Just a few short months ago, in April 2020, Hope for Hasti commissioned the manufacture of three different vectors for use in our gene therapy study, the first ever gene therapy study undertaken for CdLS. These vectors are now ready for use thanks to Vigene Biosciences and their prompt manufacturing.  These genetically engineered vectors, act as a vehicle to carry a good copy of the faulty gene which causes CdLS, into the cells of patient’s bodies. 

That bring us to a groundbreaking and exciting phase of the research; the toxicology study. During this phase, our research partners, The Jackson Laboratory, will test the gene therapy treatment on a mouse model to establish that it is safe and not toxic. 

As parents, we are very anxious about this phase. Success in this phase will take us one huge step closer to a gene therapy for our daughter Hasti, and others like her. Success means we can be almost certain that the treatment is viable, and will improve the lives of those receiving the treatment. It will validate all of our efforts to fight for healthier, longer lives for these vulnerable, but amazing children 

The results will be known in December and we will be waiting and watching anxiously for the outcome. Hopefully this will be an early Christmas gift for the entire CdLS community.​

Thanks to all our Hope for Hasti supporters and your fundraising, this has allowed us to bring this key milestone forward. Without you, we would not have been able to pay for this phase so early. Thank you for your continued support and for playing a vital part in making medical history for all the vulnerable children with CdLS.