#Spain #San Sebastián #Viralgen #CDKL5 Deficiency #Elaaj Bio

Viralgen and Elaaj Bio Initiate Gene Therapy for CDKL5 Deficiency

In an impactful partnership, Viralgen and Elaaj Bio have come together to tackle the rare pediatric neurological disorder known as CDKL5 deficiency disorder (CDD). This collaboration seeks to leverage Viralgen’s expertise in adeno-associated virus (AAV) vector manufacturing to enhance the development of an experimental gene therapy. This initiative marks a significant milestone as it aims to address a condition characterized by early onset seizures and profound developmental disabilities.

Background of CDKL5 Deficiency Disorder

CDKL5 deficiency is a severe form of encephalopathy that arises due to mutations in the CDKL5 gene. The disorder primarily affects young children and results in a range of debilitating symptoms, including refractory seizures and impaired cognitive development. Families grappling with CDD often face daunting challenges, making the need for innovative treatment options more urgent than ever.

The Collaboration between Viralgen and Elaaj Bio

Announced on May 5, 2026, this partnership is seen as a crucial step forward in advancing gene therapy to address the underlying genetic cause of CDD. Elaaj Bio, a subsidiary of the Loulou Foundation—a nonprofit dedicated to advancing research for CDD—brings a focused mission to this collaboration. By combining their development and manufacturing capabilities, the two organizations aim to accelerate the transition from laboratory research to clinical application.

As part of this collaboration, Viralgen will utilize its advanced AAV manufacturing capabilities to support key development activities. This includes process development and optimization, as well as analytical characterization and quality control measures. Initial development work has already demonstrated the technical feasibility of their manufacturing methods, and they are now focused on scaling up these processes to prepare for larger batch production.

The Positive Outlook for Gene Therapy

Jimmy Vanhove, CEO of Viralgen, emphasized the importance of partnerships in advancing complex gene therapy programs, particularly for rare pediatric diseases. He stated, "Collaborations like this are essential for the progress of complex gene therapy programs, especially in rare pediatric diseases, where the demand for new therapies and innovations remains high. We are combining our strengths to accelerate the clinical development of ELJ-101."

Russ Addis, Head of Drug Development Strategy at Elaaj Bio, reaffirmed their unwavering commitment to finding effective treatments for CDD, stating, "The team at Viralgen has embraced our mission; we are hopeful this partnership will lead to an effective gene therapy that addresses the root cause of CDKL5 deficiency."

The teams are planning to initiate the first clinical trial of the program, termed ELJ-101, by early 2027. Current efforts are centered on scaling their manufacturing processes to facilitate future clinical development.

About Viralgen and Elaaj Bio

Viralgen, founded in 2017 as a subsidiary of AskBio, Inc., operates exclusively in the realm of gene therapy manufacturing, specializing in AAV-based therapies. With its state-of-the-art facility located in San Sebastián, Spain, Viralgen supports clients from initial development phases to large-scale commercial manufacturing. Their Pro10™ cell line and Aava™ manufacturing platform facilitate scalable production across all AAV serotypes.

Elaaj Bio, as part of the Loulou Foundation, plays a pivotal role in funding and advancing research specifically aimed at understanding and treating the challenges posed by CDKL5 deficiency. The foundation has been instrumental in financing research projects across universities and institutes, thereby driving forward the quest for innovative treatments.

For more information on their progress and initiatives, interested parties can visit their respective websites and follow them on social media platforms.