Marc Lajoie (L) and Scott Boyken (Outpace Bio)

A pair of Lyell founders look to shift the cell ther­a­py par­a­digm — and they've got $30M to get their plan off the ground

Rick Klaus­ner’s Lyell has tak­en the cell ther­a­py field by storm, re­cruit­ing an All-Star team with the goal of cre­at­ing the first CAR-Ts for sol­id tu­mors. But the com­pa­ny may have been too pro­duc­tive — too good at its job — the­o­riz­ing more po­ten­tial ther­a­pies than it could han­dle alone.

As a re­sult, some of that re­search has been spun out in­to a new biotech called Out­pace Bio, with Lyell founders and for­mer co-di­rec­tors of cell and pro­tein en­gi­neer­ing Marc La­joie and Scott Boyken, get­ting things start­ed with a $30 mil­lion Se­ries A on Wednes­day. Lyell it­self is one of the lead in­vestors in the round and is al­so team­ing up with Out­pace in a col­lab­o­ra­tion for T cell ther­a­pies against sol­id tu­mors.

La­joie is tak­ing the reins as CEO with Boyken fill­ing the CSO role. The duo said they first start­ed think­ing about spin­ning out back in June, got the ini­tial op­er­a­tion up and run­ning last No­vem­ber and closed the fi­nanc­ing last month.

The quick ad­vances point to­ward an ear­ly val­i­da­tion of their ap­proach and a hefty in­vestor ap­petite for their part­ner-fo­cused busi­ness mod­el, La­joie told End­points News.

“It’s not a fa­mil­iar pat­tern to a lot of in­vestors, and we’re su­per hap­py with our syn­di­cate,” La­joie said. “They all see the broad­er vi­sion here that re­al­ly, in or­der for cell ther­a­pies to be suc­cess­ful, we’re go­ing to need to make step change in how we can pro­gram cells to be more ef­fi­ca­cious.”

Out­pace’s big idea is to pi­o­neer the next gen­er­a­tion of cell ther­a­pies through many dif­fer­ent ap­proach­es. One promi­nent area is in de no­vo pro­teins. Where­as pro­teins evolved over mil­lions of years to per­form spe­cif­ic func­tions, de no­vo pro­teins can be pro­grammed to ex­e­cute dif­fer­ent tasks, al­low­ing Out­pace to con­trol bi­ol­o­gy in a way that can’t be done nat­u­ral­ly, Boyken said.

Some of the founders’ ear­ly re­search here grabbed head­lines, earn­ing a fea­ture in the Wash­ing­ton Post two years ago — the work came from La­joie and Boyken’s time in David Bak­er’s lab at the Uni­ver­si­ty of Wash­ing­ton.

But de no­vo pro­tein de­sign is just one of the many tools in Out­pace’s tool­box. With the in­ter­dis­ci­pli­nary team they’ve as­sem­bled, La­joie and Boyken hope to shift the line of thought in the cell ther­a­py field in­to one that fo­cus­es more on how cells make de­ci­sions and how they re­spond in the right way at the right time.

“Cells are the unit of co­or­di­nat­ing all func­tions, and when you have these dis­eases like can­cer and au­toim­mu­ni­ty, those are re­al­ly caused by cells mak­ing the wrong de­ci­sions,” La­joie said. “What we want to be able to do is har­ness that com­plex­i­ty of cells in or­der to be able to ad­dress those prob­lems, which are re­al­ly just cells telling oth­er cells the wrong thing.”

The part­ner­ship with Lyell will keep things close to the orig­i­nal fam­i­ly, but al­so gives Out­pace a promi­nent col­league as it looks to make a name on its own. By lock­ing down this col­lab­o­ra­tion and al­so fo­cus­ing on fu­ture team ups, it lets the biotech “cov­er a lot of ground sci­en­tif­i­cal­ly,” La­joie not­ed.

Here, they’ll be hon­ing in on the bi­ol­o­gy of cy­tokines, shoot­ing for im­proved ef­fi­ca­cy in can­cer while try­ing to avoid the tox­i­c­i­ty that comes with it. Out­pace is still work­ing on in­ter­nal pipeline de­vel­op­ment, which La­joie says will pro­vide the foun­da­tion of its part­ner­ship mod­el.

And it’s that mod­el where Out­pace sees the field go­ing in the fu­ture.

