September 29, 2023

Viralgen Vector Core and Elpida Therapeutics partner to manufacture gene therapy medicines for anticipated Spastic Paraplegia 50 (SPG50) and Charcot-Marie-Tooth disease type 4J (CMT4J) clinical trials

Trial participants with Spastic Paraplegia 50 (SPG50) and Charcot-Marie-Tooth disease type 4J (CMT4J) could benefit from this joint effort to manufacture new gene therapies that utilize adeno-associated virus serotype 9 (AAV9) to deliver corrected genes

Sept 19, San Diego, California. Viralgen Vector Core (Viralgen) and Elpida Therapeutics (Elpida Tx) have partnered to manufacture gene therapies for use in clinical trials sponsored by Elpida Tx involving patients living with SPG50 or CMT4J. It is anticipated that these trials will take place at various sites in North America and Europe and will explore potential safety and efficacy of the new treatments to be manufactured, as well as potential quality of life improvements.

SPG50 is a rare disease caused by mutations in the adaptor protein complex-4 mu 1 subunit (AP4M1), which is a protein responsible for intracellular vesicle trafficking, specifically the sorting and transport of membrane proteins within cells. The disease symptoms of children with SPG50 begin with low muscle tone, microcephaly (small head) and epilepsy, then progress to affect the entire body, resulting in intellectual disabilities and movement/neurological abnormalities. This rare disease is known to affect only around 90 people worldwide.1

Charcot-Marie-Tooth disease type 4J (CMT4J) is an autosomal recessive, potentially severe form of CMT disease caused by mutations of the lipid phosphatase FIG4, a protein involved in the regulation of phosphoinositide lipids and membrane trafficking within cells. The same CMT4J genotype can present symptoms ranging from mild clinical signs to severe disability.2

The partnership between Viralgen and Elpida Tx is expected to enable the efficient manufacture of the adeno-associated vector serotype 9 (AAV9) based therapies for use in Elpida Tx’s anticipated multi-site clinical trials. Viralgen will manufacture these therapies using its production process with the Pro10TM cell line, which is exclusively licensed from AskBio and has been found to increase scalability, performance, and yield of AAV therapies.3

Terry Pirovolakis, Founder and CEO of Elpida Tx, remarks that “Viralgen is truly an amazing partner, demonstrating their genuine commitment to advance potential therapies for impacted patients. We feel so grateful and honored to be working with such an incredible partner.”

About Elpida Therapeutics

Elpida Tx’ s mission aims to address the current significant unmet medical needs of patients with ultra-rare diseases. Through leveraging scientific advancements and the now well-established safety and understanding of certain gene therapies, Elpida Tx aims to put cures in reach of families and children who desperately and urgently need them. Elpida Tx’s business model focuses on partnerships that promote efficiency and the chance to treat a greater number of patients, while being self-sustaining and replicable. Elpida Tx specifically focuses on advancing programs that traditional biotech companies find difficult to bring through to completion. The company pipeline includes programs that were deprioritized by biotech companies or that did not receive biotech investment to advance due to small patient populations, but which have excellent scientific data on which to build a program and springboard the science and opportunity. Elpida Tx plans to incorporate three more CNS programs into its initial program pipeline within the next six months through an application process designed for academic institutions and foundations.

About Viralgen Vector Core

Viralgen is a Contract Development and Manufacturing Organization (CDMO) founded in 2017 and exists as an independently operated subsidiary of AskBio, which is wholly owned and independently operated as a subsidiary of Bayer AG. As a manufacturer of Current Good Manufacturing Practice (cGMP) certified AAV, Viralgen offers the Pro10™ based suspension manufacturing platform, a technology licensed from AskBio and developed by Chief Technical Officer Josh Grieger, PhD, and Co-Founder R. Jude Samulski, PhD, at University of North Carolina. The Pro10™ platform has been found to increase scalability, performance, and yield of AAV therapies 3. Located in Spain, in the Gipuzkoa Science and Technology Park, Viralgen produces AAV gene therapy treatments for pharmaceutical and biotech companies with the aim of accelerating the delivery of new treatments that may improve patients’ lives.

The company's clinical facilities have four cGMP manufacturing suites, with 250-liter and 500-liter bioreactors. In 2020, Viralgen expanded within the Scientific Park by constructing a new building with three modules for large-scale commercial manufacturing. Each module of the state-of-the-art space includes three cGMP suites with a manufacturing capacity of >2,000 liters. The first module, which includes a suite dedicated to fully automated fill and finish operations, has received cGMP certification by the Spanish Agency for Medicines and Medical Devices (AEMPS) as part of the EMA network. For more information, visit


1 J Clin Invest. 2023 May 15; 133(10): e170226. 10.1172/JCI170226

2 Brain. 2011 Jul; 134(7): 1959–1971. 10.1093/brain/awr148

3 Mol Ther. 2016 Feb;24(2):287-297. doi: 10.1038/mt.2015.187. Epub 2015 Oct 6.

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Priscilla Assumpção

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