AGC Biologics partners with Child’s Cure Genetic Research to pursue mRNA-based therapeutic for severe epileptic disorder

Demonstrates new evolution in mRNA to address rare conditions

CDMO's work with parent-led, patient-focused foundation will investigate a development pathway to combat CDKL5 Deficiency Disorder

AGC Biologics is well positioned to help advance mRNA programs toward the clinic

Heidelberg, GERMANY, November 10, 2026 - In a world where patients with CDKL5 deficiencies can suffer 40 to 60 epileptic seizures a day, a contract biopharmaceutical manufacturer and a parent-founded foundation will partner on early-stage messenger RNA development to find a long-term treatment for the rare neurodevelopmental disease.

With few RNA-based programs in development for rare diseases, Child's Cure Genetic Research and AGC Biologics will collaborate to identify a viable path for a new mRNA therapeutic that mimics the protein made by the CDKL5 gene. The intent of the protein-sustaining treatment is to reduce seizures, improve neurological development, and help patients-many of whom are babies and young children-gain control from debilitating symptoms that impede their health and potential.

This early proof-of-concept work within AGC Biologics' integrated mRNA solutions will seek to identify the best pathway toward clinical development.

New rare disease frontier for mRNA

Cyclin-dependent kinase-like 5 deficiency disorder is a rare genetic disease caused by mutations or deletions of the CDKL5 gene. Current treatment for the disorder often only consists of supportive care, though the foundation is also exploring a small molecule drug that is presently in clinical trials.

For young patients like Reyna Jogani, advancement is not happening at the speed she needs to get the critical CDKL5 protein that enables routine brain activity related to movement, learning, vision, and behavior. Reyna's father, and Child's Cure co-founder, Jainu Jogani, has embraced the rapid advancement and potential of mRNA development, and this partnership with AGC Biologics aims to uncover a sustaining treatment for the rare disease.

RNA-based therapeutic programs are also in development for rare neurodevelopmental diseases such as Rett and Angelman syndromes. Child's Cure is pursuing mRNA for CDKL5 deficiency disorder as part of an effort to help advance the application of this approach to rare diseases.

mRNA therapy will complement gene therapy

Through this program, Child's Cure will explore mRNA as a potential complement to a one-time adeno-associated virus gene therapy intended to address the mutated or deleted CDKL5 gene. The mRNA therapy development will explore how to provide ongoing protein replacement infusions and could offer another option for patients with differing needs, including those for whom gene therapy may not be suitable.

"What I've learned about CDKL5 deficiency disorder is that one treatment approach may not meet every patient's needs. Gene therapy may not be an option for every patient, and questions remain about how long a one-time treatment effect will last," said Jogani. "Investing further in a complementary approach through mRNA intends to restore the missing CDKL5 protein by offering a redosable, adjustable way to support ongoing protein replacement. We are leaving no stone unturned to explore what might help."

AGC Biologics' support for early-phase mRNA programs

In 2023, AGC Biologics leveraged 40 years of experience in microbial fermentation as well as expanded plasmid DNA capabilities to create its new messenger RNA service line at its Heidelberg, Germany site. The mRNA process development services provide custom approaches to meet each specific mRNA need and can produce mRNA supply from R&D to GMP grades.

"Similar to what we've established across decades in other modalities, we are invested in delivering early-stage clinical expertise to developers with mRNA candidates looking to translate their discovery into commercial success," said Christoph Winterhalter, Chief Business Officer of AGC Biologics. "This is an inflection point for this market, where it is progressing into new arenas including personalized medicines and gene therapy. Our experts are the best in the marketplace to support this growth and expansion."

In November 2025, AGC Biologics announced it would partner with Repair Biotechnologies to develop and manufacture a novel mRNA therapy designed to combat the build-up of atherosclerotic plaques, a major contributor to cardiovascular disease, the leading cause of human mortality.

In 2027, AGC Biologics will also offer mRNA process development and commercial production at its newest location in Yokohama, Japan.