Question and Answer - International Virtual Family Conference

Please find below some of the questions that have been asked and answered so far of the conference, to be a resource for any carer or parent who has the same questions. If you do have a question still for one of our speakers, please use our online form to ask them!

Q Prof. Tim Benke – Thank you for all your informative videos. I have a question about the COVID-19 vaccine. can children with CDD be given this vaccine? In Japan, even healthy children are not taking it very often. In other countries, do they give it to children with CDD?

A We suggest that all ages should get the vaccine. At the moment, only the Pfizer vaccine is approved for 12 years and over. We hope that it will be approved for younger ages soon. Our CDD population here is getting vaccinated.  We also recommend that children with CDD get the flu vaccine. In our experience, children with CDD can become very sick with the flu and likely also with covid-19.

Q Prof. Ralf Schmid – Your slide showing the relationship of DNA to mRNA to CDKL5 protein development was very interesting. Given the huge success that programmable mRNA has had in the COVID-19 world via Moderna and Pfizer pharma, is there any way to program the CDKL5 protein delivery using their technology.

A There are key differences between a gene therapy targeting brain and COVID RNA-based immunization.

The main issue is durability. Vaccination aims to temporarily express COVID spike proteins to elicit an immune response whereas CDKL5 protein via CDKl5 gene therapy will have to be expressed permanently to provide a lasting benefit for CDKL5 deficiency. The vaccination relies on RNA delivery which is naturally unstable and already artificially modified to prolong its life time. CDD would need DNA-replacement therapy, but the liquid nanoparticle (LNP) technology used for RNA delivery is not (yet) suitable for this purpose. There may be ways in the future to use LNPs for DNA delivery but it may be a challenge to make this work for brain.

*Please note all questions are anonymised, as per guidelines of GDPR, the CCPA and other international Consumer Privacy guidelines!