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.

Pi­o­neer­ing Click Chem­istry in Hu­mans

Reimagining cancer treatments

Cancer is a leading cause of death worldwide, accounting for nearly 10 million deaths in 2020, which is nearly one in six deaths. Recently, we have seen incredible advances in novel cancer therapies such as immune checkpoint inhibitors, cell therapies, and antibody-drug conjugates that have revamped cancer care and improved survival rates for patients.

Despite this significant progress in therapeutic targeting, why are we still seeing such a high mortality rate? The reason is that promising therapies are often limited by their therapeutic index, which is a measure of the effective dose of a drug, relative to its safety. If we could broaden the therapeutic indices of currently available medicines, it would revolutionize cancer treatments. We are still on the quest to find the ultimate cancer medicine – highly effective in several cancer types, safe, and precisely targeted to the tumor site.

Justin Klee (L) and Joshua Cohen, Amylyx co-CEOs (Cody O'Loughlin/The New York Times; courtesy Amylyx)

Ad­vo­cates, ex­perts cry foul over Amy­lyx's new ALS drug, cit­ing is­sues with price, PhI­II com­mit­ment

Not 24 hours after earning the first ALS drug approval in five years, Amylyx Pharmaceuticals’ Relyvrio is already drawing scrutiny. And it’s coming from multiple fronts.

In an investor call Friday morning, Amylyx revealed that it would charge about $158,000 per year, a price point that immediately drew backlash from ALS advocates and some outside observers. The cost reveal had been highly anticipated in the immediate hours after Thursday evening’s approval, though Amylyx only teased Relyvrio would cost less than previously approved drugs.

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Land­mark Amy­lyx OK spurs de­bate; Some... pos­i­tive? Alzheimer's da­ta; Can­cer tri­al bot­tle­neck; Sanofi's CRISPR bet; 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.

After brief stops in Paris and Boston, John Carroll and the Endpoints crew are staying on the road in October with their return for a live/streaming EUBIO22 in London. The hybrid event fireside chats and panels on mRNA, oncology and the crazy public market. We hope you can join him there.

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Joshua Cohen (L) and Justin Klee, Amylyx co-CEOs

Up­dat­ed: Af­ter long and wind­ing road, FDA ap­proves Amy­lyx's ALS drug in vic­to­ry for pa­tients and ad­vo­ca­cy groups

For just the third time in its 116-year history, the FDA has approved a new treatment for Lou Gehrig’s disease, or ALS.

US regulators gave the thumbs-up to the drug, known as Relyvrio, in a massive win for patients and their families. The approval, given to Boston-area biotech Amylyx Pharmaceuticals, comes after two years of long and contentious debates over the drug’s effectiveness between advocacy groups and FDA scientists, following the readout of a mid-stage clinical trial in September 2020.

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#AAO22: J&J’s first look at com­mon eye dis­ease port­fo­lio pads the case for PhII of gene ther­a­py

CHICAGO — While the later-stage drug developers in the geographic atrophy field are near the finish line, Johnson & Johnson’s Janssen is taking a more deliberate route, with a treatment that it hopes to be a one-time fix.

The Big Pharma will take its Hemera Biosciences-acquired gene therapy into a Phase II study later this year in patients with GA, a common form of age-related macular degeneration that impacts about five million people worldwide. To get there, Janssen touted early-stage safety data at the American Academy of Ophthalmology annual conference Saturday morning, half a day after competitors Apellis and Iveric Bio revealed their own more-detailed Phase III analyses.

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Some­one old, some­one new: Mod­er­na pro­motes CTO, raids No­var­tis for re­place­ment amid pipeline push

Moderna CEO Stéphane Bancel made clear on the last quarterly call that “now is not the time to slow down.” On Thursday, he made a bit more room in the cockpit.

The company unveiled a new executive role on Thursday, promoting former chief technical operations and quality officer Juan Andres to president of strategic partnerships and enterprise expansion, and poaching a former Novartis exec to take his place.

George Church (courtesy EnPlusOne BioSciences)

George Church, Wyss sci­en­tists and North­pond chal­lenge con­ven­tion­al RNA man­u­fac­tur­ing with new biotech

RNA medicine has been at the forefront for the past few years, with the first RNA silencing therapy approved in 2018, and mRNA Covid vaccines following after. But flying under the radar has been the process of actually making RNA for these treatments.

That’s what Daniel Wiegand and Jonathan Rittichier have been working on in George Church’s lab for the past six years.

Friday morning, they unveiled EnPlusOne Biosciences, a biotech built on their RNA synthesis platform. Wiegand will serve as the Watertown, MA-based biotech’s CEO, and Rittichier will be CSO. And no different from his other startups, Church will be acting as scientific advisor. Its fourth co-founder, Dan Ahlstedt, joined through a Harvard Business School program, and will be COO.

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Up­dat­ed: Al­ny­lam re­in­forces APOL­LO-B patisir­an da­ta be­fore head­ing to the FDA

Weeks after uncorking some mostly positive data for patisiran in transthyretin-mediated (ATTR) amyloidosis with cardiomyopathy, Alnylam is bolstering its package with new exploratory and subgroup data before shipping it off to regulators.

The RNAi drug maintained “generally consistent” benefits in efficacy and quality of life across several prespecified subgroups at month 12, Alnylam announced on Friday afternoon, including age, baseline tafamidis use, ATTR amyloidosis type, baseline six-minute walk test score and others.

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Nooman Haque, head of life sciences and healthcare at Silicon Valley Bank, and John Carroll

I’m head­ed to Lon­don soon for #EU­BIO22. Care to join me?

It was great getting back to a live ESMO conference/webinar in Paris followed by a live pop-up event for the Endpoints 11 in Boston. We’re staying on the road in October with our return for a live/streaming EUBIO22 in London.

Silicon Valley Bank’s Nooman Haque and I are once again jumping back into the thick of it with a slate of virtual and live events on October 12. I’ll get the ball rolling with a virtual fireside chat with Novo Nordisk R&D chief Marcus Schindler, covering their pipeline plans and BD work.