AGC Biologics Explores mRNA Therapy For Rare Brain Disorder

KUALA LUMPUR, Nov 11 (Bernama) -- AGC Biologics, a contract biopharmaceutical manufacturer, has partnered with Child’s Cure Genetic Research, a foundation set up by parents of children with CDKL5 deficiency disorder, to explore a messenger RNA (mRNA) therapy for the rare genetic brain disorder.

Patients with the disorder, many of them babies and young children, can suffer 40 to 60 epileptic seizures a day, and treatment often consists only of supportive care, the company said in a statement.

“Similar to what we have 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.

“This is an inflection point for this market, where it is progressing into new arenas including personalised medicines and gene therapy,” said AGC Biologics Chief Business Officer, Christoph Winterhalter.

The early-stage, proof-of-concept work will seek a path towards clinical development for an mRNA therapy that mimics the protein made by the CDKL5 gene, with the aim of reducing seizures and improving neurological development.

Child’s Cure is exploring the mRNA approach as a complement to a one-time gene therapy for the disorder, as it could provide ongoing protein replacement, including for patients for whom gene therapy may not be suitable.

“Gene therapy may not be an option for every patient, and questions remain about how long a one-time treatment effect will last,” said Child’s Cure co-founder Jainu Jogani, whose daughter Reyna has the disorder.

AGC Biologics launched an mRNA service line at its Heidelberg site in 2023, and plans to offer mRNA development and commercial production at its new site in Yokohama, Japan, from 2027.

-- BERNAMA