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FAQs

What is gene and cell therapy?
What are ATMPs, and where do they fit into gene and cell therapy?
Why are animal models used when testing the safety of a new therapy?
What role do preclinical and clinical trials play in ensuring patient safety?
Why are some conditions more suitable for gene and cell therapies than others?
There is an approved treatment available for my condition - why can't I receive it?
How to look for approved medicines?
How are medicines developed and approved?

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Glossary

Accelerated assessment
Adeno-associated virus (AAV)
Adenovirus
Adult stem cells
Advanced Therapy Investigational Medicinal Product (ATIMP)
Advanced Therapy Medicinal Product (ATMP)
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Antibody
Antigen
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ATMP Engage PPI Directory

The PPI Directory contains patient and public involvement (PPI) resources for developers of cell and gene therapies, including toolkits, guidelines, advice, templates and case studies.

PPI Directory

PPI Themes
Research Field
Organisation Type
Development Stage
Audience
Country
Content Type
    Showing 5 of 174 results.
    Page 5 of 35
    2016

    Patient Involvement

    Resource by Genetic Alliance UK on PPI benefits and how to get involved
    Visit this Resource

    Research Field

    • General

    Organisation Type

    • Third Sector/Charity

    Development Stage

    • Identifying/Prioritising Patient Need
    • Research and Clinical Development
    • Regulatory Approval
    • Health Technology Assessment

    Audience

    • Researchers/Developers
    • Public/Patients
    • Charity Sector

    Country

    • UK

    Content Type

    • Resource

    NIH Toolkit for Patient-focused therapy development

    Toolkit to help patient groups support the process of developing a treatment or cure for their disease by National Institutes of Health (NIH)
    Visit this Resource

    Research Field

    • General
    • Biomedical Lab
    • ATMP/Gene Therapy Development

    Organisation Type

    • Other Organisation

    Development Stage

    • Identifying/Prioritising Patient Need
    • Research and Clinical Development
    • Regulatory Approval
    • Health Technology Assessment
    • Service Delivery

    Audience

    • Public/Patients
    • Charity Sector

    Country

    • North America

    Content Type

    • Resource
    2020

    Short Guide on Patient Partnerships in Rare Disease Research Projects

    Report on partnerships between scientists and patient organisations by European Joint Programme on Rare Diseases
    Visit this Resource

    Research Field

    • General
    • Biomedical Lab

    Organisation Type

    • Third Sector/Charity

    Development Stage

    • Research and Clinical Development

    Audience

    • Researchers/Developers
    • Public/Patients
    • Charity Sector
    • Industry
    • Other groups

    Country

    • Europe

    Content Type

    • Resource
    2008

    ISSCR Patient Handbook on Stem Cell Therapies

    Report on stem cell therapies aimed at patients by International Society for Stem Cell Research
    Visit this Resource

    Research Field

    • Other

    Organisation Type

    • Third Sector/Charity

    Audience

    • Public/Patients
    • Other groups

    Country

    • North America

    Content Type

    • Resource

    PREFER - The Patient Perspective

    PREFER study is looking at how to include patient preferences in decision making during the medicinal product lifecycle
    Visit this Resource

    Research Field

    • Other

    Organisation Type

    • Academia
    • Regulatory Body
    • HTA
    • Industry
    • Third Sector/Charity
    • Other Organisation

    Development Stage

    • Identifying/Prioritising Patient Need
    • Research and Clinical Development
    • Regulatory Approval
    • Health Technology Assessment
    • Service Delivery

    Audience

    • Researchers/Developers
    • Public/Patients
    • Charity Sector
    • Industry
    • Other groups

    Country

    • Europe

    Content Type

    • Resource
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