INTERNATIONAL PATIENT ORGANISATION FOR PRIMARY IMMUNODEFICIENCIES

Their core work

IPOPI is the international umbrella body representing national patient associations for people living with primary immunodeficiencies (PIDs) — a group of rare, often life-threatening genetic disorders affecting the immune system. In the context of H2020 research, their core contribution is not scientific execution but patient community infrastructure: they bring access to patient registries, lived-experience input into trial design, and dissemination channels directly to affected families and clinicians. In both projects they joined, IPOPI served as the patient-facing voice inside gene therapy research consortia developing treatments for Severe Combined Immunodeficiency (SCID), one of the most severe forms of PID. Their practical value to a research consortium is legitimacy with ethics boards, reach into rare disease communities across multiple countries, and the ability to help researchers understand what outcomes actually matter to patients.

What they specialise in

How they've shifted over time

IPOPI's H2020 trajectory is narrow but coherent: both projects address SCID specifically, with the earlier project (SCIDNET, 2016–2019) focused on developing genetic medicines broadly, and the later one (RECOMB, 2018–2024) narrowing further into stem-cell-based gene therapy for a defined genetic subtype of SCID. This represents a deepening of focus rather than a broadening — from rare disease advocacy in gene medicine generally to sustained, long-term engagement in a specific therapeutic modality. The shift from no explicit keyword tagging in SCIDNET to "gene therapy" in RECOMB reflects that the field itself matured and IPOPI moved with it.

IPOPI is embedding itself more deeply in gene therapy research for immunodeficiencies, suggesting future collaborations are most likely to involve advanced therapeutic consortia seeking patient advocacy and community access for SCID or related PIDs.

How they like to work

IPOPI has never led an H2020 project — they participate exclusively as consortium members, consistent with their role as a patient organization rather than a research executor. Their participation in two multi-partner RIA consortia (21 unique partners across 10 countries) suggests they are comfortable operating inside large, complex research teams without needing to drive scientific coordination. They bring a specific, bounded contribution — patient community access and advocacy — rather than technical capacity, which makes them a specialist add-on rather than a core scientific partner.

IPOPI has built connections with 21 distinct consortium partners spanning 10 countries through just two projects, indicating they join well-networked European research consortia with broad geographic footprints. Their network is likely weighted toward immunology and rare disease research institutes, clinical centers, and biotech firms active in gene therapy.

What sets them apart

IPOPI occupies a rare position in EU health research: they are one of the few international patient organizations with sustained, direct experience inside gene therapy research consortia for primary immunodeficiencies — a disease area where patient recruitment, consent, and community trust are critical bottlenecks. Unlike academic partners, they bring credibility with patients and families across multiple countries simultaneously, which no university or biotech can replicate. For any consortium working on PID treatments — especially gene or cell therapies — IPOPI is the natural first call for the patient engagement workpackage.

Highlights from their portfolio

• RECOMB
  The longer-running project (2018–2024) places IPOPI inside a stem-cell gene therapy program for SCID, one of the most ambitious therapeutic areas in rare disease research, reflecting the organization's deepest engagement with cutting-edge treatment development.
• SCIDNET
  IPOPI's only funded H2020 project (EUR 25,000), establishing their entry into EU-funded genetic medicine research and their foundational relationship with the SCID research community.