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.

Forge Bi­o­log­ics’ cGMP Com­pli­ant and Com­mer­cial­ly Vi­able Be­spoke Affin­i­ty Chro­matog­ra­phy Plat­form

Forge Biologics has developed a bespoke affinity chromatography platform approach that factors in unique vector combinations to streamline development timelines and assist our clients in efficiently entering the clinic. By leveraging our experience with natural and novel serotypes and transgene conformations, we are able to accelerate affinity chromatography development by nearly 3-fold. Many downstream purification models are serotype-dependent, demanding unique and time-consuming development strategies for each AAV gene therapy product1. With the increasing demand to propel AAV gene therapies to market, platform purification methods that support commercial-scale manufacturing of high-quality vectors with excellent safety and efficacy profiles are essential.

Cy­to­ki­net­ics’ ALS drug fails PhI­II, leav­ing the biotech with a sin­gle late-stage prospect

Cytokinetics’ candidate for the muscle disease amyotrophic lateral sclerosis, or ALS, failed a Phase III trial, the Bay Area biotech announced Friday morning.

At a second interim analysis of the trial, an independent review committee recommended that Cytokinetics discontinue its COURAGE-ALS trial for reldesemtiv, as it “found no evidence of effect” compared to placebo on the primary or key secondary endpoints.

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CHMP gives thumbs-up for We­govy use in ado­les­cents, along with nine new drug rec­om­men­da­tions

The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) recommended nine drugs for approval this week while also giving thumbs up for six expanded indications, including Novo Nordisk’s approved obesity medication Wegovy for younger people. Wegovy is already approved as an obesity treatment in the EU for adults, and the new indication would allow prescriptions for adolescents aged 12 and older.

Mathai Mammen, FogPharma's next CEO

Math­ai Mam­men hands in J&J's R&D keys to lead Greg Ver­dine’s Fog­Phar­ma 

In the early 1990s, Mathai Mammen was a teaching assistant in Greg Verdine’s Science B46 course at Harvard. In June, the former R&D head at Johnson & Johnson will succeed Verdine as CEO, president and chair of FogPharma, the same month the seven-year-old biotech kickstarts its first clinical trial.

After leading R&D at one of the largest drugmakers in the world, taking the company through more than half a dozen drug approvals in the past few years, not to mention a Covid-19 vaccine race, Mammen departed J&J last month and will take the helm of a Cambridge, MA biotech attempting to go after what Verdine calls the “true emperor of all oncogenes” — beta-catenin.

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Sen­ate Fi­nance Com­mit­tee lobs more bi­par­ti­san pres­sure on­to PBMs

Congress is honing in on how it wants to overhaul the rules of the road for pharmacy benefit managers, with a Senate Finance Committee hearing Thursday serving as the latest example of the Hill’s readiness to make changes to how pharma middlemen operate.

While pledging to ensure patients and pharmacies “don’t get a raw deal,” Finance Committee Chair Ron Wyden (D-OR) laid out the beginning of what looks like a major bipartisan effort — moves the PBM industry is likely to challenge vigorously.

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Nicklas Westerholm, Egetis Therapeutics CEO

Ac­qui­si­tion talks on­go­ing for Swedish rare dis­ease biotech Egetis, shares up al­most 40%

Shares of the Sweden-based rare disease biotech Egetis Therapeutics skyrocketed on Thursday afternoon as the company said it’s engaged in “ongoing discussion” with external parties regarding a “potential acquisition.”

Egetis confirmed rumors with a statement on Thursday while noting that there is no certainty that a takeover offer will be made.

Nonetheless, the possibility of an acquisition has shot up Egetis’ share price. By the afternoon on Thursday, its stock price was {$EGTX.ST} up over 38%. An Egetis spokesperson told Endpoints News in an email that it has no further comments.

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Lu­pus drug de­vel­op­ment mar­ket heat­ing up, while FDA links with ad­vo­ca­cy group to fur­ther ac­cel­er­ate re­search

The long-underserved systemic lupus erythematosus (SLE) market is suddenly buzzing with treatment possibilities. Less than two years after AstraZeneca’s approval for Saphnelo — the first new SLE drug in a decade and joining just one other approved in GSK’s Benlysta – the pipeline of potential drugs numbers in the dozens.

Although most are very early stage — Spherix Global Insights estimates five in Phase II/III — the pharma R&D enthusiasm is catching on among doctors, patients and advocacy groups. On Wednesday, the Lupus Research Alliance and the FDA formed a novel private-public partnership called Lupus Accelerating Breakthroughs Consortium (Lupus ABC) to help advance lupus clinical trial success.

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Stéphane Bancel, Moderna CEO (AP Photo/Markus Schreiber)

Mod­er­na so­lid­i­fies deal with Kenya to build mR­NA man­u­fac­tur­ing fa­cil­i­ty

The mRNA player Moderna is further cementing its presence on the African continent.

Moderna announced on Thursday that it has finalized an agreement with Kenya’s government to partner up and bring an mRNA manufacturing facility to the east African nation. The new facility aims to manufacture up to 500 million doses of vaccines annually. Moderna also said the new facility will have the ability to spike its production capabilities to respond to public health emergencies on the continent or globally.

Green­Light re­ceives buy­out of­fer; Apol­lomics com­pletes SPAC merg­er

RNA biotech GreenLight Biosciences has been handed an offer for potential acquisition.

GreenLight said in a release that it has received a non-binding “indication of interest” from Fall Line Endurance Fund to acquire GreenLight’s capital stock for $0.60 per share in cash. The release said any potential agreement between the two parties would depend on certain conditions.

Through a special committee, the biotech will evaluate the offer but added there’s no certainty a deal will go forward. GreenLight will also not make any more announcements until a deal comes through or “otherwise determines” a statement is necessary.