Nov 5, 2021 - Economy

VC firm behind Moderna turns sights to cystic fibrosis

Illustration of an inhaler with money sticking out of the top of it.

Illustration: Aïda Amer/Axios

Venture capital firm Flagship Pioneering is best known for creating Moderna Therapeutics on whiteboards in its Cambridge, Massachusetts, office. Now it's seeking to leverage its own portfolio to treat cystic fibrosis, a genetic disorder that causes persistent lung infections and limited breathing ability.

Driving the news: Flagship secured up to $110 million in commitments from the Cystic Fibrosis Foundation, via a unique partnership that will seek to develop multiple drug candidates and bring them through human proof of concept.

  • It will focus on cures for so-called "nonsense mutations" of CF, which account for between 10% and 20% of all cases. There are thousands of them, making for a very long tail.

How it works: This is being done via a new platform called Pioneering Medicines, led by former Bristol-Myers Squibb exec Paul Biondi.

  • "We come up with the ideas ourselves, like looking at a disease like CF and thinking about ways we could innovate where the unmet need is," Biondi tells Axios.
  • "We then look at the technologies available in the Flagship ecosystem to determine what might work best, and then go to the company and ask if they'd be interested."

How it works: Pioneering Medicines provides capital, and shares in any eventual economics (such as if the drug candidate gets sold to big pharma for commercialization). In this particular case, it also would enable collaboration with the Cystic Fibrosis Foundation.

  • The basic conceit is about working in parallel. For example, an oncology-focused portfolio company can still focus on oncology while Pioneering Medicines uses its tech to look at other therapeutic areas like CF, creating more shots on goal.
  • One Flagship portfolio company already identified to work on CF is Tessera, which focuses on programmable genetics.

The bottom line: If Pioneering Medicine is successful on CF or another target area, it could create a new operational paradigm for biotech VC. And less patient suffering.

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