Astellas absorbs U.K. ocular gene therapy player Quethera

Japan’s Astellas Pharma has delved deeper into the gene therapy sector, picking up U.K. specialist Quethera for around $109 million.

The £85 million deal—split into upfront and milestone payments—gives Astellas a recombinant adeno-associated virus (rAAV) gene therapy platform headed by a potential treatment for glaucoma in preclinical development called QTA020V.

The therapy—which acts via the neurotrophin brain-derived neurotrophic factor (BDNF) signaling pathway—showed promise in a glaucoma animal model at the Association for Research in Vision and Ophthalmology (ARVO) conference last year, achieving a 61% reduction in retinal ganglion cell (RGC) loss.

Glaucoma is one of the leading causes of vision loss and, despite the use of intraocular pressure (IOP)-lowering drugs, around 13% of patients end up going blind. It’s an interesting target for gene therapy because glaucoma seems to be closely linked to reduced serum levels of BDNF. Experimental data shows that QTA020V transfects RGCs, leading to elevated levels of BDNF and one of its receptors (TrkB) that in turn seem to reduce cell degeneration.

Quethera came out of stealth mode in 2015 with financial backing from UK Innovation & Science Seed Fund and Cambridge Enterprise, the commercialization arm of the University of Cambridge.

One of its founders, industry veteran Peter Widdowson, Ph.D., who serves as the company’s CEO, said in a release that becoming part of Astellas "enables us to accelerate our evaluation of this investigational technology program to see if we can slow or prevent disease progression."

Astellas has been building a position in gene therapy for a number of years, and the Quethera deal isn’t its first play in the eye disease category. Back in 2014, the company started working with Harvard Medical School investigator and gene therapy expert Constance Cepko, Ph.D., on a gene therapy for retinitis pigmentosa and followed that two years ago with a licensing deal for another RP therapy developed by Japan’s Clino Corp.

The Japanese pharma’s CEO, Kenji Yasukawa, Ph.D., said in the release that Quethera’s rAAV program "has potential as a new therapeutic option for the treatment of refractory glaucoma through an IOP-independent mechanism."

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