Experience with multiple modalities
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
Scientific and medical expertise
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:

试验方案开发

Long-term follow-up (LTFU)

Development of companion diagnostics

Global regulatory requirements

Vendor qualification

Medical monitoring
Global operational solutions
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
Customized training

To ensure your team keeps up with complex and rapidly evolving technologies, we provide comprehensive custom training of all team members. Our Rare Diseases, Advanced Therapies and Pediatrics Team (RAPT) and Oncology members provide targeted, study-specific training, as well as a formal Cell and Gene Therapy Training curriculum.