- AGC Biologics and Child’s Cure Genetic Research will collaborate on early-stage mRNA development to explore a potential treatment for CDKL5 deficiency disorder, a rare genetic disease that can cause frequent seizures and neurodevelopmental symptoms.
- The proof-of-concept program will investigate an mRNA therapeutic designed to mimic the protein produced by the CDKL5 gene, with the aim of identifying a viable pathway toward clinical development.
AGC Biologics, a contract biopharmaceutical manufacturer, has partnered with Child’s Cure Genetic Research to explore an mRNA-based treatment for CDKL5 deficiency disorder, a rare genetic disease caused by mutations or deletions of the CDKL5 gene. The early-stage collaboration will investigate a therapeutic designed to mimic the protein produced by the gene, with the aim of reducing seizures, supporting neurological development and addressing symptoms associated with the disorder.
The proof-of-concept work will be conducted through AGC Biologics’ integrated mRNA solutions and will seek to identify a viable path toward clinical development. The proposed approach is intended to restore the missing CDKL5 protein through ongoing protein replacement, potentially offering a redosable and adjustable treatment option. The partners say the work will explore mRNA therapy as a complement to a one-time adeno-associated virus (AAV) gene therapy approach, which may not be suitable for every patient.
CDKL5 deficiency disorder can cause severe neurological symptoms, including frequent epileptic seizures. The press release states that some patients experience 40 to 60 seizures per day. Current treatment often consists of supportive care, while Child’s Cure is also exploring a small-molecule drug that is in clinical trials. The foundation was co-founded by Jainu Jogani, whose daughter Reyna has the disorder.
AGC Biologics established its mRNA service line in 2023 at its Heidelberg, Germany, site, drawing on its experience in microbial fermentation and expanded plasmid DNA capabilities. Its mRNA process development services support customized approaches and supply from research and development through to GMP grades. The company also announced a partnership with Repair Biotechnologies in November 2025 to develop and manufacture an mRNA therapy targeting atherosclerotic plaque buildup.
The collaboration adds to AGC Biologics’ early-stage mRNA development activities as it prepares to offer mRNA process development and commercial production at its Yokohama, Japan, facility in 2027. The company said the partnership with Child’s Cure reflects its support for developers seeking to advance mRNA candidates from discovery toward clinical development.
