The University of North Carolina at Chapel Hill

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 genesSAN 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 CoreViralgen 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 viralgenvc.com. References:1 J Clin Invest. 2023 May 15; 133(10): e170226. 10.1172/JCI1702262 Brain. 2011 Jul; 134(7): 1959-1971. 10.1093/brain/awr1483 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 Assumpçãopriscilla@sg-branding.com 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 rns@lseg.com or visit www.rns.com.

WALTHAM, Mass.--(BUSINESS WIRE)-- X-Chem, a global leader in innovative drug discovery services, and the Structural Genomics Consortium (SGC), a public-private partnership dedicated to accelerating drug discovery through open science, announced that they have entered a collaboration aimed at creating new chemical tools to study human proteins. The goal is to generate DNA-encoded library (DEL) screening data for large numbers of under-addressed proteins in the human proteome — ultimately allowing drug discovery and machine learning (ML) experts from around the world to use the data to unlock disease targets of the future. Under the agreement, X-Chem will deploy its proprietary DEL platform to create datasets for proteins chosen and supplied by the SGC. These datasets, curated in an ML-ready format, will be posted to a public portal to be used for model building. These models will enable the development of tool compounds that will unlock future disease targets, a key goal of the SGC’s Target 2035 initiative. The early stages of the collaboration will be focused on screening and data generation, but X-Chem and the SGC expect the DEL-ML data portal will be ready for public access starting in early 2024. “X-Chem was the first to show that high-quality DEL datasets can support model building, and our successful collaboration with X-Chem on multiple unprecedented targets supports the scaling of their innovative approach,” said Aled Edwards, S.G., Ph.D., founder and CEO of the SGC. “We look forward to making our screening data publicly available so as to enable biological and computational researchers worldwide to collaborate openly with us to identify the disease targets of the future.” Matt Clark, Ph.D., CEO of X-Chem, said, “The SGC is dedicated to unlocking the human proteome through innovative science and open collaboration. We are thrilled that X-Chem will be a partner in their mission to understand all proteins in the human genome. Our DEL platform is the perfect technology for such high-throughput, high-quality data generation.” About X-Chem X-Chem is a leader in small molecule drug discovery services for pharmaceutical and biotech companies. As pioneers of DNA-encoded chemical library (DEL) technology, the company leverages its market-leading DEL platform to discover novel small molecule leads against challenging, high-value therapeutic targets. As experts in medicinal chemistry, X-Chem can take those leads and progress them to clinical candidates with unmatched speed. Throughout the process, X-Chem’s advanced proprietary artificial intelligence (AI) technology accelerates all steps in the process. X-Chem also provides libraries, reagents and informatic tools to allow DEL operators to get the most of their DEL platform. X-Chem empowers its partners to effectively build drug pipelines from target to clinical candidate, enhanced with AI. About the Structural Genomics Consortium The Structural Genomics Consortium is a global public-private partnership that seeks to accelerate drug discovery by fostering collaboration among a large network of scientists in academia and industry and making all research outputs openly available to the scientific community. The current SGC research sites are located at Goethe University in Frankfurt, Karolinska Institute, McGill University, University College, London, the University of North Carolina, Chapel Hill, and the University of Toronto. View source version on businesswire.com: Contacts Lindsey Langemeier SCORR Marketing 402-405-4269 lindsey@scorrmarketing.com Source: X-Chem View this news release online at:

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 SAN SEBASTIAN, SPAIN / ACCESSWIRE / September 28, 2023 / 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 viralgenvc.com. References: 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 priscilla@sg-branding.com 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 rns@lseg.com or visit . SOURCE: Viralgen Vector Core View source version on accesswire.com: