Cell and Gene Therapy

Emmes’ Cell and Gene Therapy Center

A dedicated center created to enhance Emmes’ legacy and depth in cell and gene therapy research.

We bring more than 45 years of clinical trial experience, including decades of cell and gene research in collaboration with the leading investigators in the field.

An Inflection Point

Innovations in cell and gene therapies are unlocking the research potential to test and create life-changing treatments for patients suffering from a wide range of conditions and diseases.

Cell and Gene Therapy

Why Emmes?

Proven Expertise

  • More than 125 research studies aimed at addressing questions using cell or gene therapies for various therapeutic areas including hematology/oncology, ophthalmology, cardiology, and pulmonology.
  • Full array of research services: clinical trial operations, study leadership, statistical services, and pharmacovigilance, along with regulatory, training, and educational support.
  • Depth and breadth of cell and gene therapy credentials and experience at all levels.

    Long History

    • Conducted cell and gene therapy research for biopharma and public sector clients for more than 35 years.
    • Established early leadership in the field of cord blood research, starting in 1994. Participated in the first multicenter study aimed at evaluating whether human leukocyte antigen mismatched, unrelated umbilical cord blood stem cell and progenitor cell units offered a clinically acceptable alternative to matched unrelated-donor allogeneic bone marrow for transplantation in patients with malignant and non-malignant disorders.
    • Completed the groundbreaking Age-Related Eye Disease Study (AREDS) , which began in 1990, and the subsequent AREDS 2 trial, resulting in today’s widely available vitamin supplements that can help delay progression of macular degeneration.
    • Studied the effect of graft-versus-host disease prophylaxis as part of a Phase 2 and 3 trials in recipients of unrelated donor bone marrow in 1995.
    • Designed and managed several first-in-human studies of various cell types for infusion into patients with cardiac heart failure, beginning in 2005.

    About Emmes


    Strong Relationships

    • Over 100 scientific manuscripts co-authored by Emmes staff that encompass cell and gene therapy research results and progress.
    • Viewed as a valuable partner and collaborator by influential members of the cell and gene scientific community.

    “We selected EMMES to provide these services because of their outstanding reputation and high quality of their work. Given the novelty of the study, it was critical to have the best organization evaluating and interpreting the data.”– Dr. Joanne Kurtzberg, M.D., senior author and director, Duke University Medical Center.

    Project: Umbilical Cord Blood and Motor Function Improvement in Children with Cerebral Palsy

    Emmes’ role: statistical and data coordinating center


    Complex Clinical Trials

    The Blood and Marrow Transplant Clinical Trials Network (BMT CTN)

    The BMT CTN is one of our longest-running, most collaborative and complex research projects. Funded by the National Heart, Lung, and Blood Institute (NHLBI) and the National Cancer Institute, and in collaboration with biopharma companies, other partners are the National Marrow Donor Program/Be the Match (NMDP) and the Medical College of Wisconsin (MCW). The mission of the BMT CTN is to evaluate promising therapeutic approaches in multi-institutional clinical trials to improve the outcomes of blood and marrow transplantation and other cellular therapies for patients facing life-threatening blood disorders. Over 50 clinical studies have enrolled 15,000 participants across 125 sites.

    Our role: The long-standing partnership between NMDP MCW and Emmes, launched in 2001, has served as the foundation for the overall coordination and administration of this program led by experts in the field of hematopoietic cell transplantation and cellular therapy. Responsibilities are shared across the three organizations: Emmes, managing study operations; NMDP, patient advocacy and contracting; and MCW, scientific leadership. The BMT CTN leverages processes and data from the Center for International Blood and Marrow Transplant Research (CIBMTR). The relationship with the CIBMTR has allowed the BMT CTN to effectively design and conduct these complex studies.

    Cell and Gene Therapy


    • The BMT CTN has addressed all aspects of cell transplantation: studying optimal donor sources, graft-versus-host disease prophylaxis or treatment, infection prophylaxis, mitigation of relapse, and quality of life. More recently the BMT CTN portfolio has expanded to include cellular vaccines, chimeric antigen receptor T-cells (CAR-T), and gene therapies for sickle cell disease.
    • The BMT CTN has been leveraged by multiple biopharma sponsors to efficiently run global studies using a consortium of sites and operational expertise.
    • The program’s success is reflected in our numerous practice-changing studies, such as comparing peripheral blood stem cells to bone marrow mononuclear cells and comparing haploidentical bone marrow transplantation to double umbilical cord blood transplantation. More recently, we completed a multi-center Phase 3 trial assessing two different acute-graft-versus-host disease prophylaxis regiments.
    • The onset of the pandemic prompted the need to respond even more rapidly to new requirements. We launched a study to determine how immunocompromised patients respond to COVID-19 vaccines – in only one month. The 2021 study included patients who had hematological malignancies and were undergoing cell therapies, such as blood or marrow transplantation or CAR-T infusions.

    Cell and Gene Therapy Leaders

    At Emmes, we value the open exchange of communication between our therapeutic area leaders and your team. We encourage you to contact us to discuss your research plan and determine how we can support your development pathway.

    Adam Mendizabal, Ph.D.

    Adam Mendizabal, Ph.D.

    Vice President, Transplant, Allergy, Autoimmune