Bioray Bio Announces Four-Year Milestone of World’s First CRISPR-Edited Cure for Severe β⁰/β⁰ Thalassemia Patient

This Year's Xixi(Pseudonym)Already 11 years old, and about to enter the 6th grade of elementary school,It has been 4 years since he received BRL-101 gene therapy and became free from blood transfusion dependence.Particularly, on the occasion of the 4th anniversary follow-up this year, Professor Fu Bin took Xi Xi and Wang Wang to visit the History Museum of Xiangya Hospital, Central South University, allowing them to experience the century-old history of Xiangya Hospital, which was originally established in 1906. "In the ceaseless flow of time, witness the tremendous changes in China's medical cause, and also witness the century-long dedication of generations of Xiangya people."

As introduced to the two children, Xiangya Hospital,Professor FuDeeply Moved:Since this is the world's first treatment of β-thalassemia using CRISPR gene editing technology,⁰/β⁰Successful Cases of Severe Thalassemia: A Review of the Process of Two Patients Receiving Gene Therapyg/L, OthersThe test indicators and color Doppler ultrasound results are all normal. Moreover, compared to last year, the children's height has increased by 5-6 centimeters and their weight has increased by 4-6 kilograms within this year, both returning to a normal growth state. It is heartening that gene-editing therapy has demonstrated excellent safety and efficacy as it moves into clinical use; we look forward to the future product launch, which will allow more patients with thalassemia to have a new lease on life.

Professor Fu Bin Takes Xixi and Wangwang on a Tour of the Xiangya Hospital History Museum

Yes, the treatment process was difficult. In January 2013, Xixi was born into a happy family of four in Ningyuan County, Hunan Province. However, when Xixi was only nine months old, her family accidentally noticed her unusually pale complexion and rushed her to the hospital, where she was diagnosed with severe thalassemia. Children with thalassemia are often referred to as "million-dollar babies," and for a family with an annual income of only 60,000 yuan, this was undoubtedly a heavy burden. During the long wait for a suitable bone marrow match, Xixi could only receive regular blood transfusions and take iron-chelating medication as part of her conservative treatment. Even with this approach, the annual cost of treatment was around 40,000 yuan and would increase year by year. It wasn't until 2020, when Xixi was seven years old, that Professor Fu Bin brought hope and a miracle to Xixi’s family.At that time, Professor Fu was collaborating with BRL Medicine on a clinical study for gene therapy treatment of thalassemia, and Xi Xi was assessed to be eligible to participate in this clinical trial.

In April 2020, Xixi as the first caseβ⁰/β⁰TypeSevere Thalassemia Patients Officially Receive CRISPR Gene-Editing Treatment（BRL-101）; In June 2020, Xixi successfully got rid of the dependence on blood transfusion, from receiving treatment to officially "getting rid of poverty",The treatment cycle is only 56 days.Four years have passed to date, and XiXi's physical follow-up remains completely healthy, with the most recent follow-up showing a hemoglobin level of 142.g/L。 Xixi's father excitedly stated:It has been 4 years since he successfully received gene therapy. Now, he is physically and mentally healthy, lively, and cheerful. His favorite sports are playing basketball and swimming. Seeing the child grow taller and up day by day is truly deeply moving. We are very grateful to Professor Fu from Xiangya Hospital and the team at BRL Medicine for all they have done for our child, allowing him to luckily regain a new life. We hope that he will contribute back to society in the future, and we also hope that BRL Medicine’s gene therapy drug will be launched soon to help more families with thalassemia like ours.

Recent life photos of Xixi. When talking about his dream, he said: "To become a soldier and serve the country when I grow up."

Thalassemia(Thalassemia, Mediterranean Anemia)First discovered in the Mediterranean population, it is called Mediterranean anemia, also known as thalassemia or hemoglobinopathy. It is caused by abnormal expression of globin genes, which prevents the formation of hemoglobin with normal function, and is an autosomal recessive genetic disease.It is worth mentioning that,2023Year9Month18National Health Commission of China, etc.6Departments Jointly Release the "Second Batch of Rare Disease Catalog", Thalassemia(Severe)Included therein.

Thalassemia is mainly distributed in tropical and subtropical regions with high incidences of malaria globally, including southern China. The vast area south of the Yangtze River in China is a high-incidence region for thalassemia, particularly in Guangxi, Guangdong, and Hainan provinces. Among these, Guangxi has the highest incidence of thalassemia, with 1 in every 4 to 5 people being a carrier of the thalassemia gene, and 1 in every 55 families facing the risk of having a child with severe thalassemia. Current conventional treatments for thalassemia mainly include regular blood transfusions, iron chelation therapy, and hematopoietic stem cell transplantation. Allogeneic hematopoietic stem cell transplantation is currently the only method that can cure β-thalassemia.Including fully matched and haploidentical hematopoietic stem cell transplantation, howeverHLA typing is extremely difficult and expensive, with only a small number of patients able to receive transplants; furthermore, allogeneic hematopoietic stem cell transplantation is highly prone to rejection, resulting in acute or chronic GVHD. Patients also need to use immunosuppressants long-term, which may lead to severe infections, and there are issues such as high transplant-related mortality. Therefore, the emergence of gene therapy in recent years has brought hope to patients with thalassemia.

In December 2023 and January 2024, the world's first CRISPR-based gene editing therapyCASGEVY™Used for treatmentβ-Thalassemia (TDT`) and sickle cell anemia (`SCD）Gradually in Europe and AmericaApprovedThe listing, this is undoubtedly based on pharmaceutical historyCRISPRSignificant milestone events in the technical field are visible.CRISPRHuge potential for development.  

The gene therapy product that Xixi received(Pipeline Code: BRL-101)Similar to CASGEVY™, it also utilizes CRISPR gene editing at the BCL11A site to genetically modify patients' hematopoietic stem cells. The modified hematopoietic stem cells are then infused back into the patient, where they rebuild the modified cell population through self-renewal and differentiation, thereby achieving the goal of treating hematological diseases, which can realize "One dose, lifetime cure"The Advantages."

According to the latest data released by BRL Medicine,All 15 patients who received BRL-101 gene therapy have completely eliminated their dependence on blood transfusions, achieving a one-time treatment with lifelong cure!At the same time, the average total hemoglobin of patients was greater than 120g/L, quickly restored to the level of healthy individuals, and the treatment effects were significantly better than those of products already on the market abroad and products under research in China. Moreover,BRL Medicine's BRL-101 product uses electroporation to deliver gene-editing materials, avoiding safety issues caused by random insertion of viral vectors into hematopoietic stem cells.At the same time, the target site of gene editing has been extensively studied for off-target effects, and it has been proven that this target site has no off-target effects and has high safety.

Overall, compared to other therapies, BRL Medicine's BRL-101 gene therapy is more efficient, convenient, and safer, with advantages such as good targeting, high safety, wide range of action, and significant therapeutic effects. It is expected to become a treatment that benefits the general public. In the future, BRL Medicine will continue to work with clinical experts to fully promote the transformation and implementation of this clinical research, hoping to bring benefits to patients with thalassemia and hereditary rare diseases as soon as possible.