Alexander Vos, VectorY CEO

Start­ing fresh in man­u­fac­tur­ing, For­bion start­up re­fu­els to steer next-gen gene ther­a­py ap­proach for ALS, Alzheimer's in­to clin­ic

For­bion laid out its case for a next-gen­er­a­tion gene ther­a­py ap­proach when it took the wraps off Vec­to­rY Ther­a­peu­tics and its vec­tor­ized an­ti­body tech in Feb­ru­ary. Now, the Dutch VC has tapped an ex­pe­ri­enced hand at cell and gene ther­a­py man­u­fac­tur­ing to steer the ship — and pulled a mar­quee syn­di­cate for a €31 mil­lion ($37.6 mil­lion) seed round.

Alexan­der Vos, the new CEO, is a ven­ture part­ner at Bio­Gen­er­a­tion Ven­tures and jumps im­me­di­ate­ly from Var­mX, a BGV port­fo­lio com­pa­ny de­vel­op­ing an an­ti­co­ag­u­lant. But be­fore that, he had led Dutch CD­MO Phar­ma­Cell for eight years un­til it was bought out by Lon­za.

There, Vos got a close-up look at the lim­its of the tra­di­tion­al gene re­place­ment ap­proach and vi­ral vec­tor man­u­fac­tur­ing, from lack of speci­fici­ty for cer­tain tis­sues to low ef­fi­cien­cy. He had seen com­pa­nies walk in with se­mi-aca­d­e­m­ic process­es that would take a long time to scale up.

That’s just not go­ing to cut it these days.

“If we are go­ing to ad­dress larg­er dis­ease ar­eas in the CNS and mus­cle dis­or­der space, we have to bring down the cost of goods and we have to get bet­ter at man­u­fac­tur­ing,” he said.

Sander van De­ven­ter

Those are the two dis­ease ar­eas that Vec­to­rY is pur­su­ing, with the hy­poth­e­sis that if you could just get past the clas­sic dis­tri­b­u­tion bar­ri­ers and ex­press an an­ti­body in­side cells through a vec­tor that en­codes for it, you can make a re­al dif­fer­ence in dis­eases like amy­otroph­ic lat­er­al scle­ro­sis, Alzheimer’s dis­ease and a va­ri­ety of mus­cle dis­or­ders.

In prov­ing that, they will al­so have to side­step the safe­ty is­sues that have trou­bled Voy­ager’s vec­tor­ized an­ti­body pro­grams in sim­i­lar neu­ro dis­eases in­clud­ing ALS, Parkin­son’s and Alzheimer’s.

For ALS in par­tic­u­lar, Vec­to­rY has teamed up with an un­named part­ner to vec­tor­ize their an­ti­body can­di­dates, an ap­proach that they be­lieve will be more ef­fec­tive be­cause it can get in­to the brain bet­ter.

With an­i­mal stud­ies now on­go­ing, Vos notes the ALS pro­gram will like­ly be­come the lead pro­gram with clin­i­cal stud­ies on the hori­zon for 2023.

They will pur­sue a sim­i­lar idea in Alzheimer’s — a field that’s now changed psy­cho­log­i­cal­ly, Vos re­marked, be­cause the ap­proval of Bio­gen’s Aduhelm showed it’s pos­si­ble — in terms of vec­tor­iz­ing an­ti­bod­ies that can clear pro­tein ag­gre­gates, al­though the ex­act ap­proach has not been de­cid­ed. The first mus­cle in­di­ca­tion al­so re­mains to be se­lect­ed, which will like­ly hap­pen lat­er this year.

“We see our pipeline ex­pand­ing through mul­ti­ple col­lab­o­ra­tions com­bin­ing world­class an­ti­body knowhow with our knowhow on the gene ther­a­py and vec­tor­ized an­ti­body space,” he added.

