Cell and Gene Therapy

Cell and gene therapy trials demand specialized expertise, often in small patient populations and with complex operational and regulatory requirements. For decades, Emmes has helped government and industry sponsors meet those challenges, supporting 125+ CGT trials across all phases of development, including 77 in the past 10 years. We bring scientific expertise, established investigator relationships, integrated technology, and global trial experience are built to advance promising therapies.

Experience across government and industry

Our experience spans NIH-funded research and commercial development. Through our work with NHLBI and NCI on the BMT CTN, we have developed deep expertise in transplantation and cellular therapy research. We bring that scientific and operational knowledge to biopharma programs, supporting studies from first-in-human through pivotal Phase 3 trials and BLA submission.

Extensive site and investigator networks

Through the BMT CTN, we have access to 100+ transplant and cellular therapy centers across North America, including 19 core centers, 75+ affiliate centers, and the 50+ center Pediatric Transplantation & Cellular Therapy Consortium. We have worked directly with the investigators leading these centers, so your study starts with established relationships, trained staff, and familiar systems.

Global study experience

We have managed CGT studies well beyond US borders, including a Phase 3 trial across 16 countries (the first global Phase 3 collaboration in the BMT CTN) and a program that spanned 50+ sites in 10 countries from first-in-human through registration.

Technology built into the trial

The Advantage eClinical platform embeds data science and technology within the study to reduce costs and improve the patient experience. Global Trace safeguards biospecimens, and our Datavant joint venture enables a compliant, patient-centric approach to tracking patients lost to follow-up.

Statistical rigor and speed in small populations

Small patient populations require thoughtful trial design and rigorous analysis. When patient numbers are small and timelines are tight, our biostatisticians deliver. In one pivotal Phase 3 cell therapy trial, our biostatisticians used minimization randomization to support treatment-group balance and competing-risks methods to evaluate outcomes, delivered topline data within 10 days of the last patient's last visit, and supported the sponsor's response to an FDA request for additional data.