Gene Therapy Ignites Hope: Bonovo Bio's BRL-101 Enables Another Thalassemia Child to Achieve Transfusion Independence

November 27, 2025Recently, BRL Medicine Inc. (hereinafter referred to as "BRL Medicine"), a company focused on gene and cell therapy, announced another successful case of a patient achieving "disease alleviation." Recently, in its efforts targeting...Gene Editing Therapy for Transfusion-Dependent Beta-Thalassemia (Pipeline Code: BRL-101)The multicenter Phase II pivotal clinical trial in China,AndCollaboration between Professor Lai Yongrong and Professor Liu Rongrong from the First Affiliated Hospital of Guangxi Medical UniversitySuccessfully helped one 5-year-old patient(Pseudonym "Xinxin")Free from Transfusion Dependence, Embrace a Healthy and Normal LifeFrom a moment of "despair" to one filled with "hope," the patient's family sent a heartfelt letter of gratitude to the entire research team, thanking them for the power of technology that illuminated the hope of life. This successful treatment is not only a miracle for one family but also reaffirms the safety and efficacy of BRL Medicine's BRL-101 gene therapy, offering a safer treatment option for thalassemia patients worldwide.

Figure 1: Xinxin (pseudonym) and Professor Rongrong Liu from the First Affiliated Hospital of Guangxi Medical University; Figure 2: Handwritten thank-you letter from Xinxin's parents

Technology Illuminates Hope, Gene Therapy Brings New Dawn of Rebirth

This Success"Little Patient Xinxin 'Poverty Alleviation'"(Pseudonym)She was diagnosed with severe β-thalassemia at just 6 months old. Since then, regular blood transfusions have become the only way for her to sustain life. Over the course of five long years, the little girl has had no choice but to live alongside blood bags and hospitals. While long-term transfusions keep her alive, they also bring heavy complications such as iron overload, severely affecting her growth, development, and quality of life.

"We have faced the dilemma of blood shortages time and again, and have also agonized under the threat of iron overload."XinxinThe parents wrote in the letter. In search of a cure, the family had hoped for a hematopoietic stem cell transplant, but a matching related bone marrow donor could never be found. The constant shattering of hope left the entire family almost enveloped in despair: "It felt like the sky was falling, and we thought our child would never be able to live without blood transfusions."

A turning point occurred in April 2025.BRL Medicine is collaborating with the team of Professor Lai Yongrong and Liu Rongrong from the Hematology Department of the First Affiliated Hospital of Guangxi Medical University to conduct a clinical study on the gene therapy "BRL-101" for transfusion-dependent β-thalassemia.（ⅡPeriod), Xin Xin entered the clinical project after meeting the criteria for the clinical trial. After fully informed consent and undergoing rigorous screening tests, Xin Xin was successfully admitted to the hospital and received an infusion treatment of BRL-101, a gene therapy product developed by BRL Medicine. The entire treatment process went smoothly, and the childThirteen days after the cell infusion, red blood cell transfusions were no longer needed, and one month later, Xin Xin successfully discharged from the hospital and returned to normal life. Xin Xin's parents expressed their gratitude for having the opportunity to receive this gene therapy."It's like grabbing a 'lifeline,' bringing us the dawn of hope."。

Moreover, the most encouraging result is: recently, after rigorous follow-up observations, Xin Xin has successfully achieved transfusion independence, with all key physical indicators gradually returning to normal."We dare to look forward to our children's future again!"In the letter, Xin Xin's parents mentioned that Xin Xin has finally gotten rid of the blood bags that had been troubling her for many years. Now, she can run freely and enjoy a carefree childhood like all healthy children.

Multi-party Collaboration to Tackle Challenges, Demonstrating China's Innovation in Pharmaceutical R&D

This successful treatment is also a model of the deep integration of clinical medicine and cutting-edge biotechnology.The First Affiliated Hospital of Guangxi Medical UniversityLai YongrongProfessorIndicates:"Previously, we successfully collaborated with BRL Medicine on the BRL-101 gene therapy in adult patients with severe thalassemia and sickle cell anemia, validating the therapeutic potential of its gene-editing therapy for hemoglobinopathies. I am delighted that, in this clinical study, we continue to work hand-in-hand with BRL Medicine's research team to help another young patient with thalassemia摆脱输血依赖, which not only reaffirms the safety and efficacy of their gene therapy but also strengthens my confidence in this treatment approach. I hope that, through the joint efforts of all parties, this product can be brought to market as soon as possible to help more patients worldwide overcome their dependence on blood transfusions. Additionally, I hope that beyond the field of thalassemia, gene therapy can benefit more patients suffering from other rare genetic disorders."

In response,Founder & Chairman of BRL MedicineLiu MingyaoProfessorIndicates:"We are very grateful to the clinical experts, patients, and their families who participated in and supported this research. We are delighted to see yet another patient 'free from the burden' and restart a healthy future. Currently, BRL Medicine has successfully treated multiple thalassemia patients using the BRL-101 gene therapy. Repeated clinical data has fully demonstrated the excellent efficacy of this product. From pediatric to adult thalassemia patients, BRL-101 has also broken through the longstanding treatment difficulties faced by older patients and those without matching donors in the field of thalassemia transplantation. Moving forward, we will actively collaborate with clinical experts to advance the translational application of this clinical research, hoping for its early market launch to benefit a wide range of patients."

