Skip to content
Medical Health Aged Care

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

Viralgen Vector Core 4 mins read

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

SAN DIEGO, Sept. 28, 2023 (GLOBE NEWSWIRE) -- 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.

For more information and interviews, please contact:
Priscilla Assumpção

This information is provided by Reach, the non-regulatory press release distribution service of RNS, part of the London Stock Exchange. Terms and conditions relating to the use and distribution of this information may apply. For further information, please contact or visit

Primary Logo

More from this category

  • Medical Health Aged Care
  • 29/11/2023
  • 12:07
Hola Health

Leading health tech firm named among fastest-growing Australian start-ups

Hola Health, a leading local health tech start-up providing telehealth and online script fulfillment services to a growing number of Australians, has been named in the top 5 fastest-growing new companies in the country in a prestigious annual list. The Australian Financial Review’s Fast Starters list recognises Australia’s 100 fastest-growing start-ups and young companies.It shows Hola Health grew revenue from around $100,000 in 2020/21 to $3.8 million last financial year. It is on track to exceed $10 million in revenue by the end of this financial year. In naming Hola Health at number five on the list, the AFR hailed…

  • Contains:
  • Medical Health Aged Care
  • 29/11/2023
  • 07:34
Public Health Association of Australia / Australian and New Zealand Journal of Public Health

New research reveals how babies and toddlers are targeted with unhealthy foods

Wednesday 29 November 2023 Babies and toddlers can't read or buy food yet, but new research reveals that they are being targeted with aggressive marketing in the baby and toddler aisle at the supermarket. The research, published today in theAustralian and New Zealand Journal of Public Health,has prompted health experts to call for regulation of on-pack labelling and promotional techniques used on baby and toddler food. Authors analysed 230 product packs found in two major supermarkets to uncover the marketing techniques used by manufacturers of products for children aged from just six months to three years. They found that 9…

  • Medical Health Aged Care
  • 29/11/2023
  • 06:05
Royal Australian College of GPs

RACGP backs duty of care bill for climate change harms

Australia’s largest representative body for GPs has thrown its support behind a duty of care bill requiring the Federal Government to protect young people from climate change harms in decision-making. It comes in the lead up to the first-ever Health Day at the COP28 UN Climate Conference, on Sunday 3 December, which will highlight the health impacts of climate change and the health case for climate action. Independent ACT Senator David Pocock introduced the bill which proposes government must consider the wellbeing of young people and future generations when making decisions that facilitate or fund projects that could significantly increase…

Media Outreach made fast, easy, simple.

Feature your press release on Medianet's News Hub every time your distribute with Medianet. Pay per release or save with a subscription.