Charles River and Navega Therapeutics Announce Comprehensive Gene Therapy Manufacturing Collaboration

Rhea-AI Summary

Charles River Laboratories and Navega Therapeutics announce an AAV9 production program agreement to develop a non-opioid gene therapy, NT-Z001, for chronic pain disorders. Navega's AI-enabled zinc-finger epigenome regulation platform targets rare diseases like small fiber neuropathy and primary erythromelalgia, addressing a gain-of-function mutation in the Nav 1.7 gene. With over 17 million Americans suffering from chronic pain, this therapy shows promise for various pain indications. Charles River's CGT Accelerator Program supports Navega's efforts by providing CDMO solutions and advisory services to expedite clinical trials.

Positive

• The collaboration between Charles River Laboratories and Navega Therapeutics aims to develop an AAV9-based gene therapy, NT-Z001, for chronic pain disorders.
• Navega's approach focuses on treating rare diseases like small fiber neuropathy and primary erythromelalgia by targeting the Nav 1.7 gene mutation.
• This gene therapy has the potential to address chronic pain in over 17 million Americans and could be beneficial for other pain indications.
• Charles River's CGT Accelerator Program offers CDMO solutions and advisory services to support Navega in bringing NT-Z001 to Phase I clinical trials.
• The partnership aims to simplify supply chains and enhance the global demand for plasmid DNA, viral vector, and cell therapy services.
• Navega will leverage Charles River's expertise in gene therapy manufacturing to advance NT-Z001 towards patients suffering from chronic pain.

Negative

• None.

Biotechnology Analyst

The agreement between Charles River Laboratories International and Navega Therapeutics marks a significant step in the advancement of gene therapies targeting chronic pain. Navega's utilization of Charles River's established CDMO capabilities could accelerate the development of NT-Z001, their lead gene therapy candidate. This collaboration is poised to address the unmet need for non-opioid treatments in the chronic pain market, which affects a substantial patient population in the United States.

The focus on an AAV-based gene therapy to regulate the Nav 1.7 gene implicated in pain disorders represents a novel approach within the biotechnology sector. The potential impact on the market could be considerable if clinical trials demonstrate efficacy and safety, especially given the current reliance on opioid treatments and the associated risks of addiction and overdose.

Investors should monitor the progress of NT-Z001 through clinical trials, as positive results could lead to significant shifts in the treatment paradigm for chronic pain and potentially provide a competitive advantage for Navega in a high-demand market.

Healthcare Industry Analyst

Charles River's strategic investments in CDMO solutions, including plasmid and viral vector services, position the company as a key player in the cell and gene therapy supply chain. Their comprehensive 'concept to cure' approach, which includes the CGT Accelerator Program, could streamline the path to market for emerging therapies like NT-Z001.

For stakeholders, the implications of such collaborations extend beyond the immediate partnership. They reflect a broader industry trend towards integrated services that can reduce development times and costs for biotech firms. Charles River's capabilities in expediting the supply chain process with platforms such as eXpDNA™, nAAVigation™ and Lentivation™ may attract further partnerships, potentially enhancing the company's market share and revenue growth.

Long-term, the success of such partnerships could influence the stock performance of companies like Charles River, contingent upon the clinical success of the therapies they help bring to market. As such, investors may view these collaborations as indicators of potential future growth, particularly in the burgeoning field of gene therapy.

Pharmaceutical Manufacturing Expert

The technical aspect of producing an AAV-based gene therapy is complex, involving stringent GMP-grade production standards. Charles River's production capabilities and advisory services are critical in this context, providing Navega with the necessary infrastructure to manufacture NT-Z001 at the quality required for Phase I clinical trials.

AAV9 is a vector of choice for delivering genetic material due to its lower immunogenicity and ability to target various tissues. The partnership's focus on leveraging AAV9 for NT-Z001 production could set a precedent for future gene therapies, potentially impacting the standard practices within the pharmaceutical manufacturing industry.

Furthermore, the ability to simplify supply chains while maintaining quality is a significant advantage in the competitive pharmaceutical manufacturing landscape. Charles River's approach could become a benchmark for the industry, influencing how other companies structure their manufacturing and development processes to optimize efficiency without compromising on quality.

Next-generation AAV gene therapy to target debilitating pain disorders

WILMINGTON, Mass.--(BUSINESS WIRE)-- Charles River Laboratories International, Inc. (NYSE: CRL) and Navega Therapeutics, Inc., a biotechnology company developing epigenetic gene therapies, today announced an AAV9 production program agreement. As part of Charles River’s Cell and Gene Therapy (CGT) Accelerator Program (CAP), Navega will have access to established contract development and manufacturing (CDMO) capabilities and advisory services to produce an adeno-associated virus (AAV)-based gene therapy, NT-Z001, for Phase I clinical trials.

