Voyager nabs $25M cash as Novartis runs with two AAV capsids for neuro gene therapy

After spending a year looking at Voyager’s next-gen AAV capsids, Novartis has picked two of them to be the delivery vector for its gene therapy programs targeting two neurologic diseases.

The license triggers a $25 million payment — $12.5 million for each target — and opens up a road to $600 million in total milestones. But it also leaves some money off the table as Novartis, which had the option to license capsids against a total of three targets, decided not to act on the third.

“What I imagine they’ve done is to take some of their transgenes, combine that with our capsids, and see how it does in their hands,” Al Sandrock told Endpoints News.  The former Biogen R&D chief took up the CEO mantle at Voyager just days after the Novartis deal was announced.

Coming after Pfizer also selected a capsid in a separate deal and Neurocrine paid $175 million upfront to ally on the development of certain gene therapies, Novartis’ move offers a boost to the big turnaround Sandrock was brought in to oversee. The biotech, which emerged in 2014 as one of the flashy new gene therapy players out to tackle big diseases, suffered a series of setbacks with their clinical programs and ultimately decided to go back to the drawing board in 2021, pivoting back to discovery.

At the last meeting that he joined in on, Sandrock said, scientists were interested in transduction efficiency by brain region — what proportion of neurons or glial cells gets transduced depending on the capsids.

The ability to reach the brain, including difficult-to-reach regions, and reach better gene expression there compared to traditional AAV vectors is a key selling point for the library of capsids generated from Voyager’s screening platform.

The process began with millions of variants of AAV9 and AAV5, and Voyager sifted it down to about a dozen. Sandrock reckoned that Novartis might have evaluated half a dozen or 10 capsids before deciding on the current ones.

It’s unclear what stage the Novartis programs are at currently; a Novartis spokesperson declined to provide details.

Allen Nunnally

Under the deal structure, Novartis can swap out the capsids it’s selected for a substitute over the next two years, added chief business officer Allen Nunnally, as Voyager comes up with newer ones.

Novartis may also add on options to license capsids for up to two rare central nervous system targets for an additional $18 million per target — with the exact financials down the road exactly mirroring the initial deal.

Sandrock sees Voyager inking more such deals to license out its capsids for various use. The company will still keep an internal pipeline, but it will seek to partner out programs for common diseases such as Alzheimer’s or Parkinson’s.

“We’d love it if our capsids are the most commonly used in the entire industry for CNS disorders,” he said.

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