BOSTON--(BUSINESS WIRE)-- Ensoma, a gene therapy company advancing the future of medicine through precision in vivo engineering of blood and immune cells, today announced additions to the Company’s scientific advisory board (SAB) – Crystal Mackall, M.D., of the Stanford University School of Medicine, and Harry Malech, M.D., of the National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health (NIH). They join existing SAB members, Co-founder and Chairman Hans-Peter Kiem, M.D., Ph.D., of Fred Hutchinson Cancer Research Center (Fred Hutch); Anja Ehrhardt, Ph.D., of Universität Witten/Herdecke; Vijay Sankaran, M.D., Ph.D., of Boston Children’s Hospital; and Mark Walters, M.D., of University of California, San Francisco (UCSF).

“We are excited to have assembled such an accomplished and diverse group of academic, scientific and clinical experts to serve on our scientific advisory board,” said Robert Peters, Ph.D., chief scientific officer of Ensoma. “Our scientific advisors range from gene and cell therapy innovators to clinicians with experience across an array of disease areas, including rare genetic diseases, as well as immuno-oncology and autoimmune diseases, where engineering the immune system may serve as a meaningful approach. We look forward to leveraging this group’s expansive expertise as we accelerate the development of off-the-shelf therapies that will transform the gene and cell therapy landscape by enabling healthcare providers to treat patients anywhere, including in outpatient settings.”

Additional details surrounding members of Ensoma’s scientific advisory board include:

• Hans-Peter Kiem, M.D., Ph.D., is a physician/scientist, oncologist and world-renowned pioneer in gene therapy technologies, including stem cell and gene therapies, from Fred Hutch, who also serves as president-elect of the American Society of Gene & Cell Therapy (ASGCT). His research has focused on the biology and transplantation of hematopoietic stem cells (HSCs) and on the development of novel technologies to genetically modify and select HSCs in vivo for the treatment of infectious diseases such as HIV, genetic diseases and cancer.
• Anja Ehrhardt, Ph.D., is the chair of virology and microbiology at Universität Witten/Herdecke in Germany. Dr. Ehrhardt is an internationally recognized expert in adenoviral biology and vector construction, and her lab focuses on the production and characterization of adenoviral and gene-deleted “high-capacity adenoviral vectors” (HCAdV) for gene therapeutic treatment of inherited diseases, cancer and vaccinations. Her work includes utilizing transposons and designer nucleases within adenoviral vectors for achieving permanent treatment effects.
• Crystal Mackall, M.D., is the Ernest and Amelia Gallo Family Professor of Pediatrics and Medicine at Stanford University. She has led an internationally recognized basic and translational research program focused on immune-oncology. Her group has been at the leading edge of developing and testing CAR T-cell therapies, defining resistance pathways in this setting and developing next-generation cell therapies for cancer.
• Harry Malech, M.D., chief of the Genetic Immunotherapy Section in the NIAID Laboratory of Clinical Immunology and Microbiology, is a globally recognized expert in primary immune deficiencies and gene therapy and a past president of ASGCT. At NIAID, Dr. Malech leads research and clinical development of gene therapy and HSC transplantation approaches. His lab also continues to elucidate the biology of engraftment of HSCs and the characteristics of excessive inflammation and associated autoimmune symptoms in people with primary immunodeficiencies.
• Vijay Sankaran, M.D., Ph.D., is the Lodish Family Chair for the Division of Hematology/Oncology at Boston Children’s Hospital. He is a physician-scientist focused on caring for patients with non-malignant hematologic disorders, with a special interest in diseases of red blood cell production and hemoglobin disorders. Dr. Sankaran’s laboratory uses human genetics to understand blood cell production, hemoglobin gene regulation and how this goes awry in disease.
• Mark Walters, M.D., professor of pediatrics at UCSF School of Medicine, has devoted his research career to developing and expanding curative therapies for hemoglobin disorders and non-malignant hematopoietic disorders using genome editing approaches. He has a long track record of experience in cellular therapies for pediatric acquired and hereditary conditions and in bone marrow transplantation.

Additional information on the roles and affiliations of members of the scientific advisory board may be viewed here.

Dr. Kiem added, “Beyond its gene and cell therapy expertise, this group’s clinical know-how will aid the company in its transition from the discovery stage to the clinic, where we hope to translate, for the first time, the promise of this next-generation in vivo approach into therapies for patients with a range of diseases, spanning from rare disorders to oncology and immune-mediated diseases. Ensoma’s vectors will also allow us to build upon proven ex vivo therapies, such as CAR T-cells by taking them in vivo, which would greatly simplify manufacturing and production complexities.”

About Ensoma

Ensoma believes the future of medicine lies within us. Our Engenious™ gene therapy platform is the first to precisely engineer any or all hematopoietic and immune cells with a one-time, off-the-shelf in vivo treatment. Engenious therapies have an unmatched capacity to deliver all modern genetic modification tools and set a new standard for versatile cell programming and control across a range of complex diseases. Ensoma is supported by top-tier investors, a strategic collaboration with Takeda and a passionate team committed to a bold, global vision for gene therapy. Ensoma is based in Boston. For more information, visit www.ensoma.com.

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