Bioray Biotech Announces First Adult Patient Cured and Discharged in Phase I Clinical Trial of BRL-101 for Transfusion-Dependent β-Thalassemia

ShanghaiFebruary 17, 2023PR Newswire -- February 17, 2022, BRL Medicine Inc., a Shanghai-based biotech company focusing on gene and cell therapy (hereinafter referred to as "BRL Medicine"), received another piece of good news. The multi-center Phase 1 pivotal clinical trial for its gene-editing therapeutic product (pipeline code: BRL-101) targeting transfusion-dependent β-thalassemia has been initiated at the First Affiliated Hospital of Guangxi Medical University.Completed the first cure of adult severe thalassemia, the patientSuccessfully discharged from the hospital on January 16, 2023, and has now reached the standard of摆脱输血依赖, officially "脱贫".This breakthrough news has undoubtedly provided a strong boost to adult patients with severe β-thalassemia, a historically difficult-to-treat group in China and even globally, rekindling their hope for life.

BRL-101 Granted IND Approval in China

The first patient enrolled in this Phase 1 clinical trial is a 21-year-old college student who was diagnosed with β+/β0 severe thalassemia at 8 months old. Prior to receiving gene therapy, the patient maintained regular blood transfusions and iron chelation therapy, with an average annual transfusion of whole blood as high as 15,042 ml. The patient completed the first infusion on December 8, 2022. After undergoing HSC transplantation with gene editing, the patient's hemoglobin began to increase significantly, reaching 94.4 g/L on day 37, successfully achieving hematopoietic stem cell engraftment and hematopoietic reconstitution. Notably,No serious infections or other adverse events occurred during the entire treatment process, onlySuccessfully discharged after 40 days, now reaching the standard of being free from transfusion dependence."Now able to return to a normal life, the patient also excitedly expressed: 'Finally, I can peacefully pursue my study plans!'"

Facing the successful cure of the first patient,Chief Physician Professor Lai YongrongSay"β-Thalassemia, as a genetic disorder, the most effective treatments are hematopoietic stem cell transplantation and gene therapy. The key to gene therapy is the efficiency and safety of gene-editing technology. Choosing to collaborate with BRL Medicine is because BRL Medicine has already positioned itself inWorld-leading levelThe strength of gene-editing technology is also the most powerful proof of the success of this research project. From the treatment results this time, the patient's recovery after receiving gene therapy was very ideal, becoming free from transfusion dependence within just two months, with hemoglobin continuously maintained within the normal range. This has given me more confidence and hope for this clinical project. I hope that through the joint efforts of all parties, we can help the product come to market as soon as possible, allowing more patients to overcome transfusion dependence. I also hope that in the future, beyond the field of thalassemia, gene therapy will benefit more patients with other rare genetic diseases."

This clinical trial is a multi-center, open-label clinical study aimed at evaluating the safety and efficacy of a single dose of intravenous infusion of BRL-101 in treating transfusion-dependent β-thalassemia. Previously, BRL Medicine demonstrated excellent clinical outcomes in investigator-initiated trials (IIT), successfully curing 6 patients with β-thalassemia globally, all of whom have been free from transfusion dependence for over a year. Among them, two patients have remained transfusion-independent for more than two and a half years.For the First Time in AsiaTreating thalassemia through gene editing technology is alsoFor the First Time WorldwideSuccessful Case of Treating β0/β0 Type Severe Thalassemia with CRISPR Gene Editing Technology. This early clinical research achievement was published on August 4, 2022, in a top international medical academic journal.Nature MedicineUp.

"The successful treatment and discharge of the first adult patient with severe thalassemia in the Phase 1 clinical trial of BRL-101 brings us one step closer to our goal of providing a one-time curative therapy for transfusion-dependent β-thalassemia patients, and gives us more confidence to accelerate the progress of this multi-center registration clinical trial."BRL MedicineCEO Dr. Zhengbiao Zheng"As one of the earliest companies globally to engage in the research, development, and application of gene-editing technology, we will continue to build on our increasingly robust industrial setup and global R&D network. Moving forward, we aim to advance the development of multiple product pipelines, with the goal of providing better treatment options for patients worldwide suffering from genetic disorders, malignant tumors, and autoimmune diseases."

In this regard,Professor Mingyao Liu, Chairman of BRL Medicine"We are delighted to see the launch of the BRL-101 multicenter registrational clinical trial and the first adult thalassemia patient successfully progress from enrollment to discharge after being cured. We deeply appreciate the clinical experts, patients, and their families who participated in and supported this research. According to clinical data, thalassemia patients born before 2010 are now over 10 years old, and as they grow older, the difficulty of curing them also increases. In this clinical trial, BRL Medicine expanded the patient age range to include adults and achieved such remarkable efficacy. This indicates that the BRL-101 product from BRL Medicine not only benefits young thalassemia patients but also has the potential to help the 'tough group' in the field of thalassemia transplantation—older patients and those without matching donors. Together with clinical experts, we will continue to fully advance the translation and implementation of this clinical research, hoping to benefit a wide range of β-thalassemia patients as soon as possible."