Paul Perreault, CSL Behring's chief executive (file photo)

In an un­ex­pect­ed twist, CSL buys world­wide rights to uniQure's PhI­II he­mo­phil­ia B gene ther­a­py in $2B-plus deal

In a surprise move, CSL Behring reported after the market closed on Wednesday that it is acquiring uniQure’s Phase III hemophilia B gene therapy with a $2 billion-plus deal fronted with $450 million in cash.

The money — which includes $1.6 billion in milestones — gives the Australian pharma giant global rights to the pivotal stage AMT-061 (etranacogene dezaparvovec), an AAV5 viral vector carrying a gene cassette with the Padua variant of Factor IX, which hemophilia B patients lack. Top-line Phase III data are expected before the end of this year from the HOPE-B study.

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Janet Woodcock (AP Images)

Janet Wood­cock to be act­ing FDA com­mis­sion­er while Biden team fi­nal­izes nom­i­nee — re­ports

Janet Woodcock is set to be the most powerful person at the FDA in less than a week.

The veteran regulator and longtime director of the Center for Drug Evaluation and Research has been tapped as acting commissioner of the FDA, according to reports by BioCentury’s Steve Usdin and Pink Sheet’s Sarah Karlin-Smith.

The appointment was requested by the incoming Biden team, Karlin-Smith added, as they sort out the nomination of a permanent successor to Stephen Hahn — whose one-year tenure has been defined by Covid-19.

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Janet Woodcock (AP Images)

Janet Wood­cock is in the run­ning for FDA com­mis­sion­er — what does that mean for the agen­cy's fu­ture?

Just a day after reports emerged that Janet Woodcock will serve as interim chief of the FDA, word has gotten out that she is also in the running for the permanent job.

The decision, as the initial wave of reactions suggest, could have dramatic implications for where the agency is headed in the next four years — if not beyond.

Woodcock, the longtime CDER director, is being vetted alongside former FDA principal deputy commissioner Joshua Sharfstein, Bloomberg reported. Already tapped as acting head of the agency, she’s set to take over from Stephen Hahn right after Biden’s inauguration next week.

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Steve Harr (L) and Hans Bishop

Paint­ing by the num­bers, Sana founders carve up a gi­ant uni­corn-sized IPO — for a biotech that has­n't quite made it to the clin­ic

Sana Biotechnology is one of those startups that was sketched in on the chalkboard day one in the shape of a unicorn.

A giant unicorn.

And from the numbers the cell therapy 2.0 play spelled out in their S-1 $SANA, it’s clear that the company founders — led by a pair of major VCs aligned with some high-profile industry figures — are hunting a big chunk of that value for themselves.

The raise they penciled in — $150 million — isn’t likely what they actually have in mind, and it doesn’t do justice to the size of their ambitions.

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Peter Thiel, Getty (Photographer: Kiyoshi Ota/Bloomberg)

Pe­ter Thiel's psy­che­delics-fo­cused ATAI ac­quires ma­jor­i­ty stake in Recog­ni­fy and its lead schiz­o­phre­nia can­di­date

Billionaire Peter Thiel has made significant and sometimes controversial pushes into life sciences over the past few years, and one of his startups out of Berlin has made a new acquisition less than two months after achieving unicorn status.

ATAI Life Sciences purchased a majority stake Tuesday in Recognify Life Sciences, a company focused on developing treatments for cognitive impairment associated with schizophrenia. The financial terms of the deal weren’t disclosed, but the acquisition follows up a $125 million Series C in November co-led by Thiel, leading to a post-money valuation of about $1 billion for ATAI.

Cog­nate dou­bles man­u­fac­tur­ing ca­pac­i­ties in Mem­phis, Eu­rope, as de­mand for cell and gene ther­a­pies sky­rock­ets

The marketplace for gene and cell manufacturing therapeutics continues to be scorching.

Cognate BioSciences, a leading CDMO specializing in gene and cell therapy technologies, announced plans Friday that will double its total manufacturing capacities at sites in both the US and Europe — in direct response to a “great demand of commercial capacity within the biologics industry.”

The company provided most details for its US expansion, which will take place at its current headquarters in Memphis, Tennessee near the Memphis International Airport — crucial, it said, as Memphis is one of the world’s busiest cargo airports. Cognate will add two separate facilities totaling 250,000-square-feet: a GMP distribution center to manage global supply chain needs, and a third site for commercial manufacturing.

Look­ing to build in red-hot vi­ral vec­tor space, Ther­mo Fish­er inks $878M deal for Bel­gian man­u­fac­tur­er's 2 plants

Between Covid-19 vaccines and gene therapies, the contract manufacturing market for viral vector tech has grown at a rapid clip. Thermo Fisher Scientific, already one of the biggest CDMOs on the block, has now made a move to buoy its EU footprint in that field.

Thermo Fisher will pay $878 million to acquire Henogen SA, Novasep’s viral vector manufacturing business, which comprises two Belgian locations in Seneffe and Gosselies that offer over 75,000 square feet of clinical and commercial manufacturing capacity, the Massachusetts company said Friday.

CEO Brett Monia (Ionis)

Can Brett Mo­nia push Io­n­is be­yond Spin­raza?

For 30 years, Brett Monia struggled as one of Ionis’ top scientists to get their antisense technology to work. Now, as CEO, he’s trying to use it to turn Ionis into one of the industry’s biggest biotechs.

Monia, one of the handful of young scientists who in 1989 followed Stanley Crooke across the country from SmithKline (now GSK) in Philadelphia to found Ionis in Northern California, replaced Crooke as CEO last January. By then, they had proven antisense, an RNA-based method for manipulating gene expression, could work dramatically well in at least some instances, transforming spinal muscular atrophy with the Biogen-partnered blockbuster Spinraza.

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David Kessler in April 2009 (Eric Risberg/AP Images)

Covid-19 roundup: Hack­ers start re­leas­ing 'ma­nip­u­lat­ed' Covid-19 vac­cine docs; Ex-FDA com­mish David Kessler to re­place Mon­cef Slaoui as Op­er­a­tion Warp Speed chief — re­port

There’s a new twist on the EMA Covid-19 hacking story.

Friday the European agency put out the 5th in a series of statements about the hackers who broke into their system, noting that some of the information on vaccines that was gleaned in the attack is showing up online — altered to raise questions about the Covid-19 vaccines now in use.

This included internal/confidential email correspondence dating from November, relating to evaluation processes for COVID-19 vaccines. Some of the correspondence has been manipulated by the perpetrators prior to publication in a way which could undermine trust in vaccines.

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Stefan Oelrich (Bayer)

What can a 157-year-old phar­ma gi­ant bring to the ta­ble of cell and gene ther­a­pies? Quite a bit, Bay­er says

By now, Bayer has sketched out in bold strokes some grand plans for cell and gene therapy, cemented by big-dollar acquisitions of platform companies.

But just how do you stitch together a new unit bursting with the newest ideas within a storied pharma?

Stefan Oelrich, the head of Bayer’s pharma division, briefly lifted the curtain and spotlighted three key factors as he took the stage on a virtual media day, flanked by Emile Nuwaysir and Sheila Mikhail, the chiefs of BlueRock and AskBio, who each introduced their work in a way you’d expect from a biotech CEO.