Cell Therapy
Universal Cell Platform
Quikin CAR-T Platform
Enhanced T Cell Platform
In Vivo Gene Editing CAR-T Platform

In Vivo Gene Editing CAR-T Platform

The in vivo CAR-T platform represents an innovative approach that enables direct in vivo delivery of chimeric antigen receptor (CAR) genes into a patient’s T cells, thereby generating functional CAR-T cells internally. Specifically, viral or non viral vectors (such as LNP lipid nanoparticles) are used to specifically deliver the gene fragment encoding car to T cells, which can achieve rapid, instantaneous and controllable therapeutic effect without going through the complex process of traditional in vitro separation and expansion of T cells. This platform has the core advantages of greatly simplifying the process, significantly reducing the production cost and greatly improving the accessibility. It provides a new technical path and solution for tumor and autoimmune diseases.

The development of the era of gene therapy

In 2024, Science magazine announced CAR-T therapy for autoimmune diseases as “Breakthrough of the Year”.
In 2023, FUCASO, the world's first all human targeted BCMA CAR-T therapy (Equecabtagene Autoleucel) was approved for marketing by the Chinese NMPA for the treatment of recurrent or refractory multiple myeloma (R/R MM).
In 2022, Cilta cel, the first CAR-T cell therapy for the treatment of R/R MM in China, was approved by the FDA for marketing.
In 2021, China's first CAR-T product, Aquilensai injection, was approved for marketing by the Chinese NMPA for post-treatment relapsed or refractory diffuse large B-cell lymphoma (DLBCL)
In 2020，CRISPR/Cas9 gene editing technology wins Nobel Prize in Chemistry
In June 2019, zynteglo, the world's first gene therapy drug ,was approved for marketing by the EU EMA for the treatment of thalassemia
In May 2019, Zolgensma, the world's first gene therapy drug,was approved for marketing by the FDA for the treatment of SMA
In 2018, the first CRISPR/Cas9-based in vivo gene therapy clinical trial (EDIT-101) was approved by the FDA for the treatment of Leber Congenital Amaurosis Type 10 (LCA)
In 2017, Kymriah, the world's first CAR-T product, received FDA approval to market for the treatment of relapsed or refractory acute lymphoblastic leukemia (R/R ALL)
In 2016, the first generation of single-base editors were developed
In November 2013, the third generation gene editing technology CRISPR/Cas9 (clustered regularly spaced short palindromic repeats) was introduced
In 2012, Glybera, the world's first AAV gene therapy drug, was approved for marketing by the EU EMA for the treatment of lipoprotein lipase deficiency (LPLD)
In 2010, TALENs (transcription activation-like effector nucleases), a second-generation gene editing technology, was introduced
In 2003, Gendicine, the world's first gene therapy drug, was approved for marketing by the NMPA in China for the treatment of head and neck squamous cell carcinoma (HNSCC)
In 2001, the first generation of gene editing technology ZFNs (zinc finger nucleases) was introduced
In 1990, the world's first clinical trial of gene therapy was approved by the FDA for the treatment of adenosine deaminase deficiency (ADA-SCID)
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