Initiatives

UCB; Novo Nordisk; Sanofi; Novartis; Roche; Takeda; Pfizer

Universities, hospitals, EURORDIS, technology firms and research organisations

2021-2026

Europe

IMI public-private consortium

Combines genetic newborn screening, genomic analysis and AI-supported tools to shorten the rare-disease diagnostic journey. Earlier and more accurate identification of patients also generates knowledge needed for disease characterisation, cohorts and future research.

Pre-dates Moonshot launch; active throughout the discussions

Knowledge and target understanding

Direct

AbbVie; AstraZeneca; Bayer; Biogen; Boehringer Ingelheim; BMS; Genentech/Roche; GSK; Janssen/J&J; MSD; Novartis; Pfizer; Sanofi and others

Business Finland, University of Helsinki, Finnish biobanks, hospitals and research organisations

2017-ongoing

Finland

National public-private genomics partnership

Links genomic information from 500,000 biobank participants with longitudinal health data. Although not rare-disease specific, analysis of rare and population-enriched variants can reveal disease mechanisms, validate targets and generate therapeutic hypotheses.

Established before Moonshot; expanded while Moonshot discussions continued

Knowledge and target understanding

Indirect / enabling

GSK; Sanofi; Pfizer; MSD; Genentech/Roche

EMBL-EBI and Wellcome Sanger Institute

2014-ongoing

UK / international

Precompetitive public-private discovery consortium

Integrates human genetics, genomics, experimental biology and informatics to identify and validate drug targets. Its open data and experimental outputs can help translate rare genetic findings into tractable therapeutic hypotheses.

Established precursor capability; active and expanding during Moonshot discussions

Knowledge and target understanding

Indirect / enabling

Amgen; AstraZeneca; Janssen/J&J; MSD; Novartis; Pfizer; Roche Diagnostics; BMS and other industry members

French National Cancer Institute, Health Data Hub, Bpifrance, ARIIS, France Biotech and data-producing organisations

2021-ongoing

France

National public-private AI and oncology-data consortium

Combines public cancer data with industry questions, data, AI and analytical expertise to generate knowledge, diagnostics and treatment insights across common and rare cancers. Its decentralised model and work with regulators illustrate how data can be reused for small molecularly defined populations.

Created shortly before Moonshot; active and expanding throughout the discussions

Knowledge and target understanding

Direct for rare cancers / broader oncology

Sanofi; Takeda; UCB; Novo Nordisk; EFPIA (with earlier support from Alexion/AstraZeneca)

European Reference Networks, EURORDIS, EJP RD, research infrastructures; DG SANTE observer

2021-ongoing

Europe

Multi-stakeholder rare-disease initiative

Develops practical, transparent models for collaboration between industry and European Reference Networks. It helps connect concentrated clinical expertise, cohorts and registries with drug-development, regulatory and operational expertise.

Pre-dates Moonshot launch; developed in parallel and explicitly connected to it

Translation towards development

Direct

EFPIA and participating industry experts within ERDERA

ERDERA research organisations, hospitals, patient organisations, SMEs and investors

2025-ongoing

Europe

European partnership accelerator

Provides rare-disease researchers with development roadmaps and advice in drug discovery, preclinical development, regulatory science, market access and investment readiness. It is designed to move promising academic discoveries towards investable and developable programmes.

Developed during Moonshot follow-through

Translation towards development

Direct

Boehringer Ingelheim

Universities and public/non-profit researchers worldwide

2017-ongoing

Global

Company-led open-innovation platform

Provides external scientists with free access to well-characterised molecules, associated data packages and, in selected routes, company expertise or funding. This allows rare-disease hypotheses to be tested without recreating medicinal-chemistry capabilities.

Established before Moonshot; cited by the IRDiRC study as a transferable collaboration model

Translation towards development

Indirect / enabling

Bayer; AstraZeneca; Lundbeck; Janssen/J&J; Merck KGaA; Sanofi; UCB and later partners

Universities, research organisations, SMEs and screening centres

2013-2023

Europe

IMI open drug-discovery platform

Combined pharmaceutical compound libraries, public chemistry and industrial-scale screening. Public and patient-led researchers could bring promising targets and obtain validated starting points for drug discovery, including for areas too small to sustain separate infrastructure.

Precursor platform; demonstrates the shared-capability logic later advocated by Moonshot

Translation towards development

Indirect / enabling

Bayer

Life-science start-ups; links with Charité–Universitätsmedizin Berlin, the Max Delbrück Center and the Berlin life-sciences community

2024–ongoing

Germany

Company-led life-science incubator linked to public research institutions

Provides early-stage biotechnology companies with equipped laboratories, Bayer expertise, mentoring and connections to development partners. Its first resident, MyoPax—a spin-off from Charité and the Max Delbrück Center—is developing stem-cell and gene-editing approaches for muscle regeneration, including rare muscle diseases. The model can help rare-disease innovations move from academic research towards clinical development without each company having to build the necessary infrastructure and expertise independently.

