Sem­ma steps to­ward the clin­ic af­ter demon­strat­ing ef­fect of po­ten­tial di­a­betes cure in land­mark an­i­mal stud­ies

The re­search team at the well-fund­ed Sem­ma Ther­a­peu­tics has cleared one of the last re­main­ing hur­dles to get­ting a po­ten­tial cure for di­a­betes in­to hu­man stud­ies. And if they’re right, it marks a ma­jor pre­clin­i­cal mile­stone for a resur­gent re­gen­er­a­tive med­i­cine field fo­cused on a new gen­er­a­tion of stem cell ther­a­pies.

Fe­li­cia Pagli­u­ca

Fe­li­cia Pagli­u­ca, VP of cell bi­ol­o­gy re­search and de­vel­op­ment at the Cam­bridge, MA-based biotech, told the 2019 In­ter­na­tion­al So­ci­ety for Stem Cell Re­search meet­ing in LA Sat­ur­day that their stem cell-de­rived islets per­formed as hoped for — pro­duc­ing in­sulin — in a study in­volv­ing non-hu­man pri­mates whose im­mune sys­tems had been flat­tened to pre­vent a re­jec­tion. In a sep­a­rate study in­volv­ing two pigs, a pack­age of these en­gi­neered islets con­tained in a spe­cial­ly de­signed pack­age were used suc­cess­ful­ly to gen­er­ate in­sulin with­out need­ing an im­muno­sup­pres­sant to pro­tect against a re­ac­tion.

“For the first time ever the de­vice pro­tects the cell,” Pagli­u­ca told me in a pre­view of to­day’s ses­sion, of­fer­ing ev­i­dence from a large an­i­mal mod­el that the tech­nol­o­gy func­tions with blood glu­cose lev­els, spurring in­sulin se­cre­tion as need­ed. “They re­al­ly show quite con­sis­tent re­spon­sive­ness.”

And that’s with­out fi­bro­sis, with­out cell suf­fo­ca­tion, while watch­ing the in­te­gra­tion of cell ther­a­py in­to host tis­sue.

Bas­tiano San­na

It’s ex­cit­ing, says Sem­ma CEO Bas­tiano San­na, to see the “cu­ra­tive po­ten­tial” of this cell ther­a­py.

Re­al­ly? A cure? For a mass mar­ket dis­ease like di­a­betes?

If that all seems a bit too won­der­ful to be be­lieved, think about where Pagli­u­ca is com­ing from. Stem cell ther­a­pies had their hey­day well over a decade ago as the next big thing in med­i­cine — an overnight sen­sa­tion which sput­tered out in fail­ure as the sur­vivors went back in­to the lab to do the hard work nec­es­sary to make it a re­al­i­ty. That long pe­ri­od of qui­et bred con­sid­er­able skep­ti­cism, es­pe­cial­ly af­ter the first wave of promised cures failed to ma­te­ri­al­ize. And she’s watched it play out as re­gen­er­a­tive med­i­cine made its come­back.

One of those pi­o­neer­ing sci­en­tists who stayed in the lab was Sem­ma sci­en­tif­ic founder and Har­vard pro­fes­sor Doug Melton, who pub­lished a land­mark study 5 years ago 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. And he’d been work­ing on the cure for more than 20 years, which he start­ed fol­low­ing his son’s di­ag­no­sis of Type 1 di­a­betes. 

They’ve raised $158 mil­lion over 4 years at Sem­ma to get to this stage, stand­ing on the thresh­old of a pair of small hu­man stud­ies set to launch next year. They’ll now see if they can re­pro­duce in hu­mans what they did in the non-hu­man pri­mates and pigs: first by run­ning a small study with an im­muno­sup­pres­sant, fol­lowed by the use of their de­vice in an­oth­er study lat­er next year that will avoid im­muno­sup­pres­sants.

CAR-T and gene ther­a­pies have come along to demon­strate cu­ra­tive po­ten­tial, but the CEO says it’s been frus­trat­ing to see the first wave of these ther­a­pies tar­get­ed at tiny pa­tient pop­u­la­tions. “We’re very ex­cit­ed about bring­ing the cu­ra­tive po­ten­tial of cell ther­a­pies in­to a large in­di­ca­tion.”

They’re tak­ing it step by step.

No­body is rolling out a mass tri­al for Type 2 di­a­betes. The first goal is to go af­ter some of the Type 1 di­a­betes pa­tients who are the hard­est to treat, with nowhere left to turn to rein in the au­toim­mune dis­ease. Af­ter that, they can turn to the broad­er Type 1 pop­u­la­tion be­fore mov­ing on, per­haps in­to par­tic­u­lar sub­groups of Type 2.

Ini­tial­ly, the goal will be to get the islets to do the work need­ed to safe­ly pro­duce in­sulin for these pa­tients at a re­li­able lev­el. These stem cell-de­rived islets will have to be able to be man­u­fac­tured at scale. And there’s work un­der­way to see if there’s a uni­ver­sal cell de­sign that can be used to avoid the need for the de­vice they use — some­thing Melton com­pares to a tea bag.

