Look­ing to cure Type 1 di­a­betes, in­vestors front $114M to launch a pi­o­neer­ing hu­man study at Sem­ma

Three years ago, Har­vard’s Doug Melton pub­lished a land­mark study out­lin­ing how he had suc­cess­ful­ly used stem cells to cre­ate in­sulin-pro­duc­ing pan­cre­at­ic be­ta cells that were in­sert­ed in bulk in­to mice and suc­cess­ful­ly pro­tect­ed from an im­mune re­sponse — a break­through in re­gen­er­a­tive med­i­cine that bore re­al promise to pro­vide a cu­ra­tive ap­proach for Type 1 di­a­betes that could con­ceiv­ably end a life­time of in­sulin shots.

It was the cul­mi­na­tion of 23 years of lab work, launched when his son was di­ag­nosed with Type 1 di­a­betes. And that achieve­ment marked the be­gin­ning of some­thing new in biotech.

That same year Sem­ma Ther­a­peu­tics would be launched — with a $44 mil­lion A round land­ing in 2015 — in pur­suit of a mis­sion to com­plete one of the most am­bi­tious pre­clin­i­cal pro­grams in the re­gen­er­a­tive med field. And af­ter work­ing on all the nit­ty grit­ty re­search need­ed to see if this tech could be scaled up to hu­man size, an ex­pand­ed syn­di­cate of ven­ture in­vestors have put to­geth­er a whop­ping $114 mil­lion round with plans to take this in­to hu­mans for a first-of-its-kind proof-of-con­cept study.

One of the big chal­lenges Sem­ma faced in scal­ing up, Melton tells me, was to cre­ate a mem­brane specif­i­cal­ly de­signed with pores that were large enough for mol­e­cules to pass through but too small for im­mune cells to pen­e­trate. Us­ing some cal­cu­la­tions from the lab, Melton and his col­leagues es­ti­mat­ed that they would need some 150 mil­lion cells — pos­si­bly rang­ing up to three times that amount — in or­der to pro­vide the nat­ur­al in­sulin need­ed to elim­i­nate the shots.

Melton com­pares the mem­brane to a tea bag, but one that couldn’t be over­loaded. The re­place­ment cells, he said, “will on­ly se­crete the right amount de­pend­ing on the lev­el of sug­ar in the blood.”

Mark Fish­man

The big round marks an in­flec­tion point for the 35 staffers at Sem­ma, says chair­man Mark Fish­man, who joined Har­vard af­ter a 13-year stint run­ning the No­var­tis In­sti­tutes for Bio­Med­ical Re­search.

“Un­til you get this kind of fund­ing,” says Fish­man, “you don’t know how broad your strat­e­gy can be. With this fund­ing, we can get through a proof-of-con­cept tri­al, with enough in­for­ma­tion to know whether this works.” They can fol­low par­al­lel tracks and al­so start think­ing through some new di­rec­tions to pur­sue as their di­a­betes treat­ment pro­ceeds.

Typ­i­cal­ly, you nev­er see VCs back­ing a di­a­betes play. A few ma­jor multi­na­tion­als con­trol the bulk of the de­vel­op­ment work be­cause the reg­u­la­to­ry re­quire­ments for ap­proval are daunt­ing. But that’s in Type 2, which is spread­ing at epi­dem­ic pro­por­tions. In­her­it­ed Type 1 di­a­betes has a much small­er pop­u­la­tion, adds Fish­man, which makes it pos­si­ble to con­sid­er push­ing ahead in­to late-stage de­vel­op­ment alone.

As for time­lines, Fish­man is play­ing his cards close to the vest. Ear­ly-stage re­search, as he knows all too well, has a lot of vari­ables that can af­fect time­lines. An IND is com­ing, he says, and the com­pa­ny will see how it plays out, with a spe­cial fo­cus in start­ing to look at how durable a sin­gle treat­ment can be — one of the the big is­sues that Melton is most in­trigued by.

The mega-round in play al­so un­der­scores the will­ing­ness of ven­ture back­ers to go big these days when they’re fo­cused on mak­ing a pi­o­neer­ing ad­vance.

Eight Roads Ven­tures and Cowen Health­care In­vest­ments co-led the fi­nanc­ing with help from MPM Cap­i­tal, F-Prime Cap­i­tal Part­ners and Arch Ven­ture Part­ners. Ex­ist­ing strate­gic part­ners in­clude No­var­tis, Medtron­ic and the JDRF T1D Fund, and new in­vestors jump­ing in in­clude ORI Health­care Fund, Wu Cap­i­tal, 6 Di­men­sions Cap­i­tal and SinoPharm Cap­i­tal. Sem­ma named Daniel Auer­bach from Eight Roads and Kevin Raidy from Cowen to its board of di­rec­tors.

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.

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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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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Covid-19 man­u­fac­tur­ing roundup: Mary­land looks to grow biotech ca­pac­i­ty with $400M check; Rus­sia lands sec­ond Sput­nik V part­ner this week

A Maryland real estate project has added three new biotech-focused manufacturing and research buildings to an office park to keep up with demand created by the pandemic, the Washington Business Journal reported.

The Milestone Business Park — located off of I-270 in Germantown, MD — will see the new buildings and a total of 532,000 square feet as the campus rebrands to Milestone Innovation Park.

Saurabh Saha at Endpoints News' #BIO19

On the heels of $250M launch, Centes­sa barges ahead with an IPO to fu­el its 10-in-1 Medicxi pipeline

Francesco De Rubertis made no secret of IPO plans for Centessa, his 10-in-1 legacy play. Barely two months later, the S-1 is in.

The hot-off-the-press filing depicts the same grand vision that the longtime VC touted when he did the rounds in February: Take the asset-centric mindset that he’s been preaching at Medicxi over the years, and roll up a bunch of biotech upstarts, with unrelated risk profiles, into 1 pharma company that can carry on the development at scale.

Steffen Schuster, ITM CEO

Ra­dio­phar­ma re­mains hot as Ger­many's ITM rais­es $109M to ad­vance neu­roen­docrine can­cer pro­gram

The world of radiopharmaceuticals has been heating up over the last few years, and Thursday saw another company focused on the field pull in a new nine-figure raise.

Germany’s ITM, or Isotopen Technologien München, scored a $109 million round of loan financing to push forward its precision oncology pipeline and fund late-stage development for its lead program. As part of the agreement, the loan will convert to shares in the event of future financial or corporate transactions, ITM said.

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.