'Miss­ing link': Bay­er, Morn­ing­side help cat­a­pult a new kind of de­liv­ery tech to cell and gene ther­a­py

Robert Mill­man

Robert Mill­man co-found­ed and led Sem­ma Ther­a­peu­tics as CEO while al­so a man­ag­ing di­rec­tor of MPM Cap­i­tal. By the time he left the stem cell ther­a­py pi­o­neer — two years be­fore it would be sold to Ver­tex — he had left VC life be­hind.

In­stead, he went around scout­ing new tech­nolo­gies, vis­it­ing with tech trans­fer of­fices and aca­d­e­mics in the Boston/Cam­bridge area to find wor­thy ideas that could ben­e­fit from his IP ex­per­tise.

And he found quite a few.

One of them is Vesi­gen Ther­a­peu­tics, which is launch­ing with $28.5 mil­lion to turn a new class of ex­tra­cel­lu­lar vesi­cles in­to pack­ag­ing and de­liv­ery tools for a va­ri­ety of car­gos — mR­NA, Cas9, base ed­i­tors, PRO­TACs, you name it.

“The bar­ri­er to be­ing a drug com­pa­ny right now is not tech­nol­o­gy or tar­gets,” he told End­points News. “There’s plen­ty of tar­gets, there’s plen­ty of tech­nol­o­gy, but there’s no way of get­ting the two of those in­to a pa­tient.”

Quan Lu

Vesi­gen’s plat­form stems from sci­en­tif­ic co-founder and Har­vard pro­fes­sor Quan Lu’s work on AR­MMs, or ar­restin do­main-con­tain­ing pro­tein 1 [AR­RDC1]-me­di­at­ed mi­crovesi­cles. As he wrote in Na­ture Com­mu­ni­ca­tions in March 2018, AR­MMs are “dis­tinct from ex­o­somes,” in that they lend them­selves to more con­trolled pro­duc­tion and car­gos that can be re­cruit­ed in­to the vesi­cle dur­ing the process of man­u­fac­tur­ing rather than put to­geth­er af­ter the fact.

“I don’t un­der­stand re­al­ly why a whole lot of ven­ture firms didn’t jump on it at the time,” Mill­man said. “I think it’s be­cause most were fo­cused on ex­o­somes and just thought that this was an­oth­er ex­o­some com­pa­ny, and there was al­ready Co­di­ak and Evox.”

But Lu showed in his pa­per de­liv­ery of func­tion p53 pro­tein to an p53 de­fi­cient an­i­mal and restor­ing p53 ac­tiv­i­ty: “To me it was rev­o­lu­tion­ary.”

And com­pared to the cur­rent work­hors­es of ge­net­ic med­i­cines such as vi­ral vec­tors and lipid nanopar­ti­cles, AR­MMs promise to pack­age big­ger and more var­ied pay­loads, get in­to more or­gans, and stay in cir­cu­la­tion longer with­out trig­ger­ing tox­i­c­i­ty.

Faced with the “Willy Won­ka-like” range of pos­si­bil­i­ties, Mill­man said the top or­der right now for his team of sev­en is to fig­ure out a man­u­fac­tur­ing set­up that would load the car­gos ef­fi­cient­ly and be cleared by reg­u­la­tors.

In the three years that it will like­ly take to get to the cusp of the clin­ic, Vesi­gen is al­so col­lab­o­rat­ing with re­searchers to show how AR­MMs can get in­to “tis­sues that no one can go to” — with im­pli­ca­tions in oc­u­lar and neu­ro­log­i­cal dis­eases as well as can­cer.

The Se­ries A, co-led by Leaps by Bay­er and Morn­ing­side Ven­tures and joined by Lin­den Lake Ven­tures and Alexan­dria Ven­ture In­vest­ments, should fund the com­pa­ny un­til 2025, when Mill­man ex­pects to have built out the team to 30.

Jür­gen Eck­hardt

It’s ex­act­ly the kind of projects that his group likes to in­vest in ear­ly, Leaps by Bay­er head Jür­gen Eck­hardt said, es­pe­cial­ly as big be­liev­ers in the cell and gene ther­a­py space who see de­liv­ery as the “miss­ing link.” For Vesi­gen, it al­so opens up op­por­tu­ni­ties to find part­ners with­in the phar­ma gi­ant’s net­work down the road.

While Mill­man is keen not to lose fo­cus, he al­so isn’t hid­ing am­bi­tions to make this “the next big one.”

“I saw all of the prob­lems — Al­ny­lam I was there for three years, we couldn’t get de­liv­ery. I worked very ear­ly at Cel­era and es­tab­lished col­lab­o­ra­tions with Io­n­is and again de­liv­ery was lim­it­ed. I was help­ing set up Verve Ther­a­peu­tics and again de­liv­ery of Cas9 was a prob­lem,” he said. “And here was an elo­quent so­lu­tion. I was just pleased to be at the right place at the right time.”

The Fac­tors Dri­ving a Rapid Evo­lu­tion of Gene & Cell Ther­a­py and CAR-T Clin­i­cal Re­search in APAC

APAC is the fastest growing region globally for cell & gene therapy trials representing more than a third of all cell & gene studies globally, with China leading in the region. 

