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The Readout Damian Garde & Meghana Keshavan

CRISPR could make off-the-shelf CAR-T a reality

CAR-T treatments, derived from patients’ own cells, have led to dramatic benefits for a small number of people with cancer, but the time-consuming process of crafting bespoke therapies has limited their use. CRISPR Therapeutics, using its namesake technology, believes it has a better way. And early data from a small study suggest there’s a path forward.

As STAT’s Adam Feuerstein reports, CRISPR Therapeutics’ CAR-T led to complete remissions in some patients with an advanced form of lymphoma. The study, which involved just 11 patients, resulted in four complete responses, meaning tumor cells were no longer detectable.

The results, while preliminary, are a positive sign for so-called off-the-shelf CAR-T therapies. While approved treatments from Novartis and Gilead Sciences rely on cells taken from individual patients, CRISPR Therapeutics and others are working on CAR-T treatments that can be made from the cells of healthy donors, potentially creating a more accessible medicine.

Read more.

What can biotech learn from the FDA’s calendar?

FDA decisions are high-stakes affairs for biotech investors, but the gnomic nature of the agency offers little in the way of legible tea leaves for anyone trying to guess whether a given drug is slated for approval or rejection. With that in mind, a group of analysts searched for clues in one of the few datasets we have: the FDA’s calendar.

Every would-be approved drug gets an FDA decision date, and most get scheduled for a pre-decision meeting of outside experts. Analysts at EvercoreISI wondered: Does the time between those two events tell us anything about a drug’s chances?

The answer, based on an analysis of 139 drugs, is, yeah, maybe. The shorter the period between an expert panel and a decision date, the more likely a drug is going to get rejected, according to Evercore ISI’s Umer Raffat and Mike DiFiore. And if you plot the numbers on a graph, every 100 days between a panel and a decision date adds 20% to the odds a drug wins approval, they concluded.

The brave new world of medical AI that teaches itself

To date, the FDA has only approved medical artificial intelligence products that work off of a fixed set of data, but the rapid development of AIs that adapt to their real-world use forces the agency to tackle a thorny question: How do you regulate a product that changes after it’s approved?

As STAT’s Casey Ross reports, that’s the topic of an FDA meeting slated for Thursday, when the agency will hear from patients and physicians about the issues of privacy, transparency, and oversight of so-called adaptive AI.

The agency has proposed regulating such products through a “total life-cycle approach,” which would mean keeping tabs on accuracy even after an AI is approved for clinical use. But just how that should play out — and how much say doctors and patients should have — remains to be decided.

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A plea for more Covid-19 vaccine transparency

In recent months, drug companies have taken the unusual step of publicizing the clinical trial protocols behind Covid-19 studies. But to a group of more than two dozen academics and health policy experts, they can — and should — do more.

As STAT’s Ed Silverman reports, the group is calling for an unprecedented amount of transparency, requesting protocols, amendments, meeting minutes, and statistical analysis plans, among other details. Complete disclosure will only bolster public faith in the eventual result, they argue, and, because most of the vaccines are being developed with federal funding, the companies have an obligation to be open.

“Transparency is one of the few tools at our disposal for increasing public trust in any vaccine proved safe and effective,” the group wrote in an open letter. “It will also help protect the long-term reputation of our nation’s public health agencies and enable researchers to better contribute at this moment of great public need.”

Read more.

More reads

  • AstraZeneca U.S. Covid-19 vaccine trial may resume as soon as this week. (Reuters)
  • A conversation with Exuma Biotech’s CEO about CAR-T and China’s strategic advantages. (STAT Plus)
  • Here's how Bristol Myers' CEO Giovanni Caforio completed a $13 billion buyout. (Endpoints)
  • As election nears, Trump leans on health care agencies to aid his campaign. (STAT)

Thanks for reading! Until tomorrow,

Wednesday, October 21, 2020

STAT

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