Vigene Biosciences, Inc.

Charles River will help bring NT-Z001 to the clinic by leveraging its off-the-shelf plasmid products, custom plasmid capabilities and adeno-associated virus (AAV) production. After charting multiple personalized medicine expansions in recent years, contract manufacturer Charles River Laboratories is back with another gene therapy pact—this time centered on an adeno-associated virus 9 (AAV9) candidate meant to serve as a non-opioid alternative for rare disease patients living with chronic pain. Charles River on Thursday announced a new accord with epigenetic gene therapy outfit Navega Therapeutics as part of the CDMO’s Cell and Gene Therapy Accelerator Program. The deal will grant Navega access to Charles River’s contract development and manufacturing capabilities and advisory services to produce the AAV gene therapy candidate NT-Z001 for phase 1 clinical trials, the companies said in a press release. Navega is positioning its gene therapy candidate to help treat chronic pain associated with rare diseases like small fiber neuropathy and primary erythromelalgia. The drug leverages Navega’s artificial intelligence-enabled zinc-finger gene-editing platform, the company explained. NT-Z001 specifically addresses a gain-of-function mutation in the Nav 1.7 gene, which is linked to inherited erythromelalgia, small fiber neuropathy and other chronic, debilitating pain disorders. Aside from pain tied to rare diseases, Navega figures its gene therapy could eventually address other pain indications, including neuropathic and inflammatory pain. Charles River, for its part, will help bring NT-Z001 to the clinic by leveraging its off-the-shelf plasmid products, custom plasmid capabilities and AAV production. Over the years, Charles River says it has significantly enhanced its cell and gene portfolio through a mix of acquisitions, capacity expansions and manufacturing platform launches, including eXpDNA for plasmids, nAAVigation for AAV and Lentivation for lentiviral vectors. Back in November 2022, the company debuted the expansion of its cell therapy manufacturing facility in Memphis, Tennessee. The plant reveal came just weeks after the CDMO opened a separate plasmid production facility in the U.K. Under the expansion, Charles River added nine processing suites to its 16 clean rooms at the Memphis site. The additions were designed to handle high-volume production—plus options for dual production lines—for late-stage clinical and commercial cell therapies, the company said at the time. Charles River also recently acquired a site in Rockville, Maryland, through its $350-million buyout of gene therapy CDMO Vigene Biosciences back in 2021. The site largely helps with the production of viral vectors, which have experienced supply constraints among advanced cell and gene therapies for several years. Elsewhere on the personalized medicines front, Charles River last year linked up with South Korea’s Rznomics to develop and crank out viral vectors for its partner’s experimental liver cancer gene therapy RZ-001. RZ-001 marks the first ribozyme-based RNA reprogramming approach to receive FDA authorization for evaluation in humans, the companies said last January.

Adenovirus production underpins preclinical trials targeting genes responsible for tumor growth ROCKVILLE, Md.--(BUSINESS WIRE)-- Charles River Laboratories International, Inc. (NYSE: CRL) and Curigin, a Korean biotechnology company developing innovative oncolytic ribonucleic acid interference (RNAi) gene therapies, today announced a collaboration for adenoviral vector production. The gene therapy developer will leverage Charles River's market-leading expertise in contract development and manufacturing organization (CDMO) solutions to support its preclinical and clinical trials. Curigin develops anticancer gene therapy products that utilize innovative genetically engineered viruses and novel RNAi technology to quickly and accurately block key disease-specific genetic signal pathways, effectively switching off genes responsible for tumor growth. These gene therapies serve an unmet clinical need and can be offered to patients who have not been treated with conventional cancer drugs. Developing a Treatment for Bladder Cancer Curigin’s lead candidate is CA102, a genetically engineered adenovirus for bladder cancer which, according to the World Cancer Research Fund, is the 10th most common type of cancer worldwide. Based on preclinical evaluation data for CA102, Curigin expects to submit an Investigational New Drug (IND) application to the Food and Drug Administration (FDA) within the year. Adenoviral Vector Manufacturing Services Charles River has standardized protocols for cell culture, transfection, and downstream purification, as well as a validated platform process with a proven track record. These high-yield, optimized methods increase speed to clinical manufacturing by reducing process development time and costs while ensuring the highest quality production. Expanding its comprehensive cell and gene therapy portfolio to span viral vector, plasmid DNA, and cell therapy production, through the acquisitions of Vigene Biosciences, Cobra Biologics, and Cognate BioServices in 2021, Charles River offers end-to-end support and supply chain simplification for gene therapy developers. Approved Quotes “This collaboration with Curigin will tap into our industry-leading gene therapy CDMO capabilities and we are thrilled that our expertise will help bring potentially curative therapies to patients.” - Kerstin Dolph, Corporate Senior Vice President, Biologics Solutions, Charles River “Developing innovative therapeutics is our mission and we are steadily working towards that goal. We are excited to work with Charles River in the manufacturing phase as we continue to race on the path to drug development for oncology patients.” - Jae-Gyun Jeong, President, Curigin About Charles River Charles River provides essential products and services to help pharmaceutical and biotechnology companies, government agencies and leading academic institutions around the globe accelerate their research and drug development efforts. Our dedicated employees are focused on providing clients with exactly what they need to improve and expedite the discovery, early-stage development and safe manufacture of new therapies for the patients who need them. To learn more about our unique portfolio and breadth of services, visit . About Curigin Curigin develops innovative therapies using novel bi-specific shRNA technology to simultaneously knockdown two different disease-causing pathways. Our lead pipelines target cancer pathways such as mTOR and STAT3 and deliver our bi-specific shRNA through an oncolytic virus genetically engineered to exclusively target and replicate in cancer cells. To learn more about Curigin, visit .

