EdiGene raises $67M to take gene-editing therapies into humans

EdiGene has raised around $67 million to advance work on gene-editing therapies. The Beijing-based biotech will use the series B funding to progress a pipeline led by a beta-thalassemia treatment and an allogeneic CAR-T therapy.

Over the past five years, EdiGene has built a preclinical pipeline using several gene-editing platforms. The work has culminated in EdiGene having an ex vivo gene-editing treatment for beta-thalassemia, ET-01, at the pre-IND stage and an off-the-shelf CAR-T prospect, ET-02, following close behind. With clinical development on the horizon, EdiGene sought funding for the next stage of its evolution.

3H Health Investment answered the call, leading a $67 million series B round with assists from new investors Sequoia Capital China, Alwin Capital and Kunlun Capital. IDG Capital, Lilly Asia Venture and other investors that pumped more than $30 million into EdiGene in earlier rounds also participated. 

The funding will support EdiGene as it becomes a clinical-phase biotech and leverages sites in China and Massachusetts to execute its vision of becoming a globally competitive gene-editing player. 

ET-01 is EdiGene’s most advanced candidate. EdiGene makes ET-01 by editing autologous CD34+ cells using CRISPR/Cas9 to disrupt the BCL11A erythroid enhancer. In doing so, EdiGene aims to elevate fetal hemoglobin to levels needed to ease the clinical symptoms of beta-thalassemia.

BCL11A is also the focus of Vertex and CRISPR Therapeutics’ CRISPR/Cas9 therapy CTX001. Earlier this year, the partners shared data showing the fetal hemoglobin levels of the first two beta-thalassemia patients to receive CTX001 rose after administration of the therapy and remained elevated for the up to 15 months of follow-up then available.

EdiGene is advancing ET-01 alongside its allogeneic CAR-T cell therapy. By editing immune-rejection molecules in T cells from healthy donors, EdiGene is aiming to create an off-the-shelf CAR-T for use in patients with hematological malignancies. A solid tumor program is at the early research stage, too.

ET-01 and the CAR-T therapy, ET-02, are based on EdiGene’s ex vivo platforms. Edigene also has an in vivo platform for RNA base editing, which it is applying to diseases such as Hurler syndrome, and a high-throughput gene editing technology designed to identify targeted therapies.