Genezen Partners with Nevena Lešević and University of Missouri to Advance Personalized AAV9 Gene Therapy for Her Son with Ultra-Rare ARCA2

INDIANAPOLIS, Ind. and LAZAREVAC, Serbia, Sept. 29, 2026 /PRNewswire/ -- Genezen, a gene therapy contract development and manufacturing organization (CDMO), today announced a partnership with Nevena Lešević of Lazarevac, Serbia to support the manufacturing of an AAV serotype 9 gene therapy for an N-of-1 clinical trial to address her son Kosta's autosomal recessive spinocerebellar ataxia-2 (ARCA2) disorder caused by a PMPCA gene mutation.

At just 2 years old, Kosta was diagnosed with ARCA2 disorder in 2023. ARCA2 disorder is caused by a mutation in the gene PMPCA and is a rare condition that leads to symptoms such as unsteady walking and loss of balance, epilepsy and tremors, slurred speech and other developmental delays. According to medical literature, PMPCA-related cerebellar ataxia affects only approximately 25 patients worldwide. There is currently no established disease-modifying or curative therapy in routine clinical practice for PMPCA-related disease.

In early 2023, Kosta and his parents underwent full exome sequencing in Macedonia, which led to the discovery that both parents are carriers for two mutations. Later that year Kosta underwent whole genome sequencing, which confirmed the previous findings. He was then enrolled in a clinical study where he began to show signs of improvement, but since 2024 has been regressing in his condition. Nevena is now working closely with Professor Smita Saxena, PhD, and her team at the University of Missouri's NextGen Precision Health initiative to develop an AAV9 gene therapy for an N-of-1 clinical trial for Kosta. Dr. Saxena's work is focused on mechanisms of neuronal vulnerability in neurodegenerative diseases with a strong focus on spinocerebellar ataxias.

Single-patient (N-of-1) gene therapies offer a pragmatic pathway to deliver potentially life-saving treatments where no standard of care exists. However, conventional manufacturing models—built for scalability and population-based trials—are not designed for the speed, economics, and personalization required for N-of-1 investigational new drug (IND) applications, such as Kosta's. Through this collaboration, Genezen will apply its viral vector manufacturing expertise and comprehensive in-house analytical capabilities to support an accelerated AAV9 manufacturing and release timeline, building on its experience advancing N-of-1 therapies to the clinic.

Nevena and Kosta have demonstrated remarkable resilience throughout their ongoing journey to address his condition. They travel frequently to Barcelona, Spain, where Kosta receives specialized medical care and undergoes regular examinations and monitoring. Beyond the clinic, they are supported by a close-knit village, including Nevena's sisters and parents, whose encouragement and support have been an important part of their journey. Together, that support has helped sustain Nevena's determination to pursue every possible avenue to advance a potential treatment for her son.

"As a parent of a child with an ultra-rare disease, you quickly become an expert in ways and subjects you never imagined," said Nevena Lešević, Kosta's mother. "Over the last few years, I have taught myself to be a geneticist, scientist, and a drug developer. All in the name of helping Kosta become himself once again. I am so grateful to be working with Dr. Saxena, with her focus in this disease area, and to partner with Genezen to help us produce a clinical trial for Kosta as quickly as we can."

"Nevena and Kosta's journey to diagnosis and now development of a therapy for Kosta's condition underscores how we define resilience here at Genezen, and I am proud to support them with manufacturing a therapy for Kosta," said Steve Favaloro, Chairman and Chief Executive Officer at Genezen. "Genezen has deep experience supporting parent-led foundations to bring N-of-1 or small patient population therapies into the clinic and eventually into commercial. We are honored to be a part of this collaboration for Kosta and all parent-founded biotechs to help cure rare and ultra-rare diseases."

"Collaborations like this remind us why it is essential for the scientific community to come together," said Smita Saxena, PhD, who is also a faculty member with the University of Missouri's Center for Translational Neurogenetics. "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 will give Kosta back what he has lost to this disease."

The partnership highlights Genezen's mission to support parent-led foundations and develop efficient, scalable pathways for N-of-1 and ultra-rare disease programs, backed by proven end-to-end capabilities from concept through commercialization.

About Genezen
Genezen is a best-in-class gene therapy CDMO with over 12 years of experience and state-of-the-art viral vector manufacturing facilities. From concept to commercial scale, Genezen partners with innovators to deliver life-saving gene and cell therapies worldwide. Learn more at genezen.com or follow Genezen on LinkedIn.

About Kosta's Journey 
Kosta and his mother Nevena live in Lazarevac, Serbia. Kosta was diagnosed with an ultra-rare form of ARCA2 caused by mutations in the PMPCA gene following a COVID-19 infection and significant developmental regression in 2023, at just 2 years old. Since then, Nevena has worked tirelessly to pursue answers and potential treatment options for her son—including research collaborations in the US and on-going medical treatments and monitoring in Spain—with support from her sisters, parents and a dedicated community. Their efforts have led to the active development of a personalized AAV9 gene therapy clinical trial for Kosta. For more information and to support Kosta's journey, please visit https://gofund.me/4de78739f

About NextGen Precision Health 
At the University of Missouri, we are taking on society's biggest health care challenges and expanding our collaborative research efforts to bring precision health to patients, no matter where they live. From major urban centers to underserved rural communities, NextGen Precision Health investigators are dedicated to delivering the next generation of personalized health care solutions. To learn more, please visit precisionhealth.missouri.edu or medicine.missouri.edu/neurogenetics.

SOURCE Genezen