Genome Editing for a Better Life
We are developing ex vivo and in vivo gene-editing therapies for a range of diseases. Our lead program, ET-01 for patients with b-thalassemia, expects to enter clinic in 2019, offering the potential for a novel, safe and tolerable therapy for patients without HLA-matching donors through autologous hematopoietic stem cell transplant.
Our lead program, ET-01, is a CRISPR-based gene-editing therapy being developed for patients with b-thalassemia, through raising the level of fetal hemoglobin. Prior clinical observation has suggested that in patients with b-thalassemia, increased production of fetal hemoglobin can compensate for the defective production of the b-hemoglobin molecules.
We are currently building a GMP manufacturing facility in Guangzhou, China, and expect to advance ET-01 to clinic in 2019.
ET-02 is a CRISPR-based gene-editing therapy to produce Universal Chimeric Antigen Receptor T cell
Therapy (U-CAR-T). One of the most exciting treatment breakthroughs in recent years, autologous
CAR-T therapies have been approved to treat multiple cancers. By knocking out selected genes
involved in immunogenicity, it is possible to produce U-CAR-T from healthy donors as an
“off-the-shelf” product to treat cancer patients, potentially resulting in readily
availability, more consistent quality and lower cost.
In vivo Therapeutics
We are developing novel gene-editing technology-based therapies to treat selected diseases. To that end,
we have also been developing in vivo drug delivery platforms, including virus-based systems and lipid nanoparticles.
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