APAC is the leading location globally for CAR-T trials with China attracting ~60% of all CAR-T trials globally between 2015-2022. The number of CAR-T trials initiated by Western companies has rapidly increased in recent years (current CAGR of about 60%), with multiple targets being explored including CD19, CD20, CD22, BCMA, CD30, CD123, CD33, CD38, and CD138.

The End­points 11; blue­bird's $3M gene ther­a­py; Bio­gen tout new neu­ro da­ta; Harsh re­views for can­cer drugs; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

Reading about John Carroll’s pick of biotech’s most promising startups has become a treasured tradition. If you ever get curious about previous classes of the Endpoints 11, you can find all of them (plus a number of our other regular specials) here.

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EMA warns of short­ages of two Boehringer heart drugs due to a spike in de­mand

The EMA is putting EU member states on alert over the shortage of two drugs that counter heart attacks due to an uptick in demand.

On Friday, the EMA sent out a warning that two Boehringer Ingelheim drugs are experiencing a shortage: Actilyse and Metalyse. The drugs are used as emergency treatments for adults experiencing acute myocardial infarction, or a heart attack, by dissolving blood clots that have formed in the blood vessels.

The End­points 11: The top pri­vate biotechs in pur­suit of new drugs. Push­ing the en­ve­lope with pow­er­ful new tech­nolo­gies

Right around the beginning of the year, we got a close-up look at what happens after a boom ripples through biotech. The crash of life sciences stocks in Q1 was heard around the world.

In the months since, we’ve seen the natural Darwinian down cycle take effect. Reverse mergers made a comeback, with more burned out shells to go public at a time IPOs and road shows are out of favor. And no doubt some of the more recent arrivals on the investing side of the business are finding greener pastures.

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Solicitor General Elizabeth Prelogar

Should SCO­TUS hear Am­gen's Repatha case? So­lic­i­tor gen­er­al says no

Back in April, Amgen said it was encouraged by the solicitor general’s anticipated review of its Supreme Court petition to rehear a Repatha patent case. They’re likely much less optimistic about the outcome now.

Solicitor General Elizabeth Prelogar wrote in a recent 27-page brief that Amgen’s arguments “lack merit and further review is not warranted.”

The case traces back to a suit filed in 2014 against Sanofi and Regeneron’s Praluent, which ended up beating Amgen’s PCSK9 blockbuster Repatha to market by a month just a year later.

Phil Sharp, Nobel Prize laureate (L), and John Carroll, Endpoints News co-CEO (via Michael Last)

The End­points 11: Fire­side chat with No­bel Prize lau­re­ate Phil Sharp

On Thursday evening in Boston I had the great good fortune to talk about the creation of the biotech industry with Nobel Prize-winning scientist Phil Sharp. I learned quite a bit about the early days of Genentech, Biogen and Alnylam, which all helped birth this unusual drug development ecosystem. And that’s why we can do things like the Endpoints 11. Here’s my talk with Phil Sharp, which you can either watch or read below.

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Klick Health gath­ers biotech and phar­ma lu­mi­nar­ies to dis­cuss in­dus­try in­no­va­tions, in­vest­ments and fu­ture

At Klick Health’s first Ideas Exchange conference with biotech and pharma industry insiders since before the pandemic began, it was no surprise many conversations included Covid topics. Yet while vaccines and treatments were discussed, so too were the effects on drug development, federal responses, health inequities — and what to do now and next.

George Yancopoulos, chief scientist and cofounder of Regeneron, opened the conference responding to a question from Acorda CEO Ron Cohen about the spotlight on the industry during Covid and some of the “flak” biopharma has taken in the past.

Neil Desai, Aadi Bioscience CEO (via YouTube)

Pre­ci­sion on­col­o­gy biotech agrees to $72M cash in­fu­sion as it seeks to in­crease rev­enue

Almost a year after the FDA gave the green light to LA-based Aadi Bioscience’s first drug, the biotech is looking to private investors to keep itself going.

The oncology player announced Thursday that it has engaged with both new and existing investors in a PIPE financing — selling 3.3 million shares at $12.50 a share, the biotech’s closing price at Nasdaq on Wednesday. The company is also selling off pre-funded warrants to purchase over 2.4 million more shares at $12.4999 per pre-funded warrant.

FDA's out­side ex­perts vote in fa­vor of Fer­ring's fe­cal trans­plant for C. dif­fi­cile, set­ting the stage for Seres

FDA’s outside advisors voted in favor of Ferring Pharmaceuticals’ RBX2660, an experimental poop-based drug implant that the company says would be the first microbiota-based live biotherapeutic to receive an FDA green light.

That was a point repeatedly discussed during the Vaccines and Related Biological Products Advisory Committee, or VRBPAC, meeting Thursday when evaluating Ferring’s fecal microbiota transplant, or FMT, for reducing the recurrence of Clostridioides difficile infection in adults who have received antibiotics. Multiple members brought up the need for a regulated product amid a landscape of unregulated FMTs already happening in clinical care.

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