Armed with CRISPR and a $38M round, eGe­n­e­sis tack­les the Holy Grail of xeno­trans­plan­ta­tion

Luhan Yang, eGe­n­e­sis

The first crude re­search in­volv­ing xeno­trans­plan­ta­tion goes back to the 1960s, when in­ves­ti­ga­tors first ac­tive­ly con­sid­ered the pos­si­bil­i­ties of har­vest­ing or­gans from pri­mates for use in hu­mans. That failed, and sub­se­quent at­tempts run­ning through the mid-90s al­so flopped, with in­com­pat­i­bil­i­ty prov­ing im­pos­si­ble to over­come, while al­so rais­ing some big fears about trans­fer­ring pig virus­es to hu­mans with po­ten­tial­ly cat­a­stroph­ic re­sults.

But George Church and his cel­e­brat­ed team at Har­vard have been us­ing a hot new lab tool to give xeno­trans­plan­ta­tion an­oth­er shot at the re­al world. And now the team has raised $38 mil­lion from some for­ward-think­ing in­vestors to see if yes­ter­day’s sci­ence fic­tion can be­come to­mor­row’s land­mark re­al­i­ty.

Xeno­trans­plan­ta­tion “has been aban­doned for the past 15 years,” says Luhan Yang, an award-win­ning young sci­en­tist who’s the CSO and co-founder at eGe­n­e­sis. But Yang and her 10-mem­ber crew are us­ing CRISPR gene edit­ing tech­nol­o­gy to knock out prob­lem anti­gens to ad­dress the re­jec­tion is­sue while prov­ing in a pre­clin­i­cal set­ting that you can erad­i­cate the prob­lem virus from the pig genome, hope­ful­ly clos­ing the door on any pos­si­ble plague that could be trig­gered.

George Church

Yang says she’s al­ready pub­lished her work show­ing the pos­si­bil­i­ties there, but she’s al­so quick to ac­knowl­edge just how ear­ly-stage the pre­clin­i­cal an­i­mal re­search is, and the long road ahead be­fore this can be test­ed in hu­mans.

“We have to see whether the or­gan is safe, com­pat­i­ble with the hu­man host, be­fore we move in­to the pa­tient,” says the CSO. “2017 is a very im­por­tant year for us” as eGe­n­e­sis con­tin­ues its work on Pigs 2.0 to ad­dress both is­sues.

It took some vi­sion­ary VCs will­ing to fund some rad­i­cal ideas to make this pos­si­ble, says Yang. Bio­mat­ics Cap­i­tal and Arch Ven­ture Part­ners co-led the round with par­tic­i­pa­tion from Khosla Ven­tures, Al­ta Part­ners, Alexan­dria Eq­ui­ties, Her­itage Provider Net­work, Berggru­en Hold­ings North Amer­i­ca Ltd., Up­ris­ing, and Fan Ven­tures. In ad­di­tion, Daniel S. Lynch — the for­mer CEO of Im­Clone — has joined eGe­n­e­sis as ex­ec­u­tive chair­man. They’ve be­gun the process of look­ing for a CEO while the team is set to grow to about 15 to 20 now that the new fi­nanc­ing has ar­rived.

I wrote about Bio­mat­ics and its two man­ag­ing part­ners yes­ter­day. They’ve put to­geth­er a $200 mil­lion fund look­ing for some break­through sci­ence to sup­port. And it doesn’t get much more am­bi­tious than this. Arch, mean­while, has prid­ed it­self in bankrolling break­through ideas in biotech. This was tai­lor made for them.

Xeno­trans­plan­ta­tion may sound a lit­tle far-fetched to some, con­cedes Yang. But if they can get this to work, the con­se­quences of farm­ing tis­sue and or­gans like hearts and kid­neys are enor­mous. In her na­tive Chi­na, she says, the cul­ture and re­li­gion make or­gan do­na­tions rare. Xeno­trans­plan­ta­tion would save a large num­ber of lives.

For now, though, the team is keep­ing its time­lines to it­self. How much time will it take to try this in hu­mans? It’s still to ear­ly to say, says Yang, who al­so didn’t want to spec­u­late. But they’ve just tak­en some big steps for­ward with the fi­nanc­ing.

It’s fi­nal­ly over: Bio­gen, Ei­sai scrap big Alzheimer’s PhI­I­Is af­ter a pre­dictable BACE cat­a­stro­phe rais­es safe­ty fears

Months after analysts and investors called on Biogen and Eisai to scrap their BACE drug for Alzheimer’s and move on in the wake of a string of late-stage failures and rising safety fears, the partners have called it quits. And they said they were dropping the drug — elenbecestat — after the independent monitoring board raised concerns about…safety.

We don’t know exactly what researchers found in this latest catastrophe, but the companies noted in their release that investigators had determined that the drug was flunking the risk/benefit analysis.

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It's not per­fect, but it's a good start: FDA pan­elists large­ly en­dorse Aim­mune's peanut al­ler­gy ther­a­py

Two days after a fairly benign review from FDA staff, an independent panel of experts largely endorsed the efficacy and safety of Aimmune’s peanut allergy therapy, laying the groundwork for approval with a risk evaluation and mitigation strategy (REMS).

