Surgeons at University of Maryland Medical Center performing the first pig-to-human transplant (University of Maryland School of Medicine)

In his­toric first, sur­geons trans­plant a ge­net­i­cal­ly mod­i­fied pig heart in­to a dy­ing pa­tient

Two and a half months af­ter an NYU team du­bi­ous­ly claimed to have per­formed the first ever pig-to-hu­man or­gan trans­plant, a team at the Uni­ver­si­ty of Mary­land Med­ical Cen­ter ap­pears to have ac­tu­al­ly done it.

The cen­ter an­nounced Mon­day evening that sur­geons there suc­cess­ful­ly trans­plant­ed a ge­net­i­cal­ly mod­i­fied pig heart in­to a 57-year-old pa­tient with ter­mi­nal heart dis­ease. The pa­tient, David Ben­nett, was too sick to qual­i­fy for a hu­man donor and had run out of oth­er op­tions.

“I con­sid­er this a tremen­dous break­through for the field,” Jim Mark­mann, head of trans­plant surgery at MGH and a xeno­trans­plant ex­pert not in­volved in the UMD case, said in an email.

In the decades-long ef­fort to make xeno­trans­plan­ta­tion a re­al­i­ty, the new re­port dif­fered from Oc­to­ber’s in key re­spects. Most glar­ing­ly, the pa­tient at NYU was al­ready brain-dead. Doc­tors su­tured a porcine kid­ney to the out­side of her body and could on­ly track whether the pa­tient im­me­di­ate­ly re­ject­ed the for­eign or­gan. As pre­dict­ed by years of mon­key ex­per­i­ments, she didn’t.

Ben­nett, with the doc­tor who per­formed the surgery, Bart­ley Grif­fith

The new pro­ce­dure may of­fer the first test of whether porcine or­gans, when bred with the right bat­tery of ge­net­ic ed­its, can ac­tu­al­ly serve as a re­place­ment or sup­ple­ment to hu­man or­gan do­na­tion and al­low pa­tients to live func­tion­al lives.

So far, so good, ac­cord­ing to UMD, al­though doc­tors cau­tioned that it’s still ear­ly. Ben­nett is do­ing well three days af­ter the pro­ce­dure, the cen­ter said. The New York Times re­port­ed he was still on a heart-lung by­pass ma­chine, but that is com­mon for any new heart trans­plant re­cip­i­ent.

“It was ei­ther die or do this trans­plant. I want to live. I know it’s a shot in the dark, but it’s my last choice,” Ben­nett said, in a state­ment pro­vid­ed by UMD. “I look for­ward to get­ting out of bed af­ter I re­cov­er.”

The news comes as xeno­trans­plan­ta­tion, af­ter years on the med­ical mar­gin, ap­peared to be inch­ing clos­er to re­al­i­ty, po­ten­tial­ly of­fer­ing an al­ter­na­tive to the hun­dreds of thou­sands of pa­tients in the US and around the world wait­ing for or­gans. Af­ter a high-pro­file at­tempt to trans­plant a ba­boon heart in­to a ba­by with a con­gen­i­tal heart de­fect failed in 1983, re­searchers and sev­er­al com­pa­nies tried to use re­com­bi­nant DNA tech­niques to make pigs with or­gans that hu­mans can ac­cept.

Those ef­forts dis­in­te­grat­ed vir­tu­al­ly overnight af­ter the dis­cov­ery of a retro­virus com­mon to pigs, ter­ri­fy­ing health of­fi­cials then deal­ing with the height of the US’s HIV cri­sis. But in the last decade, as fur­ther re­search low­ered con­cerns that porcine en­doge­nous retro­virus, or PERV as the retro­virus is known, can trans­mit to hu­mans and as new genome-en­gi­neer­ing tools be­came avail­able, the field has re­vived it­self.

