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.

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.

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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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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Masayoshi Son, SoftBank CEO (glen photo/Shutterstock)

Japan's Soft­Bank plots bil­lions in biotech in­vest­ments in move that could keep the val­u­a­tion flood ris­ing — re­port

The valuation crazy train in biotech continues to roll into the new year with more than a dozen companies taking a chance on Nasdaq and money flowing in from all sides. Now, a Japanese institutional investor is reportedly weighing an entry into the market in a big way — will it keep the bitcoin-esque flood rising?

Already a part-time investor in biotech, SoftBank could drop billions of dollars into the industry as part of helmsman Masayoshi Son’s plan to spend around $80 billion of the firm’s own assets, according to a report from Bloomberg citing people familiar with the plan.

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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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Biden is look­ing be­yond Wood­cock or Sharf­stein for FDA com­mis­sion­er — re­port

Neither Janet Woodcock nor Joshua Sharfstein is likely to be nominated as the permanent FDA commissioner, Steve Usdin at BioCentury reports.

The White House is looking for alternatives to Woodcock, the acting chief and longtime CDER director, after opposition from several Democratic senators who are calling on others to block her nomination if her name is put forth, according to Usdin. Sharfstein, the former principal deputy FDA commissioner and current Johns Hopkins professor, is out of the running altogether.