In July 2020, BRL Medicine Inc. (referred to as "BRL Medicine") collaborated with Xiangya Hospital of Central South University to successfully complete Asia's first clinical trial using gene-editing technology to treat thalassemia (referred to as "thalassemia"). In this clinical study, the two patients treated were boys aged 7 and 8 with transfusion-dependent severe β-thalassemia, both of whom successfully became transfusion-independent after treatment. Notably, one of the boys (pseudonym "Xi Xi") is the world’s first patient with β0/β0 type severe thalassemia to be cured using CRISPR gene-editing technology—this type represents the most severe form of β-thalassemia.Five years have passed, BRL Medicine announced,This is the world's first patient to receive CRISPR gene-editing therapy (BRL Medicine pipeline code: BRL-101) treatment.β0/β0 Type Severe Thalassemia Patients Have Been Free of Transfusion Dependence for Over Five Years。From being tethered to a blood transfusion bag for life to running freely, his life has been completely transformed. This marks a significant milestone in the gene-editing treatment of hereditary blood disorders.Figure: Recent life photo of "Xi Xi," the first thalassemia child cured by BRL-101From "Million Babies" to Running Free: Xixi's Road to Rebirth
In 2013, Xixi was born in Hunan and was diagnosed with the most severe β0/β0 type of thalassemia at just 9 months old. This means he needs lifelong regular blood transfusions and iron chelation therapy to sustain his life. The heavy medical burden turned him into a "million-dollar baby" and plunged his family into despair—finding a fully matched hematopoietic stem cell transplant is as difficult as climbing to the sky.In 2020, the fate of 7-year-old Xixi was rewritten. Professor Fu Bin, Xixi's attending physician from Xiangya Hospital of Central South University, brought news of a revolutionary clinical study: BRL Medicine was conducting research using CRISPR gene-editing technology (pipeline code: BRL-101) to treat severe thalassemia. Holding on to their last hope, Xixi’s parents agreed without hesitation, making Xixi the world’s first β0/β0 type severe thalassemia patient to receive CRISPR gene-editing treatment.The entire treatment process was astonishing: After just 56 days, Xixi successfully "overcame the poverty" — completely freeing herself from blood transfusion dependency! Now, five years have passed, and at the age of 12, Xixi is about to enter middle school. Xixi's parents expressed their deep emotions: "The heavy burden in our hearts has finally been lifted! Life is full of hope. Watching Xixi brimming with energy every day makes us incredibly happy. We are grateful to Professor Fu Bin and the scientists at BRL Medicine for giving our child a new lease on life!"The principal investigator (PI) of this clinical trial, Professor Fu Bin, stated:"The recovery of Xixi after receiving BRL-101 gene therapy has been very ideal! She was off transfusions within just 2 months, and her hemoglobin levels have consistently remained within the normal range. The treatment process was safe, with no severe complications or significant chronic complications, and her physical development is completely normal. Recent follow-ups show that both Xixi and another boy named Wangwang (pseudonym), who received gene therapy at the same time, are in excellent health, with hemoglobin levels reaching around 140g/L, allowing the children to grow up healthy. The successful implementation of this clinical project is inseparable from BRL Medicine's world-leading gene-editing technology. This is a triumph of science and technology, as well as a response to the suffering of patients, continuously pushing the boundaries of what was once thought 'impossible.'"
