- Genezen has partnered with Nevena Lešević of Lazarevac, Serbia to support manufacturing of an AAV9 gene therapy for an N-of-1 clinical trial targeting her son Kosta’s autosomal recessive spinocerebellar ataxia-2 (ARCA2).
- Genezen will provide viral vector manufacturing and analytical capabilities to support the development and release of the investigational therapy.
Genezen, a gene therapy contract development and manufacturing organization (CDMO), has entered a partnership with Nevena Lešević to support manufacturing of an AAV serotype 9 (AAV9) gene therapy for an N-of-1 clinical trial for her son Kosta. Kosta has ARCA2, an ultra-rare genetic disorder caused by a mutation in the PMPCA gene.
ARCA2 can cause unsteady walking and loss of balance, epilepsy, tremors, slurred speech, and developmental delays. The press release states that PMPCA-related cerebellar ataxia affects approximately 25 patients worldwide and that there is currently no established disease-modifying or curative therapy in routine clinical practice for the disease. The investigational therapy is being developed with Professor Smita Saxena, Ph.D., and her team at the University of Missouri’s NextGen Precision Health initiative.
Under the partnership, Genezen will apply its viral vector manufacturing expertise and in-house analytical capabilities to support an accelerated AAV9 manufacturing and release timeline. The company said the approach builds on its experience supporting N-of-1 therapies and small patient population programs toward clinical development.
The program is intended to support an N-of-1 clinical trial for Kosta, who was diagnosed with ARCA2 in 2023. His whole genome sequencing later confirmed the genetic findings identified through earlier testing. The research team led by Saxena is focused on mechanisms of neuronal vulnerability in neurodegenerative diseases, with a focus on spinocerebellar ataxias.
“Collaborations like this remind us why it is essential for the scientific community to come together. By combining Nevena’s commitment with the scientific knowledge of our university, and working with an experienced manufacturer like Genezen, we are going to bring to life a therapy trial that might very well give Kosta back what he has lost to this disease.”
Smita Saxena, Ph.D., a faculty member with the University of Missouri’s Center for Translational Neurogenetics
