Beginning of a Phase 1 clinical trial: what to expect?

The first phase should last about 6 months. If there are no signs of toxicity, it will be followed by a second phase, focused on testing efficacy and understanding at what dosage the therapy works best for patients depending on their overall situation. 

All stakeholders have prepared this trial for it to be successful. But research is never as linear and simple as we want it to be. It is our duty to stay focused, keep a cool mind and prepare for different scenarios. 

If everything works well, it could lead to other trials for additional exons and other diseases. Indeed, as the dystrophin gene is one of the longest in the DNA, it represents an excellent case study and the solutions currently being tested could have numerous applications for many genetic diseases. As the technology developed is very promising, partnerships have already been sealed to test the technology on other diseases such as epidermolysis bullosa or spinal muscular atrophy. 

If, despite all the efforts made to ensure safety and effectiveness, this first trial was not conclusive, the process would be restarted at the laboratory research level with the aim to launch a second trial. Although it is not an exciting scenario, it is one that our organization is considering in order to be able to support the project in the long term.