National Brain Tumor Society (NBTS) today announced a research grant to KiraGen Bio, a Boston-based biotechnology company developing gene-edited cell therapies for glioblastoma (GBM), the most common and aggressive malignant brain tumor in adults.
The grant supports a study in patient-derived glioblastoma models — tumor tissue taken directly from patients and grown in the brains of laboratory animals — that will test whether KiraGen’s engineered immune cells, called CAR-T cells, can achieve sustained control of tumor growth. Successful results from this testing is intended to help the company move its lead candidate, KGEN-001, into the clinic.
Recent early-phase clinical trials have established that CAR-T therapies, which reprogram immune cells to recognize and attack cancerous cells, can be efficiently generated, delivered to glioblastoma tumors, and have shown tumor-shrinking capability. However, the durability of the treatment effect has remained a persistent challenge for the field.
“CAR-T therapy has demonstrated meaningful activity against glioblastoma,” said David Arons, JD, president and chief executive officer of the National Brain Tumor Society. “Efforts to optimize and bolster the potency and effect of these treatments are the next steps toward realizing the full potential for patients with brain tumors. KiraGen has developed a novel approach to that problem, and we believe it holds exciting potential. National Brain Tumor Society invests in innovation at precisely this stage, when a promising idea needs rigorous preclinical evaluation to establish whether it is ready for testing in patients.”
KiraGen’s approach leverages an emerging body of research on the elements that hinder the effectiveness and longevity of T cells in a suppressive glioblastoma tumor microenvironment. The company uses multiplex gene editing — making several precise changes to a cell’s DNA at the same time rather than one at a time — to potentially overcome multiple challenges to CAR-T cell durability in glioblastoma. KiraGen’s cells are made from healthy donor T cells rather than a patient’s own, an approach intended to produce a therapy that can be manufactured in advance and available when needed versus in real-time.
“Patient-derived models are expensive and difficult to run, and they are the closest thing we have to a real test before the clinic,” said Aaron Edwards, MSc, MBA, co-founder and chief executive officer of KiraGen Bio. “We’ve been grateful to receive advice and guidance from the team at the National Brain Tumor Society to navigate the technical and funding path required to establish rigorous preclinical data, and with this grant such work is now underway. For an early-stage company, this kind of support does more than fund an experiment. It makes it possible to generate the evidence that patients, researchers, and investors should expect before a program moves forward.”
About National Brain Tumor Society
Building on over 30 years of experience, the National Brain Tumor Society (NBTS) unrelentingly invests in, mobilizes, and unites the brain tumor community to discover a cure, deliver effective treatments, and advocate for patients and caregivers. Our focus on defeating brain tumors and improving the quality of patients’ lives is powered by our partnerships across the science, health care, policy, and business sectors. We fund treatments-focused research and convene those most critical to curing brain tumors — once and for all. Join us at BrainTumor.org.