Launched during Moonshot implementation discussions; complements the Berlin Center for Gene and Cell Therapies

Translation towards development

Direct through participating companies / broader enabling platform

Bayer through Leaps by Bayer

DEEP – Institute for Deep Tech Innovation at ESMT Berlin; Creative Destruction Lab-Berlin; biotechnology and health-technology start-ups

2023–2026 three-year partnership

Germany / international

Company–academic innovation and start-up acceleration partnership

Supports the identification, mentoring, financing and scaling of science-based biotechnology and health-technology ventures. The partnership explicitly covers innovations addressing genetic diseases, alongside tissue replacement, cancer, autoimmune diseases and data-driven health solutions. It can help promising technologies relevant to rare genetic diseases cross the gap between academic innovation, company formation and investable development programmes.

Launched shortly after the Moonshot and active alongside its implementation discussions

Translation towards development

Indirect / enabling, with an explicit focus on genetic diseases

Pfizer; AstraZeneca; Novo Nordisk and other industry/technology partners

Structural Genomics Consortium, Goethe University Frankfurt, UCL, UHN, University of Campinas and others

2025-2030

Europe / Canada / Brazil

IHI open-science and AI consortium

Aims to create an open, AI-ready protein-ligand resource at unprecedented scale and experimentally validated chemical starting points for hundreds of proteins. It can open drug discovery around understudied proteins implicated in rare diseases.

Launched during Moonshot follow-through

Technology platforms

Indirect / strong enabling

Astellas; Bayer; BMS; Janssen/J&J; Novartis; Novo Nordisk and other partners

Academic gene-therapy centres, regulators, patient and research organisations

2020-2025

Europe

IMI regulatory-science and advanced-therapy consortium

Builds shared knowledge, tools and standards for understanding and managing immune responses to adeno-associated virus and other advanced therapies. These reusable approaches reduce scientific and regulatory uncertainty for rare-disease gene-therapy programmes.

Pre-dates Moonshot; active during its development and featured on the Moonshot website

Technology platforms

Direct / strong enabling

Pfizer; Takeda; Novartis and other life-science partners

NIH, FDA, FNIH, patient organisations, academic centres and NIIMBL

2021-ongoing

United States

Accelerating Medicines Partnership consortium

Develops a common AAV platform, preclinical approaches, manufacturing processes, regulatory templates and clinical pathways for ultra-rare diseases that are too small for conventional commercial development. It replaces a one-disease-at-a-time model with reusable capabilities.

Pre-dates Moonshot launch; became a central global analogue in Moonshot discussions

Technology platforms

Direct

Bayer

Charite - Universitatsmedizin Berlin; Federal Government of Germany; State of Berlin

2024-ongoing

Germany

Public-private translational centre and incubator

Combines a clinical development unit with an incubator for emerging gene and cell therapy companies. It is intended to move advanced therapies from research towards patients and create reusable expertise, infrastructure and company formation capacity relevant to rare genetic diseases.

Launched alongside Moonshot implementation discussions

Technology platforms

Direct / enabling

Bayer; Boehringer Ingelheim; Janssen/J&J; MSD; Novartis; Pfizer and other partners over time

Universities and public research institutes across several countries

2004-ongoing; Target 2035 since 2019

Global

Open-science public-private research platform

Creates open structural biology, chemical probes, assays and data for understudied proteins, while Target 2035 seeks a pharmacological tool for every human protein. This reduces the discovery disadvantage faced by rare diseases linked to poorly studied biology.

Long-standing precursor; continuing platform for Moonshot white spots

Technology platforms

Indirect / enabling

AstraZeneca; Bayer; GSK; Janssen/J&J; Novartis; Pfizer; Roche; Takeda and others over time

University of Strathclyde and wider academic, government and technology partners

2011-ongoing

UK / international

Precompetitive manufacturing research hub

Develops advanced crystallisation, formulation, process analytics, digital tools and continuous-manufacturing capability. Its relevance to rare diseases is indirect: flexible and more efficient manufacturing can improve the feasibility of diverse, lower-volume products.

Established precursor; active during Moonshot discussions

Manufacturing capability

Indirect / enabling

Pfizer

CPI and the UK Medicines Manufacturing Innovation Centre

2021-ongoing

UK

Public-private manufacturing technology collaboration

Develops a continuous direct-compression platform supported by digital twins and predictive models. It aims to reduce materials, development time and formulation risk, providing an example of reusable manufacturing capability rather than a disease-specific programme.

