Chengdu YouSino Biosciences Announces China IND Approval for UC101, the World’s First Umbilical Cord Blood-Derived Allogeneic Anti-CD19 CAR-T Therapy

1. Technological Innovation: Dual Breakthroughs in Cord Blood Sourcing and Gene Editing

UC101 for newbornsUmbilical cord blood serves as the starting raw material, and through multi-gene editing technology, an allogeneic universal design is achieved, overcoming the core bottleneck of traditional CAR-T therapy.：

1.FreeImmunogenicity Regulation：Umbilical cord blood T cells have naturally low immunogenicity. By combining with CRISPR/Cas9 gene editing technology to knock out the T cell receptor (TCR) and major histocompatibility complex (MHC) genes, the risks of graft-versus-host disease (GvHD) and host-versus-graft reaction (HvGR) can be eliminated from the source.

2.Cell Activity Advantage: 90% of the cells in cord blood are naive T cells, and after in vitro expansion, they still retain 80% of stem memory T cells (Tscm) and central memory T cells (Tcm). Their in vivo expansion capability and persistence are significantly better than traditional CAR-T cells, achieving longer-lasting therapeutic effects.

3.Technological Innovation in Production: Globally pioneering the use of stable cell lines to produce lentiviral vectors, eliminating the need for plasmid materials. The process is simple and reduces costs by over 70%. A single batch can meet the needs of more than 100 patients, significantly improving drug accessibility.

2. Clinical Value: Wide Potential from Hematological Tumors to Autoimmune Diseases

The core indication of UC101 is acute B lymphoblastic leukemia, and its early investigator-initiated clinical study (IIT) has shown significant results.Efficacy:

1.Hematological Oncology Field: In patients with acute B lymphoblastic leukemia, the complete remission rate of UC101 is as high as 90%, with manageable adverse reactions, and key indicators surpassing those of similar international products.

2.Indication Expansion: Based on its unique B-cell depletion capability, UC101 is planned for exploratory research in autoimmune diseases.

3. Internationalization Process: Dual Filing in China and the US Demonstrates Global Competitiveness

The approval of UC101's IND in China and the United States has multiple strategic significances:

1.FDA-Recognized Milestones：The FDA approval in January 2025 marks not only the world's first regulatory breakthrough for an allogeneic CAR-T derived from umbilical cord blood but also validates UCELLO’s internationally leading capabilities in gene editing, production processes, and quality control.

2.CDE Approval of Local Value：This CDE approval marks the authoritative recognition of China's independent innovation capability in the universal CAR-T field, paving the way for subsequent indication expansion and commercialization.

IV. Industrial Synergy: Full-chain Ecosystem Boosts Technology Transformation

The development of UC101 has received deep support from the biopharmaceutical industry chain in China:

1.CDMO Partner：Many partners have provided strong support during the development process, such as: Shenzhen UCELLO Biotech Co., Ltd. contributed to the construction and screening of stable cell lines, Obio Technology provided GMP-level auxiliary cell production services, VectorBuilder offered clinical-grade lentiviral vector production services, and GenScript's eSpCas9 gene editing tool ensured the precision of gene knockout, among others.

2.Production Platform Support：Relying on the BIO-LINK Biopharmaceutical Incubator and International Parallel Laboratories of Chengdu Tianfu International Bio City, UC101 significantly reduced costs during the process development and quality research phases, accelerating the transition from laboratory to clinical application.

V. Corporate Strategy: Driving Cell Therapy from "Customized" to "Universal"

“The approval of UC101's dual IND submission in China and the U.S. marks a significant implementation of UCELLO's strategy of 'Globalization, Universality, and Accessibility.'”Professor Qiang Zou, the founder of UCELLO, stated, "Starting with acute B lymphoblastic leukemia, we will gradually expand to indications such as multiple myeloma and systemic lupus erythematosus, while exploring the potential application of CAR-T derived from umbilical cord blood in solid tumors. In the future, through technological innovation and process optimization, we hope to reduce the cost of CAR-T therapy to less than 100,000 RMB, allowing more patients to benefit from this revolutionary treatment."

UC101 Project IND Receives Approval from Both Chinese and U.S. Regulatory Agencies, Demonstrating UCELLO's Allogeneic Universal CAR-T Cell Development Technology PlatformIt has gained recognition, which means that adopting this technology platform can lead to the development of more immunologically compatible allogeneic cell therapy drugs. This breakthrough not only brings new hope to patients with acute B lymphoblastic leukemia but also marks China's transformation from "catching up" to "leading" in the global cell therapy field, bringing hope to millions of patients and their families worldwide.

E.N.D

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