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Next-Gen Gene Therapy to Navigate Debilitating Pain Disorders
Developing a non-opioid treatment for chronic pain from rare diseases
Pursuing a different approach for the treatment of chronic pain associated with rare diseases such as primary erythromelalgia and small fiber neuropathy, Navega Therapeutics will leverage our off-the-shelf plasmid products, custom plasmid capabilities, and Good Manufacturing Practice (GMP)-grade adeno-associated virus (AAV) production to manufacture its AAV-based gene therapy.
As part of the Cell and Gene Therapy (CGT) Accelerator Program (CAP), Navega will benefit from access to our contract development and manufacturing organization (CDMO) capabilities and Scientific Advisory Services to produce NT-Z001 for Phase I clinical trials.
"The selection of Charles River as our CDMO resulted from the additional benefits of its CGT Accelerator Program, including manufacturing prioritization for Nav 1.7, introduction to key industry players, scientific advisory and consulting services, plus co-marketing efforts,” commented Ana Moreno, PhD, CEO, Navega Therapeutics, in a press release announcing the collaboration. “We are delighted to have been selected from a pool of talented applicants.”
Epigenetic Gene Therapies
Harnessing the precision of an AI-enabled zinc-finger epigenome regulation platform, the biotechnology company’s epigenetic gene therapy addresses a gain-of-function mutation in the Nav 1.7 gene, linked to inherited erythromelalgia, small fiber neuropathy plus other chronic and debilitating pain disorders.
With over 17 million people in the United States alone living with high-impact chronic pain, Navega’s non-opioid gene therapy may also be used in other intractable pain indications, not curable by other known means, such as neuropathic and inflammatory pain.
To help bring NT-Z001 to clinic, our CDMO team will undertake an AAV9 production program, encompassing a broad portfolio of plasmid DNA and viral vector products and services, including the nAAVigation® AAV production platform.
“The collaboration with Navega will tap into our premier gene therapy CDMO capabilities and robust AAV offerings,” added Kerstin Dolph, CSVP, Global Manufacturing. “We are thrilled that our expertise will help bring Navega’s gene therapy closer to patients suffering with chronic pain.”
For more information on our integrated plasmid DNA, viral vector, and cell therapy development and manufacturing platforms and services, please visit Cell and Gene Therapy CDMO Solutions.
2024 Cell & Gene Therapy Summit
The Company Pitches session showcased the “who, what, why, and how,” highlighting technologies, milestones, path to clinic, and patient outcomes – explore key takeaways from Ana Moreno, CEO, Navega Tx, or watch on-demand.
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