Pre-dates Moonshot launch; active during discussions

Manufacturing capability

Indirect / enabling

AstraZeneca; GSK; Janssen/J&J; Merck/MSD; Pfizer; Sanofi; Takeda and other biopharma members

US Department of Commerce/NIST, universities, non-profits and technology companies

2017-ongoing

United States

Public-private advanced biomanufacturing institute

Develops and disseminates platform technologies, standards, workforce skills and process capabilities for biopharmaceutical manufacturing. It can support scalable and more standardised manufacture of biologics and gene therapies relevant to rare diseases; it also participates in BGTC.

Established before Moonshot; global enabling infrastructure active throughout discussions

Manufacturing capability

Indirect / enabling

Fresenius/Fresenius Kabi; Philips and other industrial technology partners; supported through IHI private-member contributions

Fraunhofer IZI and IESE, hospitals, universities, EBMT, research organisations and technology partners

2025-2030

Europe

IHI advanced-therapy manufacturing consortium

Develops an integrated, automated, non-viral platform for hospital-based CAR-T manufacturing, alongside quality control, digital integration, regulatory, health-economic and workflow solutions. Although initially focused on cancer, decentralised and faster manufacturing can support personalised cell and gene therapies relevant to rare diseases.

Launched during Moonshot follow-through

Manufacturing capability

Indirect / strong enabling

Bayer; with exchange of experience involving Orion, Revvity and technology partners

University of Turku; Finland Futures Research Centre; Turku School of Economics; Business Finland; research and technology partners

2024–2027, following a 2023 co-creation phase

Finland

Publicly funded company–academic manufacturing innovation consortium

Develops reusable digital-manufacturing capabilities, including a digital twin, standardised production-data models, contextualised manufacturing data and AI-assisted batch-record verification and problem-solving in a regulated environment. The solutions are intended to be scaled beyond the initial Bayer Turku pilot. Although not rare-disease specific, adaptable and transferable manufacturing methods can reduce the need to recreate manufacturing capabilities for every product and may improve the feasibility of low-volume rare-disease medicines.

Developed from the Turku–Bayer strategic partnership during Moonshot discussions and implemented during Moonshot follow-through

Manufacturing capability

Indirect / enabling

AstraZeneca (industry lead); Sanofi and other pharmaceutical partners

Sigmund Freud Private University, EMA, IQWiG, EURORDIS, ERNs, hospitals and research infrastructures

2024-2029

Europe

IHI rare-disease clinical-trial consortium

Develops and tests innovative trial designs, analysis methods, RWE approaches, digital tools and operational playbooks for rare and ultra-rare diseases. It combines methodological innovation with regulator, HTA, patient and clinical-network participation to support acceptance and use.

Launched alongside Moonshot implementation; featured on the Moonshot website

Evidence generation and development methods

Direct

Roche; Novartis

INSERM, universities, Health Data Hub, ECRIN, regulators, hospitals and research organisations

2023-2028

Europe

Horizon Europe public-private research consortium

Develops modelling, simulation, extrapolation and evidence-synthesis tools for regulatory evaluation of treatments in rare and paediatric diseases. Industry use cases and datasets help ensure methods address real development decisions and small-population constraints.

Launched alongside Moonshot discussions

Evidence generation and development methods

Direct

Bayer; Roche; GSK; Servier; Janssen/J&J; Novartis; Pfizer; Sanofi; UCB; Lilly and others

Paediatric hospitals, academic networks and patient organisations across Europe

IMI project 2018-2024; Stichting c4c operational from 2024

Europe

IMI paediatric trial network followed by an independent non-profit foundation

Created a pan-European paediatric trial network with common processes, expert advice, site capacity and methods relevant to rare paediatric diseases. Stichting c4c carries the capability forward as a sustainable legal entity supporting trials, strategic feasibility and expert advice after the IMI grant.

Established before Moonshot; active at launch and institutionalised during Moonshot follow-through

Evidence generation and development methods

Direct / enabling

Janssen/J&J; Novartis; Takeda and other EFPIA partners

University Medical Center Utrecht, ECRIN, academic trial groups, patient organisations and SMEs

2020-2023; designs being implemented

Europe

IMI platform-trial methodology consortium

Created master-protocol and integrated research-platform designs across disease areas, including neurofibromatosis. Its importance for the Moonshot lies in the subsequent use of these designs: the capability moved from methodology development into CTF-led rare-disease trials.

