Charlie Albright and Petra Kaufmann

Gene edit­ing ex­pert Char­lie Al­bright tells us why he hopped from Ed­i­tas to a new role as CSO of an up­start biotech out to en­gi­neer gene ther­a­py 2.0

We now know where Char­lie Al­bright was head­ed when he jumped ship at CRISPR gene edit­ing pi­o­neer Ed­i­tas a few weeks ago.

Al­bright has skipped across town to Waltham, MA-based Affinia, the new­ly hatched gene ther­a­py biotech — from AveX­is vets Sean Nolan and Rick Mo­di — look­ing to de­vel­op next-gen AAV de­liv­ery ve­hi­cles for a new wave of once-and-done ther­a­pies. And he’s been joined on the team by Pe­tra Kauf­mann, a vet from No­var­tis Gene Ther­a­pies — un­til re­cent­ly known as AveX­is.

“We’re re­al­ly at the be­gin­ning of a new era of drug de­vel­op­ment with gene ther­a­py,” Al­bright tells me, point­ing to some big ad­vances, as well as ac­knowl­edg­ing some daunt­ing set­backs that have oc­curred.

“It’s not sur­pris­ing we’re see­ing both suc­cess­es and some set­backs,” he adds. “I came to Affinia be­cause I thought we had the po­ten­tial to ad­dress some of the fun­da­men­tal is­sues in AAV gene ther­a­py. And af­ter my first 4 weeks here I be­lieve that even more than when I took the job.”

The big idea at Affinia, what at­tract­ed Al­bright to the com­pa­ny, is the be­lief that the in­sights Luk Van­den­berghe made in his Har­vard lab can trans­late in­to new and much bet­ter cap­sids that can sig­nif­i­cant­ly im­prove ef­fi­ca­cy, guard against tox­i­c­i­ty and tar­get a va­ri­ety of new tis­sues that can open the door to a host of dis­eases now out­side the reach of the cur­rent­ly avail­able tech. The tech al­so has the po­ten­tial to avoid re­sis­tance mech­a­nisms, nat­u­ral­ly re­oc­cur­ring an­ti­bod­ies, and help solve man­u­fac­tur­ing chal­lenges.

“My job is to take that tech­nol­o­gy from an aca­d­e­m­ic set­ting and in­dus­tri­al­ize that tech­nol­o­gy and at the same time point it to­ward the right com­bi­na­tion of where the tech­nol­o­gy works and the un­met need is high,” says Al­bright.

“The plat­form is based on AAV cap­sids that come from the re­con­struc­tion of the an­ces­tral se­quences… We take this li­brary of po­ten­tial an­ces­tors and we bar­cod­ed them so we can see which an­ces­tor we’re look­ing at, or which cap­sids we’re look­ing at, and then we can in­ject them in­to an­i­mals and see which ones get more broad­ly dis­trib­uted in the cen­tral ner­vous sys­tem, which ones al­low bet­ter pen­e­tra­tion in­to car­diac tis­sue, and there­by iden­ti­fy cap­sids that al­low us to get the dose down and the amount of gene ther­a­py we de­liv­er way up.”

These vari­ants based on an­ces­tral se­quences have high func­tion­al­i­ty, he notes. “We have ev­i­dence that that ac­tu­al­ly will work. In par­tic­u­lar, we found a cap­sid that de-tar­gets the liv­er, one of the ma­jor sites of tox­i­c­i­ty that comes from high dos­es of sys­temic AAV. An­oth­er dis­trib­utes gene ther­a­py more broad­ly in the brain, though lum­bar punc­ture, that out­per­forms AAV9.” Both are ex­am­ples of what dif­fer­en­ti­ates Affinia from the rest of the field, which has been ex­plod­ing with new pro­grams over the last few years.

Affinia still has a long way to go, but at this point in the jour­ney, Al­bright feels he’s found a spot right on the cut­ting edge of the gene ther­a­py boom. And he plans to ride it out to the clin­ic.

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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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.

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.

Vicente Anido (University of West Virginia via YouTube)

Aerie fires CEO af­ter lead pro­gram flop, com­ments about pri­ma­ry end­points be­ing 'not re­quired'

Aerie Pharmaceuticals CEO Vicente Anido has left the company less than a week after trying to chart a Phase III study in the wake of a serious Phase IIb flop.

Anido’s last day at Aerie was Friday, the biotech announced in a news release Tuesday morning, and Benjamin McGraw is taking his place in an interim role. The now former CEO was terminated without cause, according to an SEC filing.

The board has started looking for a full-time chief to take his place.

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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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Lat­est news: It’s a no on uni­ver­sal boost­ers; Pa­tient death stuns gene ther­a­py field; In­side Tril­li­um’s $2.3B turn­around; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

Next week is shaping up to be a busy one, as our editor-in-chief John Carroll and managing editor Kyle Blankenship lead back-to-back discussions with a great group of experts to discuss the weekend news and trends. John will be spending 30 minutes with Jake Van Naarden, the CEO of Lilly Oncology, and Kyle has a brilliant panel lined up: Harvard’s Cigall Kadoch, Susan Galbraith, the new head of cancer R&D at AstraZeneca, Roy Baynes at Merck, and James Christensen at Mirati. Don’t miss out on the action — sign up here.

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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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