Traditionally, peanut allergies are managed by avoidance, but the threat of accidental exposure cannot be nullified. Some allergists have devised a way to dose patients off-label with peanut protein derived from supermarket products to wean them off their allergies. But the idea behind Aimmune’s product was to standardize the peanut protein, and track the process of desensitization — so when accidental exposure in the real world invariably occurs, patients are less likely to experience a life-threatening allergic reaction.

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Lisa M. DeAngelis, MSKCC

MSK picks brain can­cer ex­pert Lisa DeAn­ge­lis as its next CMO — fol­low­ing José Basel­ga’s con­tro­ver­sial ex­it

It’s official. Memorial Sloan Kettering has picked a brain cancer expert as its new physician-in-chief and CMO, replacing José Baselga, who left under a cloud after being singled out by The New York Times and ProPublica for failing to properly air his lucrative industry ties.

His replacement, who now will be in charge of MSK’s cutting-edge research work as well as the cancer care delivered by hundreds of practitioners, is Lisa M. DeAngelis. DeAngelis had been chair of the neurology department and co-founder of MSK’s brain tumor center and was moved in to the acting CMO role in the wake of Baselga’s departure.

Penn team adapts CAR-T tech, reengi­neer­ing mouse cells to treat car­diac fi­bro­sis

After establishing itself as one of the pioneer research centers in the world for CAR-T cancer therapies, creating new attack vehicles to eradicate cancer cells, a team at Penn Medicine has begun the tricky transition of using the basic technology for heart repair work.

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Tal Zaks. Moderna

The mR­NA uni­corn Mod­er­na has more ear­ly-stage hu­man da­ta it wants to show off — reach­ing new peaks in prov­ing the po­ten­tial

The whole messenger RNA field has attracted billions of dollars in public and private investor cash gambled on the prospect of getting in on the ground floor. And this morning Boston-based Moderna, one of the leaders in the field, wants to show off a few more of the cards it has to play to prove to you that they’re really in the game.

The whole hand, of course, has yet to be dealt. And there’s no telling who gets to walk with a share of the pot. But any cards on display at this point — especially after being accused of keeping its deck under lock and key — will attract plenty of attention from some very wary, and wired, observers.

“In terms of the complexity and unmet need,” says Tal Zaks, the chief medical officer, “this is peak for what we’ve accomplished.”

Moderna has two Phase I studies it wants to talk about now.

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Sanofi takes a $260M hit to ex­tri­cate it­self from a dis­as­trous al­liance with Lex­i­con

Sanofi spent $300 million in cash to get into a $1.7 billion alliance with Lexicon on their SGLT1/2 diabetes drug sotagliflozin. And now that the drug has been spurned by the FDA after burning through a program that provided mixed late-stage data and a late shot at a last-place finish, the French pharma giant is forking over another $260 million to get out of the deal.

Sanofi’s unhappiness was already apparent when the company — now under new CEO Paul Hudson — posted a statement back in July that they were dropping the deal. But it wasn’t that simple. 

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Rit­ter bombs fi­nal PhI­II for sole lac­tose in­tol­er­ance drug — shares plum­met

More than two years ago Ritter Pharmaceuticals managed to find enough silver lining in its Phase IIb/III study — after missing the top-line mark — to propel its lactose intolerance toward a confirmatory trial. But as it turned out, the enthusiasm only set the biotech and its investors up to be sorely disappointed.

This time around there’s little left to salvage. Not only did RP-G28 fail to beat placebo in reducing lactose intolerance symptoms, patients in the treatment group actually averaged a smaller improvement. On a composite score measuring symptoms like abdominal pain, cramping, bloating and gas, patients given the drug had a mean reduction of 3.159 while the placebo cohort saw a 3.420 drop on average (one-sided p-value = 0.0106).

Ear­ly snap­shot of Ad­verum's eye gene ther­a­py sparks con­cern about vi­sion loss

An early-stage update on Adverum Biotechnologies’ intravitreal gene therapy has triggered investor concern, after patients with wet age-related macular degeneration (AMD) saw their vision deteriorate, despite signs that the treatment is improving retinal anatomy.

Adverum, on Wednesday, unveiled 24-week data from the OPTIC trial of its experimental therapy, ADVM-022, in six patients who have been administered with one dose of the therapy. On average, patients in the trial had severe disease with an average of 6.2 anti-VEGF injections in the eight months prior to screening and an average annualized injection frequency of 9.3 injections.

Alex Ar­faei trades his an­a­lyst's post for a new role as biotech VC; Sanofi vet heads to Vi­for

Too often, Alex Arfaei arrived too late. 

An analyst at BMO Capital Markets, he’d meet with biotech or pharmaceutical heads for their IPO or secondary funding and his brain, trained on a biology degree and six years at Merck and Endo, would spring with questions: Why this biomarker? Why this design? Why not this endpoint? Not that he could do anything about it. These execs were coming for clinical money; their decisions had been made and finalized long ago.