As well as working on AKU, we will focus on other rare genetic diseases. We are confident that our strategy will become a paradigm for the management of rare monogenic diseases in general.
Our aim is to work with rare disease patient groups to kick-start and accelerate their research into mRNA treatments. If you represent a rare disease patient group, please do get in touch so we can discuss whether and how we can work together. We will start by identifying your need, discussing your rare disease and then seeing whether mRNA might be a suitable therapy. If it is, then we will devise a programme of research.
It will be up to you as the patient group to fundraise for this. In exchange, we will carry out the research and provide a fair share of any revenue we ultimately receive once it reaches the clinic, which you can reinvest in more research and patient support.
A rare disease patient group and Sireau Labs discuss the potential to use mRNA therapy as a treatment for their rare disease.
If it looks possible, the rare disease patient group and Sireau Labs agree a programme of pre-clinical and early clinical work using mRNA technology, with milestones, deliverables and a budget.
The rare disease patient group raises the funds needed.
Sireau Labs and the rare disease patient group collaborate with a pharma company to take the treatment through late phase clinical trials.
If the studies are successful and marketing authorisation is obtained, Sireau Labs provides a fair share of the profit it receives to the rare disease patient group.
The rare disease patient group can reinvest this money into other research and patient support.