GenScript USA Inc., the world's leading life-science service provider, recently expanded its global current Good Manufacturing Practices (cGMP) single-guide RNA (sgRNA) manufacturing capabilities to meet the growing demand for cell and gene therapies. GenScript's 400,000 square-foot cGMP facility enables next-generation gene and cell therapy R&D programs to advance IND filings and clinical trials.

"We are pleased to partner with these prestigious biotechnology companies in advancing cell therapies for the betterment of those that are sick and in need," said Michael Lau Ph.D., head of GMP business development at GenScript. "Our goal is to provide, with the fastest turnaround time, high-quality reagents that conform to the regulatory requirements necessary to support regulatory filings, clinical trials, and commercialization of these very important drug therapies."

One example is Shanghai BRL Medicine, which was recently granted an investigational new drug (IND) application for its BRL-201 in relapsed/refractory B-cell non-Hodgkin's lymphoma (r/r B-NHL) by the China Center for Drug Evaluation, National Medical Products Administration (CDE, NMPA).

BRL-201 was developed using Quikin CART®, BRL's non-viral site-specific integration platform, which can produce CAR-T cell products using one-step, non-viral preparation. Advantages of this approach include: the CAR sequence is precisely inserted into a specific site, avoiding the risk of tumorigenesis caused by random insertion; CAR knock-in and PD1 knock-out can be completed in one step, allowing the continuous expression of CAR and releasing the inhibition of T cell function; and shortened preparation time using a simpler and more cost-effective process.

This is BRL Medicine's second IND clearance from the CDE in 2022, following just four months behind its first gene therapy product, BRL-101, for transfusion-dependent β-thalassemia.

In addition, using GenScript's cGMP sgRNA, Nanjing BioHeng Biotech Co. Ltd. (BioHeng) also received CDE approval in March 2022 for its universal UCAR-T drug, CTA101, which targets CD19 and CD22 for the indication of adult recurrent or refractory B-cell acute lymphoblastic leukemia (r/ r B-ALL). Earlier this year, another China-based cell therapy company also utilized GenScript's cGMP sgRNA capability and received IND clearance for an autologous hematopoietic stem-cell gene-editing therapy.

"We congratulate BRL Medicine and BioHeng on receiving IND approval from the CDE," said Ray Chen Ph.D., president of GenScript's Life Sciences Group. "We are encouraged by these partnerships and will continue our mission to offer world-class facilities and expertise, along with an unwavering focus on service quality. GenScript is committed to advancing non-viral gene and cell therapy development by providing cGMP materials that meet our clients' demanding timelines."