Pharmaphorum: ViGeneron to develop next-gen eye gene therapies after funding round

Germany’s ViGeneron is to advance development of its next-generation ophthalmology gene therapy pipeline after a series A financing round.

The privately-owned Munich-based biotech did not say how much it had raised from the financing round, which was led by WuXi AppTec and Sequoia Capital China.

But ViGeneron said the proceeds will allow it to accelerate its proprietary viral vector-based gene therapy platforms and drive product development in its two lead ophthalmic gene therapy drugs.

Founded in 2017, ViGeneron is a spin-off from the Ludwig-Maximilians-University in Munich, and is led by CEO and co-founder Dr Caroline Man Xu, and two scientific co-founders, Professor Dr Martin Biel and Professor Dr Stylianos Michalakis.

ViGeneron said that its pipeline addresses ophthalmic diseases that badly need new treatments, with the two lead drugs being for undisclosed indications for which there are no currently approved treatment options.

But it did say that the company’s next-generation gene therapy platforms are geared towards addressing the limitations of adeno-associated virus-based (AAV) gene therapies.

AAV-based vectors don’t tend to produce a strong immune response, and can accurately insert a DNA sequence into the host’s genome.

This makes their behaviour more predictable than retroviruses, which have a habit of inserting DNA at random points, potentially causing cancer – but AAVs cannot carry large genes limiting their use in gene editing technology.

ViGeneron has a platform dubbed vgAAV, where the company has found a way to pack split genes into the vectors, with full-length proteins produced by splicing with mRNA.

This allows for more flexibility in terms of the gene being inserted, and the technology also works in a broad range of cell types in the retina and other tissues such as the central nervous system.

A Chinese biotech, WuXi AppTec has been hunting for new investments since its first listing on the Hong Kong stock exchange late last year.

WuXi managed to raise almost $1 bn after an initial listing on the Shanghai exchange midway through last year.

Spark Therapeutics, which is in the process of being bought by Roche, paved the way for ophthalmology gene therapies with its Luxturna, approved at the end of 2017 for a rare eye disorder.