2022-02-10| China

EdiGene Digs Into Cell Therapies with Arbor and Neukio Partnerships

by Joy Lin
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Beijing-based EdiGene has announced two separate partnerships with Arbor Biotechnologies and Neukio Biotherapeutics that will see it develop, or help to develop, ex vivo cell therapies. 

Founded in 2015, EdiGene is headquartered in Beijing, with offices in Guangzhou, Shanghai in China and Cambridge, Massachusetts in the US. It has spent the last seven years developing a pipeline of preclinical and clinical candidates using its ex vivo genome-editing platform, in vivo therapeutic programs and high throughput genome-editing screening. 

EdiGene’s ex vivo genome editing resulted in ET-01, an investigational autologous stem cell therapy that attempts to treat patients with severe beta-thalassemia by increasing fetal hemoglobin levels in red blood cells. EdiGene last November enrolled its first patient in a Phase 1 trial assessing ET-01 for severe beta-thalassemia.

Related Article: The 6 Companies Leading the Resurgence in RNA Editing R&D

Using CRISPR for Ex Vivo Engineered Cell Therapies 


EdiGene will make undisclosed upfront and milestone payments to Arbor in exchange for access to Arbor’s CRISPR gene editing technology. The company will make use of the CRISPR tech to develop ex vivo engineered cell therapies in the field of oncology. 

If the fruits of their partnership are commercialized, Arbor could stand to receive tiered royalties on net sales of the products. 

“Partnering with leading engineered cell therapy developers is a key component of Arbor’s strategy to broaden the potential of our CRISPR discovery engine beyond our wholly-owned in vivo genetic medicine approaches for the benefit of even more patients,” said Devyn Smith, CEO of Arbor.

Arbor closed $215 million in an oversubscribed Series B financing round last November, bringing its total fundraising to $300 million since its founding in 2016. It has an ongoing collab with Vertex Pharmaceuticals worth $1.2 billion across seven potential programs. The programs cover treatments ranging from type 1 diabetes to sickle cell diseases and beta thalassemia.

Related Article: Cartesian’s Off-the-shelf RNA Therapy for Myeloma Enters Clinical Trials

High-Throughput Genome Editing Screening for Immune Cell Therapies 


In its second partnership, EdiGene will leverage its high-throughput genome-editing screening platform to help Neukio develop allogeneic induced pluripotent stem cell (iPSC) and CAR natural killer (NK) cell therapies. 

Dong Wei, CEO of EdiGene, said the company’s screening platform allows genome-wide exploration of key genes in the growth and differentiation of specific cells, which could help establish a link between genetic variation and therapeutic development. 

Richard Wang, founder and CEO of Neukio, believes that the collaboration with EdiGene will yield new therapeutic mechanisms and targets and allow the company’s NK cells to treat solid tumors. 

The two companies did not disclose which indications they are pursuing, but Neukio will pay EdiGene milestone payments for clinical progress of the programs, as well as post-commercialization royalties. 

Founded last June, Shanghai-based Neukio is one of a growing number of biotech startups pursuing iPSC-derived CAR NK cell therapies. Other companies in the field include Century Therapeutics and Fate Therapeutics. 

Last July, Neukio raised $40 million in an angel round led by Lilly Asia Ventures. 

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