細胞療法・遺伝子治療薬

• CAR-T and AAV
• Other viral and non-viral delivery systems (e.g., adenovirus, LNP)
• Cell therapies (immune cell/stem cell, autologous/allogeneic, modified/non-modified)
• Gene editing (e.g., CRISPR, meganucleases)
• Advanced and complex therapeutics such as RNA-based therapies (e.g., ASOs, siRNAs), T-cell engagers and genetically modified organisms

Not only do we know how to work with these complex entities and recognize real or potential impacts on patient well-being, but we also understand the science behind how they work. We understand how these treatments are developed and manufactured, and how these complex characteristics impact the conduct of a clinical trial including:

• プロトコルの開発
• Long-term follow-up (LTFU)
• Development of companion diagnostics
• Global regulatory requirements
• Vendor qualification
• Medical monitoring

The complexity of these therapies can involve numerous operational challenges defined by the asset in development and reflected in the trial footprint. We offer best practices, solutions and technologies and operational flexibility to optimize the conduct of your clinical trial, including, but not limited to:

• Providing logistics support for autologous CAR-T cell therapies
• Navigating site selection, activation, and overall study setup
• Defining regulatory and GMO pathways timelines along with approvals
• Reducing the patient burden for LTFU studies
• Designing and implementing a program’s regulatory strategy, including early engagement (e.g., INTERACT, pre-IND), and when working with rare diseases and pediatrics