Ben­nett’s new heart came from Re­vivi­cor, a sub­sidiary of the $9 bil­lion rare dis­ease biotech Unit­ed Ther­a­peu­tics. It’s the same com­pa­ny that pro­vid­ed the NYU kid­ney, but Ben­nett’s heart came from pigs with far more ge­net­ic ed­its: 10 in to­tal — four pig genes knocked out to pre­vent re­jec­tion, six hu­man genes knocked in to pro­mote tol­er­ance, and one ad­di­tion­al pig gene knocked out to pre­vent ex­ces­sive tis­sue growth.

Sci­en­tists are di­vid­ed on the ex­act num­ber of ed­its need­ed to pre­vent re­jec­tion. Re­vivi­cor has been com­pet­ing with a cou­ple aca­d­e­m­ic labs and eGe­n­e­sis, the ven­ture cap­i­tal fa­vorite, to make xeno­trans­plan­ta­tion a re­al­i­ty. eGe­n­e­sis, found­ed by ge­net­ic en­gi­neer Luhan Yang and backed with over $260 mil­lion from pri­vate in­vestors, makes an even high­er num­ber of ed­its — they’ve nev­er said quite how many — as they try to elim­i­nate PERV en­tire­ly.

To be clear, though, Ben­nett’s surgery is still a re­mark­ably ear­ly step by the stan­dard process of drug de­vel­op­ment. The FDA OK’d the pro­ce­dure un­der com­pas­sion­ate use, an emer­gency mea­sure to make ex­per­i­men­tal ther­a­pies avail­able to pa­tients with no oth­er op­tions.

For xeno­trans­plan­ta­tion to ac­tu­al­ly be­come a med­ical­ly ap­proved prod­uct, one com­pa­ny or aca­d­e­m­ic team will have to show the mod­i­fied porcine or­gans are safe and ef­fec­tive in a clin­i­cal tri­al.

Re­vivi­cor, eGe­n­e­sis and oth­ers are try­ing to com­plete the mon­key stud­ies nec­es­sary to re­ceive clear­ance for such a tri­al. They will like­ly need to show the non-hu­man pri­mates have con­sis­tent sur­vival af­ter trans­plant. Ex­perts sug­gest one could be­gin in 2023.

IDC: Life Sci­ences Firms Must Em­brace Dig­i­tal Trans­for­ma­tion Now

Pre-pandemic, the life sciences industry had settled into a pattern. The average drug took 12 years and $2.9 billion to bring to market, and it was an acceptable mode of operations, according to Nimita Limaye, Research Vice President for Life Sciences R&D Strategy and Technology at IDC.

COVID-19 changed that, and served as a proof-of-concept for how technology can truly help life sciences companies succeed and grow, Limaye said. She recently spoke about industry trends at Egnyte’s Life Sciences Summit 2022. You should watch the entire session, free and on-demand, but here’s a brief recap of why she’s urging life sciences companies to embrace digital transformation.

Martin Landray, Protas CEO (Illustration: Assistant Editor Kathy Wong for Endpoints News)

Those big bil­lion-dol­lar PhI­II stud­ies? Mar­tin Lan­dray says they can be done for a tiny frac­tion of the cost

Martin Landray knows what controversy in clinical drug development feels like, from first-hand experience.

Landray was the chief architect of RECOVERY, a study that pitted a variety of drugs against Covid-19. And he offered some landmark data that would help push dexamethasone out into broader use as a cheap treatment, while helping ice hydroxy’s reputation as a clear misfire.

“Lots of people told us we shouldn’t use it,” Landray says about dexamethasone and Covid-19. “It was dangerous. We shouldn’t even do a trial. They also cared about hydroxychloroquine and lots of people said we shouldn’t do a trial because it must be used. I’ve got the letters from both sets of people.”

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FDA ap­proves one of the prici­est new treat­ments of all time — blue­bird's gene ther­a­py for be­ta tha­lassemia

The FDA on Wednesday approved the first gene therapy for a chronic condition — bluebird bio’s new Zynteglo (beti-cel) as a potentially curative treatment for those with transfusion-dependent thalassemia.

