BPI Theater Interview: Ramin Baghirzade, Senior Director of Gene Therapy, Charles River Laboratories

Baghirzade began by discussing Charles River Laboratories’ (CRL’s) role in the viral-vector space and the concept-to-cure portfolio envisioned by the company. “We provide support from early discovery all the way to commercialization, and then we work side by side with our partners throughout that entire drug-development journey.”

CRL has launched three complementary viral-vector platforms. The first two, the nAAVigation and Lentivation platforms, were launched based on client feedback and the needs of the market. They were designed to support early stage drug developers that were still 18 to 24 months away from their first human studies. Baghirzade said that the industry has matured recently, with more products making it to market. To meet increased needs for technology transfers into contract development and manufacturing organizations (CDMOs), in May 2024, CRL announced its Modular and Fast Track viral-vector technology-transfer frameworks.

CRL had a hand in the success of Casgevy (exagamglogene autotemcel), a cell-based gene therapy used to treat sickle-cell disease. Baghirzade cautioned that it’s important to ask questions to try to determine why some drugs succeed where others fail, even when the science is sound. He cited manufacturing challenges as one major cause, adding that making it to market doesn’t automatically lead to success. “If other elements are not there, including things like reimbursement, market access, robust manufacturing processes, and chemistry, manufacturing, and control (CMC) elements, you still might fail.” He said that CRL strives to standardize manufacturing practices to make them more reproducible, which leads to improved accessibility. He emphasized that patient access is close to his heart and that CRL has a long history of working with patient advocacy groups to make treatments more readily available.

In the gene-therapy space, CRL has two centers of excellence. The viral-vector center is based in Rockville, MD. It has been open for nearly two decades, producing adenoassociated viruses (AAVs), lentiviral vectors (LVVs), and retroviral vectors (RVVs), among other molecules. The company also has centers for plasmid DNA (pDNA) production based in Alderley Park and Keele, UK. Those two facilities are important because plasmids are used as a starting material for viral-vector manufacturing. One facility is a fully good manufacturing practice (GMP) facility for plasmid production and the other specializes in high-quality “GMP-like” production, and the other. The gene-therapy facilities are powered by CRL’s global facility network of testing capabilities. All analytics are performed in house.

Baghirzade explained that as a global company, CRL continues to explore expansion through acquisitions and partnerships. “We have quite a few partners that we haven’t fully acquired, but in which we have a minority stake. We also continue expanding internally.”

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