Figure: Xixi's past photo with Professor Bin FuFrom 7 years old to now a healthy 12 years old,Xixi excitedly expressed her dream: "I hope to study hard and become a doctor when I grow up, just like Uncle Fu, to help more people."Scientific Breakthrough: Gene Therapy Breaks the "No Match" Dilemma
Thalassemia is a hereditary blood disease caused by defects in globin genes that lead to impaired hemoglobin synthesis, and patients with severe forms experience extremely poor quality of life.Traditional radical methods rely on allogeneic hematopoietic stem cell transplantation, but face two major challenges: extremely high costs and the great difficulty in matching., and there are high risks such as transplant rejection, graft-versus-host disease (GVHD), and infection.The emergence of BRL Medicine's BRL-101 gene therapy has brought hope.Its principle is similar to that of CASGEVY™, the world's first marketed CRISPR therapy: using CRISPR gene editing on the BCL11A site to genetically modify the patient’s hematopoietic stem cells, which are then reinfused into the patient. Through self-renewal and differentiation, the modified cell population is rebuilt to achieve the goal of treating hematological diseases.PossibleAchieve"The Advantage of 'One-Time Dosing, Lifetime Cure'"。Moreover, BRL Medicine's BRL-101 product uses electroporation to deliver gene-editing materials, avoiding the safety issues caused by random insertion of viral vectors into hematopoietic stem cells. Previously, on October 31, 2023, regarding the off-target issue of CASGEVY™, at the FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee meeting in the United States, studies published by BRL Medicine in 2019 and 2022 were cited.Nature MedicineTwo references to substantiate the safety of this product.
Figure: BRL-101 Treatment Strategy
From Lab to Global Layout: BRL Medicine’s Gene Therapy Rewrites the "Code of Life"
BRL Medicine has been deeply engaged in the gene therapy field for many years, with a solid and rapid development process for its first gene therapy product:
2019:《Nature Medicine》publishes research, laying the theoretical foundation for gene therapy to cure hereditary blood diseases.
2020:AndCentral South UniversityProfessor Bin Fu from Xiangya Hospital Collaborates on the World's First Successful Cure of a Severe β0/β0 Type Thalassemia Patient; Clinical Results Published.
2021:Collaborated with Professor Zhang Xinhua from the 923rd Hospital of the Joint Logistics Support Force of the People's Liberation Army to achieve clinical cure for three thalassemia patients in Guangxi for the first time.
2022:*Nature Medicine* published a paper reporting the significant efficacy of the first successful treatment of a patient with severe thalassemia for over two years. In the same year, the BRL-101 gene therapy product received clinical trial (IND) approval from the China National Medical Products Administration (NMPA).
2023: Collaborated with Professor Lai Yongrong from the First Affiliated Hospital of Guangxi Medical University to achieve the first successful cure of an adult patient with severe thalassemia in China, overcoming the global treatment challenge for adult thalassemia.
2024: Completed the Phase 1 registrational clinical study of the BRL-101 gene therapy product, successfully curing 15 patients with thalassemia worldwide. In the same year, collaborated with Professor Lai Yongrong from the First Affiliated Hospital of Guangxi Medical University to achieve the first successful case of gene therapy for a foreign patient with sickle cell anemia (sickle cell disease) in China.
2025: Complete the Phase 2 pivotal registration clinical study for BRL-101 gene therapy product, accelerating the product's market entry process.
Figure: In August 2022, BRL-101 received IND approval in China
Professor Mingyao Liu, Founder & Chairman of BRL Medicine, stated:"Clinical data fully demonstrates that BRL Medicine's gene therapy is highly targeted, safe, significantly effective, and long-lasting. It can greatly reduce the economic burden of long-term treatment for patients, making it a more accessible gene therapy. Currently, we are also exploring the therapy's application in treating sickle cell anemia (pipeline code: BRL-102) and have successfully completed the first case of treatment for a foreign sickle cell anemia patient in China. In the future, BRL Medicine will continue to collaborate with global clinical experts to advance the implementation of this gene therapy, with the hope of benefiting patients with thalassemia, sickle cell anemia, and other hereditary blood disorders worldwide as soon as possible."Currently, BRL Medicine's BRL-101 gene therapy has been consecutively selected for presentation at several top international academic conferences, including the American Society of Gene & Cell Therapy (ASGCT) Annual Meeting, the European Hematology Association (EHA) Annual Meeting, and the American Society of Hematology (ASH) Annual Meeting, due to its groundbreaking results. It has also been honored with the first award in China’s rare disease field for 2024—the Golden Snail Award for "Rare Disease Industry Advancement." Relevant research findings have been included in reports such as the *Blue Book of China’s Thalassemia Prevention and Treatment* (2020) and *Rare Disease Drug Payment under Common Prosperity in China* (2022), showcasing significant influence.