The thumbs-up from the FDA follows a unanimous adcomm vote in June, with outside experts pointing to extraordinary efficacy, with 89% of subjects with TDT who received beti-cel having achieved transfusion independence.

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Geoffrey Porges, new Schrödinger CFO

Long­time an­a­lyst Ge­of­frey Porges de­parts SVB to lead fi­nances at a drug dis­cov­ery shop

Geoffrey Porges has ended his two-decade run as a biotech analyst, as the former SVB Securities vice chair began as CFO of Schrödinger on Thursday.

The long-running analyst, who previously headed up vaccines marketing at Merck before the turn of the millennium, will lead the financial operations of the 700-employee company as Schrödinger broadens its focus from a drug discovery partner to also building out an in-house pipeline, with clinical trial No. 1 set to begin next quarter.

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James Sabry, Roche global head of pharma partnering

Roche, Genen­tech plunk down $60M up­front to part­ner with Chi­nese phar­ma on PRO­TAC-based prostate can­cer drug

Roche and Genentech are always on the hunt for deals, and on Thursday they found their newest partner.

The pair will team up with the Chinese pharma company Jemincare to push forward a new program for prostate cancer, the companies announced. Roche is ponying up $60 million upfront to get its hands on the candidate and promising up to $590 million in biobucks, plus royalties, down the line.

In return, Genentech will get a worldwide license to develop the program, known as JMKX002992, and bring it to market.

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Andrew Hopkins, Exscientia CEO

Ex­sci­en­tia ter­mi­nates Bay­er pact half a year ear­ly, col­lect­ing small por­tion of €240M promised

Bayer and Exscientia are winding down their three-year collaboration, leaving the big German pharma to take the AI-designed compounds born out of the pact further.

London-based Exscientia revealed in its Q2 update that the partners have “mutually agreed to end” their collaboration, which kicked off in early 2020, after recently achieving a drug discovery milestone. In an SEC filing, Exscientia said it terminated the pact on May 30, about six months early.

Bayer's first DTC ad campaign for chronic kidney disease drug Kerendia spells out its benefits

Bay­er aims to sim­pli­fy the com­plex­i­ties of CKD with an ABC-themed ad cam­paign

Do you know the ABCs of CKD in T2D? Bayer’s first ad campaign for Kerendia tackles the complexity of chronic kidney disease with a play on the acronym (CKD) and its connection to type 2 diabetes (T2D).

Kerendia was approved last year as the first and only non-steroidal mineralocorticoid receptor antagonist to treat CKD in people with type 2 diabetes.

In the TV commercial launched this week, A is for awareness, B is for belief and C is for cardiovascular, explained in the ad as awareness of the connection between type 2 and kidney disease, belief that something can be done about it, and cardiovascular events that may be reduced with treatment.

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James Mock, incoming CFO at Moderna

Mod­er­na taps new CFO from PerkinElmer af­ter for­mer one-day CFO oust­ed

When Moderna hired a new CFO last year,  it didn’t expect to see him gone after only one day. Today the biotech named his — likely much more vetted — replacement.

The mRNA company put out word early Wednesday that after the untimely departure of then brand-new CFO Jorge Gomez, it has now found a replacement in James Mock, the soon-to-be former CFO at diagnostics and analytics company PerkinElmer.

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Etleva Kadilli, director of UNICEF’s supply division

GSK lands first-ever UNICEF con­tract for malar­ia vac­cine worth $170M

GSK has landed a new first from UNICEF the first-ever contract for malaria vaccines, worth up to $170 million for 18 million vaccine doses distributed over the next three years.

The vaccine, known as Mosquirix or RTS,S, won WHO’s backing last October after a controversial start, but UNICEF said these doses will potentially save thousands of lives every year.

“We hope this is just the beginning,” Etleva Kadilli, director of UNICEF’s supply division, said. “Continued innovation is needed to develop new and next-generation vaccines to increase available supply, and enable a healthier vaccine market. This is a giant step forward in our collective efforts to save children’s lives and reduce the burden of malaria as part of wider malaria prevention and control programmes.”