描述
Cell and Gene Therapy
R&D and Clinical Translations Solutions for CGT
As a revolutionary biomedical technology, Cell and Gene Therapy (CGT) faces unique challenges in drug R&D, manufacturing processes, and clinical translation. These "living" drugs possess dynamic characteristics, which place higher demands on product quality research and safety evaluation, requiring the establishment of a rigorous quality control system throughout the entire R&D process. Leveraging its forward-looking layout and technical accumulation in the CGT field, Sequanta has established a comprehensive solution covering the entire drug R&D cycle. In the process development and quality control phase, the company provides core services such as vector integration site analysis, gene editing efficiency assessment (including on-target/off-target effect detection), and cell genomic stability monitoring; it also possesses robust capabilitiesin vector sequence release testing, as well as cell diferentiation characterization and population analysis. In the clinicalresearch stage, key studies such as cytokine immune response detection and in vivo cell distribution tracking can beconducted. By building a standardized technical service platform, Sequanta provides comprehensive technical support for CGT drugs from early R&D to commercial production, accelerating the translation of innovative therapies.
 Comprehensive Solutions Covering the Entire Drug R&D Cycle
描述
Project Experience: Filing and Clinical Trials
  • 3 +BLA/NDA Projects
    Autologous CAR-T
  • 30 +Phase l/II/III Clinical Trials
    Autologous CAR-T, Autologous HSC (CRISPR edited), Autologous HSC (Base edited), TIL, mDAP (iPSC-derived), MSC, mRNA Tumor Vaccine, siRNA
  • 100+ IND/IIT Projects
    Autologous CAR-T, Allogeneic Universal CAR-T (CRIS, Autologous HSC (Base edited),PR edited), In Vivo CAR-T, Autologous HSC (CRISPR editedTIL, mDAP (iPSC-derived), MSC, mRNA Tumor Vaccire, siRNA, Gene Therapy (Base edited), AAV
Project Experience: Drug Types and Indications

描述
Project Experience: Cases Studies
IND Filing
In 2026, the UX-GIP001 injection, anallogeneic iPSC (Induced Pluripotent Stem Cell)-derived cell drug independently developed by Unixell for epilepsy, successfully received IND approval from the U.S. Food and Drug Administration (FDA).
In 2025, Ucello Therapeutics announced that its independently developed CD19-targeted chimeric antigen receptor (CAR) allogeneic universal T-cellinjection (UC101) received Investigational New Drug (IND) application approval from the U.S. Food and Drug Administration(FDA) on January 11, 2025.
In 2025, the IND application for UX DA001 injection, an autologous iPSC (Induced Pluripotent Stem cel) derived cell drug independenty developed by Unixellfor Parkinson's disease, was officially approved by the U.S. Food and Drug Administration (FDA), making it the world's first iPSC-derived autologous cell therapy for Parkinson's disease to enter the clinical stage in both China and the U.S.
IIT Research
In 2026, the top international academic journal Nature published online the collaborative clinical research results of CorrectsequenceTherapeutics with the The First Affiliated Hospitalof Guangxi Medical University, ShanghaiTech University, Fudan University, and ShanghaiClinical Research center, titled "Clinical application of base editing for treating B thalassaemia." This study introduced the lIT clinical trialresults of the base -editing drug CS-101 injection, developed using the transformer Base Editor (tBE), for the treatment of patients with B-thalassemia.
References: 1. happy newspaper| Congratulations to partner Yuesai Biotech's iPSC cell treatment pipeline for epilepsy receiving FDA IND approval https://mp.weixin.gq.com/s/CVD1INBBOV5oGVxPMtwnZg2. Happy news| Congratulations to partner Chengdu Yousanuo's blood-derived allogeneic universal CAR-T approval of the US FDA IND https:/mp.weixin.qq.com/s/Anc9AnlwoiHmirBiV8JG9Q3. Happy news| Congratulations to the cooperation of Tongyuesai Biological Parkinson's disease autologous cell drug UXDA001 for obtaining FDA clinical trial approval and embarking on a dual clinical journey between China and the United States https://mp.weixin.qq.com/sy9vOuWCIMWhPf9bU.Yaig4www.example.com. Clinical application of base editing for treating B-thalassaemia. Nature.2026. https://www.nature.com/articles/s41586-026-10342-9