Summary: TI12 primary efficacy evaluation met the study's prespecified requirements; all 30 patients have achieved transfusion independence

Shanghai, China, August 25, 2026 —— BRL Medicine today announced important progress in the pivotal clinical study of its independently developed gene-edited hematopoietic stem cell therapy BRL-101. The study is a pivotal clinical trial for transfusion-dependent β-thalassemia (TDT) approved by the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA). Based on data as of August 20, 2026, the proportion of patients achieving transfusion independence for 12 months (TI12) has met the study's prespecified primary efficacy endpoint requirements.

BRL-101 Pivotal Study Met Prespecified Primary Efficacy Endpoint

A total of 30 TDT patients in this study have completed dosing. All 30 patients have achieved transfusion independence; as of the data cutoff date of August 20, 2026, all 30 patients remain transfusion-independent without requiring red blood cell infusions. All patients will continue to complete subsequent follow-ups per the study protocol to further evaluate durability of efficacy, long-term safety, and other clinical parameters.

Key Pivotal Clinical Validation Achieved

Transfusion-dependent β-thalassemia is a severe hereditary blood disorder. Due to abnormal β‑globin production, patients typically require regular, long‑term red blood cell transfusions and face iron overload as well as related multi-system complications affecting the heart, liver, endocrine system, and other organs, along with significant long-term disease burden.

BRL-101 is an autologous hematopoietic stem cell therapy developed using gene-editing technology. By editing key regulatory regions in the patient's own hematopoietic stem cells, it promotes re-expression of fetal hemoglobin (HbF), thereby restoring the patient's effective hematopoietic capacity. The therapy is designed to help TDT patients achieve long-term transfusion independence with a single administration.

For TDT gene therapy, achieving transfusion independence is a critical clinical goal, and the long-term, stable sustainability of this clinical benefit is key to evaluating the long-term value of a one-time treatment.

Compared with globally marketed viral-vector-based gene-editing therapies for thalassemia, a core advantage of BRL-101 lies in its non-viral vector gene-editing technology. Unlike conventional viral-vector gene therapies, the non-viral delivery approach eliminates the potential oncogenic risk associated with viral integration into the host genome. The application of non-viral vector technology enables BRL-101 to achieve high editing efficiency while offering a potentially superior safety profile compared to viral-vector products, providing patients with a more reliable gene therapy option.

The achievement of the prespecified primary efficacy endpoint in this pivotal study marks the expansion of BRL-101’s clinical evidence from early-stage proof-of-concept and long-term follow-up in a small number of patients to systematic pivotal clinical validation in a larger sample size, providing an important clinical basis for subsequent regulatory submissions of BRL-101.

Continued Long‑term Follow‑up and Regulatory Submission Preparation

Achieving the prespecified primary efficacy endpoint in the pivotal study is a significant milestone in BRL-101’s clinical development. For a one-time gene-edited hematopoietic stem cell therapy, long-term follow-up remains critically important for ongoing evaluation of durability of efficacy and long-term safety.

BRL Medicine will continue to complete subsequent follow-ups for all patients per the study protocol, continuously evaluating durability of transfusion independence, hemoglobin levels, hematopoietic function, long-term safety, and other clinical parameters. As more data mature and complete statistical analyses are finalized, the Company will announce full clinical results at an appropriate time and actively advance preparatory work for BRL-101’s regulatory submission.

Dr. XIANG Yu, CEO of BRL Medicine, commented:

"For thalassemia patients, we care not only about whether they can achieve transfusion independence after treatment, but also whether this benefit can be sustained long‑term, whether patients can establish stable and sufficient autonomous hematopoiesis, and whether they can truly return to normal study, work, and daily life. This TI12 primary efficacy evaluation meeting the study’s prespecified requirements provides further important evidence for BRL-101’s long-term clinical value."

"As the Company enters a new stage of development, the new management team will continue to adhere to the primary principles of patient safety and high-quality clinical evidence, rigorously advancing long-term patient follow-up, regulatory submissions, quality systems, and industrialization preparation, while actively exploring global collaboration opportunities."

About BRL Medicine

BRL Medicine is a platform-based biotechnology company focused on the research, development, and clinical application of gene and cell therapies. Leveraging our capabilities in gene editing technologies and a combination of six modularized platforms, we have independently developed a comprehensive product pipeline of innovative therapeutic solutions for conditions in the areas of genetic diseases, autoimmune diseases, hematologic malignancies and solid tumors, each with significant medical needs but yet effective solutions, thereby leading to vast market potential. Since our inception, BRL Medicine has evolved from building our core technology platforms and research capabilities to advancing our product candidates toward the commercialization stage.

Forward-looking Statements