Heidelberg:AGC Biologics, a contract biopharmaceutical manufacturer, has announced a partnership with Child's Cure Genetic Research. This collaboration aims to explore a messenger RNA (mRNA) therapy for CDKL5 deficiency disorder, a rare genetic brain condition that significantly impacts patients, many of whom are infants and young children. These patients can experience 40 to 60 epileptic seizures daily, with treatments often limited to supportive care.

According to BERNAMA News Agency, AGC Biologics is committed to delivering early-stage clinical expertise to developers with mRNA candidates, aiding in the transition from discovery to commercial success. Christoph Winterhalter, AGC Biologics Chief Business Officer, emphasized the significance of this development as an inflection point for the market, which is progressing into new areas such as personalized medicines and gene therapy.

The partnership's initial focus will be on proof-of-concept work, aiming to progress towards clinical development of an mRNA therapy that replicates the protein produced by the CDKL5 gene. This approach seeks to decrease seizures and enhance neurological development. Child's Cure views the mRNA strategy as a complement to one-time gene therapy, offering ongoing protein replacement for patients for whom gene therapy may not be suitable.

Jainu Jogani, co-founder of Child's Cure and parent of a child with the disorder, highlighted the uncertainty regarding the duration of the effects of one-time gene treatments and the necessity for alternative options. AGC Biologics has already launched an mRNA service line at its Heidelberg facility and plans to extend mRNA development and commercial production to a new site in Yokohama, Japan, by 2027.