Gene Therapy
The Gene Editing Technology Innovation Platform
Hematopoietic Stem Cell Platform

Gene Editing Technology Innovation Platform

Gene Editing Technology Innovation Platform

The Gene Editing Technology Innovation Platform (CRISTARS®) is dedicated to developing international leading gene editing tools, obtaining core technologies with independent intellectual property rights, and paving the important foundation for gene editing technology translation. The platform has successfully created the world-leading dual base editor and the ultra-high activity cytidine single-base editor (hyper CBE, HyCBE), which can efficiently convert two bases, improve editing activity and broaden the target range. The research results have been published in high-impact peer-review journals such as Nature Biotechnology, Nature Cell Biology, and Cell Research.

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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