Genome Editing for a Better Life
We are developing ex vivo and in vivo genome-editing therapies for a range of diseases. Our lead program, ET-01 for patients with beta thalassemia, expects to be the first to enter clinic, offering the potential for a novel, one time treatment for patients without HLA-matching donors through autologous hematopoietic stem cell transplant.
Ex vivo: ET-01
Our lead program, ET-01, is an autologous hematopoietic stem cell transplant (HSCT) therapy being developed for patients with beta thalassemia major, through genome-editing of HSCs to raise the level of fetal hemoglobin in red blood cells. Prior clinical observation has suggested that in patients with beta thalassemia, increased production of fetal hemoglobin in red blood cells can significantly ameliorate the morbidity of the disease. It is estimated that there are over 50,000 patients with beta thalassemia major in China, and 1% of them expect to live beyond 20 years old.
We have built a GMP manufacturing facility in Guangzhou, China, and expect to advance ET-01 to clinic soon.
Ex vivo: ET-02
ET-02 is a Universal (a.k.a, allogeneic) Chimeric Antigen Receptor T cell Therapy (U-CAR-T). One of the most exciting treatment breakthroughs in recent years, autologous CAR-T therapies have the potential to revolutionize our ways to treat multiple cancers. By editing selected genes involved in immunogenicity and immune-rejection, it is possible to produce U-CAR-T from healthy donors as an “off-the-shelf” therapeutic product to treat cancer patients, potentially resulting in better availability, more consistent quality and lower cost.
In vivo Therapeutics
We are developing novel RNA base-editing technologies to treat selected genetic diseases. Recently published in Nature Biotechnology by Professor Wensheng Wei’s group in Peking University in collaboration with EdiGene, the new technology, leveraging endogenous ADAR for programmable editing of RNA (LEAPER), is ideally suited for in vivo genome-editing approaches, with potential to work with multiple delivery platforms including virus-based systems and lipid nanoparticles.
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