Mar­co Boors­ma

Join­ing the group of gene ther­a­py vets al­ready on the team will be Sander van De­ven­ter, co-founder of For­bion, who is per­haps best known for his work on the gene ther­a­py Gly­bera and then help­ing cre­ate uniQure — a place both CSO Pavli­na Kon­stan­ti­no­va and VP of vec­tor de­vel­op­ment Bar­bara Sanders have spent time at. Sanofi Gen­zyme vet Car­lo In­cer­ti con­tin­ues to chair the board while Mar­co Boors­ma, for­mer­ly in­ter­im CEO, will now step back and take a board seat.

Build­ing an in-house man­u­fac­tur­ing ca­pac­i­ty with this ex­pe­ri­enced crew is key to Vos’ plan for the com­pa­ny, ini­tial­ly with a CD­MO part­ner whom it will help on the process de­vel­op­ment front in ex­change for a ded­i­cat­ed part of the fa­cil­i­ty, so that they won’t be at the mer­cy of sched­ul­ing.

In the long term, the plan — backed by For­bion and new in­vestors at BGV as well as Eli Lil­ly — will be to es­tab­lish its own plant in the Nether­lands.

“The truth al­ways is that you have enor­mous la­ten­cy in the in­dus­try, be­cause peo­ple are locked in­to their process­es be­cause of the CMC and reg­u­la­to­ry en­vi­ron­ment, right? They can­not change away from that. So it’s al­ways slow,” he said. “I think it’s some­times eas­i­er to just start fresh.”

Adap­tive De­sign Meth­ods Of­fer Rapid, Seam­less Tran­si­tion Be­tween Study Phas­es in Rare Can­cer Tri­als

Rare cancers account for 22 percent of cancer diagnoses worldwide, yet there is no universally accepted definition for a “rare” cancer. Moreover, with the evolution of genomics and associated changes in categorizing tumors, some common cancers are now characterized into groups of rare cancers, each with a unique implication for patient management and therapy.

Adaptive designs, which allow for prospectively planned modifications to study design based on accumulating data from subjects in the trial, can be used to optimize rare oncology trials (see Figure 1). Adaptive design studies may include multiple cohorts and multiple tumor types. In addition, numerous adaptation methods may be used in a single trial and may facilitate a more rapid, seamless transition between study phases.

Marianne De Backer (L) and Jeff Hatfield

Bay­er nabs star biotech Vi­vid­ion with a $2B buy­out and an ‘arms-length’ pact, pulling a part­ner out of the IPO con­ga line

Vividion is canceling that IPO it filed. Instead of following the industry-wide migration to Nasdaq, the biotech that has captured considerable attention for its still-preclinical work finding cryptic pockets to bind to on proteins is going to work for Bayer now.

The pharma giant is putting out word today that it has bought out Vividion for $1.5 billion in cash and another half-billion dollars in milestones.

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Tadataka Yamada (Photographer: Kiyoshi Ota/Bloomberg via Getty Images)

Sci­ence pi­o­neer, phar­ma re­search chief, glob­al health ad­vo­cate and biotech en­tre­pre­neur Tadata­ka ‘Tachi’ Ya­ma­da has died

Tadataka Yamada, a towering physician-scientist who made his name in academia before transforming drug development at GlaxoSmithKline and developing vaccines for malaria and meningitis at the Gates Foundation, died unexpectedly of natural causes at his home in Seattle Wednesday morning.

He was 76. Frazier Healthcare Partners’ David Socks confirmed his death.

Known widely by the mononym “Tachi,” Yamada had a globetrotting career and arrived in industry relatively late in life. A 2004 Independent article noted GSK had asked Yamada to stay on beyond his approaching 60th birthday, the company’s usual retirement age. Yamada would continue working for the next 17 years, steering the Gates Foundation’s global health division for 6 years, funding Jim Wilson’s gene therapy work when few would touch it, launching Takeda Vaccines and co-founding a series of high-profile biotechs.

Josh Hoffman, outgoing Zymergen CEO (Zymergen)

UP­DAT­ED: Syn­bio uni­corn Zymer­gen jet­ti­sons found­ing CEO, cuts guid­ance as cus­tomers re­port lead prod­uct does­n't work

Zymergen, just months off a $500 million IPO that put the synthetic bio firm in rarified air, has now ejected its founding CEO and downgraded its revenue forecasts after customers reported its lead film product doesn’t work as advertised, the company said Tuesday afternoon.