Pre-dates Moonshot; completed as Moonshot launched, with implementation continuing

Evidence generation and development methods

Indirect / enabling

Takeda in INTUITT-NF2; additional pharmaceutical companies can contribute assets to platform arms

Children's Tumor Foundation, Global Coalition for Adaptive Research, European clinical centres and patient community

2020-ongoing; EU NF1 platform launched 2026

United States / Europe

Patient-foundation-led adaptive platform trials

Applies shared master protocols to evaluate multiple therapies and disease manifestations within continuing trial infrastructures. The 2026 European NF1 platform basket trial explicitly builds on EU-PEARL, showing how an IMI methodology can be deployed and extended in rare diseases.

Implementation evolved during Moonshot discussions; European follow-up launched in 2026

Evidence generation and development methods

Direct

EFPIA and participating pharmaceutical companies

European Forum for Good Clinical Practice, patient organisations, academia, research networks, ethics committees, regulators, CROs and payers

2023-ongoing

Europe

Multi-stakeholder public-private initiative

Develops recommendations and country information to enable patients to join trials in another European country. This is especially important for rare and paediatric diseases, where the relevant study or specialist site may not exist in a patient's home country.

Announced immediately after the Moonshot launch and developed concomitantly

Evidence generation and development methods

Direct / enabling

Multiple pharmaceutical companies contribute approved targeted medicines, including EFPIA-member assets

Netherlands Cancer Institute, Center for Personalized Cancer Treatment, Dutch hospitals, molecular tumour boards and researchers

2016-ongoing

Netherlands

National precision-oncology access and evidence protocol

Matches patients who have exhausted standard options to approved targeted therapies based on molecular tumour profiles, while systematically collecting outcomes and biomarker data. The model is particularly valuable for orphan cancers and very small molecular subgroups that cannot support conventional trials.

Established before Moonshot; active evidence-and-repurposing model throughout discussions

Evidence generation and development methods

Direct for rare molecular subgroups / broader oncology

Multiple EFPIA companies

Academic toxicology, genomics and pathology research groups

2008-2013

Europe

IMI biomarker and safety-science consortium

Developed molecular biomarkers and mechanistic approaches for detecting non-genotoxic carcinogenesis. It demonstrates the precompetitive creation of shared safety tools that can reduce duplicative work and improve evidence packages, including for rare-disease programmes using novel modalities.

Early precursor drug-development tool highlighted in the IRDiRC report

Evidence generation and development methods

Indirect / enabling

Roche; Servier; Pfizer historically, with other biopharma partners

Critical Path Institute, Parent Project Muscular Dystrophy, FDA, EMA, universities and patient organisations

2015-ongoing

United States / international

Disease-specific regulatory-science consortium

Pools and standardises patient-level data to build disease-progression models, clinical-trial simulation tools, biomarkers and outcome measures for Duchenne muscular dystrophy. The outputs are intended for regulatory use and are made available to the wider development community.

Established before Moonshot; active global evidence capability throughout discussions

Evidence generation and development methods

Direct

Biogen; Servier; PTC Therapeutics and other industry members

Critical Path Institute, ataxia patient organisations, universities and FDA advisers

2021-ongoing

United States / international

Disease-area regulatory-science consortium

Aggregates and shares data, evaluates endpoints and biomarkers, and develops tools and strategies for inherited ataxias. It offers a neutral forum in which companies, researchers, patients and regulators address common trial and evidence bottlenecks.

Pre-dates Moonshot launch; active and expanding during discussions

Evidence generation and development methods

Direct

Janssen/J&J (IMI project lead) and multiple EFPIA partners

Erasmus MC, universities, data partners, SMEs and healthcare organisations

IMI project 2018-2024; EHDEN Foundation ongoing

Europe

IMI federated health-data network followed by a sustainable foundation

Standardised heterogeneous health datasets by mapping them to the OMOP common data model and built a federated network, analytical tools and training capability. The EHDEN Foundation sustains this work. For rare diseases, the core contribution is making fragmented real-world data interoperable, analysable and reusable across institutions and countries.

Established before Moonshot; active at launch and institutionalised during follow-through

Evidence generation and development methods

Indirect / strong enabling

IHI/EFPIA industry contribution and an industry advisory group; individual company roles should be updated as publicly confirmed

TREAT-NMD, patient organisations, Amsterdam UMC, SMEs and rare neuromuscular-disease organisations

2024-2027

Europe / United States

IHI patient-centred data-platform consortium

Is developing the Interactium, a FAIR-by-design platform for patient-reported and clinical data in rare neuromuscular diseases. It aims to improve outcomes, trial planning, drug development and decision-making, with tools designed for reuse in other rare diseases.

Launched alongside Moonshot implementation discussions

Evidence generation and development methods

Direct