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AF
Academic

AGORA Foundation

Leiden, NETHERLANDSNot for ProfitReg: 927015094421-69Since 30/10/2024

Budget

€10 — €0

EP Access

0

accredited persons

Staff

5

0.5 FTE

EU Grants

None

Mission & Goals

Our mission is to facilitate sustainable access to effective gene therapies for treatment of patients with ultra-rare diseases in Europe focusing on Inborn Errors of Immunity (IEI) and Inborn Errors of Metabolism (IEM). In recent years it has become evident that effective stem cell gene therapies are not reaching patients in need for non-medical reasons. To achieve sustainable and affordable access to these life-changing gene therapies for patients with rare inherited diseases (r)evolution is needed to provide alternatives for the conventional commercialization model when not fit for purpose.

EU Legislative Interests

Health Technology Assessment Stakeholder Network European Reference Network - ERN RITA (the European Reference Network that aims at improving the care of patients with Rare Immunological Disorders)

Communication Activities

We plan to engage in organised events related to ensuring access to effective and safe advanced therapies for patients with inherited rare and ultra-rare diseases We plan to also engage with EMA in this context. We have already contributed to an EU event organised by IPOPI at the European Parliament to inform members of parliament on challenges and potential solutions in this field.

Interests Represented

Promotes their own interests or the collective interests of their members

Member Of

AGORA is not a member of any associations or bodies

Organisation Members

Prof - Leiden University Medical Centre, Netherlands Prof - UCL Great Ormond Street Institute of Child Health, Great Ormond Street Hospital London, UK Prof - San Raffaele Hospital TIGET, Milan, Italy Prof - University of Lausanne Representative - International Patient Organisation for Primary Immunodeficiencies (IPOPI)

Additional Information

To date, no costs have been made a/o reported

Connected Legislation

Commissioner Meetings

No recorded meetings with EU commissioners.

AGORA Foundation — EU Lobby Register | GovLens