Af­ter a long, hard slog to the clin­ic, stem cell play­er Vi­a­Cyte fu­els up with $105M for its next big at­tack on di­a­betes

Over the 14 years since 3 com­pa­nies merged to­geth­er to cre­ate the stem cell play­er we now call Vi­a­Cyte in San Diego, the biotech has been op­er­at­ing on a steady stream of cash from the Cal­i­for­nia In­sti­tute of Re­gen­er­a­tive Med­i­cine as well as the non-prof­it JDRF or­ga­ni­za­tion. 

But with one stem cell prod­uct in the clin­ic and a next-gen prod­uct about to go back in­to hu­man test­ing af­ter a re­vamp — plus a new­ly formed col­lab­o­ra­tion with the gene edit­ing spe­cial­ists at CRISPR Ther­a­peu­tics — CEO Paul Laikind is ready to dou­ble down on a strate­gic bet that the crew at Vi­a­Cyte can make some ma­jor clin­i­cal progress over the next 2 to 3 years.

Paul Laikind, Vi­a­Cyte

To­day Laikind is un­veil­ing an $80 mil­lion ven­ture round, the first in years, that brings to­geth­er a set of new in­vestors — Bain Cap­i­tal Life Sci­ences and joined by TPG and RA Cap­i­tal Man­age­ment —who are join­ing Sander­ling and some in­di­vid­ual back­ers on the next leg of the jour­ney. To­geth­er with the $15 mil­lion CRISPR paid them to forge their tie-up and an­oth­er $10 mil­lion from part­ners at WL Gore and As­so­ci­ates, Laikind’s team now has $105 mil­lion to fund this next leg of prod­uct de­vel­op­ment.

“With this new fund­ing we’re re­al­ly open­ing the gate, say­ing ‘lets get this done,’” says the CEO.

“We stuck to our guns of what we want to ac­com­plish here,” adds Laikind. “It’s tak­en 7-8 years to de­vel­op the tech­nol­o­gy for dif­fer­en­ti­at­ing the cells in a reg­u­la­to­ry-com­pli­ant way.” 

Pe­ter Kolchin­sky

Laikind has been woo­ing RA’s Pe­ter Kolchin­sky for some time now. And Kolchin­sky and Bain’s Adam Kop­pel have a long­stand­ing re­la­tion­ship forg­ing deals to­geth­er, which helped get this syn­di­cate to gel.

Like a lot of the stem cell sur­vivors in Cal­i­for­nia, Vi­a­Cyte has seen the burst of glo­ry and hype suc­ceed­ed by the bleak­ness of the marathon run that stem cell R&D has proven to be — just like many oth­er new tech­nolo­gies in biotech. And it’s man­aged to keep plug­ging away at it to get to the re­newed era of op­ti­mism that has been lift­ing the sur­vivor’s prospects over the last cou­ple of years.

Adam Kop­pel

The lead prod­uct at Vi­a­Cyte is called PEC-Di­rect, de­signed to fer­ry pan­cre­at­ic prog­en­i­tor cells from stem cells that can se­crete in­sulin. It’s prob­lem­at­ic, though, trig­ger­ing the kind of im­mune re­sponse that re­quires im­muno­sup­pres­sion — which nec­es­sar­i­ly lim­its its us­es to 10% of high-risk Type 1 di­a­betes pa­tients will­ing to put up with the risks.

PEC-En­cap is an en­cap­su­lat­ed prod­uct that us­es Gore-tex like ma­te­r­i­al to de­liv­er the same pan­cre­at­ic prog­en­i­tor cells with­out spark­ing the for­eign body re­sponse that spurs the body to coat their de­vice with cells. And this can be used with­out im­mune sup­pres­sion, which would sig­nif­i­cant­ly ex­pand the mar­ket for Vi­a­Cyte.

CRISPR Ther­a­peu­tics $CR­SP, one of the lead­ers in CRISPR/Cas9 gene edit­ing with a big in­ter­est in re­gen­er­a­tive med­i­cine, is step­ping in to use their im­mune-eva­sive tech to cre­ate a new line of off-the-shelf cells that can do the job with­out kick­ing in the re­jec­tion re­sponse.

There are 55 staffers at the biotech, which the CEO says will grow now that he has his big fi­nanc­ing pack­age com­plet­ed. Vi­a­Cyte does its own man­u­fac­tur­ing work, and that team will need to ex­pand along with a spe­cial group ded­i­cat­ed to the part­ner­ship with CRISPR.

5AM Ven­tures: Fu­el­ing the Next Gen­er­a­tion of In­no­va­tors

By RBC Capital Markets
With Andy Schwab, Co-Founder and Managing Partner at 5AM Ventures

Key Points

Prescription Digital Therapeutics, cell therapy technologies, and in silico medicines will be a vital part of future treatment modalities.
Unlocking the potential of the microbiome could be the missing link to better disease diagnosis.
Growing links between academia, industry, and venture capital are spinning out more innovative biotech companies.
Biotech is now seen by investors as a growth space as well as a safe haven, fuelling the recent IPO boom.

