Lorenz Mayr, Vector BioPharma CEO (PhoreMost)

Up­dat­ed: 'Close to the mid­dle': Ver­sant launch­es Vec­tor with 'gut­less ade­n­ovirus' de­liv­ery method

Most of the gene ther­a­py world cen­ters around vi­ral vec­tors, but a few biotechs have sprung up in re­cent years to try and avoid the safe­ty is­sues that can come with ade­no-as­so­ci­at­ed virus­es.

Alex May­weg

A new biotech has emerged to strad­dle some­where “close to the mid­dle,” in the words of Alex May­weg, Ver­sant man­ag­ing di­rec­tor and board mem­ber of the in­cu­ba­tor’s lat­est start­up, Vec­tor Bio­Phar­ma.

Built out of the work of the lab of An­dreas Plück­thun at the Uni­ver­si­ty of Zurich, Vec­tor’s vec­tors in­volve “gut­less ade­n­ovirus,” May­weg told End­points News.

“It’s a gut­less vi­ral cap­sid com­po­nent of an ade­n­ovirus, but it’s a gut­less ade­n­ovirus so you could ar­gue it’s not re­al­ly a virus any­more,” May­weg said.

Put an­oth­er way, in the words of Ver­sant part­ner and Vec­tor board mem­ber Markus En­zel­berg­er, there is “no virus ge­net­ic in­for­ma­tion any­more.”

The apt­ly named biotech emerged Wednes­day with $30 mil­lion from Ver­sant’s Basel, Switzer­land dis­cov­ery lab, which four months ago churned out cell ther­a­py start­up Cimeio Ther­a­peu­tics.

There are three key hur­dles to vi­ral vec­tors that Vec­tor thinks it can over­come: pay­load size lim­i­ta­tions, off-tar­get­ing and im­mune shield­ing.

Markus En­zel­berg­er

“The worst thing in cur­rent ge­net­ic med­i­cines is that you can have al­most cu­ra­tive ef­fects in some dis­eases that the cur­rent vec­tors can do, but you can’t re-dose, so it’s al­most heart­break­ing that you’re so lim­it­ed with re-dos­ing,” May­weg said.

Com­pared to con­ven­tion­al AAV gene ther­a­pies that have a de­liv­ery ca­pac­i­ty of about 4.7kb, Vec­tor aims to de­liv­er as much as 36kb. The en­hanced pay­load ca­pac­i­ty could at­tract phar­ma part­ners, May­weg said.

“Phar­ma of­ten when we, un­der stealth mode, reached out, they said, ‘Oh, we’d love to de­liv­er this pro­tein gene, but it’s way too big. Can you do it?’” the Ver­sant man­ag­ing di­rec­tor said. “We can do dy­s­trophin or oth­ers that are re­al­ly mas­sive, and that’s no prob­lem. So we are do­ing that.”

The im­mune shield­ing as­pect in­volves pro­teins that “to the hu­man im­mune sys­tem, this thing looks re­al­ly hu­man,” May­weg said.

Amid a broad­er biotech bear mar­ket, gene ther­a­py star­tups have been par­tic­u­lar­ly af­fect­ed, as cash crunch­es have led to mul­ti­ple rounds of lay­offs. Vec­tor ap­pears to have se­cured its funds pri­or to the win­ter dol­drums; the Se­ries A came through last year, ac­cord­ing to the start­up’s web­site.

CEO Lorenz Mayr, for­mer­ly CTO of GE Health­care’s life sci­ences unit, said in a state­ment that the biotech’s plat­form can “tar­get vir­tu­al­ly any cell sur­face epi­tope.”

An­dreas Plück­thun

May­weg, En­zel­berg­er and Mayr told End­points the vec­tors can be ap­plied to mul­ti­ple in vi­vo pro­grams across CAR-T, CAR-NK, CRISPR gene edit­ing, bis­pe­cif­ic T-cell en­gagers (BiTEs) and oth­ers.

The next step in the process is con­duct­ing pri­mate stud­ies, May­weg said.

The non-vi­ral route has spurred a few new biotechs in re­cent years. Take­da-part­nered Code Bio­ther­a­peu­tics un­veiled its $75 mil­lion Se­ries A in June for its type 1 di­a­betes and Duchenne mus­cu­lar dy­s­tro­phy pro­grams. An­oth­er biotech, Xalud Ther­a­peu­tics, is test­ing its non-vi­ral ap­proach in a mid-stage tri­al in os­teoarthri­tis.

Vec­tor’s 40-em­ploy­ee team will have tech­ni­cal proof-of-con­cept da­ta in im­muno-on­col­o­gy and genome edit­ing this year, the com­pa­ny said. In vi­vo da­ta for undis­closed lead pro­grams will be in hand be­fore next sum­mer.

Ed­i­tor’s note: This ar­ti­cle has been up­dat­ed with in­for­ma­tion from an in­ter­view with Vec­tor CEO and Ver­sant lead­ers and to clar­i­fy that the com­pa­ny’s vec­tor is vi­ral-like, not non-vi­ral.  

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 150,800+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

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.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 150,800+ biopharma pros reading Endpoints daily — and it's free.