“We re­al­ly think that this tech­nol­o­gy and this mod­el is re­al­ly need­ed for the cell ther­a­py space right now,” Boyken said. “In ad­di­tion to the Lyell part­ner­ships, all the con­ver­sa­tions we’ve been hav­ing as well as our ini­tial Se­ries A has re­al­ly proved that that is the case. And we feel re­al­ly val­i­dat­ed that this is a mod­el that’s go­ing to work.”

In ad­di­tion to Lyell, Wednes­day’s round was al­so led by AR­TIS Ven­tures. Ad­di­tion­al par­tic­i­pa­tion came from Ab­stract Ven­tures, Civ­i­liza­tion Ven­tures, Mubadala Cap­i­tal, Play­ground Glob­al, Sah­sen Ven­tures, and WRF Cap­i­tal.

BY­OD Best Prac­tices: How Mo­bile De­vice Strat­e­gy Leads to More Pa­tient-Cen­tric Clin­i­cal Tri­als

Some of the most time- and cost-consuming components of clinical research center on gathering, analyzing, and reporting data. To improve efficiency, many clinical trial sponsors have shifted to electronic clinical outcome assessments (eCOA), including electronic patient-reported outcome (ePRO) tools.

In most cases, patients enter data using apps installed on provisioned devices. At a time when 81% of Americans own a smartphone, why not use the device they rely on every day?

Voting in the 2020 election (AP Images)

The right to vote is fun­da­men­tal — a let­ter from biotech­nol­o­gy in­dus­try lead­ers

Biotech Voices is a collection of exclusive opinion editorials from some of the leading voices in biopharma on the biggest industry questions today. Think you have a voice that should be heard? Reach out to senior editors Kyle Blankenship and Amber Tong.

We oppose all attempts to introduce laws that reduce the rights of US citizens to vote or that restrict them from exercising that right. The right to vote is fundamental to democracy. States that have enacted, or are proposing to enact, legislation to restrict voting are undermining our democracy and posing a threat to our nation. As leaders of the life sciences industry, we stand for what we believe is right for our country, our enterprises, our employees and those who benefit from our work. We join the first groups of business leaders who have challenged these laws and will continue to make our collective voices heard on this matter.

Near­ly a year af­ter Au­den­tes' gene ther­a­py deaths, the tri­al con­tin­ues. What hap­pened re­mains a mys­tery

Natalie Holles was five months into her tenure as Audentes CEO and working to smooth out a $3 billion merger when the world crashed in.

Holles and her team received word on the morning of May 5 that, hours before, a patient died in a trial for their lead gene therapy. They went into triage mode, alerting the FDA, calling trial investigators to begin to understand what happened, and, the next day, writing a letter to alert the patient community so they would be the first to know. “We wanted to be as forthright and transparent as possible,” Holles told me late last month.

The brief letter noted two other patients also suffered severe reactions after receiving a high dose of the therapy and were undergoing treatment. One died a month and a half later, at which point news of the deaths became public, jolting an emergent gene therapy field and raising questions about the safety of the high doses Audentes and others were now using. The third patient died in August.

“It was deeply saddening,” Holles said. “But I was — we were — resolute and determined to understand what happened and learn from it and get back on track.”

Eleven months have now passed since the first death and the therapy, a potential cure for a rare and fatal muscle-wasting disease called X-linked myotubular myopathy, is back on track, the FDA having cleared the company to resume dosing at a lower level. Audentes itself is no more; last month, Japanese pharma giant Astellas announced it had completed working out the kinks of the $3 billion merger and had restructured and rebranded the subsidiary as Astellas Gene Therapies. Holles, having successfully steered both efforts, departed.

Still, questions about precisely what led to the deaths of the 3 boys still linger. Trial investigators released key details about the case last August and December, pointing to a biological landmine that Audentes could not have seen coming — a moment of profound medical misfortune. In an emerging field that’s promised cures for devastating diseases but also seen its share of safety setbacks, the cases provided a cautionary tale.

Audentes “contributed in a positive way by giving a painful but important example for others to look at and learn from,” Terry Flotte, dean of the UMass School of Medicine and editor of the journal Human Gene Therapy, told me. “I can’t see anything they did wrong.”

Yet some researchers say they’re still waiting on Astellas to release more data. The company has yet to publish a full paper detailing what happened, nor have they indicated that they will. In the meantime, it remains unclear what triggered the events and how to prevent them in the future.

“Since Audentes was the first one and we don’t have additional information, we’re kind of in a holding pattern, flying around, waiting to figure out how to land our vehicles,” said Jude Samulski, professor of pharmacology at UNC’s Gene Therapy Center and CSO of the gene therapy biotech AskBio, now a subsidiary of Bayer.

