Marco Boorsma, Forbion

With a ros­ter of gene ther­a­py vets, For­bion makes a bet on 'rel­a­tive­ly emp­ty space' of vec­tor­ized an­ti­bod­ies

The fu­ture waves of gene ther­a­py could look quite dif­fer­ent from the gene re­place­ment of­fer­ings cur­rent­ly on the mar­ket, For­bion reck­ons.

It’s the key idea be­hind Vec­to­rY, a start­up seed­ed by the Eu­ro­pean VC firm that’s out to de­vel­op vec­tor­ized an­ti­bod­ies for mus­cu­lar and neu­rode­gen­er­a­tive dis­or­ders.

Voy­ager Ther­a­peu­tics, which briefly pushed the tech­nol­o­gy to the lime­light with its short-lived Ab­b­Vie col­lab­o­ra­tion, is among the “sev­er­al oth­er com­pa­nies work­ing on it too, but def­i­nite­ly it’s a pret­ty rel­a­tive­ly emp­ty space,” said Mar­co Boors­ma — a gen­er­al part­ner at For­bion who’s step­ping in as in­ter­im CEO.

At the in­ter­sec­tion of vi­ral gene ther­a­pies and an­ti­bod­ies, vec­tor­ized an­ti­bod­ies are AAV vec­tors that car­ry genes en­cod­ing for an­ti­bod­ies. With some cap­sid en­gi­neer­ing and “in­ter­est­ing ef­fi­ca­cy en­hanc­ing tools,” they promise to treat a wider range of dis­eases and do a more thor­ough job at, say, clean­ing up mis­fold­ed pro­teins in­side and out­side of cells than ei­ther of the tra­di­tion­al modal­i­ties can.

“With naked an­ti­bod­ies you hard­ly get in­to the brain,” Boors­ma said. “Al­so dif­fi­cult to get in­to mus­cle.”

Build­ing the com­pa­ny re­quired ex­per­tise in both gene ther­a­py and an­ti­bod­ies, he added, and Vec­to­rY co-founders bring a di­verse skill set: Chair­man Car­lo In­cer­ti is an op­er­at­ing part­ner and vet­er­an of Sanofi Gen­zyme; Pavli­na Kon­stan­ti­no­va spent some years at uniQure (an­oth­er For­bion-backed gene ther­a­py ven­ture) be­fore tak­ing up the CSO post; COO An­tho­ny New­combe learned the ins and outs of man­u­fac­tur­ing through stints at Pfiz­er, Glax­o­SmithK­line, Pfiz­er and Sam­sung Bi­o­log­ics; and Bar­bara Sanders, the VP of vec­tor de­vel­op­ment, cut her teeth at J&J and held a sim­i­lar role at uniQure.

“I have been work­ing in the gene ther­a­py field for over 30 years, and, from ex­pe­ri­ence, know that ac­cu­rate­ly tar­get­ing the right cells with the trans­gene has al­ways been of para­mount im­por­tance,” In­cer­ti said in a state­ment. “Us­ing vi­ral vec­tors de­vel­oped at Vec­to­rY to de­liv­er the ge­net­ic se­quence for ther­a­peu­tic an­ti­body ex­pres­sion in tar­get cells has the po­ten­tial to ad­dress some of the key short­com­ings of in­tra­venous­ly in­ject­ed mol­e­cules.”

With ac­cess to the deep pock­ets of For­bion — which re­cent­ly added a $557 mil­lion new fund — one thing the Am­s­ter­dam-based biotech has de­cid­ed to do ear­ly is to set up its own man­u­fac­tur­ing fa­cil­i­ties.

“Def­i­nite­ly CD­MOs are ex­pand­ing ca­pac­i­ty, they are de­vel­op­ing well al­so in gene ther­a­pies,” Boors­ma said. “Still I think they are not there where we think they should be to serve a com­pa­ny like Vec­to­rY.”

The is­sues can range from var­ied stan­dards to wait­ing times up to one and a half years that are “sim­ply too long for a young biotech com­pa­ny.”

Hav­ing start­ed op­er­a­tions last Oc­to­ber, the team is now aim­ing to dou­ble the head­count to about 40 by the end of the year.

“Vec­to­rY is work­ing very hard, but al­so the field is work­ing on that very hard,” he added. “The first wave of gene ther­a­pies was more in kind of re­pair­ing genes, now you al­so see a lot of cap­sid en­gi­neer­ing to get your pay­load to the right tis­sues, and then of course al­so man­u­fac­tur­ing that’s be­ing scaled up […] be­ing more ro­bust.”

Tar­get­ing a Po­ten­tial Vul­ner­a­bil­i­ty of Cer­tain Can­cers with DNA Dam­age Re­sponse

Every individual’s DNA is unique, and because of this, every patient responds differently to disease and treatment. It is astonishing how four tiny building blocks of our DNA – A, T, C, G – dictate our health, disease, and how we age.

The tricky thing about DNA is that it is constantly exposed to damage by sources such as ultraviolet light, certain chemicals, toxins, and even natural biochemical processes inside our cells.¹ If ignored, DNA damage will accumulate in replicating cells, giving rise to mutations that can lead to premature aging, cancer, and other diseases.

