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.

How Pa­tients with Epilep­sy Ben­e­fit from Re­al-World Da­ta

Amanda Shields, Principal Data Scientist, Scientific Data Steward

Keith Wenzel, Senior Business Operations Director

Andy Wilson, Scientific Lead

Real-world data (RWD) has the potential to transform the drug development industry’s efforts to predict and treat seizures for patients with epilepsy. Anticipating or controlling an impending seizure can significantly increase quality of life for patients with epilepsy. However, because RWD is secondary data originally collected for other purposes, the challenge is selecting, harmonizing, and analyzing the data from multiple sources in a way that helps support patients.

Jason Kelly, Ginkgo Bioworks CEO (Kyle Grillot/Bloomberg via Getty Images)

UP­DAT­ED: Gink­go Bioworks re­sizes the de­f­i­n­i­tion of go­ing big in biotech, rais­ing $2.5B in a record SPAC deal that weighs in with a whop­ping $15B-plus val­u­a­tion

Ginkgo Bioworks execs always thought big. But today should redefine just how big an upstart biotech player can dream.

In the largest SPAC deal to clear the hurdles to Nasdaq, the biotech that envisioned everything from remaking synthetic meat to a whole new approach to developing drugs has joined forces with one of the biggest disruptors in biotech to slam the Richter scale on dealmaking.

Soon after becoming the darling of the VC crew and clearing the bar on a $4 billion valuation, Ginkgo — a synthetic biotech player out to reprogram cells with industrial efficiency — has now struck a deal to go public in the latest leviathan SPAC that sets its pre-money valuation at $15 billion. In one swift vault, Ginkgo will combine with Harry Sloan’s Soaring Eagle Acquisition Corp. and leap into the public markets.

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FDA un­veils six ICH guide­lines ahead of meet­ing with Health Cana­da

A sign that the FDA’s non-Covid-related processes are beginning to normalize: The release of six guidelines from the International Council of Harmonisation.

Years in development, the ICH documents offer an international perspective on drug development, with these latest guidelines covering everything from recommendations to support the classification of drug substances, featured in the M9 guidance, to standards for nonclinical safety studies for pediatric medicines in the S11 guideline.

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Sanofi, Glax­o­SmithK­line, Boehringer ac­cused of play­ing games, de­stroy­ing emails re­lat­ed to law­suit over con­t­a­m­i­nat­ed Zan­tac

A recent court filing raises new questions about how major pharma companies like Sanofi, GlaxoSmithKline, and Boehringer Ingelheim have dealt with a lawsuit related to recalls of certain over-the-counter heartburn drugs due to the presence of a potentially cancer-causing substance found in them.

More than 70,000 people who took Sanofi’s Zantac and other heartburn drugs containing ranitidine, which have been recalled over the past two years, have sued the manufacturers, including generic drugmakers, and other retailers and distributors as part of a consolidated suit before US District Court Judge Robin Rosenberg in Florida.

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Alvotech takes Ab­b­Vie to court over al­leged patent 'mine­field' sur­round­ing megablock­buster Hu­mi­ra

AbbVie has so far been successful in shooing away competition to its megablockbuster Humira, deploying a number of patents and settlements to keep biosimilars off the US market until 2023. But one Icelandic drugmaker doesn’t want to wait — and on Tuesday, it filed a lawsuit challenging what it called a patent “minefield.”

Alvotech has accused AbbVie of trying to “overwhelm” and “intimidate” it with “an outrageous number of patents of dubious validity,” according to court documents. The company is currently seeking approval for its Humira copycat AVT02, which AbbVie says would infringe upon 62 patents.

Chris Garabedian (Xontogeny)

Per­cep­tive Ad­vi­sors, Xon­toge­ny bring the band back and then some with a $515M sec­ond fund sniff­ing out lead com­pounds

When Perceptive Advisors and startup accelerator Xontogeny initially teamed up on an early-stage VC round in 2019, the partners hoped to prove their investments could be a force multiplier for early-stage companies. Now, with that proof of concept behind them, the pair have closed a second VC round worth more than double the money.

Dubbed PXV Fund II and headed by Xontogeny CEO and former Sarepta head Chris Garabedian, the $515 million fund will target 10 to 12 early-stage preclinical companies with Series A rounds in the $20 million to $40 million range with opportunities for Series B follow-ups. The oversubscribed fund is bringing the band back with initial investors from PXVI as well as new investors that include “top-tier” asset managers, endowments, foundations, family offices, and individual investors.

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A clos­er look at the FDA’s more than 700 pan­dem­ic-re­lat­ed record re­quests to re­place on­site in­spec­tions

As the pandemic constrained the FDA’s ability to travel for onsite manufacturing inspections, the agency increasingly turned to requesting records to fill the gap, even for hundreds of US-based facilities.

FDA explains in its guidance on manufacturing inspections during the pandemic that the agency can request records (not to be confused with the FDA’s remote interactive evaluations) directly from facilities “in advance of or in lieu of” certain onsite inspections. Companies are legally required to fulfill those requests because a denial may be considered limiting an inspection, which could lead to the FDA deeming a drug made at that site to be adulterated.

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Stephen Squinto, Gennao Bio CEO (Gennao)

Alex­ion co-founder Stephen Squin­to is back in the game as CEO, this time for a small gene ther­a­py play­er

With his name already behind a rare disease success story in Alexion, Stephen Squinto was looking for a great story to drive him to jump back into the biotech game. He found that in a fledging non-viral gene therapy company, and now he’s got a few backers on board as well.

On Tuesday, Gennao Bio launched with a $40 million Series A co-led by OrbiMed and Logos Capital with participation by Surveyor Capital. The biotech, which is looking to use its cell-penetrating antibody platform to deliver nucleic acid “payloads” during into the nucleus, had to rush for its initial series — and had a name change along the way.

UP­DAT­ED: Feds charge an­oth­er CRO staffer with fak­ing da­ta in a Glax­o­SmithK­line pe­di­atric asth­ma study

A Florida woman has been indicted as part of a clinical trial fraud scheme over a GlaxoSmithKline pediatric asthma study, the Justice Department announced Tuesday, the latest development in a case where three individuals have already pleaded guilty.

Jessica Palacio was charged with participating in a plot to falsify medical records, giving off the appearance that trial participants were making their scheduled visits to a Miami CRO and taking an experimental asthma medication as required. Palacio was also charged with lying to FDA investigators about her conduct.