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.”

Health­care Dis­par­i­ties and Sick­le Cell Dis­ease

In the complicated U.S. healthcare system, navigating a serious illness such as cancer or heart disease can be remarkably challenging for patients and caregivers. When that illness is classified as a rare disease, those challenges can become even more acute. And when that rare disease occurs in a population that experiences health disparities, such as people with sickle cell disease (SCD) who are primarily Black and Latino, challenges can become almost insurmountable.

Jacob Van Naarden (Eli Lilly)

Ex­clu­sives: Eli Lil­ly out to crash the megablock­buster PD-(L)1 par­ty with 'dis­rup­tive' pric­ing; re­veals can­cer biotech buy­out

It’s taken 7 years, but Eli Lilly is promising to finally start hammering the small and affluent PD-(L)1 club with a “disruptive” pricing strategy for their checkpoint therapy allied with China’s Innovent.

Lilly in-licensed global rights to sintilimab a year ago, building on the China alliance they have with Innovent. That cost the pharma giant $200 million in cash upfront, which they plan to capitalize on now with a long-awaited plan to bust up the high-price market in lung cancer and other cancers that have created a market worth tens of billions of dollars.

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So what hap­pened with No­var­tis' gene ther­a­py group? Here's your an­swer

Over the last couple of days it’s become clear that the gene therapy division at Novartis has quietly undergone a major reorganization. We learned on Monday that Dave Lennon, who had pursued a high-profile role as president of the unit with 1,500 people, had left the pharma giant to take over as CEO of a startup.

Like a lot of the majors, Novartis is an open highway for head hunters, or anyone looking to staff a startup. So that was news but not completely unexpected.

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Who are the women su­per­charg­ing bio­phar­ma R&D? Nom­i­nate them for this year's spe­cial re­port

The biotech industry has faced repeated calls to diversify its workforce — and in the last year, those calls got a lot louder. Though women account for just under half of all biotech employees around the world, they occupy very few places in C-suites, and even fewer make it to the helm.

Some companies are listening, according to a recent BIO survey which showed that this year’s companies were 2.5 times more likely to have a diversity and inclusion program compared to last year’s sample. But we still have a long way to go. Women represent just 31% of biotech executives, BIO reported. And those numbers are even more stark for women of color.

David Meek, new Mirati CEO (Marlene Awaad/Bloomberg via Getty Images)

Fresh off Fer­Gene's melt­down, David Meek takes over at Mi­rati with lead KRAS drug rac­ing to an ap­proval

In the insular world of biotech, a spectacular failure can sometimes stay on any executive’s record for a long time. But for David Meek, the man at the helm of FerGene’s recent implosion, two questionable exits made way for what could be an excellent rebound.

Meek, most recently FerGene’s CEO and a past head at Ipsen, has become CEO at Mirati Therapeutics, taking the reins from founding CEO Charles Baum, who will step over into the role of president and head of R&D, according to a release.

Jay Bradner (Jeff Rumans for Endpoints News)

Div­ing deep­er in­to in­her­it­ed reti­nal dis­or­ders, No­var­tis gob­bles up an­oth­er bite-sized op­to­ge­net­ics biotech

Right about a year ago, a Novartis team led by Jay Bradner and Cynthia Grosskreutz at NIBR swooped in to scoop up a Cambridge, MA-based opthalmology gene therapy company called Vedere. Their focus was on a specific market niche: inherited retinal dystrophies that include a wide range of genetic retinal disorders marked by the loss of photoreceptor cells and progressive vision loss.

But that was just the first deal that whet their appetite.

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Dave Lennon, former president of Novartis Gene Therapies

Zol­gens­ma patent spat brews be­tween No­var­tis and Re­genxbio as top No­var­tis gene ther­a­py ex­ec de­parts

Regenxbio, a small licensor of gene therapy viral vectors spun out from the University of Pennsylvania, is now finding itself in the middle of some major league patent fights.

In addition to a patent suit with Sarepta Therapeutics from last September, Novartis, is now trying to push its smaller partner out of the way. The Swiss biopharma licensed Regenxbio’s AAV9 vector for its $2.1 million spinal muscular atrophy therapy Zolgensma.

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The biggest ques­tions fac­ing gene ther­a­py, the XLMTM com­mu­ni­ty, and Astel­las af­ter fourth pa­tient death

After three patients died last year in an Astellas gene therapy trial, the company halted the study and began figuring out how to safely get the program back on track. They would, executives eventually explained, cut the dose by more than half and institute a battery of other measures to try to prevent the same thing from happening again.

Then tragically, Astellas announced this week that the first patient to receive the new regimen had died, just weeks after administration.

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When ef­fi­ca­cy is bor­der­line: FDA needs to get more con­sis­tent on close-call drug ap­provals, agency-fund­ed re­search finds

In the exceedingly rare instances in which clinical efficacy is the only barrier to a new drug’s approval, new FDA-funded research from FDA and Stanford found that the agency does not have a consistent standard for defining “substantial evidence” when flexible criteria are used for an approval.

The research comes as the FDA is at a crossroads with its expedited-review pathways. The accelerated approval pathway is under fire as the agency recently signed off on a controversial new Alzheimer’s drug, with little precedent to explain its decision. Meanwhile, top officials like Rick Pazdur have called for a major push to simplify and clarify all of the various expedited pathways, which have grown to be must-haves for sponsors of nearly every newly approved drug.

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