Cell and Gene Therapy Drug Developer

2024 Shanghai Science and Technology Award

August 26, 2025, "2024 Shanghai Science and Technology Awards Conference"Was grandly convened in Shanghai. ByBRL Medicine Inc.(hereinafter referred to as "BRL Medicine"), East China Normal UniversityLiu Mingyao, Du Bing, Li DaliProfessor TeamProjects jointly developed“Development and Transformation Application of Key Technologies in Gene Editing and Cell Therapy”AwardedFirst Prize of Shanghai Municipal Science and Technology Progress Award。

Professor Liu Mingyao (1st from the left) and Professor Du Bing (2nd from the left) receive the honor on behalf of the team.
"Shanghai Science and Technology Progress Award"It is one of the highest honors in Shanghai's technology field, aiming to recognize individuals and organizations that have achieved significant breakthroughs or made outstanding contributions in scientific and technological research, technology development and innovation, achievement transformation, and high-tech industrialization. The selection criteria for this award are strict, emphasizing not only the advancement and innovation of the technology but also its practical application value, industry-driving capability, and actual contribution to economic and social development. Projects awarded the first prize represent international advanced or domestically leading levels in their respective fields, showcasing major scientific and technological achievements with significant social or economic benefits, and are highly recognized for their value.
This award marksBRL MedicineThe innovation capability and transformation achievements in the field of cutting-edge biomedicine technology have received dual recognition from the government and academia.As the core completing unit of the award-winning project,BRL MedicineWith profound accumulation in gene editing technology and cell therapy product development, in collaboration with East China Normal UniversityLiu Mingyao、Du Bing、Li DaliThe professor's team worked closely together over several years, successfully overcoming multiple technical bottlenecks.Achieved original breakthroughs and clinical translation in three core technologies.

Thalassemia Gene Therapy:"One treatment, lifelong cure"
BRL MedicineThe project team, as one of the earliest research teams in China to develop CRISPR-Cas9 gene editing technology, began its work before 2013.Nature Biotechnology JournalReportedThe world's first successful application of CRISPR-Cas9 in mammalian gene editing. For many years, relying on core gene editing technology, with β-thalassemia(Thalassemia)Focusing on monogenic blood hereditary diseases, the project team developed the first gene therapy for thalassemia.

In 2022,Nature Medicine Issuance
In 2020,This therapy has completed the world's first gene editing treatment for a patient with β0/β0 type severe thalassemia.。As of now,This gene therapy has helped 15 patients with thalassemia completely摆脱输血依赖,Achieved"One treatment, lifelong cure."Relevant Research AchievementsPublished in 2019 and 2022, respectively, inNature Medicine Journal, and has been included in reports such as the *Blue Paper on the Prevention and Treatment of Thalassemia and Anemia in China* (2020) and *Rare Disease Drug Payment in China under Common Prosperity* (2022), becoming a benchmark solution in the field of international thalassemia treatment.
Non-viral Targeted IntegrationPD1-CAR-T: A Safe, Efficient, and Accessible New Choice for Cancer Treatment
Traditional CAR-T cell therapy mainly relies on viral vectors, with a price exceeding one million yuan, and carries the risk of gene insertion causing cancer, which limits clinical accessibility. The project team utilizes the CRISPR-Cas9 gene editing technology, which won the Nobel Prize in 2020.World's First Non-Viral Targeted Integration PD1-CAR-T Technology Applied in Clinical Tumor Treatment, Completely Eliminating Dependence on Viral Vectors, Significantly Reducing Production Costs and Cancer Risk。Clinical studies show that this CAR-T product is effective in relapsed/refractory B-cell non-Hodgkin lymphoma.(R/R B-NHL)During the patient's treatment,Objective Response Rate(ORR)Up to 100%, Complete Remission Rate(CR)Reaching 85.7%, median progression-free survival(mPFS)More than 20 months,Excellent safety and efficacy,Patients' long-term benefits are significantly higher than those of currently marketed CAR-T products.Relevant research achievements have been successively published in top journals.Nature(2022)And eClinicalMedicine(2023), and has successively been awarded"Top Ten Research Advances in Hematology in China 2022", "Important Medical Advances in China 2022"Such重磅 honors.

