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Bridging the gap between scientific discovery and investment-ready innovation for rare disease breakthroughs.

ERDERA Technology Accelerator

The ERDERA Technology Accelerator advances the enabling technologies that make rare-disease therapies possible. We bring public and private partners together to mature, validate, and streamline advanced therapy platforms so promising discoveries can move faster, safer, and more efficiently toward the clinic.

By strengthening shared technological toolboxes for the rare disease community, we help reduce development risk and accelerate translation across multiple indications.

You are a technology developer…

…working on enabling technologies for rare-disease therapies within academia, a research infrastructure, an SME, or industry.

…developing platforms such as viral or non-viral delivery systems, gene-editing tools, therapeutic RNAs, or analytical and manufacturing solutions.

…looking to improve robustness, scalability, comparability, or clinical relevance of your technology through collaboration and real-world feedback.

If this sounds like you, the Technology Accelerator can help.

The Technology Accelerator supports partners by:

Advancing enabling ATMP technologies, including:

Adeno-associated Virus (AAVs)

Messenger RNA (mRNA)

Lipid Nanoparticles, Extracellular Vesicles, Biohybrids

Gene Editing Tools

Addressing key technical bottlenecks across:

Delivery efficiency and tissue targeting

Manufacturing and scale-up

Analytical characterisation and quality control

Evaluation of therapy response, safety, and immunogenicity

Facilitating pre-competitive collaboration between public and private stakeholders to strengthen shared technology platforms.

Aligning technology development with translational and clinical needs in rare diseases.

Advancing enabling ATMP technologies, including:

  • Adeno-associated Virus (AAVs)
  • Messenger RNA (mRNA)
  • Lipid Nanoparticles, Extracellular Vesicles, Biohybrids
  • Gene Editing Tools

Addressing key technical bottlenecks across:

  • Delivery efficiency and tissue targeting
  • Manufacturing and scale-up
  • Analytical characterisation and quality control
  • Evaluation of therapy response, safety, and immunogenicity

Networking

Facilitating pre-competitive collaboration between public and private stakeholders to strengthen shared technology platforms.

Alignment

Aligning technology development with translational and clinical needs in rare diseases.

Robust, well-validated enabling technologies are essential for translating rare-disease discoveries into viable therapies. By strengthening these foundations, the Technology Accelerator supports not just individual projects but the entire rare-disease innovation ecosystem.

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Understand the concept of ATMPs with this Knowledge Pill

The role of National Mirror Groups (NMGs)

Publicaciones (1)

EU rare disease action plan- European Added Value Assessment

This study investigates possible measures that could be taken at EU level to address these challenges. It finds significant European added value in harmonising coordination and access across the 27 Member States, mainly in terms of improved diagnostic tools and availability of medical treatment, better health outcomes, particularly lower infant mortality, and improved well-being of family members and caregivers.
Scientific paper

Human genomics technologies in clinical studies – the research landscape: report on the 1990–2024 period

Published on 14 December, this WHO technical document maps global trends in registered clinical studies using human genomic technologies from 1990 to 2024, including patterns of inclusion and equity.
abstract publication image on science health

Standardized transcriptome analysis improves rare disease diagnosis in the pan-European Solve-RD consortium

Long-read genome sequencing (lrGS) has the potential to consolidate current standard-of-care (SoC) diagnostics into a single assay, but its accuracy and clinical utility in routine practice have not been established at large scale.
Publications

Clinical long-read genome sequencing for rare disease diagnostics

Long-read genome sequencing (lrGS) has the potential to consolidate current standard-of-care (SoC) diagnostics into a single assay, but its accuracy and clinical utility in routine practice have not been established at large scale.

Latest relevant publications

abstract publication image on science health

Standardized transcriptome analysis improves rare disease diagnosis in the pan-European Solve-RD consortium

Long-read genome sequencing (lrGS) has the potential to consolidate current standard-of-care (SoC) diagnostics into a single assay, but its accuracy and clinical utility in routine practice have not been established at large scale.
Publications

Clinical long-read genome sequencing for rare disease diagnostics

Long-read genome sequencing (lrGS) has the potential to consolidate current standard-of-care (SoC) diagnostics into a single assay, but its accuracy and clinical utility in routine practice have not been established at large scale.
Molecular therapy

Tailored antisense oligonucleotides for ultrarare CNS diseases: An experience-based best practice framework for individual patient evaluation

Outside traditional drug development pathways, clinicians and scientists face the challenge of systematically evaluating whether individual patients with severe ultrarare diseases might be eligible for and potentially benefit from individualized mutation-specific RNA therapies.
bioinfo_41_12cover

PROTRIDER: protein abundance outlier detection from mass spectrometry-based proteomics data with a conditional autoencoder

PROTRIDER is a method for detecting aberrant protein expression from mass spectrometry data that outperforms existing approaches and identifies enrichments of pathogenic variants, supporting its application in rare disease diagnostics and cancer proteomics.

ERDERA's

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