USA – Avista Therapeutics, a University of Pittsburgh Medical Center (UPMC) spinout, has entered a collaboration with Roche for developing new AAV gene therapy vectors for eyes.

The alliance plans to leverage the single-cell adeno-associated virus (AAV) engineering (scAAVengr) platform technology of Avista to develop intravitreal AAV capsids that match Roche-defined profile.

According to the deal, if Roche takes up its right to license the vectors, it will handle further work, from preclinical to commercialization, and hand over milestones that could swell the deal value to north of US$1 billion.

Roche will use these new capsids to carry out preclinical, clinical and marketing activities for gene therapy programmes.

These capsids will be different from the internal pipeline of Avista. Roche sees promise in the approach, leading it to pay US$7.5 million upfront to work with Avista.

Roche will also make further payments to Avista during the collaboration’s research phase and clinical, sales-based milestone and royalty payments for resulting products.

Avista’s computationally steered, in vivo scAAVengr platform uses a high-throughput method with the integrated quantitative validation of new cell-specific AAVs.

The alliance plans to leverage the single-cell adeno-associated virus (AAV) engineering (scAAVengr) platform technology of Avista to develop intravitreal AAV capsids that match Roche-defined profile.

This aids in quickly advancing transformative gene therapies to clinical trials for ophthalmic ailments.

In addition, the company will create a pipeline based on AAV variants’ toolkit that can act on gene delivery to separate retinal cell types.

By tagging AAV vectors with genetic barcodes, scAAVengr enables Avista to track and quantify multiple vectors simultaneously in cells and animal models.

The quantification supports the ranking of the vectors by their ability to infect different cell types and deliver their genetic payloads.

Avista Therapeutics CEO Robert Lin said: “We are excited to enter into this collaboration with Roche, a global leader in health care.

“This collaboration will complement our in-house pipeline and will accelerate the delivery of transformative therapies to patients.

The company raised US$10m in seed funding and received UPMC Enterprises’ foundational support.

Meanwhile, Roche’s fast-growing eye drug Vabysmo is set to debut in the EU market after the European Medicines Agency (EMA) gave it a positive vote, portending a duel with Bayer and Regeneron’s Eylea in the US and Europe.

Vabysmo provides a new approach to treating vision-threatening retinal conditions by targeting and inhibiting two disease pathways linked to these conditions by neutralizing angiopoietin-2 (Ang-2) and vascular endothelial growth factor-A (VEGF-A).

Ang-2 and VEGF-A are thought to contribute to vision loss by destabilizing blood vessels, which may cause new leaky blood vessels to form and increase inflammation.

Additional research is being conducted to explore the Ang-2 pathway and its role in retinal disease.

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