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, Nov. 10, 2026 (GLOBE NEWSWIRE) — 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.

Frequently Asked Questions (FAQs):

Why is Child’s Cure exploring mRNA alongside gene therapy for CDKL5 deficiency disorder?

The approaches are intended to serve different roles. The AAV gene therapy is intended to address the mutated or deleted CDKL5 gene, while the mRNA program is intended to provide the CDKL5 protein that patients are missing. Child’s Cure is exploring mRNA as a potential complementary or alternative approach because gene therapy may not be suitable for every patient, and the long-term durability of a one-time gene therapy remains an important question. Repeatable mRNA dosing to restore CDKL5 protein production could provide another way to support patients over time, subject to further development and clinical evidence.

What experience does AGC Biologics have in working with rare disease therapeutics?

Amidst a complicated landscape for rare conditions with small patient populations, therapy development demands innovation and new ways of thinking. Organizations tackling some of the rarest of diseases are choosing AGC Biologics because of its ability to support unconventional development models:

  • AGC Biologics is a part of the Orphan Therapeutics Accelerator, a non-profit biotech focused on completing development and enabling commercial access to stalled rare disease treatments.
  • In 2025, AGC Biologics partnered with public benefit corporation, Rarity PBC, to provide end-to-end manufacturing services for Rarity’s gene therapy for ADA-SCID, an immunodeficiency disorder also known as “Bubble Baby Disease.”
  • When WASKYRA was approved in December 2025, Fondazione Telethon became the first non-profit organization to receive FDA approval of a gene therapy, with AGC Biologics as its chosen manufacturer.
  • AGC Biologics has produced multiple FDA-approved therapies already in commercial supply, with additional programs currently under review. This track record makes AGC Biologics an ideal partner for a broad range of organizations, from public benefit corporations to non-profits and institutions funded by public initiatives.

What experience does AGC Biologics have in mRNA development and manufacturing?

In 2023, AGC Biologics established its mRNA platform services in Heidelberg, Germany, where it already had a longstanding commercial plasmid DNA operation serving multiple customers. This services expansion also leveraged four decades of microbial manufacturing in addition to pDNA to develop its mRNA production capabilities.

Which AGC Biologics sites have mRNA services?

AGC Biologics offers mRNA development and manufacturing services in Europe and Asia at its Heidelberg mRNA Center of Excellence in Germany and Yokohama site in Japan.

About Child’s Cure Genetic Research

Child’s Cure Genetic Research is a parents-founded organization dedicated to developing innovative, precision treatment opportunities for rare genetic disorders in children. Driven by the urgent need to find cures, the organization envisions a future where every child has access to transformative treatments that offer hope for a better tomorrow. Through cutting-edge research and strategic collaborations, Child’s Cure is committed to reshaping the future of pediatric rare disease care. Through cutting-edge research, Child’s Cure is committed to reshaping the future of pediatric rare disease care—and actively welcomes strategic collaborations across other pediatric genetic conditions.

About AGC Biologics

AGC Biologics is a leading global biopharmaceutical Contract Development and Manufacturing Organization (CDMO) with a strong commitment to delivering the highest standard of service as we work side-by-side with our clients and partners, to provide friendly and expert services. We provide world-class development and manufacturing of mammalian and microbial-based therapeutic proteins, plasmid DNA (pDNA), messenger RNA (mRNA), viral vectors, and genetically engineered cells. Our global network spans the U.S., Europe, and Asia, with locations in Seattle, Washington; Copenhagen, Denmark; Heidelberg, Germany; Milan, Italy; and Chiba and Yokohama, Japan. AGC Biologics is a part of AGC Inc.’s Life Science Business. The Life Science Business runs eight facilities focused on biopharmaceuticals, advanced therapies, small molecule active pharmaceutical ingredients, and agrochemicals. To learn more, visit www.agcbio.com.

Photos accompanying this announcement are available at:

https://www.globenewswire.com/NewsRoom/AttachmentNg/81f0193f-0c10-4233-b072-ca4cc13654bb

https://www.globenewswire.com/NewsRoom/AttachmentNg/2fb9fb62-bb73-40c7-a305-09b857937127


AGC Inc. corporate contact: info-pr@agc.com 

AGC Biologics media contact: kati.sills@agc.com

Primary Logo

Media gallery

About The Author
AGC Biologics, Inc.