Op­ti­miz­ing Cell and Gene Ther­a­py De­vel­op­ment and Pro­duc­tion: How Tech­nol­o­gy Providers Like Corn­ing Life Sci­ences are Spurring In­no­va­tion

Remarkable advances in cell and gene therapy over the last decade offer unprecedented therapeutic promise and bring new hope for many patients facing diseases once thought incurable. However, for cell and gene therapies to reach their full potential, researchers, manufacturers, life science companies, and academics will need to work together to solve the significant challenges facing the industry.

Pfiz­er, Sarep­ta and two oth­ers sug­gest Duchenne drug safe­ty is­sues tied to "class ef­fect"

Since the first experimental Duchenne gene therapy programs came about, the space has proven rife with safety issues and patient deaths in clinical trials. Pfizer and three biotechs now think they’ve found a reason why.

The four companies suggested there may be a “class effect” causing the adverse events in Duchenne gene therapies, they wrote in a new study. They specifically highlighted how side effects in five patients across three trials, who all showed muscle weakness with cardiac involvement, were “strikingly similar.”

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Pre­sent­ing a live End­points News event: Man­ag­ing a biotech in tur­bu­lent times

Biotech is one of the smartest, best educated industries on the planet. PhDs abound. We’ve had a long enough track record to see a new generation of savvy, experienced execs coming together to run startups.

And in these times, they are being tested as never before.

Biotech is going through quite a rough patch right now. For 2 years, practically anyone with a decent resume and some half-baked ideas on biotech could start a company and get it funded. The pandemic made it easy in many ways to pull off an IPO, with traditional road shows shut down in exchange for a series of quick Zoom meetings. Generalist investors flocked as the numbers raised soared into the stratosphere.

Pearl Huang, former Cygnal Therapeutics president and CEO

UP­DAT­ED: Flag­ship builds a new start­up out of pieces from 2 of its biotechs. And a Roche vet leaves to do some­thing new

Flagship has crafted a new startup out of pieces from a pair of fledglings in the VC’s nest. And a prominent Roche veteran who ran one of the biotechs won’t be making the next leg of the journey.

The new company is called Sonata Therapeutics, which is picking up the work that Inzen was doing related to the cellular microenvironment and combining with Flagship’s Cygnal Therapeutics, which came out of stealth more than 3 years ago and put Pearl Huang — the BeiGene founder and former Roche SVP — at the helm.

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Amidst R&D reshuf­fle, Ver­tex ex­pands its pres­ence in Boston, aim­ing to be­come num­ber one

Vertex Pharmaceuticals has been one of the buzzier names in the bustling Boston biotech scene, but now the company is looking to vault to number one status — at least in terms of physical footprint.

At a ribbon cutting on Tuesday for its new Jeffrey Leiden Center for Cell and Genetic Therapies at the Boston Seaport, Vertex announced it would embark on a new project: The company will build a 344,000 square foot facility in the seaport to accommodate the company’s growing R&D needs, especially in its cell and gene therapies program.

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Martin Shkreli (Dennis Van Tine/MediaPunch/IPX)

In­fa­mous biotech ex­ec Mar­tin Shkre­li gets out of prison, hits the street

Martin Shkreli, the infamous biotech CEO who made headlines for his jeering assault on a legion of critics in and out of Congress, is back on the streets after 4 years inside a federal penitentiary.

Shkreli’s attorney put out a statement Wednesday afternoon saying that the “pharma bro” had been transferred to a halfway house in New York with a few more months to go under federal custody, slated to end September 14. Attorney Benjamin Brafman acknowledged the release and vowed that he and Shkreli are keeping quiet.

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De­spite fed­er­al ef­forts to di­ver­si­fy clin­i­cal tri­als, progress re­mains 'stag­nan­t' — re­port

While calls to diversify clinical trials have grown louder in recent years — gaining support from federal agencies such as the FDA and NIH — progress has largely stalled, according to a new report from the National Academies of Sciences, Engineering and Medicine.

Swaths of patients in racial and ethnic minority groups, as well as LGBTQIA+, pregnant and older adult populations continue to be left out of clinical trials. While some advances have been made in the last 30 years — women now account for roughly half of clinical trial participants — growth in other areas remains stagnant, according to the report, which was mandated by Congress and sponsored by the NIH.

Paul Chaplin, Bavarian Nordic president and CEO

Bavar­i­an Nordic se­cures BAR­DA con­tract for small­pox vac­cine

It seems that smallpox vaccination production is weighing on the mind of the US government. And manufacturer Bavarian Nordic is the latest company to benefit.

Just a few days after Emergent, a company that has made government contracts its lifeblood, acquired the exclusive rights to Tembexa from Chimerix, with a $225 million cash payment and an expected BARDA contract, the agency has offered a contract for smallpox vaccine production.

Frank Pallone (D-NJ), House Energy and Commerce Committee chair (Kevin Dietsch/Pool via AP Images)

House com­mit­tee unan­i­mous­ly ad­vances FDA user fee leg­is­la­tion with ac­cel­er­at­ed ap­proval tweaks

The House Energy and Commerce Committee on Wednesday offered a rare show of bipartisan support for a bill that would provide the FDA with user fees for the next five years.

The committee voted 55-0 to advance the quinquennial user fee bill to the full House floor, which if approved, will allow the FDA to use biopharma funds to hire new reviewers, and hit new marks as outlined in the user fee deals that the FDA and biopharma companies forged over the past several years.