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The Readout Damian Garde & Meghana Keshavan

Novartis and the Gates Foundation want to find a cheap cure for sickle cell disease

After decades of scientific neglect, there are finally therapies in development that might effectively cure sickle cell disease. But each is bespoke, complex, and, if successful, likely to be unattainable in sub-Saharan Africa, where the disease is particularly prevalent. That’s a problem Novartis and the Bill and Melinda Gates Foundation want to solve.

As STAT’s Elizabeth Cooney reports, the two announced this morning they are working together to create a treatment that can match the promise of sickle cell gene therapies now in development but without the costly scientific steps that make those treatments so expensive. Where current approaches rely on removing, engineering, and then reinfusing a patient’s own cells, the partners want to create an off-the-shelf treatment that could be readily administered.

There’s no straight line to getting there. They’ll need to invent technological solutions in order to replace each step along the way, solving for delivery, gene transfer, tolerability, and durability. As Novartis Institutes for BioMedical Research President Jay Bradner put it, “we’re going to have to reimagine what it means to be a gene therapy for this project.”

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Bluebird’s safety problem is worrisome for gene therapy

Bluebird Bio paused clinical trials for a gene therapy after two patients in its studies were diagnosed with cancer, a development that underlines long-term safety concerns affecting the whole field.

As STAT’s Adam Feuerstein reports, it remains unknown whether the two cases of blood cancer are related to Bluebird’s therapy, which uses a re-engineered HIV virus used to deliver corrective genes. It’s also possible that a chemotherapy called busulfan, used to prepare patients for gene therapy, is to blame.

Either outcome is potentially worrisome for gene therapy research. If Bluebird’s chosen virus carries the risk, however rare, of cancer, it could impact patients’ willingness to opt for gene therapy. And if the problem is busulfan, used as a pretreatment for many gene therapies and genome editing medicines, the reverberations could be wider.

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Woodcock: There’s no need for a ‘firewall’ between FDA and industry

Among the many facets of the seemingly endless controversy over Biogen’s Alzheimer’s disease treatment aducanumab is this: Is the FDA too cozy with the drug makers it’s meant to regulate?

As STAT’s Ed Silverman reports, that criticism has been led by the advocacy group Public Citizen, which, after November’s polarized FDA discussion of aducanumab, demanded the agency erect a “firewall” between its staff and employees of drug companies. Janet Woodcock, the longtime leader of the FDA’s drug-reviewing division, disagrees, arguing in a public response that doing so “would cause significant negative repercussions for public health.”

That’s not a surprising statement from Woodcock given her current job, but it’s impossible not to consider it in the context of what might be her next one. Woodcock is widely considered to be a leading candidate for FDA commissioner, a role that will likely be filled before the agency makes its final decision on Biogen’s drug. Her reported closest rival is Johns Hopkins University professor Joshua Sharfstein, a public health expert perceived to be less open to collaborating with the drug industry. And thus the debate over aducanumab, and the FDA’s process for reviewing it, has become a subplot in the process for choosing a commissioner.

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Neil Woodford is ready to invest in biotech again

Neil Woodford, the famed British stockpicker whose biotech investments proved so misguided that he lost his job and saw his fund entirely liquidated, is giving it another go.

Woodford, whose 2019 downfall was the rare story that united the Financial Times and the Daily Mail, knows he has some ground to make up when it comes to public perception. “You can imagine lots of people who have read the media about me wouldn’t want to touch me with a ten-foot disinfected bargepole,” he told the Telegraph in a lengthy and similarly colorful interview. 

But he insists he’s learned the lessons of forced liquidation. For one, he will no longer put public investors’ money into private biotech startups, which contributed to the cash crunch that doomed his earlier fund. And he won’t be raising funds from retail traders anymore, focusing instead on institutional investors. It’s important to note that this all depends on the results of an ongoing investigation by the U.K.’s Financial Conduct Authority, which is looking into Woodford’s work in recent years and reserves the right to ban him from investing outright. 

More reads

  • The myth of ‘good’ and ‘bad’ Covid vaccines: Why false perceptions overlook facts, and could breed resentment. (STAT)
  • Nektar Therapeutics holders hit board with insider trading suit. (Bloomberg Law)
  • Backed by big hospitals, a former Microsoft executive wades into the messy business of selling patient data. (STAT+)

Thanks for reading! Until tomorrow,

Wednesday, February 17, 2021

STAT

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