Gracell Initiates Investigational Study of the Technological Breakthrough TruUCAR™ Therapy for Relapsed or Refractory T-cell Malignancies

SUZHOU,Chinaand SHANGHAI,Jan. 7,2020 -- 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 CRISPRgenome 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."

About GC027

GC027 is an investigational,off-the-shelf CAR-T cell therapy for T cell malignancies,derived from healthy donors. The use of healthy donor's cells are preferential to a patient's own with potential to improve efficacy,reduce production time,and lower cost of goods.

About T-ALL

T lymphoblastic leukemia (T-ALL) is an aggressive form of T cell malignancies,with a diffuse invasion of bone marrow and peripheral blood. In 2015,ALL affected around 876,000 people globally and resulted in 110,000 deaths worldwide. T-ALL compromises about 15%-20% children and adults[1].Current standard therapies for T-ALL are chemotherapies and stem cell transplantation. A large portion of these patients will experience relapse within two years following treatment by conventional therapies.

About T-LBL

T lymphoblastic lymphoma (T-LBL) is an aggressive form of T cell malignancies,with rare lymphoproliferative neoplasm of mature T cells caused by infection with the retrovirus human T lymphotropic virus. T-LBL compromises about 2% of adult non-Hodgkin's lymphoma (NHL) and 30% of pediatric NHL patients[2]. Five-year overall survival is only 14% in adults.Although first-line treatment using cytotoxic combination chemotherapy can achieve 70% ORR,nearly 90% of patients relapse,often within months of completing chemotherapy.

About Gracell

Gracell Biotechnologies Co.,Ltd. ("Gracell") is a clinical-stage biopharma company,committed to developing highly reliable and affordable cell gene therapies for cancer. Gracell is dedicated to resolving the remaining challenges in CAR-T,such as high production costs,lengthy manufacturing process,lack of off-the-shelf products,and inefficacy against solid tumors. Led by a group of world-class scientists,Gracell is advancing FasTCAR™,TruUCAR™ (off-the-shelf CAR),Dual CAR and Enhanced CAR-T cell therapies for leukemia,lymphoma,myeloma,and solid tumors.

CONTACT:

Linc HE

Business Development and Public Relations

Linc.he@gracellbio.com

Dr.William CAO

Founder and CEO

william.cao@gracellbio.com

[1]Pediatric hematologic Malignancies: T-cell acute lymphoblastic Leukemia,Hematology 2016

[2]Clinical Review: Adult T-cell Leukemia/lymphoma,Journal ofOncology Practice 2017

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