Viralgen Partners with Elaaj Bio to Advance Gene Therapy for Rare Pediatric Disorder CDKL5 Deficiency
Key Insights
Viralgen (search), a leading AAV gene therapy (search) CDMO, has partnered with Elaaj Bio (search) to advance ELJ-101 (search), an investigational gene therapy targeting CDKL5 Deficiency Disorder (search), a rare pediatric neurological condition.
The collaboration leverages Viralgen (search)'s AAV manufacturing expertise to support process development, scale-up manufacturing, and analytical characterization for the preclinical-stage program.
CDKL5 Deficiency Disorder (search) is a severe developmental and epileptic encephalopathy (search) characterized by early-onset seizures (search) and significant neurodevelopmental impairment (search) caused by CDKL5 gene (search) alterations.
Viralgen (search), a leading contract development and manufacturing organization specializing in recombinant adeno-associated virus (rAAV) gene therapies, has announced a strategic partnership with Elaaj Bio (search) to advance an investigational gene therapy program for CDKL5 Deficiency Disorder (search) (CDD). The collaboration brings together complementary expertise in AAV vector development and manufacturing to accelerate the ELJ-101 (search) program toward clinical development, with the first clinical trial planned for early 2027.
Addressing a Critical Unmet Medical Need
CDKL5 Deficiency Disorder (search) represents a rare and severe developmental and epileptic encephalopathy (search) caused by alterations in the CDKL5 gene (search). The condition is characterized by early-onset seizures (search) and significant neurodevelopmental impairment (search), creating a profound impact on affected individuals and their families. The investigational gene therapy program is being developed to target the underlying genetic cause of the disease, offering potential hope for patients with this devastating condition.
"Elaaj Bio (search) and the Loulou Foundation are mission-driven: everything we do is focused on finding treatments for CDD," said Russ Addis, Chief of Pipeline Strategy and Head of Genetic Medicine at the Loulou Foundation. "The Viralgen (search) team have embraced that mission; we have great hopes that this collaboration will result in an effective gene therapy that treats the underlying cause of CDKL5 (search) Deficiency."
Manufacturing Expertise and Technical Progress
Under the partnership agreement, Viralgen (search) will leverage its specialized AAV vector manufacturing capabilities to support critical development activities, including process development and optimization, manufacturing across multiple scales, and analytical characterization and quality control. The collaboration has already achieved significant technical milestones, with initial development work successfully completed, demonstrating the technical feasibility of the manufacturing approach.
Current activities are focused on process scale-up, including the preparation of larger-scale batches to support the planned clinical development timeline. Viralgen (search)'s state-of-the-art facility in San Sebastian, Spain, features three cGMP suites with 2,000-liter capacities each, certified by the AEMPS/EMA, and utilizes the company's proprietary Pro10™ suspension cell line and Aava™ manufacturing platform.
"Collaborations like this are essential to advancing complex gene therapy programs, particularly in rare pediatric diseases where the need for new therapies and innovations remains high," said Jimmy Vanhove, Chief Executive Officer at Viralgen (search). "By working with Elaaj Bio (search) and the Loulou Foundation, we're uniting complementary strengths in development and manufacturing to help accelerate ELJ-101 (search) toward clinical development."
Foundation-Driven Research Initiative
Elaaj Bio (search) operates as a wholly owned subsidiary of the Loulou Foundation, a private non-profit UK foundation dedicated to advancing research into the understanding and development of therapeutics for CDKL5 deficiency disorder (search). The Foundation has established an extensive research network, funding 31 separate projects across 41 laboratories at 30 different institutions, enabling focused research by over 120 scientists worldwide.
The Loulou Foundation also hosts the CDKL5 (search) Forum, an annual conference that has become the flagship meeting for CDKL5 deficiency disorder (search) basic and clinical science, further demonstrating the organization's commitment to advancing the field through collaborative research efforts.
Manufacturing Capabilities and Track Record
Viralgen (search) brings significant manufacturing expertise to the partnership, having produced over 1,500 AAV batches since its founding in 2017 as a subsidiary of AskBio, Inc. within the Bayer AG group. The company delivers end-to-end support from early development to large-scale commercial manufacturing, achieving industry-leading, high-yield, scalable manufacturing across all AAV serotypes.
The collaboration represents a strategic alignment of mission-driven research with proven manufacturing capabilities, positioning the ELJ-101 (search) program for advancement through clinical development as the teams work toward their early 2027 clinical trial initiation target.