MEMBERS' HIGHLIGHTS

Retroviruses are presently amongst the most widely used vectors in gene therapy clinical trials to target pathologies of different origins, such as cancers, genetic diseases or neurological disorders. One of the characteristics of retroviral vectors is their ability to integrate the genetic material transferred into the host-cell resulting in long-term transgene expression. This makes them particularly suited for the treatment of monogenic diseases. Indeed, retroviruses have been the vector of choice for ex vivo transduction of hematopoetic stem cells.

Retroviruses present several attributes making them attractive to be further applied: large safety record in clinical application, reduced vector genome mobilization and recombination in the host-cell and pre-existing immune response against the retroviral vector particle and easiness to produced at large scale from stable packaging cell lines. Lentiviruses, which are a subclass of Retroviruses have recently been adapted as gene delivery vehicles not only to their ability to integrate into the genome of dividing cells (as retroviruses), but also of non-dividing cells, where they can also achieve long-term stable transgene expression allowing for the development of stable cell lines for in vitro research. Moreover, due to their low immunogenicity, lentiviral vectors have also been delivered in vivo repeatedly in non-human primates with no treatment-related serious adverse events. Together, these vectors show a wide range of potential applications, whether for R&D or therapeutic purposes.

 

IBET has proven its capability and expertise in the field of viral vector development including
• Vector design and construction
• Vector manufacturing in adherent or suspension cell culture, including down-stream purification
• Analytical assays, including functional titering (FACS, qPCR) and  particle titering (ELISA, qPCR)
• Development of stable producer cell lines
• Testing viral vector constructs in mammalian cell lines
• Genibet (partially owned by IBET) is developing the facilities and technologies for preparation of the purified products for phase I and phase II clinical trials.

 

Main Equipments:

- Liquid Chromatography (ÄKTAexplorer 10 and 100) Micro/Ultra/Diafiltration (ÄKTAcrossflow) and Quixstand
- Quantitative PCR Light Cycler
- Leica Fluorescence, phase contrast, dark and bright field microscope
- Luminescence/Fluorescence 96-well plates Reader
- Flow cytometer coupled with a 96-well plate autosampler
- Maldi-Tof/Tof (4800 ABI)
- Biacore

Intended collaboration:

• Service contract : controlled access to equipment
• Joint R&D
• Service contract : full service
  
Application or research field :  Gene therapy
Gene therapy may provide a way to cure genetic disorders, such as severe combined immunodeficiency, cystic fibrosis or even Haemophilia A. Because these diseases result from mutations in the DNA sequence for specific genes, gene therapy trials have used retroviral vectors to deliver unmutated copies of these genes to the cells of the patient's body. There have been a huge number of clinical successes with gene therapy with retroviral vectors, namely in ADA deficiency and SCID-X1.

Potential Users:
Academic laboratories: production of vectors for gene therapy
Industry: pharmaceutical companies, biotechnology.

 

Contact:  info.ibet@ibet.pt ; real@ibet.pt

Useful links:
http://www.ibet.pt
http://tca.itqb.unl.pt
http://www.ibet.pt/Industry/Biopharmaceuticals_and_novel_therapies/
http://www.genibet.com