CEO Josh Hoffman will leave his role and sacrifice his board seat immediately in favor of Jay Flatley, the former CEO of Illumina who will take the lead role on an interim basis as the company conducts a search for its next leader.

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Zymergen co-founders Zach Serber, Josh Hoffman, and Jed Dean (Zymergen via website)

Zymer­gen's sud­den im­plo­sion shocked biotech. A lin­ger­ing loan could make things even worse

As former synbio unicorn Zymergen picks up the pieces from its spectacular implosion Tuesday, an outstanding loan from Perceptive Advisors — the only blue-chip biotech crossover investor to touch Zymergen’s fundraising efforts — could make the situation worse, according to public documents.

In December 2019, more than a year before Zymergen filed for what would eventually become a $500 million IPO, the “biofacturing” firm signed a $100 million credit facility with Perceptive to help supplement the nearly $700 million the company had raised across four VC rounds.

UK re-in­ves­ti­gates Pfiz­er's eye-pop­ping price goug­ing on an epilep­sy drug

When a drugmaker raises the price of a drug in the US by more than 2,000% overnight, and without any particular reason for that increase, nothing typically happens to the company. No fines, no court orders, just business as usual.

Martin Shkreli’s decades-old anti-parasitic drug Daraprim was the perfect example — massive price spike on an old drug, lots of media attention, public outcry, Congressional committees dragging his former company through multiple hearings, and at the end of it? Nothing happened to the price or the company (until generic competition came).

Thomas Lingelbach, Valneva CEO

A small vac­cine de­vel­op­er fa­vored by the UK gov­ern­ment in Covid-19 touts a PhI­II first in chikun­gun­ya

Before Valneva garnered the favor of the UK government as a potential supplier of Covid-19 vaccines, the French biotech prided itself on being the first company to bring a chikungunya vaccine into Phase III.

It now has positive pivotal results to back up the breakthrough therapy designation the FDA granted just weeks ago.

There are currently no approved jabs to prevent chikungunya virus infection despite decades of R&D efforts, a fact that underscores just how arduous traditional vaccine development can be, particularly for neglected tropical disease. In a absence of a major commercial market, the US government and NGOs such as CEPI have deployed various grants and incentives to spur on a small crew of academics and industry players, with Merck, via its acquisition of Themis, claiming a spot in that race.

Bio­gen, Ei­sai are push­ing for an­oth­er ac­cel­er­at­ed Alzheimer's OK — this time for BAN2401

Now that the door at the FDA has been opened wide for Alzheimer’s drugs that can demonstrate a reduction in amyloid, Biogen and its partners at Eisai are pushing for a quick OK on the next drug to follow in the controversial path of aducanumab.

In a presentation to analysts, Eisai neurology chief Ivan Cheung outlined some bullish expectations for their newly-approved treatment and set the stage for what he believes will be a fast follow for BAN2401 (lecanemab) — after a dry spell in new drug development that’s lasted close to 20 years.

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Paul Hudson, Sanofi CEO (Eric Piermont/AFP via Getty Images)

UP­DAT­ED: Sanofi buys mR­NA play­er Trans­late Bio for $3.2B. And the price fits a pop­u­lar range for biotech M&A

Sanofi CEO Paul Hudson is dead serious about his intention to vault directly into contention for the future of mRNA vaccines.

A year after paying Translate Bio $TBIO a whopping $425 million in an upfront and equity payment to help guide the pharma giant to the promised land of mRNA vaccines, Sanofi closed the deal with a buyout early Tuesday, spending $38 a share in a $3.2 billion buyout.

Translate’s stock $TBIO soared after the market closed Monday when Reuters reported the first word of the acquisition just hours ahead of the formal announcement. The wire service, though, didn’t have a price to report in its scoop, and investors chased the stock up 78% in the wild ride that followed. Once the price was announced, gains shriveled to 29% ahead of the bell.

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