In 2022,Nature Issuance
Currently, BRL Medicine is focusing on relapsed/refractory B-cell non-Hodgkin lymphoma based on this non-viral targeted integration PD1-CAR-T technology.(R/R B-NHL)Moderate or Severe Refractory Systemic Lupus Erythematosus(SLE)Two registrational clinical studies are currently underway, aiming to bring patients a next-generation PD1-CAR-T drug that offers advantages in terms of safety, efficacy, and cost.
Allogeneic Universal TypeCAR-T: Launching a New "Cell Bank" Treatment Model
To address the issues of long personalized treatment cycles and high costs associated with existing autologous CAR-T therapies, as well as the persistent problem of excessive immune suppression in traditional allogeneic CAR-T treatments that poses significant infection risks to patients.After 8 years of technical research, the project team developed the world's first allogeneic universal CAR-T cell therapy targeting CD19.(TyU19)。It completely solves host rejection and graft-versus-host disease through multiple gene editing strategies.(GvHD)Risk, achieving "off-the-shelf" availability, with a single batch capable of meeting the treatment needs of over 200 cases. This allogeneic CAR-T not only significantly reduces production costs but also markedly shortens patient waiting times, making clinical treatment more accessible.
In 2024,BRL MedicineIn collaboration with Shanghai Changzheng HospitalXu HujiProfessor Team and East China Normal UniversityLiu MingyaoTeam, completedThe world's first in an internationally top-tier academic journalCell Successfully Treated 3 Cases of Refractory Patients with Allogeneic UCAR-T Published in [Journal Name]Autoimmune DiseasesPatient Outcomes。The research achievement was successively rated as"News of the Top Ten Scientific and Technological Advances in China in 2024 Selected by the Two Academies of China", "Top Ten Advances in China's Pharmaceutical and Biotechnology in 2024", "Top Ten Scientific Advances in China in 2024"and other important honors, and obtained"Father of CAR-T" Professor Carl June inCell The article highly commented: "It is expected to rewrite the treatment landscape of autoimmune diseases."
Allogeneic UCAR-T Partial Honors
Currently, BRL Medicine, based on this allogeneic UCAR-T, is focusing on the field of hematological tumors, specifically targeting relapsed/refractory B-cell acute lymphoblastic leukemia.(R/R B-ALL)Relapsed/Refractory B-cell Non-Hodgkin Lymphoma(R/R B-NHL)Two registration clinical studies for dual indications are currently underway, further expanding the therapy's application in solid tumors and autoimmune diseases such as systemic lupus erythematosus, systemic sclerosis, and dermatomyositis. The aim is to enable a broad patient population to fully benefit from the high-tech advantages of CAR-T treatment.
From Following to Leading, from Lab to Clinical Translation, and Future Commercialization: the Achievements of This Project Not Only Enable China to "Overtake on a Curve" in the Gene and Cell Therapy Field, but Also Hold the Promise of Making "Incurable" Diseases History and Bringing "Sky-High Priced Drugs" to Ordinary Households. Where Technology Meets Humanity, Every Precise Beat of Gene Editing Resonates with the Strong Melody of Life First. In the Future, BRL Medicine Will Continue to Focus on the Gene and Cell Therapy Field, Striving to Become a Globally Leading Innovative Pharmaceutical Enterprise and Contributing Technological Strength to Realize the "Healthy China" Strategy.

Project Team Photo:Liu MingyaoProfessor (3rd from the left),Du BingProfessor (left 1),Li DaliProfessor (4th from the left),Zhang JiqinPh.D. (2nd from left)
Award-Winning Team Members:
Liu Mingyao, Du Bing, Wu Yuxuan, Li Dali, Zhang Jiqin, Tan Binghe, Zhang Na, Liao Jiaoyang, Wang Liren, Qin Juliang
Award-winning Completion Unit:
East China Normal University
BRL Medicine Inc.
Yaotang (Shanghai) Biotechnology Co., Ltd.

