The Gene Editing Technology Innovation Platform -BRL Medicine

Gene Editing Technology Innovation Platform

As our core foundational technology platform, the gene editing technology innovation platform encompasses the research and application of various gene editing tools such as large fragment integration and gene activation/silencing. The platform has also carried out key process modifications, significantly improving editing efficiency and specificity. In addition, we are developing large fragment targeted integration editing technology, which has completed preliminary in vivo functional validation, creating a new foundational gene editing technology and forming a gene editing tool system. Currently, the platform demonstrates significant competitive advantages in key metrics such as editing efficiency, specificity, and safety.

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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Gene Therapy: Gene Editing Technology Innovation Platform; Hematopoietic Stem Cell Platform

Cell Therapy: Allogeneic Universal Cell Platform; Non-viral site-specific integration CAR-T platform; Enhanced T Cell Platform; In Vivo Gene Editing CAR-T Platform

Contact Us: Tel： 021-64340008; Email： info@brlmed.com; Address： Science&Technology Park, 3699 Yuanjiang Road, Minhang District, Shanghai

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