Sarepta’s gene therapy stumbles in a key trial

Sarepta Therapeutics’ one-time treatment for Duchenne muscular dystrophy failed to meaningfully improve patients’ physical function in a key clinical trial, casting doubt on whether the gene therapy will pass FDA scrutiny.

As STAT’s Adam Feuerstein reports, a single infusion of Sarepta’s therapy, SRP-9001, led to increases in a crucial muscle protein typically missing in children born with Duchenne, but those effects didn’t translate to observable benefits one year after treatment.

The news is a bitter disappointment for families dealing with Duchenne, a rare disease with few effective treatments. It may also derail Sarepta’s plans to seek FDA approval for SRP-9001 later this year.

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What does a Democratic Senate portend for the drug industry?

Is software eating health care? And can Covid-19 vaccines withstand viral mutations?

We discuss all that and more on this week’s episode of “The Readout LOUD,” STAT’s biotech podcast. First, STAT Washington correspondent Lev Facher joins us to break down the implications of a Democrat-controlled Senate — and the aftermath of pro-Trump rioting at the Capitol. Then, our colleague Erin Brodwin calls in to talk about CES, the massive tech conference taking place next week, and what it portends for that sector’s embrace of health care. Finally, we chat with Bob Nelsen, a biotech venture capitalist and one of the few people who saw the coming pandemic with clarity months before everyone else, about what's ahead in 2021.

Listen here.

Covid-19 proved mRNA could be a vaccine. Now we’ll find out if it can treat autoimmune disease

BioNTech, inventor of a leading Covid-19 vaccine, has demonstrated that messenger RNA can rev up the immune system to fight infection. Now the company is testing whether it can do the opposite: tamp down the bodily overreactions that cause diseases like multiple sclerosis.

In a study published in Science yesterday, BioNTech’s scientists reported that infusions of synthetic mRNA led to dramatic benefits in mice bred to have autoimmune disease. The treatment, which uses the same technology behind the company’s Pfizer-partnered Covid-19 vaccine, prevented disease progression when given immediately and arrested any worsening when administered after the onset of symptoms.

BioNTech is years away from testing the idea in a human trial, and success in mice is notoriously difficult to translate when it comes to multiple sclerosis. But outside experts said the underlying idea presents a new way to correct a misfiring immune system.

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How to cope with conference separation anxiety

Last year at this time, as the denizens of the biotech industry once again pondered whether the chaos and expense of attending the forthcoming JPM week was worth it, no one imagined that these questions would be resolved by Covid-19, which was on the radar of relatively few in the U.S. 

With a virtual JPM week, there's little chaos and less expense. But it also precludes chances to make, or deepen, the personal relationships that Carin Canale-Theakston calls the "glue that helps hold the industry together" in a First Opinion. 

To counter that, she's identified a dozen or so people she would have reached out to meet at JPM for coffee or a drink, not "people I don't know but would like to," but "existing touchpoints and true industry partners," and recommends that you do the same.

Read more.

More reads

• With biotech funding at ‘all-time high,’ Scorpion scoops up $162 million. (Boston Globe)
• Sinovac coronavirus vaccine 78% effective in Brazil trial, details sparse. (Reuters)
• Generic drug makers fear a ‘skinny labeling’ court ruling will pose a huge threat. (STAT Plus)
• Biopharma leaders condemn mob violence on Capitol Hill. (Endpoints)