Developing a Non-Opioid Treatment for Chronic Pain

Navega is pursuing an approach for treatment of chronic pain associated with rare diseases such as small fiber neuropathy and primary erythromelalgia by harnessing the precision of its AI-enabled zinc-finger epigenome regulation platform. Navega’s epigenetic therapy addresses a gain-of-function mutation in the Nav 1.7 gene, linked to inherited erythromelalgia, small fiber neuropathy and other chronic, debilitating pain disorders. With over 17 million Americans living with high-impact chronic pain, Navega’s non-opioid gene therapy for chronic pain may also be used in other intractable pain indications, including neuropathic and inflammatory pain.

Plasmid and Viral Vector CDMO Solutions

To bring NT-Z001 to clinic, Navega will leverage Charles River’s off-the-shelf plasmid products, custom plasmid capabilities, and Good Manufacturing Practice (GMP)-grade AAV production.

Simplifying complex supply chains, without compromising on quality, Charles River provides phase-appropriate solutions to meet the growing global demand for plasmid DNA, viral vector, and cell therapy services. Through several acquisitions, capacity expansions, and the establishment of manufacturing platforms including eXpDNA^™ (plasmid), nAAVigation^™ (AAV), and Lentivation^™ (lentiviral vector) to expedite and standardize supply, Charles River’s CGT portfolio has been significantly enhanced. Combined with the company’s legacy testing services, Charles River offers an industry-leading “concept to cure” solution for advanced therapies, helping developers to navigate the path to clinic and beyond.

New for the 2024 Charles River Cell & Gene Therapy Summit, the Company Pitches session showcases the “who, what, why, and how,” highlighting technologies, milestones, path to clinic, and patient outcomes. Join Ana Moreno, CEO, Navega Therapeutics on March 19 in San Francisco – see the full agenda and save your seat: https://bit.ly/3SW3VOV

Approved Quotes

• “The collaboration with Navega will tap into our premier gene therapy CDMO capabilities and robust AAV offerings. We are thrilled that our expertise will help bring Navega’s gene therapy closer to patients suffering with chronic pain.” - Kerstin Dolph, Corporate Senior Vice President, Biologics Solutions, Charles River
• "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. We are delighted to have been selected from a pool of talented applicants.” - Ana Moreno, PhD, Chief Executive Officer, Navega Therapeutics, Inc.

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 www.criver.com.

About Navega Therapeutics Inc.

Navega is a preclinical stage company developing epigenetic-regulation gene therapies to tackle common and complex diseases via a proprietary AI-enabled zinc finger platform. Due to the involvement of NaV1.7 in multiple chronic pain states, Navega is harnessing the power of the epigenome to modify its expression and treat multiple chronic pain indications, including chemotherapy-induced peripheral neuropathy. Other indications in Navega’s pipeline include neurological and ophthalmic diseases.

To learn more visit https://navegatx.com/.

View source version on businesswire.com: https://www.businesswire.com/news/home/20240314552741/en/

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.

FAQ

What is the gene therapy being developed for chronic pain disorders by Charles River Laboratories and Navega Therapeutics?

The gene therapy being developed is NT-Z001, targeting rare diseases like small fiber neuropathy and primary erythromelalgia.

What is the approach taken by Navega Therapeutics to address chronic pain?

Navega is utilizing an AI-enabled zinc-finger epigenome regulation platform to target the Nav 1.7 gene mutation associated with chronic pain disorders.

How many Americans are estimated to be living with high-impact chronic pain?

Over 17 million Americans are living with high-impact chronic pain, making the development of non-opioid gene therapy crucial.

What solutions does Charles River Laboratories provide to support the development of NT-Z001?

Charles River offers CDMO capabilities, including plasmid products, custom plasmid capabilities, and GMP-grade AAV production.

What program within Charles River is assisting Navega in the development of the gene therapy?

Navega is part of Charles River's Cell and Gene Therapy Accelerator Program (CAP), which provides advisory services and manufacturing solutions.

Who is the CEO of Navega Therapeutics, and what benefits did they mention in partnering with Charles River?

The CEO is Ana Moreno, who highlighted benefits like manufacturing prioritization, industry introductions, advisory services, and co-marketing efforts from the collaboration with Charles River.