FDA Approves IND for Base-Edited NK Cell Therapy by Base Therapeutics

GenScript Biotech, a major player in global biotechnology, has announced a significant milestone in its collaboration with Base Therapeutics. The FDA has approved the investigational new drug (IND) application for Base Therapeutics' NK510 program, a groundbreaking NK cell therapy designed for treating advanced solid tumors. This approval is noteworthy as it marks the first instance of a base-edited NK cell program receiving IND approval from the FDA. Prior to this, the NK510 program had also secured clinical trial approval from China's National Medical Products Administration (NMPA) in October 2024.

Base Therapeutics, a company at the forefront of gene editing and cell-based therapies, is dedicated to addressing cancer and genetic diseases. The NK510 program utilizes advanced base editing technology, facilitated by GenScript's cGMP sgRNA, to modify NK cells. This process is further supported by GenScript’s CytoSinct™ Cell Isolation Nanobeads and CytoSinct™ 1000 instruments, which are pivotal for cell isolation. These technologies ensure that Base Therapeutics can maintain high standards of precision and reliability in their innovative therapeutic approaches.

Dr. Tianhong Xu, the founder of Base Therapeutics, expressed gratitude for the partnership with GenScript, highlighting the importance of their collaboration in successfully achieving regulatory approvals in both the US and China. This achievement underscores the advancements in base editing technology and reflects the companies' dedication to innovation, safety, and efficacy in gene therapy solutions.

Dr. Ray Chen, President of GenScript's Life Science Group, remarked on the successful collaboration, emphasizing GenScript’s role in providing essential materials and platforms for Base Therapeutics. This partnership highlights GenScript's capabilities in supporting gene and cell therapy initiatives, facilitating expedited regulatory approvals, and contributing to making innovative therapies more globally accessible.

The NK510 program is an allogeneic NK cell initiative developed by Base Therapeutics, featuring the proprietary AccuBase® technology. This zero off-target base editor is designed to precisely modify key genes in NK cells, achieving over 90% editing efficiency. Such precision is crucial in developing effective and safe cell therapies.

GenScript's expertise in nucleic acid synthesis spans over two decades, offering a wide range of gene editing materials from research-use-only (RUO) to cGMP grade. Their portfolio includes chemically synthesized guide RNA (gRNA) and templates for HDR knock-in. GenScript is capable of producing cGMP gRNA at significant scales, supporting various editing technologies like Cas9, Cas12a, base editing, and prime editing. This capacity is vital for preparing submission materials for regulatory bodies in the US, EU, and APAC, providing a comprehensive support system for clients seeking regulatory approvals.

As of mid-2024, GenScript has successfully delivered over 120 batches of cGMP gRNA and HDR templates, supported over 40 regulatory submissions, and facilitated 13 IND approvals globally. This track record solidifies GenScript's position as a reliable partner in accelerating the development of cell therapies.

GenScript's CytoSinct™ platform, utilizing advanced nanobead-based cell isolation technology, further enhances their offerings. This platform ensures precise isolation of specific cell populations, with automation capabilities designed to meet FDA regulations. The CytoSinct™ technology has completed FDA DMF filing, demonstrating its preparedness for clinical applications.

Base Therapeutics, founded in 2021, is committed to pioneering in the field of gene editing and cell therapies. The company boasts extensive GMP-compliant manufacturing and laboratory facilities, meeting stringent regulatory standards in the US and China. With substantial funding from top-tier investors and a portfolio of over 30 global patents, Base Therapeutics is positioned as a leader in innovative therapy development, aiming to deliver cutting-edge treatments to patients worldwide.