BRL-101: The Hope of Gene-Edited Life

BRL-101 is a hematopoietic stem cell platform (ModiHSC) independently developed by BRL Medicine.^®) A gene therapy product developed by BRL Medicine, indicated for transfusion-dependent β-thalassemia.ModiHSC^®Mainly utilizing gene editing systems to genetically modify hematopoietic stem cells of patients, the modified hematopoietic stem cells are then infused back into the patient’s body. Through self-renewal and differentiation, the modified cell population is rebuilt, thereby achieving the goal of treating hematological diseases. In July 2020, BRL Medicine collaborated with Xiangya Hospital of Central South University to announce the world's first successful treatment of β-thalassemia using CRISPR gene editing.⁰/β⁰Clinical data of type severe thalassemia; In August 2022, BRL MedicineNature MedicineBreaking News: The World's First Successful Case of BRL-101 Gene Therapy in a Child with Severe Thalassemia Has Been Free from Blood Transfusion Dependence for Over Two Years, Marking a Breakthrough in Clinical Research for Gene-Editing Treatment in China; On August 16 of the Same Year, the Investigational New Drug (IND) Application for BRL-101 Was Officially Approved by the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) of China.

BRL-101 Approved for IND in China

According toBRL MedicineThe latest clinical results show that,BRL-101Gene Therapy:

All 15 patients with thalassemia who received BRL-101 gene therapy worldwide have successfully eliminated their dependence on blood transfusions, with the first patient having been free of transfusions for over 5 years! Additionally, the patients' average total hemoglobin level is greater than 120g/L, rapidly restoring them to healthy levels, demonstrating excellent clinical efficacy.

BRL-101 Gene Therapy Product Uses Electroporation to Deliver Gene Editing Materials, Avoiding Safety Issues Caused by Random Insertion of Virus Vector-Based Cell Gene Therapy Products. Meanwhile, the Gene Editing Target Has Been Proven to Have No Off-Target Effects Through Extensive Off-Target Research, Demonstrating High Safety.

Overall,BRL Medicine's gene therapy is efficient, convenient, and safe, with advantages such as good targeting, high safety, wide range of action, and significant therapeutic effects, and is expected to become a more accessible treatment for the public.Currently, BRL-101 is undergoing pivotalⅡThe clinical research was registered on schedule; the relevant research results have been successively included in reports such as the *Blue Paper on the Prevention and Treatment of Thalassemia in China* (2020) and *Payment for Rare Disease Drugs under Common Prosperity in China* (2022) due to their breakthrough nature, becoming a benchmark solution in the international thalassemia treatment field. In 2024, the outstanding achievements of this product were honored with the first-ever award in China's rare disease field—the "Golden Snail" Award·"Rare Disease Industry Promotion Award." Notably, BRL Medicine has also explored the treatment of sickle cell anemia based on this gene therapy and has successfully completed the first-ever treatment of a foreign sickle cell anemia patient in China. In the future, BRL Medicine will continue to collaborate with clinical experts to fully advance the transformation and implementation of this clinical research, with the hope of benefiting thalassemia and sickle cell anemia patients worldwide as soon as possible.

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About BRL Medicine

BRL Medicine Inc. is committed to becoming a global leading cell and gene pharmaceutical company in the new era of commercial civilization. With the mission of "leading innovation with gene editing technology, developing breakthrough therapies, and benefiting all humanity," BRL Medicine, relying on its self-developed center and the "Shanghai Gene Editing and Cell Therapy Research Center" co-built with universities, has generated more than 100 patent achievements so far. Currently, 19 projects are undergoing clinical trials in more than 20 well-known hospitals, 5 projects have been approved for IND, officially entering the registration clinical trial stage, and several other projects are in the IND application stage. Among them, gene editing therapy for β-thalassemia, non-viral...Targeted IntegrationPD1-Projects such as CAR-T and UCAR-T have achieved excellent clinical outcomes, demonstrating global leadership, and inNature、Cell、Nature Medicine、Nature biotechnology...and published multiple academic papers in well-known academic journals. BRL Medicine has established six proprietary technology platforms: a gene-editing technology innovation platform, a hematopoietic stem cell platform, a non-viral targeted integration CAR-T platform, a universal cell platform, an enhanced T-cell platform, and an in vivo gene-editing CAR-T platform. With a 7,000-square-meter GMP pilot production base and a nearly 100-person operations team, BRL Medicine ensures that innovative research outcomes can be quickly transformed and applied. By continuously driving rapid updates and iterations of its R&D products based on patient needs and clinical feedback, BRL Medicine adheres to an open, shared, and win-win philosophy. Collaborating with global innovative biopharmaceutical ecosystem companies, BRL Medicine is accelerating the transformation and application of innovative drugs for the benefit of patients worldwide suffering from genetic disorders, malignant tumors, and autoimmune diseases!