Book ex­cerpt: How Hen­ri Ter­meer built Gen­zyme and pi­o­neered the rare dis­ease in­dus­try

It wasn’t that long ago when the notion of a startup company going after a rare disease with a total addressable market of a few thousand sick patients was a non-starter.

It was Genzyme founder Henri Termeer who changed all that. And today I’m pleased to share with readers an excerpt from a new book that examines how he built his groundbreaking company.

Termeer’s legacy rests on growing Genzyme into a world leader in orphan drug R&D, developing drugs for tiny patient populations, saving lives and pioneering an aggressive pricing strategy that made the field a popular focus among a whole generation of startups.

He was a mentor and friend to many leaders in the industry today. After selling Genzyme to Sanofi, he stayed active in biotech, serving on boards at Moderna, Verastem and others and investing part of the $158 million windfall of cash he earned in the Sanofi buyout in a long lineup of upstarts.

The book is titled Conscience and Courage: How Visionary CEO Henri Termeer Built a Biotech Giant and Pioneered the Rare Disease Industry and you can buy it from Amazon here or directly from the publisher. — Arsalan Arif

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Qual­i­ty Con­trol in Cell and Gene Ther­a­py – What’s Re­al­ly at Stake?

In early 2021, Bluebird Bio was forced to suspend clinical trials of its gene therapy for sickle cell disease after two patients in the trial developed cancer. As company scientists rushed to assess whether there was any causal link between the therapy and the cancer cases, Bluebird’s stock value plummeted – as did those of multiple other biopharma companies developing similar therapies.

While investigations concluded that the gene therapy was unlikely to have caused cancer, investors and the public may be more skittish regarding the safety of gene and cell therapies after this episode. This recent example highlights how delicate the fields of cell and gene therapy remain today, even as they show great promise.

Law pro­fes­sors call for FDA to dis­close all safe­ty and ef­fi­ca­cy da­ta for drugs

Back in early 2018 when Scott Gottlieb led the FDA, there was a moment when the agency seemed poised to release redacted complete response letters and other previously undisclosed data. But that initiative never gained steam.

Now, a growing chorus of researchers are finding that a dearth of public data on clinical trials and pharmaceuticals means industry and the FDA cannot be held accountable, two law professors from Yale and New York University write in an article published Wednesday in the California Law Review.

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Novavax CEO Stanley Erck at the White House in 2020 (Andrew Harnik, AP Images)

As fears mount over J&J and As­traZeneca, No­vavax en­ters a shaky spot­light

As concerns rise around the J&J and AstraZeneca vaccines, global attention is increasingly turning to the little, 33-year-old, productless, bankruptcy-flirting biotech that could: Novavax.

In the now 16-month race to develop and deploy Covid-19 vaccines, Novavax has at times seemed like the pandemic’s most unsuspecting frontrunner and at times like an overhyped also-ran. Although they started the pandemic with only enough cash to last 6 months, they leveraged old connections and believers into $2 billion and emerged last summer with data experts said surpassed Pfizer and Moderna. They unveiled plans to quickly scale to 2 billion doses. Then they couldn’t even make enough material to run their US trial and watched four other companies beat them to the finish line.

FDA of­fers scathing re­view of Emer­gent plan­t's san­i­tary con­di­tions, em­ploy­ee train­ing af­ter halt­ing pro­duc­tion

The FDA wrapped up its inspection of Emergent’s troubled vaccine manufacturing plant in Baltimore on Tuesday, after halting production there on Monday. By Wednesday morning, the agency already released a series of scathing observations on the cross contamination, sanitary issues and lack of staff training that caused the contract manufacturer to dispose of millions of AstraZeneca and J&J vaccine doses.

Brad Bolzon (Versant)

Ver­sant pulls the wraps off of near­ly $1B in 3 new funds out to build the next fleet of biotech star­tups. And this new gen­er­a­tion is built for speed

Brad Bolzon has an apology to offer by way of introducing a set of 3 new funds that together pack a $950 million wallop in new biotech creation and growth.

“I want to apologize,” says the Versant chairman and managing partner, laughing a little in the intro, “that we don’t have anything fancy or flashy to tell you about our new fund. Same team, around the same amount of capital, same investment strategy. If it ain’t broke, don’t fix it.”

But then there’s the flip side, where everything has changed. Or at least speeded into a relative blur. Here’s Bolzon:

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Jenny Rooke (Genoa Ventures)

Ear­ly Zymer­gen in­vestor Jen­ny Rooke re­flects on 'chimeras' in biotech, what it takes to spot a $500M gem

When Jenny Rooke first heard of Zymergen back in 2014, she knew she was looking at something different and exciting. The Emeryville, CA biotech held the promise of blending biology and technology to solve a huge unmet need for cost-effective chemicals — of all things — and a stellar founding team to boot.

But back then, West Coast venture capitalists didn’t see in Zymergen the one thing they were looking for in a winning biotech: therapeutic potential. Rooke, however, saw an opportunity and made her bets. Seven years later, that bet is paying off in a big way.

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Sushil Patel, Replimune

Ex­clu­sive: Genen­tech's for­mer Tecen­triq 'CEO' jumps ship to head up com­mer­cial at an on­colyt­ic virus play­er

Genentech has long been a headhunter’s paradise with biotechs shopping for top execs with premium résumés, so it’s not unusual to see some long-time employees jumping ship. Now, one of the commercial leads on the company’s oncology portfolio is leaving after a 20-year stint to try his hand at a tiny oncolytic virus biotech.

Sushil Patel, Genentech’s former global oncology franchise head for lung and skin cancer and rare/agnostic tumor types, will leave his post at the world’s first biotech to head commercial at a much smaller operation in Replimune, which is using oncolytic viruses to hunt solid tumors.

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Sen. Patty Murray (D-WA) (Graeme Sloan/Sipa USA/Sipa via AP Images)

Sen­a­tors to NIH: Do more to pro­tect US bio­med­ical re­search from for­eign in­flu­ence

Although Thursday’s Senate health committee hearing was focused on how foreign countries and adversaries might be trying to steal or negatively influence biomedical research in the US, the only country mentioned by the senators and expert witnesses was China.

Committee chair Patty Murray (D-WA) made clear in her opening remarks that the US cannot “let the few instances of bad actors” overshadow the hard work of the many immigrant researchers in the US, many of which have won Nobel prizes for their work. But she also said, “There is more the NIH can be doing here.”

Kåre Schultz, Teva CEO (Christopher Goodney/Bloomberg via Getty Images)

Te­va CEO Kåre Schultz sees a 35% raise de­spite the com­pa­ny's con­tin­ued le­gal woes

Amid a year where revenues were mostly flat but lawsuits continued piling up, Teva CEO Kåre Schultz saw himself getting a raise.

In shareholder documents filed to the SEC on Wednesday, Teva revealed its executive compensation packages for 2020. Though his salary remained the same as each of the last two years, Schultz’s stock awards increased by about $4 million from 2019, accounting for almost all of the additional pay he received in his $15.7 million package.