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Roche, aiming to make better gene therapies, turns to an emerging startup

Startup Dyno Therapeutics has signed a deal with Roche to develop gene therapy delivery tools that the Swiss drugmaker aims to use to make long-lasting treatments for neurological and liver diseases.The startup uses artificial intelligence to design capsids, or viral shells, capable of delivering genetic material into cells. These capsids are meant to improve upon ones found naturally, potentially leading to more effective gene therapies that are less likely to trigger an immune response. Dyno could get more than $1.8 billion in milestone payments in the alliance, though those payouts may never materialize.

Dyno is part of an emerging crop of companies, like Generation Bio, Affinia Therapeutics, StrideBio and Taysha Gene Therapies, aiming to use new technologies to overcome gene therapy’s current limitations. Prior to the Roche deal, the company had already attracted partnerships with Novartis and Sarepta, two other gene therapy leaders, that could be worth more than $2 billion combined.

Several recent setbacks notwithstanding, the gene therapy field continues to move forward, with pharmaceutical companies investing in more and more programs while simultaneously searching for ways to make the technology better.

The technical challenges are particularly complex. For instance, the most commonly used gene therapy delivery tool, the adeno-associated virus, has several flaws. Some people have pre-existing immunity to certain AAVs, rendering them ineligible for treatment. Others might produce immune responses after treatment that can blunt the gene therapy’s effects, make it impossible to receive a second dose and potentially cause worrisome side effects. Some AAVs also have trouble getting to the right tissues in the body.

While scientists and drugmakers have found workarounds, those hurdles may limit the reach and potency of AAV gene therapies for certain diseases. At the same time, however, the challenges have created opportunities for new companies to advance the field further, leading to a growing crop of startups.

Generation Bio, for instance, is developing non-viral gene therapy vectors. Affinia and StrideBio are developing new AAV vectors. Each have raised money from venture investors, gone public or formed partnerships with larger drugmakers.

Enter Dyno, which uses computational tools to attempt to precisely measure how a gene therapy capsid would fare if tested in humans. Dyno uses this technology, which it calls CapsidMap, to make synthetic AAV capsids that are meant to be optimized for gene therapy and superior to those found in nature.

Those would-be advantages haven’t been proven, but Dyno has garnered an unusual amount of industry support for a company that’s largely been in stealth mode since spinning out of Harvard in 2018 with $9 million in seed funding.

By the time Dyno formally unveiled itself in May, the company already had two deals in place with Novartis and Sarepta, aimed at developing gene therapies for eye and muscle diseases, respectively. Dyno isn’t yet making its own gene therapies, just licensing out its technology.

Now Roche, whose gene therapy ambitions became clear when it acquired Spark Therapeutics in 2019, has been added to the list.

The Swiss pharma will lean on Dyno to design capsids with “improved functional properties for gene therapy,” and then use those capsids to develop gene therapies for neurological and liver diseases, the company said. Roche noted Dyno’s improved capsids might help reduce immune responses to treatment and be easier to manufacture.

Roche said the future payments promised to Dyno could ultimately exceed $1.8 billion, consisting of payouts tied to research progress, regulatory milestones and product sales. The company didn’t break down any of those figures, however, or mention how much it is paying Dyno upfront.