Tom Barnes (Orna)

The mR­NA era is here. MPM be­lieves the fu­ture be­longs to oR­NA — and Big Phar­ma wants a seat at the ta­ble

If the ultra-fast clinical development of Covid-19 vaccines opened the world’s eyes to the promises of messenger RNA, the subsequent delays in supply offered a crash course on the ultra-complex process of producing them. Even before the formulation and fill-finish steps, mRNA is the precious end product from an arduous journey involving enzyme-aided transcription, modification and purification.

For Bristol Myers Squibb, Novartis Institutes for Biomedical Research, Gilead’s Kite and Astellas, it’s time to rethink the way therapeutic RNA is engineered.

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Fol­low biotechs go­ing pub­lic with the End­points News IPO Track­er

The Endpoints News team is continuing to track IPO filings for 2021, and we’ve designed a new tracker page for the effort.

Check it out here: Biopharma IPOs 2021 from Endpoints News

You’ll be able to find all the biotechs that have filed and priced so far this year, sortable by quarter and listed by newest first. As of the time of publishing on Feb. 25, there have already been 16 biotechs debuting on Nasdaq so far this year, with an additional four having filed their S-1 paperwork.

Steve Cutler, Icon CEO (Icon)

In the biggest CRO takeover in years, Icon doles out $12B for PRA Health Sci­ences to fo­cus on de­cen­tral­ized clin­i­cal work

Contract research M&A had a healthy run in recent years before recently petering out. But with the market ripe for a big buyout and the Covid-19 pandemic emphasizing the importance of decentralized trials, Wednesday saw a tectonic shift in the CRO world.

Icon, the Dublin-based CRO, will acquire PRA Health Sciences for $12 billion in a move that will shake up the highest rungs of a fragmented market. The merger would combine the 5th- and 6th-largest CROs by 2020 revenue, according to Icon, and the merger will set the newco up to be the second-largest global CRO behind only IQVIA.

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S&P ex­pects steady ero­sion in Big Phar­ma's cred­it pro­file in 2021 as new M&A deals roll in — but don't un­der­es­ti­mate their un­der­ly­ing strength

S&P Global has taken a look at the dominant forces shaping the pharma market and come to the conclusion that there will be more downgrades than upgrades in 2021 — the 8th straight year of steady decline.

But it’s not all bad news. Some things are looking up, and there’s still plenty of money to be made in an industry that enjoys a 30% to 40% profit margin, once you factor in steep R&D expenses.

Tal Zaks, Moderna CMO (AP Photo/Rodrique Ngowi, via still image from video)

CMO Tal Zaks bids Mod­er­na a sur­prise adieu as biotech projects $18.4B in rev­enue, plots post-Covid ex­pan­sion

How do you exit a company after six years in style? Developing one of the most lucrative and life-saving products in pharma history is probably not the worst way to go.

Tal Zaks, Moderna’s CMO since 2015, will leave the mRNA biotech in September, the biotech disclosed in their annual report this morning. The company has already retained the recruitment firm Russell Reynolds to find a replacement.

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Ken Frazier, Merck CEO (Bess Adler/Bloomberg via Getty Images)

UP­DAT­ED: Mer­ck takes a swing at the IL-2 puz­zle­box with a $1.85B play for buzzy Pan­dion and its au­toim­mune hope­fuls

When Roger Perlmutter bid farewell to Merck late last year, the drugmaker perhaps best known now for sales giant Keytruda signaled its intent to take a swing at early-stage novelty with the appointment of discovery head Dean Li. Now, Merck is signing a decent-sized check to bring an IL-2 moonshot into the fold.

Merck will shell out roughly $1.85 billion for Pandion Pharmaceuticals, a biotech hoping to gin up regulatory T cells (Tregs) to treat a range of autoimmune disorders, the drugmaker said Thursday.

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Joe Nolan, Jaguar

Sean Nolan gath­ers AveX­is alum­ni to launch an­oth­er biotech. Is it an­oth­er race-to-IPO?

The old AveXis crew is launching another company. And they may once again look to cash in quick.

Armed with patents and a steady stream of cash from Deerfield Management, Sean Nolan and five ex-Avexis executives have launched Jaguar Gene Therapy to develop AAV9 gene therapies for a small constellation of disparate disorders, from autism to diabetes. Jaguar remains over a year from the clinic, but CEO Joe Nolan hinted they could look to follow a pace set by Tasyha, the biotech Sean Nolan and other AveXis alumni launched last year and took from Series A to IPO in less than 6 months.

Glax­o­SmithK­line re­thinks strat­e­gy for Covid-19 an­ti­body — not the Vir ones — af­ter tri­al flop. Is there hope in high-risk pa­tients?

In the search for a better Covid-19 therapeutic, GlaxoSmithKline and Vir have partnered up on two antibodies they hope have a chance. GSK is also testing its own in-house antibody, and early results may have shut the door on its widespread use.

A combination of GSK’s monoclonal antibody otilimab plus standard of care couldn’t best standard of care alone in preventing death and respiratory failure in hospitalized Covid-19 patients after 28 days, according to data from the Phase IIa OSCAR study unveiled Thursday.