Joël de Beer, Anjarium Biosciences CSO

Pfiz­er backs a small Swiss up­start's at­tempt to dis­cov­er a non-vi­ral gene ther­a­py

Joël de Beer made it just a few months in­to his PhD pro­gram at ETH Zurich be­fore he dropped out to join the hunt for the holy grail of gene ther­a­py — a non-vi­ral de­liv­ery mech­a­nism.

Now that he’s raised $61 mil­lion from some big-name back­ers — in­clud­ing Pfiz­er’s ven­ture arm — to aid that quest, the pres­sure is on.

An­jar­i­um Bio­sciences un­cloaked Thurs­day morn­ing af­ter four years in stealth mode, sport­ing a Se­ries A round co-led by Abing­worth and Gimv. The team of five has set up shop in a pic­turesque vil­lage about 40 min­utes out­side of Zurich to solve the cur­rent chal­lenges of AAV us­ing DNA-based gene vec­tors.

“Our am­bi­tion now is to re­al­ly paint the fu­ture of the field our­selves,” de Beer, An­jar­i­um’s founder and CSO, told End­points News. 

One of the ma­jor ad­van­tages of An­jar­i­um’s ap­proach is that DNA-based vec­tors, un­like AAV, don’t trig­ger an im­mune re­sponse. With AAV, some pa­tients have a nat­ur­al im­mu­ni­ty to the vi­ral vec­tors, or, once they’re dosed with it, they mount an im­mune re­sponse, mak­ing it dif­fi­cult to re-dose.

“We don’t have any vi­ral com­po­nents in it, and hence the pre-ex­ist­ing im­mu­ni­ty, or maybe an ac­quired im­mu­ni­ty, is some­thing that we are not ex­pect­ing,” de Beer told End­points News. “Mean­ing that you could start to have in­di­vid­u­al­ized dos­ing, de­pend­ing on, let’s say, (the) age of a pa­tient, (or) their lifestyle.”

The abil­i­ty to re-dose could al­low sci­en­tists to treat some peo­ple with a low­er dose to start, po­ten­tial­ly avoid­ing some of the safe­ty is­sues in­volved with giv­ing high­er dos­es. There’s al­so the po­ten­tial for an in­creased pay­load ca­pac­i­ty and more tar­get­ed de­liv­ery, de Beer ex­plained.

An­jar­i­um’s de­liv­ery plat­form in­cludes what the com­pa­ny calls its Hy­bri­do­some tech, which com­bines a lipid nanopar­ti­cle and an ex­tra­cel­lu­lar vesi­cle.

“Of­ten­times when you’re try­ing to tack­le very com­plex prob­lems, see what Moth­er Na­ture has giv­en you as a so­lu­tion … and try to mim­ic that,” de Beer said. “So that’s where the ex­tra­cel­lu­lar vesi­cle com­po­nent comes in­to play, where we can hope­ful­ly bank on 3 bil­lion years of evo­lu­tion to re­al­ly im­prove that de­liv­ery, which you can then … fur­ther en­gi­neer in view of that syn­thet­ic bi­ol­o­gy rev­o­lu­tion, which is go­ing on right now.”

An­jar­i­um is far from alone in the search for gene ther­a­py 2.0. Just a few months ago, Glax­o­SmithK­line vet Bri­an McVeigh launched Code Bio­ther­a­peu­tics with $10 mil­lion in seed fund­ing to de­vel­op a syn­thet­ic DNA-based vec­tor. Gen­er­a­tion Bio closed a $230 mil­lion IPO last June to fund its pre­clin­i­cal push for a non-vi­ral gene ther­a­py to treat dis­eases of the liv­er and reti­na. And Michael Ehlers is lead­ing a new ef­fort over at Ap­ple Tree Ven­tures.

“The bright­est minds in the world are try­ing to tack­le ex­act­ly that is­sue of not be­ing able to ap­ply such med­i­cine twice, hav­ing to get it right the first time,” de Beer said.

The Se­ries A round, which al­so saw par­tic­i­pa­tion from Omega Funds and Sur­vey­or Cap­i­tal, will be used to ex­pand An­jar­i­um’s team and progress sev­er­al pro­grams to­ward the clin­ic. De Beer de­clined to re­veal the com­pa­ny’s tar­gets just yet, adding that they won’t be in the clin­ic for at least an­oth­er two years.

Biotech and Big Phar­ma: A blue­print for a suc­cess­ful part­ner­ship

Strategic partnerships have long been an important contributor to how drugs are discovered and developed. For decades, big pharma companies have been forming alliances with biotech innovators to increase R&D productivity, expand geographical reach and better manage late-stage commercialization costs.

Noël Brown, Managing Director and Head of Biotechnology Investment Banking, and Greg Wiederrecht, Ph.D., Managing Director in the Global Healthcare Investment Banking Group at RBC Capital Markets, are no strangers to the importance of these tie-ups. Noël has over 20 years of investment banking experience in the industry. Before moving to the banking world in 2015, Greg was the Vice President and Head of External Scientific Affairs (ESA) at Merck, where he was responsible for the scientific assessment of strategic partnership opportunities worldwide.

Credit: Shutterstock

How Chi­na turned the ta­bles on bio­phar­ma's glob­al deal­mak­ing

Fenlai Tan still gets chills thinking about the darkest day of his life.

