ElevateBio Announces the Formation of a New Company With George Daley, M.D., Ph.D., and Boston Children’s Hospital to Develop iPSC-Derived Allogeneic Immune Therapies

WALTHAM, Mass.–()–ElevateBio, LLC (ElevateBio), a technology-driven company focused on powering transformative cell and gene therapies, today announced that it has formed a new company co-founded by George Daley, M.D, Ph.D., and Boston Children’s Hospital to develop allogeneic immune cell therapies based on a novel platform that generates functionally mature immune cells from induced pluripotent stem cells (iPSCs). This proprietary differentiation process overcomes the tendency of iPSCs to generate immature, embryonic blood cell types, and enables the generation of multiple subtypes of immune cells that display mature molecular signatures similar to T cells from adult blood. The peer-reviewed publication in the journal Cell Stem Cell showed that iPSC-derived mature αβ T cells exhibited antitumor activity and cytokine secretion and could serve as an ideal source for the development of allogeneic “off-the-shelf” therapies.

This is the first company to emerge from the previously announced five-year collaboration between Boston Children’s Hospital and ElevateBio to accelerate the development of novel cell and gene therapies.

“At ElevateBio, we have been rapidly building fully integrated end-to-end cell and gene therapy enabling technologies and capabilities to enable the world’s most prolific scientific innovators and visionaries, such as Dr. Daley, to push the boundaries of the field and deliver powerful new therapeutic modalities for patients,” said David Hallal, Chairman and CEO of ElevateBio. “This exciting new company is the first from our collaboration with Boston Children’s Hospital and is a prime example of how our integrated ecosystem – our iPSC cell lines and process development capabilities, our gene editing technology, our CAR and TCR constructs, and our scale-up manufacturing capabilities – creates the perfect foundation needed to turn these scientific breakthroughs into transformational medicines.”

“CAR-T therapies have revolutionized the treatment of certain blood cancers, with complete responses in many patients undergoing this type of treatment. However, current treatment strategies involve manufacturing a customized CAR-T cell product for each individual patient, which is cumbersome and labor-intensive,” said Daley, senior author of the newly published Cell Stem Cell paper and whose lab is at Boston Children’s Hospital. “Our science offers an approach to circumvent these hurdles by offering an entirely new way of making allogeneic immune cell therapies that could pave the way for powerful treatments for a wide range of cancers.”

Key findings shared in Cell Stem Cell publication: “EZH1 repression generates mature iPSC-derived CAR T cells with enhanced antitumor activity” (1):

  • A novel differentiation process discovered in Dr. George Daley’s lab at Boston Children’s Hospital has been shown to promote definitive hematopoiesis and supports efficient production of mature T cells

    • This novel differentiation process incorporates repression of the histone methyltransferase EZH1 during iPSC differentiation to facilitate T cell maturation. The Daley lab showed previously that EZH1 is a negative regulator of lymphoid potential during embryonic blood development.
    • iPSC-T cells derived in a stroma-free, serum-free system following repression of EZH1, referred to as EZ-T cells, showed a molecular signature more closely resembling mature TCRαβ T cells found in adult blood. Single cell RNA sequencing showed that activated EZ-T cells give rise to high levels of memory T cells, which promotes T cell longevity and may be essential for durable remissions in cancer patients.
  • In vitro studies showed EZ-T cells engineered to express anti-CD19 Chimeric Antigen Receptors (CARs) exhibited cytotoxic and cytokine-producing effector functions against CD19+ lymphoid tumor cells comparable to CAR-T cells engineered from adult blood.
  • In a xenograft mouse model injected with CD19+ diffuse large B-cell lymphoma (DLBCL) cells, EZ-T cells expressing anti-CD19 CARs demonstrated increased anti-tumor activity versus traditional iPSC-T cells generated without EZH1 knockdown.

(1) Jing, R., Scarfo, I., Daley, G., EZH1 repression generates mature iPSC-derived CAR T cells with enhanced antitumor activity, Cell Stem Cell, 2022

About ElevateBio:

ElevateBio is a technology-driven company built to power the development of transformative cell and gene therapies today and for many decades to come. The company has assembled industry-leading talent, built state-of-the-art facilities, and integrated diverse technology platforms, including gene editing, induced pluripotent stem cells (iPSCs), and protein, vector, and cellular engineering, necessary to drive innovation and commercialization of cellular and genetic medicines. In addition, BaseCamp is a purpose-built facility offering process innovation, process sciences, and current Good Manufacturing Practice (cGMP) manufacturing capabilities. Through BaseCamp and its enabling technologies, ElevateBio is focused on growing its collaborations with industry partners while also developing its own portfolio of cellular and genetic medicines. ElevateBio’s team of scientists, drug developers, and company builders are redefining what it means to be a technology company in the world of drug development, blurring the line between technology and healthcare.

For more information, visit us at www.elevate.bio, or follow Elevate on LinkedIn , Twitter, or Instagram.

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