Gracell Biotechnologies Co., Ltd. ("Gracell"), a clinical-stage immune cell therapy company, today announced the initiation of an investigational study of GC027, the first product candidate developed using TruUCAR^™ to treat relapsed or refractory (R/R) T-cell malignancies.

T-cell acute lymphoblastic leukemia or T-ALL is an aggressive form of ALL, which affects white blood cells and the bone marrows ability to generate healthy blood cells. About 15-20% of people with ALL have T-ALL. While T-ALL is treatable by chemotherapy and stem cell transplant, around 75% of patients will relapse within two years^[1]. T-cell lymphoblastic lymphoma (T-LBL) is another devastating T-cell malignancies. For patients who develop R/R T-ALL or T-LBL, there are few options for treatment.

Autologus CAR-T therapies rely on patients' own T cells, which have been affected by prior therapies; thus, cell quality as well as efficacy remains questionable. Allogenic CAR-T therapies made of healthy donors' T cells would be characterized as being of consistently good quality with the potential to improve efficacy. Unlike autologous CAR-T cells, allogeneic CAR-T cells can be made as off-the-shelf product which means patients do not have to wait for lengthy production time. Furthermore, the cost of production can be significantly lower. Allogenic CAR-T therapies also provide a vital treatment option for patients with viral infections and/or other conditions prohibiting access to autologous cell therapies.

TruUCAR^™ based GC027 is designed to meet the above unmet needs. Its cells are made of T cells from healthy donors, genetically edited and inserted with chimeric antigen receptor (CAR) ex vivo, which can specifically bind to and eliminate target T malignant cells. Different from industry leaders' off-the-shelf CAR-T design, Gracell's proprietary and patented TruUCAR^™ technology requires no co-administration of anti-CD52, a cytotoxic agent for ablating cancerous cells while inducing long term immune depletion in the patient. Instead, GC027 utilizes CRISPR genome editing strategy that is expected to avoid graft-versus-host disease (GvHD) as well as graft rejection caused by the patients' immune system.

The prudent preclinical studies provide substantial evidence to trigger GC027 moving into a non-IND (investigational new drug) clinical trial to evaluate the safety, pharmacokinetics and pharmacodynamics of GC027 therapy in patients suffering from relapsed and refractory T lymphocyte malignancies.

TruUCAR^™ is another technological breakthrough developed by Gracell following the recent announcement of FasTCAR^™ technology and products. It enables producing off-the-shelf CAR-T cells from healthy MHC (major histocompatibility complex) mismatched donors with a large number of doses readily to be dispatched to patients in need.

"Launch of the investigational GC027 study as the first-of-its-kind therapy marks another significant milestone for Gracell," said Dr. William CAO, Founder and CEO of Gracell. "Once the concept is well-proved with solid evidence for safety and efficacy, we will immediately deploy development of a series of TruUCAR^™ products for other medical unmet needs, including B cell malignancies."

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