CDMO will perform HQ plasmid production in support of Phase I/II clinical trials for Infantile Neuroaxonal Dystrophy ALDERLEY PARK, England--(BUSINESS WIRE)-- Charles River Laboratories International, Inc. (NYSE: CRL) and the INADcure Foundation, a nonprofit organization whose mission is to support the development of treatments for Infantile Neuroaxonal Dystrophy (INAD) and other forms of PLA2G6-related neurodegeneration (PLAN), today announced a plasmid DNA manufacturing alliance. The collaboration will leverage Charles River’s market leading contract development and manufacturing organization (CDMO) expertise in High Quality (HQ) plasmid DNA production, to manufacture its leading candidate for Phase I/II clinical trials. In INAD patients, a mutation or genetic alteration in the PLA2G6 gene causes mitochondrial and axonal membrane defects that lead to neuronal damage with axonal swellings and accumulation of pre-synaptic spheroids. Leena and Anil Panwala founded the INADcure Foundation in 2016, following the diagnosis of their two-year-old daughter, Ariya. Upon discovering there was no dedicated organization or centralized effort driving INAD research forward, they launched the Foundation with the mission to accelerate research and drive drug discovery. After seven years of unwavering dedication and with the tremendous support of INAD-afflicted families worldwide, the Foundation is furthering the development of a gene therapy treatment specifically designed for children affected by this rare neurodegenerative disease. What is INAD? INAD, otherwise known as phospholipase associated neurodegeneration (PLAN), is a rare disease with no known treatment or cure. Like other neurodegenerative diseases, INAD is progressive, and symptoms include poor motor function, involuntary eye movements, seizures, hearing loss, and difficulty swallowing and breathing. INAD affects over 150 children worldwide with a life expectancy of 10 or less years. Plasmid DNA Manufacturing Services Charles River’s plasmid DNA production, specifically HQ plasmid, combines key features of Good Manufacturing Practice (GMP) manufacture with rapid turnaround times to accelerate the timeline to clinic. Plasmid DNA is a critical starting material for many cell and gene therapy therapeutics and demand continues to outstrip supply. In response to this, Charles River recently announced the opening of a state-of-the-art HQ plasmid manufacturing center of excellence to address these supply shortages and support the growing needs of the cell and gene therapy field. Charles River, with the acquisitions of Cobra Biologics, Vigene Biosciences, and Cognate BioServices, in 2021, has extended its comprehensive cell and gene therapy portfolio to include CDMO capabilities spanning plasmid DNA, viral vector, and cellular therapy production for clinical through to commercial supply. To learn more about plasmid DNA production, watch Andrew Frazer, PhD, Associate Director, Scientific Solutions, Gene Therapy CDMO Services’ on-demand webinar: Advancements in Manufacturing for Rapid Delivery of pDNA Starting Materials. Approved Quotes “The opportunity to work with INADcure is exactly why we do the work we do. Their work is incredibly important to patients with this ultra-rare disorder. We are excited for the INADcure team to achieve this next stage of treatment development.” - Kerstin Dolph, Corporate Senior Vice President, Biologics Solutions, Charles River “Our collaboration with Charles River brings us closer to accomplishing our goal of delivering breakthrough treatments for INAD. We are excited to begin HQ plasmid DNA manufacturing to further develop our treatment with their experienced team. Their invaluable expertise will undoubtedly contribute to the advancement of our gene therapy program.” - Leena Panwala, Founder & Executive Director at INADcure. About INADcure The INADcure Foundation is a 501(c)(3) nonprofit charity whose mission is to support the development of treatments, including a cure, for Infantile Neuroaxonal Dystrophy (INAD) and other forms of PLA2G6-related neurodegeneration (PLAN). Its work includes advancing our understanding of this devastating disease, educating the public about INAD and other forms of PLA2G6-related neurodegeneration, advocating for better access to treatment, and supporting the families and caregivers of children diagnosed with INAD worldwide. For more information, visit . About Charles River Charles River provides essential products and services to help pharmaceutical and biotechnology companies, government agencies and leading academic institutions around the globe accelerate their research and drug development efforts. Our dedicated employees are focused on providing clients with exactly what they need to improve and expedite the discovery, early-stage development and safe manufacture of new therapies for the patients who need them. To learn more about our unique portfolio and breadth of services, visit . View source version on businesswire.com: Contacts Charles River Investor Contact: Todd Spencer Corporate Vice President, Investor Relations 781.222.6455 todd.spencer@crl.com Charles River Media Contact: Amy Cianciaruso Corporate Vice President, Chief Communications Officer 781.222.6168 amy.cianciaruso@crl.com Source: Charles River Laboratories International, Inc. View this news release online at: