Paul Sekhri, eGenesis CEO

George Church spin­off swal­lows a key man­u­fac­tur­ing part­ner as it preps CRISPR-edit­ed pig or­gans for an­i­mal test­ing

Re­search­ing, cloning and breed­ing gene-edit­ed pigs are core to eGe­n­e­sis’ au­da­cious mis­sion to grow or­gans that can be xeno­trans­plant­ed in­to hu­mans. But per­haps un­sur­pris­ing­ly, lit­tle of that ac­tu­al­ly hap­pens at its Cam­bridge, MA labs.

Rather, the biotech works with ex­ter­nal par­ties for that part of the R&D and man­u­fac­tur­ing work. As eGe­n­e­sis ap­proach­es a fi­nal ver­sion of the gene-edit­ed or­gans that it will test in an­i­mals, it’s ac­quir­ing one of those part­ners and in­te­grat­ing it as a sub­sidiary.

ICBiotec’s 15-per­son team will stay in the Mid­west. In ad­di­tion to the ex­ist­ing fa­cil­i­ties and equip­ment, eGe­n­e­sis al­so gains ac­cess to un­de­vel­oped land they can build on.

The way they work to­geth­er — with eGe­n­e­sis study­ing fi­brob­last cells in dish­es and turn­ing over gene-edit­ing pay­loads they de­vel­op in the lab to ICB for fur­ther test­ing — won’t change, said CEO Paul Sekhri; eGe­n­e­sis is mere­ly tran­si­tion­ing from the largest client of ICB to its own­er.

“They have a few oth­er cus­tomers that we’ll prob­a­bly en­sure that there’s a prop­er tran­si­tion, but to be hon­est with you I think we will need all of their ca­pa­bil­i­ties, which is one of the rea­sons why we did the trans­ac­tion in the first place,” he said.

De­spite pan­dem­ic-re­lat­ed re­stric­tions, eGe­n­e­sis is still on track to fi­nal­ize the recipe for ge­net­i­cal­ly en­gi­neer­ing the pigs from which they will har­vest or­gans to test in an­i­mal mod­els for safe­ty and ef­fi­ca­cy — be­gin­ning with the kid­ney. In the US alone, there are al­most 100,000 peo­ple on the kid­ney wait­ing list but on­ly around 14,000 trans­plants per­formed each year.

“The re­search nev­er stopped, it just was slowed down a bit be­cause you just lit­er­al­ly couldn’t have the same con­cen­tra­tion of peo­ple in the lab as we had in the past,” Sekhri said.

The slog to­ward the clin­ic will in­volve many more reg­u­la­tor hur­dles, some of which would be un­fa­mil­iar even to the FDA as it deals with the eth­i­cal and tech­ni­cal im­pli­ca­tions of this sci-fi wor­thy un­der­tak­ing. The chief re­quire­ment for a green light to the clin­ic, Sekhri has pre­vi­ous­ly not­ed, is con­sis­tent pre­clin­i­cal re­sults among non-hu­man pri­mates.

Mov­ing ICB ca­pa­bil­i­ties in-house adds band­width for this cru­cial ex­er­cise, he added. Hav­ing bagged $100 mil­lion last No­vem­ber, the deal “doesn’t sig­nif­i­cant­ly im­pact our cash run­way.”

5AM Ven­tures: Fu­el­ing the Next Gen­er­a­tion of In­no­va­tors

By RBC Capital Markets
With Andy Schwab, Co-Founder and Managing Partner at 5AM Ventures

Key Points

Prescription Digital Therapeutics, cell therapy technologies, and in silico medicines will be a vital part of future treatment modalities.
Unlocking the potential of the microbiome could be the missing link to better disease diagnosis.
Growing links between academia, industry, and venture capital are spinning out more innovative biotech companies.
Biotech is now seen by investors as a growth space as well as a safe haven, fuelling the recent IPO boom.

Biohaven CEO Vlad Coric (Photo Credit: Andrew Venditti)

Pssst: That big Bio­haven Alzheimer's study? It was a bust. Even the sub­group analy­sis ex­ecs tout­ed was a flop

You know it’s bad when a biopharma player plucks out a subgroup analysis for a positive take — even though it was way off the statistical mark for success, like everything else.

So it was for Biohaven $BHVN on MLK Monday, as the biotech reported on the holiday that their Phase II/III Alzheimer’s study for troriluzole flunked both co-primary endpoints as well as a key biomarker analysis.

The drug — a revised version of the ALS drug riluzole designed to regulate glutamate — did not “statistically differentiate” from placebo on the Alzheimer’s Disease Assessment Scale-Cognitive Subscale 11 (ADAS-cog) and the Clinical Dementia Rating Scale Sum of Boxes (CDR-SB).  The “hippocampal volume” assessment by MRI also failed to distinguish itself from placebo for all patients fitting the mild-to-moderate disease profile they had established for the study.

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Janet Woodcock and Joshua Sharfstein (AP, Images)

Poll: Should Joshua Sharf­stein or Janet Wood­cock lead the FDA from here?

It’s time for a new FDA commissioner to come on board, a rite of passage for Joe Biden’s administration that should help seal the new president’s rep on seeking out the experts to lead the government over the next 4 years.

As of now, the competition for the top job appears to have narrowed down to 2 people: The longtime CDER chief Janet Woodcock and Joshua Sharfstein, the former principal deputy at the FDA under Peggy Hamburg. Both were appointed by Barack Obama.

Dan Skovronsky, Eli Lilly CSO (Lilly via Facebook)

Eli Lil­ly tees up dis­cov­ery pact worth more than $1.6B with Merus for T cell-fo­cused bis­pe­cif­ic an­ti­bod­ies

Under science chief Dan Skovronsky, Eli Lilly has taken some big swings at next-gen therapies, including trying to find the next big thing in oncology. Now, after one early failure in the field, Lilly is going back to the bispecific antibody well with a new deal with a Dutch biotech.

Lilly will pay $40 million upfront with an additional $20 million equity stake in Merus NV to identify and develop three bispecific antibodies looking to engage the CD3 antigen on T cells and redirect immune cells, the Indianapolis pharma giant said Tuesday.

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Lon­za hits the ex­it on gel and liq­uid cap­sules, of­fload­ing 2 pro­duc­tion sites to NextPhar­ma

With the contract manufacturing market on a hot streak with the Covid-19 vaccine rollout, Lonza has opened its war chest in recent months for a suite of expansions. But now, with its focus elsewhere, the company is looking to contract its capsule offerings for pharma.

The CDMO giant will offload its lipid capsule production sites in Ploërmel, France, and Edinburgh, UK, as part of a plan to exit the pharmaceutical capsules space while maintaining its market foothold in consumer health and nutrition, the company said Tuesday.

Andrew Allen (Gritstone)

As coro­n­avirus vari­ants trig­ger new alarms, the NIH is putting an un­der-the-radar ‘next-gen’ vac­cine in­to PhI

Over the past year, the world has been transfixed by the development of new vaccines to fight SARS-CoV-2. In a frenzy of activity, the new mRNA approach has delivered pioneering emergency approvals in record time. And with some setbacks, the more traditional big players are coming along with added jabs as the most affluent nations in the world begin to vaccinate large portions of their populations.

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The IPO queue adds 5 more biotechs hop­ing to ring in 2021 by blitz­ing Nas­daq

Following a record year for IPOs — in terms of both proceeds and count — there’s already a long lineup of biotechs ready to jump onto Nasdaq in the new year. The companies are likely looking for much higher raises than they initially projected on their S-1s. Now it’s time to see if investors are still hungry for another round of biotech stocks.

Sana helped set the pace early on, as its founders look to divvy up a fortune from their IPO. And late last week 5 more biotechs crowded in, looking to pick up the pace where 2020 left off. Here they are:

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News brief­ing: Beam bags a $260M pri­vate place­ment; mi­Ra­gen re­brands to Virid­i­an Ther­a­peu­tics

Agios vet John Evans has demonstrated how to raise big money for a little biotech.

The Beam Therapeutics CEO — and ARCH partner — has pieced together a $260 million private placement from a group of backers that includes Perceptive Advisors, Farallon Capital, Casdin Capital, Redmile Group and Cormorant Asset Management. And there are 3 main goals they’ll pursue with it: clinical development, strategic partnerships and general corporate purposes.

As­traZeneca keeps the ball rolling on Dai­ichi-part­nered En­her­tu, pick­ing up 2nd in­di­ca­tion in gas­tric can­cer

AstraZeneca’s big gamble on Daiichi Sankyo’s antibody-drug conjugate Enhertu has already paid off with a big approval in breast cancer more than a year ago. But the partners have big plans for their blockbuster in the making, and a new nod in gastric cancer will raise their spirits even higher.

The FDA on Friday approved Enhertu to treat locally advanced or metastatic HER2-positive gastric or gastroesophageal junction adenocarcinoma in patients who have previously undergone at least one round of treatment with a Herceptin-based regimen, AstraZeneca said in a release.