Jainu Jogani on the Potential to Address CDKL5 Deficiency With Gene Therapy

“There is this sense of urgency that comes with rare disorders. Many in the industry understand this. It would be phenomenal if everybody could understand this and give support at that level and with that sense of urgency so that we don’t have to wait the traditional 7 or 10 or 15 years for development of a medicine for our children.”

Cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder is a rare genetic disease caused by mutations or deletions of the CDKL5 gene. The condition is devastating and causes developmental delay, epilepsy, sleep issues, and other symptoms. Currently, there is no cure available for CDKL5 deficiency disorder and treatment largely consists of supportive care.

At the American Society of Gene & Cell Therapy (ASGCT) 28th Annual Meeting, held May 13 to 17, 2025, in New Orleans, Louisiana, CGTLive spoke with Jainu Jogani, the cofounder of Child’s Cure Genetic Research and father of a child with CDKL5 deficiency disorder, to learn more about the disease and current efforts to expand treatment options. Jogani provided some context for the cause and symptoms of CDKL5 and went over the details of his daughter’s experience with the disease. He also discussed current standard-of-care treatment for CDKL5, which is largely supportive, and emphasized the need for new treatment options.

Jogani then explained that his organization is working on an investigational adeno-associated virus vector-based treatment for CDKL5. He noted that the therapy has not yet entered clinical development but that a lot of the preclinical work has been completed. Jogani also touched upon a small-molecule treatment that is already commercially available and can provide some help to patients with CDKL5 deficiency disorder. He concluded by highlighting the sense of urgency that surrounds rare and serious disorders such as CDKL5 deficiency disorder and shared his view on the necessity of working to accelerate development timelines for relevant therapeutics.

Click here to view more coverage of the 2025 ASGCT Annual Meeting.