SAN DIEGO, CA – February 4, 2025 – Navega Therapeutics, a pioneering biotechnology company developing cutting-edge epigenetic gene therapies, today announced a significant milestone with the receipt of a $4 million Translational Science grant from the California Institute for Regenerative Medicine (CIRM). This funding will support the continued development of NT-Z001, Navega’s lead therapeutic candidate designed to provide long-lasting relief from neuropathic pain. This award marks the second CIRM grant received by Navega, underscoring the agency’s confidence in the company’s innovative approach and potential to address the significant unmet need in chronic pain management.
Navega’s core technology centers around a proprietary AI-enabled platform that allows for the design and engineering of novel epigenetic editors. These editors offer a new level of precision in gene regulation, enabling the targeted modification of gene expression without altering the underlying DNA sequence. NT-Z001, the company’s lead candidate, exemplifies this approach by targeting the SCN9A gene, which encodes for the Nav1.7 sodium channel, a critical component in pain signal transmission. By precisely downregulating SCN9A expression, NT-Z001 aims to provide safe and effective pain relief for a range of chronic pain conditions, including primary erythromelalgia and small fiber neuropathy.
“We are incredibly grateful for this second grant from CIRM,” said Ana Moreno, PhD, Chief Executive Officer of Navega Therapeutics. “This funding will be instrumental in accelerating the preclinical development of NT-Z001, enabling us to conduct the necessary studies to support an Investigational New Drug (IND) application and ultimately bring this much-needed therapy to patients.”
A Non-Opioid Solution for a Growing Crisis
The prevalence of chronic pain is staggering, affecting an estimated 1.5 billion people worldwide, including over 100 million individuals in the United States alone. Current treatment options for chronic pain are limited, with opioids often being the mainstay of therapy for severe cases. However, the use of opioids carries significant risks, including addiction, overdose, and other adverse effects. The opioid epidemic has tragically impacted countless lives and communities, highlighting the urgent need for safer and more effective alternatives for pain management.
Navega’s epigenetic gene therapy approach offers a potential breakthrough in addressing this crisis. By precisely targeting the genetic mechanisms underlying pain, NT-Z001 has the potential to provide long-lasting pain relief without the risks associated with opioid use. This innovative therapy could significantly improve the quality of life for millions of patients suffering from chronic pain.
About Navega Therapeutics
Navega Therapeutics is a preclinical-stage biotechnology company dedicated to developing transformative epigenetic gene therapies for a wide range of diseases. The company’s proprietary AI-enabled platform allows for the design and engineering of novel epigenetic editors, providing a new level of precision in gene regulation. Navega’s lead program, NT-Z001, targets the SCN9A gene for the treatment of chronic pain. The company’s pipeline also includes programs for neurological and inflammatory disorders, demonstrating the broad potential of its epigenetic platform.
About the California Institute for Regenerative Medicine (CIRM)
The California Institute for Regenerative Medicine is a world-leading organization dedicated to accelerating the development of transformative regenerative medicine treatments. CIRM’s mission is to fund and support innovative research and clinical trials to bring these life-changing therapies to patients with unmet medical needs.
Looking Ahead
Navega Therapeutics is poised to make significant strides in the field of epigenetic gene therapy. With the support of CIRM, the company is well-positioned to advance NT-Z001 through preclinical development and towards clinical trials, offering hope for a new era of safer and more effective chronic pain management.
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