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. 

Virtual Event

Fierce Medtech Innovation Week

An exclusive virtual experience to explore the latest innovations in Medtech from device design to market.

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.

Source link