Three out of eight lung cancer patients who received a tyrosine kinase inhibitor developed by his company, Betta Pharma, died in the span of a month. Tan, the chief medical officer, was summoned to Peking Union Medical College Hospital, where the head of the clinical trial department told him that the trial investigators would be conducting an autopsy to see if the patients had died of the disease — they were all very sick by the time they enrolled — or of interstitial lung disease, a deadly side effect tied to the TKI class that’s been reported in Japan.

No­var­tis' sec­ond at­tempt to repli­cate a stun­ning can­cer re­sult falls flat

Novartis’ hopes of turning one of the most surprising trial data points of the last decade into a lung cancer drug has taken another setback.

The Swiss pharma announced Monday that its IL-1 inhibitor canakinumab did not significantly extend the lives or slow the disease progression of patients with previously untreated locally advanced or metastatic non-small cell lung cancer when compared to standard of-care alone.

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Peter Nell, Mammoth Biosciences CBO

UP­DAT­ED: Jen­nifer Doud­na spin­out inks a Mam­moth CRISPR deal with Ver­tex worth near­ly $700M

When a company gets its start in gene editing pioneer Jennifer Doudna’s lab, it’s bound to make headlines. But three years in, the fanfare still hasn’t died down for Mammoth Biosciences. Now, the Brisbane, CA-based company is cheering on its first major R&D pact.

Mammoth unveiled a nearly $700 million deal with Vertex on Tuesday morning, good for the development of in vivo gene therapies for two mystery diseases. The stars of the show are Mammoth’s ultra-small CRISPR systems, including two Cas enzymes licensed from Doudna’s lab over the past couple years, Cas14 and Casɸ.

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James Peyer, Cambrian CEO

Brent Saun­ders joins $100M Se­ries C for a com­pa­ny out to be the Bridge­Bio of ag­ing

About a year ago, James Peyer, a CEO and co-founder of the little known longevity biotech Cambrian Biopharma, was trying to find some R&D talent last year when he met with more than a bit of experience in that department: David Nicholson, the former R&D chief of the erstwhile pharma giant Allergan.

It turned out Nicholson already had an interest in Peyer’s field. In their Allergan days, he and COO Brent Saunders held weekly meetups where they tried to figure out how to take the company’s dominance in aesthetics — which, until recently, was often what people meant by anti-aging science — and expertise with more traditional drug development, and use it to make drugs that extend people’s lifespan.

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An­gion's or­gan dam­age drug strikes out again, this time in high-risk kid­ney trans­plant pa­tients

After flopping a test in Covid-19 earlier this year, Angion’s lead organ damage drug has now hit the skids again in kidney transplant patients.

Angion and partner Vifor Pharma’s ANG-3777 failed to beat out placebo in terms of improving eGFR, a measure of kidney function, in patients who had received a deceased donor kidney transplant and were at high risk of developing what is known as delayed graft function, according to Phase III results released Tuesday.

(Photo courtesy Pfizer)

FDA's vac­cine ad­comm votes al­most unan­i­mous­ly in fa­vor of Pfiz­er's Covid-19 vac­cine for younger chil­dren

The FDA’s Vaccines and Related Biological Products Advisory Committee on Tuesday voted 17-0, with one panelist abstaining, that the benefits of the Pfizer-BioNTech Covid-19 vaccine outweigh the risks for children between the ages of five and 12.

The vote will likely trigger a process that could allow the shots to begin rolling out as early as next week.

The vaccine, which is one-third of the adult Pfizer dose, proved to be about 90% effective in a placebo-controlled trial in which about 1,500 kids in this age range received the vaccine, and only about 12% of those receiving the vaccine had any adverse event. All serious adverse events in the trial were unrelated to the vaccine.

Stéphane Bancel, Moderna CEO (Steven Ferdman/Getty Images)

Mod­er­na chips in fur­ther on African vac­cine sup­ply — but ad­vo­cates are call­ing for even more

In a sign of its growing commitment to the continent, Moderna will supply up to 110 million doses of its Covid-19 vaccine to the African Union, the company announced Tuesday. And CEO Stéphane Bancel said it’s just the first step.

“We believe our vaccine can play an important role in addressing the needs of low-income countries given its combination of high Phase 3 efficacy against COVID-19, strong durability in the real-world evidence, and superior storage and handling conditions. We recognize that access to COVID-19 vaccines continues to be a challenge in many parts of the world and we remain committed to helping to protect as many people as possible around the globe,” Bancel said in a statement.

An image of Alzheimer's brain tissue. The red show gingipains, a protein from P. gingivalis, intermixing with neurons (yellow) and glial cells (green)

An Alzheimer's dark­horse fails its first big tri­al, but of­fers hope for a long-over­looked hy­poth­e­sis

Three years ago, Cortexyme emerged out of obscurity with some big-name backers and an unorthodox approach to treating Alzheimer’s.

They moved their drug into a pivotal study the next year, offering one of the first major tests for a hypothesis that has fluttered on the outskirts of Alzheimer’s research for decades: that, in many cases, the disease is driven by infectious agents — the havoc they wreak in the brain and the inflammation the body uses to try to fend them off. And that quashing the infection could slow patients’ cognitive decline.

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