A patient with heavily pretreated relapsed/refractory diffuse large B-cell lymphoma (DLBCL) has achieved complete remission following treatment with a novel in vivo CD19 CAR-T therapy developed by Genocury Biotech. The groundbreaking results, reported from an investigator-initiated trial at Tongji Hospital in China, demonstrate the therapy's efficacy without requiring lymphodepletion—a toxic preconditioning step standard in conventional CAR-T protocols.
The male patient with advanced DLBCL achieved complete hematological remission within 28 days after receiving a single dose of the investigational therapy. Flow cytometric analysis revealed robust expansion of CAR-T cells, with the response remaining durable through the 90-day follow-up period.
"In this groundbreaking case, we observed the patient treated with Genocury's CD19 in vivo CAR-T achieved complete remission through 90-day follow-up," said Dr. Jia Wei, principal investigator of the trial and professor in the Department of Hematology at Tongji Hospital. "This was achieved without any lymphodepletion, which fundamentally challenges current cellular therapy dogma."
Unprecedented Safety Profile
The novel approach demonstrated a remarkable safety profile, with complete absence of cytokine release syndrome (CRS), immune effector cell-associated neurotoxicity syndrome (ICANS), and lymphodepletion-related complications. This stands in stark contrast to conventional CAR-T therapies, which carry approximately 50% risk of CRS and neurotoxicity.
These complications typically require intensive monitoring and management, often necessitating hospitalization and specialized care. The elimination of these side effects represents a significant advancement in CAR-T therapy safety.
Reimagining CAR-T Delivery
Genocury's platform addresses two major limitations in current CAR-T therapy approaches. First, it eliminates the need for ex vivo manipulation of T cells. Traditional CAR-T protocols require harvesting, modifying, and expanding a patient's T cells outside the body—a process that typically takes 3-4 weeks and costs approximately $400,000.
In contrast, Genocury's proprietary in vivo CAR-T vector delivers the CAR payload directly into circulating T cells within the patient's body, enabling functional CAR-T generation without the extensive laboratory processing.
Second, the lymphodepletion-free protocol removes the need for harsh preconditioning chemotherapy, which is a leading cause of hospitalization in conventional CAR-T therapy. This approach reduces treatment-related complications and logistical burdens while still enabling significant CAR-T cell expansion.
Implications for Global Access to Advanced Cancer Therapy
Dr. Wei emphasized the potential broader impact of this approach: "This therapy combines the benefits of autologous CAR-T with the accessibility of universal therapies, potentially ending the era of unaffordable cancer treatments. This could democratize access to CAR-T globally."
DLBCL is an aggressive form of non-Hodgkin lymphoma affecting B cells. While CAR-T cell therapy has revolutionized treatment for patients with relapsed or refractory disease, the complex manufacturing process, high costs, and significant toxicities have limited its widespread adoption.
Genocury Biotech's technology utilizes its proprietary Vivoexpress platform, a modular delivery system that enables targeted delivery of genetic payloads with organ-specific tropism. The company focuses on developing next-generation immunotherapies for oncology and autoimmune disorders.
The trial was conducted at Tongji Hospital, one of China's most prestigious hematology centers and the largest cell immunotherapy center in Hubei Province. The hospital has been recognized as a national key discipline since 2007 and a national clinical key specialty since 2011.
While these early results are promising, larger clinical trials will be necessary to confirm the efficacy, safety, and durability of this novel approach across a broader patient population. Nevertheless, this breakthrough represents a potential paradigm shift in how CAR-T therapy might be delivered to patients worldwide, potentially making this life-saving treatment more accessible and less burdensome for patients with aggressive blood cancers.
