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.

The FDA’s Oncology Center of Excellence has been a bright spot within the agency in terms of speeding new treatments to patients. That flexibility was on full display this morning as FDA released new draft guidance spelling out exactly how oncology drug developers can fulfill both the accelerated and full approval’s requirements with just a single randomized controlled trial.

While Congress recently passed legislation that will allow FDA to require confirmatory trials to be recruiting and ongoing prior to granting an accelerated approval, the agency is now making clear that the initial trial used to win the AA, if designed appropriately, can also serve as the trial for converting the accelerated approval into a full approval.

Digital therapeutics company Better Therapeutics announced on Thursday that it’s cutting 35% of its staff as it awaits FDA clearance for its first product.

The company, which launched eight years ago, is one of a growing group of companies seeking a digital alternative to traditional medicine. The space saw a record $7.5 billion in investments in 2021, according to Chris Dokomajilar at DealForma, with uses spanning ADHD, PTSD and other indications. However, private insurers have been slow to hop on board.