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The Readout Damian Garde & Meghana Keshavan

How CRISPR could help fight hard-to-treat cancer

CAR-T cancer therapy has led to previously unthinkable benefits for certain blood cancers, but translating that promise to solid tumors has been a rocky scientific road. Researchers have come up with a new idea that might succeed where CAR-T has faltered: Using CRISPR to turn a different kind of immune cell into a cancer-killing agent.

As Emily Mullin writes in STAT, the idea involves isolating patients’ tumors and extracting T cells called tumor-infiltrating lymphocytes, or TILs, which, true to their name, have gotten beyond cancer’s defenses. Then they use CRISPR to knock out a gene called CISH, which acts as a checkpoint on the immune system. The result: a super-powered TIL that might be potent against tumors that evade other treatments.

To find out, researchers at the University of Minnesota are recruiting patients with advanced gastrointestinal cancers to see if the CRISPR’d cells can make a difference. So far, only two patients have undergone treatment, but the researchers are optimistic they can recruit many more.

Read more.

Biogen might have wasted $800 million

Back in 2019, when Biogen traded $800 million for a company called Nightstar Therapeutics, the plan was to “advance potentially transformative gene therapy programs for rare retinal diseases.” Two years and two clinical failures later, the whole acquisition might be a write-off.

Yesterday, Biogen said a Phase 3 study of a Nightstar gene therapy did not meet its goal of improving vision for patients with choroideremia, a rare, inherited disorder that degrades eyesight. The news comes a month after Biogen disclosed that another Nightstar gene therapy, targeting the eye disease retinitis pigmentosa, failed in a mid-stage study

This news would have led to familiar questions about Biogen’s pipeline, its business development track record, and its intensifying need to find new sources of growth. But, as you might have heard, the company just won approval for an Alzheimer’s disease treatment that could become a multibillion-dollar product in the coming years. So the fate of the Nightstar acquisition is unlikely to affect the narrative around Biogen.

There’s no going back from Warp Speed

Operation Warp Speed brought us a bunch of world-changing vaccines and one indelible question: Why can’t we do this all the time?

To BIO CEO Michelle McMurry-Heath, Warp Speed proved that with enough money and political will, the slow pace of creating new medicines becomes optional. “I hope we will never be satisfied with pre-Covid research and development timelines,” McMurry-Heath said yesterday, the first day of BIO’s 2021 conference. “We’d often assumed that science was the barrier. Now, we know better. And we must never forget.”

As STAT’s Kate Sheridan points out in her pop-up BIO newsletter, that sounds great in the abstract, but it’s worth considering that regulators have been stretched beyond their limits over the past year to make Warp Speed possible. Turning that experiment into the new normal would require sweeping investment and regulatory overhaul.

For more from the BIO conference, sign up for Kate’s free daily newsletter.

Steve Hahn, biotech VC

Former FDA Commissioner Steve Hahn is joining Flagship Pioneering, the firm said yesterday, news that will give new life to the debate over whether it’s OK for federal officials to take jobs in the industries they once regulated.

This is a common practice. Over the past 18 months alone, former FDA Commissioner Scott Gottlieb joined the board of Pfizer, and his former deputy, Amy Abernethy, took a job at Verily. Many more FDA officials spend their post-agency careers as paid consultants, advising the very companies they previously policed.

But Hahn going to Flagship, which was first reported by the Washington Post, brings an added wrinkle. Flagship founded and incubated Moderna, and Noubar Afeyan, founder of the VC firm, is chairman of Moderna’s board. Hahn led the FDA during the review and emergency authorization of Moderna’s Covid-19 vaccine. It’s easy to imagine how someone might connect the dots and draw some unflattering conclusions.

More reads

  • Why two scientific powerhouses are teaming up to tackle rare cancers long overlooked in research. (STAT)
  • Biotech rally sparked by Alzheimer’s drug has staying power. (Wall Street Journal)
  • FDA’s Cavazzoni calls for adcomms to ‘get back to the substance.' (Endpoints)
  • The approval of aducanumab isn’t the end of the story. It’s just the beginning. (STAT)

Thanks for reading! Until tomorrow,

Tuesday, June 15, 2021


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