BRL Medicine Inc. Named Shanghai Municipal Enterprise Technology Center, Advancing Global Gene and Cell Therapy Innovation

Source: Publicity List of Shanghai Municipal Commission of Economy and Informatization

Shanghai Enterprise Technology Center RecognitionIt is a high-standard review process organized and implemented by Shanghai to enforce the innovation-driven development strategy, led by the Shanghai Municipal Commission of Economy and Informatization, and generated through strict screening and expert evaluation. This recognition aims to encourage and support enterprises in building technology centers, with extremely high standards for reviewing companies' innovation capabilities, talent teams, technical accumulation, R&D investment, and more. It can be said that receiving this honor not only represents the company's outstanding performance in technological innovation system construction and innovative achievements but also serves as an important indicator of the company’s comprehensive innovation capability reaching a leading level in Shanghai.

BRL Medicine Shanghai Headquarters

This successful selection of BRL Medicine clearly marks that BRL Medicine has gained authoritative recognition in organizational systems, management mechanisms, innovation efficiency, talent development, and technological achievements, and is now in a leading position in the industry.

BRL Medicine, with the mission of "leading innovation with gene editing technology, developing breakthrough therapies, and benefiting all humanity," is committed to becoming a global leading cell and gene pharmaceutical company. Since 2016, relying on its self-developed research center and university collaboration to build the "Shanghai Gene Editing and Cell Treatment Research Center," it has established...5A large technology platform with independent intellectual property rights:Gene Editing Technology Innovation Platform (CRISTARS)^®）、Hematopoietic Stem Cell Platform (ModiHSC^®), Non-viral Site-specific Integration CAR-T Platform (Quikin CART^®）、Universal Cell Platform (TyUCell^®）、Enhanced T-cell Platform (HyperTCell^®). Relying on these platforms, BRL Medicine has generated more than 100 patent achievements and laid out over 10 innovative drug pipelines, among which...5 itemsApproved in ChinaIND(Investigational New Drug Application), officially entering the registration clinical trial phase.

It is worth mentioning that BRL Medicine, having deeply engaged in the CGT field for many years, has garnered repeated recognition from international top-tier academic journals and major awards both in China and globally, demonstrating strong R&D capabilities and global influence.

1、The First Gene Therapy for Thalassemia (Pipeline Code:BRL-101）

This is the world's first gene therapy product to successfully treat β0/β0 type severe thalassemia using CRISPR/Cas9 technology,Received IND Approval in China in 2022According to the currently available data, BRL-101 gene therapy has helped 15 patients with thalassemia completely摆脱输血依赖, and the first patient has been cured for over 5 years. Relevant research findings have been successively published inPublished in 2019 and 2022 inNature MedicineJournal, and was written into"China Thalassemia Prevention and Anemia Blue Paper"(2020)、"Payment for Rare Disease Drugs in China under Common Prosperity"2022）In the report, it has become a benchmark solution in the field of international thalassemia treatment. In 2024, this product was awarded the first prize in China's rare disease field due to its outstanding results——"Golden Snail" Award · "Rare Disease Industry Promotion Award"。

BRL-101 Approved for IND in China

2. Non-viral targeted integration PD1-CAR-T therapy (Pipeline code: BRL-201/BRL-203)

This is the world's first non-viral targeted integration CAR-T product successfully applied in clinical tumor treatment. It has been developed in 2022 and 2024, respectively, for relapsed/refractory B-cell non-Hodgkin lymphoma (pipeline code: BRL-201) and moderate to severe refractory systemic lupus erythematosus (pipeline code: BRL-203).Received Two IND Approvals in ChinaThis CAR-T therapy has broken through the limitations of using viruses and random integration in traditional CAR-T cell preparation, and has also lifted the immune suppression of CAR-T cells by tumors. Existing clinical data shows that this CAR-T product is effective in treating relapsed/refractory B-cell non-Hodgkin lymphoma patients.The objective response rate (ORR) reached 100%, the complete response rate (CR) was 85.7%, the median progression-free survival (mPFS) exceeded 20 months, demonstrating excellent safety and efficacy.Relevant research achievements have been successively published in top journals.Nature（2022）AndeClinicalMedicine（2023）, and has successively been awarded"Top Ten Research Advances in Hematology in China 2022"、"China's Important Medical Advances of 2022"Such prestigious honors.

BRL-201 and BRL-203 were approved for IND in China in 2022 and 2024, respectively.

3. Allogeneic Universal CAR-T Therapy (Pipeline Code: BRL-301/BRL-303)

This is a globally pioneering allogeneic universal CAR-T therapy targeting CD19, developed by the project team after 8 years of technical research. It primarily eliminates the risks of host rejection and graft-versus-host disease (GVHD) through a multi-gene editing strategy, enabling "off-the-shelf" use, with a single batch capable of meeting the treatment needs of over 200 patients. This allogeneic CAR-T not only significantly reduces production costs but also greatly shortens patient waiting times, making clinical treatment more accessible. The product has now been expanded to the field of autoimmune disease treatment (pipeline code: BRL-303). In 2024, BRL Medicine Inc. will collaborate with Shanghai Changzheng Hospital.Professor Xu HujiTeam and East China Normal UniversityProfessors Liu Mingyao and Du BingTeam,The world's first in an international top-tier journalCellPublication of Successful Treatment of 3 Refractory Autoimmune Disease Patients with Allogeneic UCAR-T. The research achievement has successively been rated as"News of the Top Ten Scientific and Technological Advances in China in 2024 Selected by Academicians of the Two Academies", "Top Ten Scientific Advances in China in 2024"and other honors,AndAwarded by "Father of CAR-T" Professor Carl JuneCellJournal publication highly commented: "Expected to reshape the treatment landscape of autoimmune diseases". In addition, this therapyRespectivelyAndThe First Affiliated Hospital of Zhejiang University School of MedicineProfessor Tong HongyanAnd Shanghai Changzheng HospitalProfessor Xu Huji, Nanjing Drum Tower HospitalSun LingyunProfessorCooperation,Significant clinical treatment effects for systemic lupus erythematosus,Relevant achievements have been successively published in academic journals.Cell ResearchAndMed。

BRL-301 Approved for 2 INDs in China

It can be said that the selection of BRL Medicine Inc. as a municipal enterprise technology center in Shanghai is not only an affirmation of BRL Medicine's past scientific research achievements but also an expectation for its future development. In the future, the company will continue to collaborate with global partners to empower basic scientific research and promote the clinical progress of innovative products. It will also further expand its global presence, working alongside global innovative biopharmaceutical companies to advance China’s CGT sector, benefiting more patients with genetic disorders, cancer, and autoimmune diseases.

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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 and have officially entered the registration clinical trial stage, and several other projects are in the IND application stage. Among them, gene editing treatment 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 five 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, and an enhanced T-cell platform. It also owns a 7000-square-meter GMP pilot production base and a nearly 100-person operations team, strongly ensuring that innovative research achievements can be quickly transformed and applied. BRL Medicine continuously drives the rapid updating and iteration of its R&D products based on patient needs and clinical feedback. With an open, sharing, and win-win attitude, BRL Medicine is working with global innovative biopharmaceutical ecosystem companies to accelerate the transformation and implementation of innovative drugs for the benefit of patients worldwide suffering from genetic diseases, malignant tumors, and autoimmune diseases!