Hunting for next big eye disease drug, Regeneron gets deeper into gene therapies with ViGeneron pact

Regeneron is dipping its toe deeper into the gene therapy waters, teaming up with ViGeneron to work on a retinal disease prospect based on engineered recombinant adeno-associated virus vectors (vgAAVs). 

While best known for its antibody work, Regeneron has a history of exploring other modalities through dealmaking, inking a $640 million gene therapy pact with Avalanche Biotechnologies back in 2014 and, more recently, partnering with Decibel Therapeutics on a hearing loss candidate. The Avalanche deal failed to give Regeneron the hoped-for successor to Eylea but it clearly remains open to the modality.

That openness has manifested in a deal with ViGeneron, a German biotech with a platform designed to overcome the limitations of AAV-based gene therapies. ViGeneron and Regeneron will work to develop vgAAV candidates against an undisclosed inherited retinal disease target.

The agreement gives Regeneron the option to exclusively license a product against the target. In return for that option, Regeneron is making an upfront payment and providing research funding. Down the line, the big biotech could pay an option exercise fee, development and commercial milestones, and royalties on net sales. Neither party has disclosed the financial figures involved.

Given the early-stage nature of the single-target deal, Regeneron is likely making a fairly small wager for the chance to get a close look at one of an emerging clutch of technologies designed to improve on the vectors currently used to deliver gene therapies. In the case of ViGeneron’s vgAAVs, the vectors are tailored to the specific challenges of using gene therapies to treat eye diseases. 

As ViGeneron sees things, the sector is currently hindered by subretinal vector delivery, an approach that can get the therapy to the targeted photoreceptors but carries risks such as retinal detachment. Using its engineered vgAAVs, ViGeneron hopes to create gene therapies that cross biological barriers, enabling intravitreal injections that minimize the risks while making transduction of target cells more efficient.

Led by CEO Caroline Man Xu, formerly of Novartis, ViGeneron is using the technology to develop a gene therapy treatment for retinitis pigmentosa that is at the IND-enabling stage. ViGeneron has earlier-stage programs targeting inherited retinal diseases and age-related macular degeneration.