherlands
• THE ANNA FREUD CENTRE · LondonUnited Kingdom
• THE UNIVERSITY OF LIVERPOOL · LIVERPOOLUnited Kingdom
• UNIVERSIDAD POMPEU FABRA · BarcelonaSpain
• UNIVERSITA DEGLI STUDI DI ROMA LA SAPIENZA · RomaItaly
• UNIVERSITAT DE BARCELONA · BarcelonaSpain
• UNIVERSITAT ZURICH · ZurichSwitzerland
• UNIVERSITEIT VAN AMSTERDAM · AmsterdamNetherlands
• UNIVERSITY COLLEGE LONDON · LondonUnited Kingdom
• UNIVERSITY OF NEWCASTLE UPON TYNE · Newcastle Upon TyneUnited Kingdom
• UNIVERSITY OF YORK · YORK NORTH YORKSHIREUnited Kingdom
• VRIJE UNIVERSITEIT BRUSSEL · Bruxelles / BrusselBelgium
• Vereniging Innovatieve Geneesmiddelen · Den HaagCity levelNetherlands
• Zantiks Limited · CambridgeCity levelUnited Kingdom

Links

• View on CORDIS
• DOI: 10.3030/101311855