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Pascal Soriot (AstraZeneca via YouTube)

Af­ter be­ing goad­ed to sell the com­pa­ny, Alex­ion's CEO set some am­bi­tious new goals for in­vestors. Then Pas­cal So­ri­ot came call­ing

Back in the spring of 2020, Alexion $ALXN CEO Ludwig Hantson was under considerable pressure to perform and had been for months. Elliott Advisers had been applying some high public heat on the biotech’s numbers. And in reaching out to some major stockholders, one thread of advice came through loud and clear: Sell the company or do something dramatic to change the narrative.

In the words of the rather dry SEC filing that offers a detailed backgrounder on the buyout deal, Alexion stated: ‘During the summer and fall of 2020, Alexion also continued to engage with its stockholders, and in these interactions, several stockholders encouraged the company to explore strategic alternatives.’

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Barbara Weber, Tango Therapeutics CEO (Tango)

It takes two to Tan­go: The biotech us­ing CRISPR to dis­cov­er new can­cer gene tar­gets rides a $353M SPAC deal to Nas­daq

Editor’s note: Interested in following biopharma’s fast-paced IPO market? You can bookmark our IPO Tracker here.

The latest biotech-SPAC deal has arrived, and it’s dancing its way to Nasdaq to the tune of several hundred million dollars.

Tango Therapeutics and its CRISPR-focused search for new cancer genes is reverse merging with Boxer Capital’s blank-check company, the biotech announced Wednesday morning. With a spotlight on three lead programs, Tango expects total proceeds to equal about $353 million in the deal, which includes the roughly $167 million held in the SPAC and an additional $186 million in PIPE financing.

Anand Shah (FDA)

For­mer head of FDA’s med­ical and sci­en­tif­ic af­fairs on Covid: ‘FDA has nev­er been test­ed like this’

Anand Shah has served the American public in a unique way, crisscrossing over the last two administrations between serving as an attending radiation oncologist focused on prostate cancer at NIH, serving as CMO at the Center for Medicare and Medicaid Innovation, and most recently, leading the FDA’s operations on medical and scientific affairs from within the commissioner’s office.

Shah, who stepped down from the FDA in January, caught up with Endpoints News in a phone interview on Tuesday afternoon, offering his thoughts on the agency’s latest decision to pause the J&J vaccinations in the US, and reflecting on his time at an agency during this once-in-a-lifetime pandemic.

Launched by MIT grads, a small start­up gets $20M to back a ro­bot­ics rev­o­lu­tion in cell ther­a­py man­u­fac­tur­ing

As co-director of an experimental cellular therapy process development and manufacturing group at UCSF specializing in T cell therapies for autoimmune conditions, Jonathan Esensten has learned a lot about the challenges involved when his group hand-fashions a cell therapy. Esensten — who was a postdoc in Wendell Lim’s lab and counts the legendary Jeffrey Bluestone as a mentor — gives them all high marks at being great at what they do, but time and again there are variations in the treatments they construct.

UP­DAT­ED: J&J paus­es vac­cine roll­out as feds probe rare cas­es of blood clots

The FDA and CDC have jointly decided to stop administering J&J’s Covid-19 vaccine after reviewing data involving six reported US cases of a rare and severe type of blood clot in individuals after receiving the vaccine.

CDC will convene a meeting of its Advisory Committee on Immunization Practices on Wednesday to further review these cases and assess their potential significance. “FDA will review that analysis as it also investigates these cases. Until that process is complete, we are recommending a pause in the use of this vaccine out of an abundance of caution,” Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research and Anne Schuchat, Principal Deputy Director of the CDC, said in a joint statement Tuesday morning.

Patrizia Cavazzoni, new CDER director

Pa­trizia Cavaz­zoni named per­ma­nent di­rec­tor of CDER, adding to ques­tions around where Wood­cock will end up

Patrizia Cavazzoni on Monday became the permanent director of the FDA’s Center for Drug Evaluation and Research, which puts to rest the idea that Janet Woodcock, Cavazzoni’s predecessor, might return to lead CDER if she isn’t made permanent commissioner.

Woodcock, who’s currently serving as acting commissioner and principal medical advisor to the commissioner, a position she was detailed to last year, may not make the move to permanent commissioner because of lingering questions from Senate Democrats. She previously served as director of CDER since 1994. Cavazzoni took over as acting director of CDER when Woodcock moved over to Operation Warp Speed to run the therapeutics side of the Trump-era program.

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