• FAQs
  • Glossar
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FAQs

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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Glossar

Adeno-associated virus (AAV)
Adenovirus
Adult stem cells
Antibody
Base editors
Cas9 nuclease
Chimeric Antigen Receptor (CAR)
Chromosome
Clinical trial
CRISPR-Cas

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Case Studies

This section contains case studies on ATMP development. Follow the links below to learn about the development processes, the keys to success, the challenges and how developers overcame them: 

Case study: Holoclar

learn about the development of Holoclar, a Tissue Engineered Product (TEP)
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Case Study: Strimvelis

learn about the development of Strimvelis, a Gene Therapy Medicinal Product (GTMP)
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Case Study: Therapy Classification

Early therapy classification considerations in developing skin equivalents
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