Hal Barron, GSK via YouTube

What does $29B buy you in Big Phar­ma? In Glax­o­SmithK­line’s case, a whole lot of un­com­fort­able ques­tions about the pipeline

Talk about your bad timing.

A little over a week ago, GSK R&D chief Hal Barron marked his third anniversary at the research helm by taking a turn at the virtual podium during JP Morgan to make the case that he and his team had built a valuable late-stage pipeline capable of churning out more than 10 blockbusters in the next 5 years.

And then, just days later, one of the cancer drugs he bet big on as a top prospect — bintrafusp, partnered with Merck KGaA — failed its first pivotal test in non-small cell lung cancer.

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Janet Woodcock (AP Images)

End­points poll: Janet Wood­cock takes the (in­ter­im) helm at the FDA. And a large ma­jor­i­ty of our read­ers want her to stay there

It’s official: Janet Woodcock is now the acting chief of the FDA.

And — according to an Endpoints poll — most industry readers would like her to stay there, although a significant minority is strongly opposed.

To recap: Joe Biden is reportedly choosing between Woodcock and former deputy FDA commissioner Joshua Sharfstein as his nominee for the permanent position. Given their respective track records, the decision is set to determine the agency’s lodestar for years to come.

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An Endpoints Zoom meeting; and the email header employees will see if your company is a Premium subscriber

What’s next for End­points — and how to sup­port our in­de­pen­dent bio­phar­ma news mis­sion

The firehose of biopharma news is gushing these days.

That’s why broader and deeper is the theme for 2021 at Endpoints. You can expect new coverage outside our core R&D focus, with deeper reporting in some key areas. When John Carroll and I launched Endpoints nearly five years ago, we were wading in waist-high waters. Now we’re a team of 25 full-time staffers (and growing) with plans to cover the flood of biopharma news, Endpoints-style.

Janet Woodcock and Joshua Sharfstein (AP, Images)

Poll: Should Joshua Sharf­stein or Janet Wood­cock lead the FDA from here?

It’s time for a new FDA commissioner to come on board, a rite of passage for Joe Biden’s administration that should help seal the new president’s rep on seeking out the experts to lead the government over the next 4 years.

As of now, the competition for the top job appears to have narrowed down to 2 people: The longtime CDER chief Janet Woodcock and Joshua Sharfstein, the former principal deputy at the FDA under Peggy Hamburg. Both were appointed by Barack Obama.

Fast on Glax­o­SmithK­line's heels, Au­rinia wins OK to steer a sec­ond lu­pus nephri­tis drug straight to the mar­ket

GlaxoSmithKline’s Benlysta isn’t alone in the small circle of approved lupus nephritis drugs anymore.

Little Aurinia Pharmaceuticals has gotten the green light from the FDA to start marketing its first and only program, voclosporin, under the brand name Lupkynis — something CEO Peter Greenleaf says it’s been ready to do since December.

Regulators went right down to the wire on the decision, keeping the company and the entire salesforce it’s already assembled on its toes.

Charlie Fuchs, Roche and Genentech global head of product development for oncology and hematology (Yale Cancer Center)

Yale can­cer spe­cial­ist Char­lie Fuchs tapped as new glob­al de­vel­op­ment chief for Roche/Genen­tech

Roche and their big sub Genentech have just recruited a top cancer specialist at Yale to head up global product development in oncology and hematology.

I just got word that the pharma giant, which leads one of the most active cancer research operations in the world, recruited Charlie Fuchs, director of the Yale Cancer Center and physician-in-chief of Smilow Cancer Hospital. He’ll join the global operation March 1 and will be based in South San Francisco, where Genentech is based.

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Jonathan Weissman (MIT)

Can a new CRISPR tech­nique un­lock the se­crets of how can­cer spreads?

Jonathan Weissman’s team watched the cancer cells spread across the doomed mouse. Engineered with a bioluminescent enzyme, they appeared in scans first as a small navy blue diamond lodged near the heart; a week later, as a triangle splayed across the mouse’s upper body, with streaks of green and two distinct bright red hubs of activity. By day 54, the mouse resembled a lava lamp.

The images would have been familiar to any cancer biologist, but they didn’t actually tell you much about what was going on: why the cancer was metastasizing or which cells were responsible. For that, Weissman’s team had designed a new tool. Inside the original navy blue diamond, they had engineered the microbiological equivalent of an airplane’s black box — a “molecular recorder” that, after the mouse’s death, could allow them to extract the cells and wind back intimate footage of a single cancer’s ascent.

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Kimberly Smith, ViiV R&D chief (ViiV Healthcare)

Af­ter sting­ing FDA set­back, Glax­o­SmithK­line's Vi­iV fi­nal­ly notch­es US ap­proval for long-act­ing HIV in­jec­tion

GlaxoSmithKline’s HIV unit ViiV was dealt a stinging loss back in late 2019 when the FDA slammed the brakes on its application for a once-monthly injection based on manufacturing issues. Now, with that roadblock in the rearview, ViiV has finally made good on its promise to change the HIV game.

The FDA on Thursday approved ViiV’s Cabenuva (cabotegravir and rilpivirine) as a long acting, once-monthly therapy for HIV-positive adults who are virologically suppressed and on a stable antiviral regimen.