ATMP in Ireland is a national network of industry, academia, and government agency representatives. Our goal is to promote, support and facilitate research and education in the area of Cell and Gene Therapies with a view to making Ireland a world-leading location for the production of these revolutionary new medicines
What is CGT
Cell therapy and gene therapy are overlapping fields of biomedical research and treatment. Both therapies aim to treat, prevent, or potentially cure diseases, and both approaches have the potential to alleviate the underlying cause of genetic diseases and acquired disease. But, cell and gene therapies work differently.
The difference between cell therapy and gene therapy?
Cell therapy aims to treat diseases by restoring or altering certain sets of cells or by using cells to carry a therapy through the body. With cell therapy, cells are cultivated or modified outside the body before being injected into the patient. The cells may originate from the patient (autologous cells) or a donor (allogeneic cells).
Gene therapy aims to treat diseases by replacing, inactivating or introducing genes into cells— either inside the body (in vivo) or outside of the body (ex vivo).
Some therapies are considered both cell and gene therapies. These therapies work by altering genes in specific types of cells and inserting them into the body.
- • Academic labs (links to research groups in)…TCD, UCC, UCD, NUIG, DCU, etc
• Company links?
• Government agencies?
• Regulator? EU? Irish?
• Professional bodies – IPHA, BPCI, Ibec, etc
CGT research expertise at NIBRT
- Upstream processes for viral vector
- Production (AAV and LV)
- Producer cell line engineering/optimisation
- Viral genome engineering
- Media development and optimisation
- Viral yield optimisation
- In-line culture monitoring
- Molecular profiling (omics) of GT production processes and modified and unmodified CTs
- Analytics of AAV gene therapy vectors
Training & Education
Allergan/Abbvie-NIBRT gene therapy research collaboration
The collaboration is addressing key challenges in the manufacture and characterisation of Adeno-Associated Virus (AAV)-based gene therapies.
Adeno-Associated virus (AAV)-based gene therapies represent an exciting new wave of advanced therapy medicinal products (ATMPs) that are being generated for the treatment of ailments caused by a defective gene within certain patient populations. AAV based gene therapy is used to deliver a correctly functioning copy of the gene to the patient. Very promising results have been reported in a number of genetic diseases including haemophilia and ocular degeneration.
However, current yields of AAV from the manufacturing process mean that supplying patients with the required quantities of medicine will be challenging. Furthermore, ensuring the quality and efficacy of the AAV material also requires further technology development. This collaboration between NIBRT and Allergan/Abbvie is addressing the following challenges in AAV production:
- Optimising cell culture process for generation of infectious AAV particles of various serotypes
- Establishing vectors and transfection/transduction conditions to minimise incorrect packaging events
- Investigation of sensitive analytical methods for the characterisation of the resulting AAV particles that enable differentiation between full and empty AAV capsids
The collaboration is funded by Enterprise Ireland.
• Information on clinical trials (ICORG etc)?
• Info for patients/clinicians???
• Other resources???