Shelia Violette (Q32)

For a post-Soliris world, At­las-backed Q32 Bio out­lines $46M next-gen com­ple­ment play

Long be­fore Alex­ion kin­dled a re­nais­sance of com­ple­ment ther­a­peu­tics with the in­tro­duc­tion of the first an­ti-C5 an­ti­body, Mike Holers — a long­time pro­fes­sor at the Uni­ver­si­ty of Col­orado School of Med­i­cine — be­came fas­ci­nat­ed with the host de­fense sys­tem as he com­plet­ed his rheuma­tol­ogy train­ing. As Soliris be­gins to fade and fol­low-on, ri­vals and even gener­ics catch up to the stan­dard bear­er. Holers is de­but­ing a next-gen­er­a­tion ap­proach he’s been re­fin­ing with At­las Ven­ture over the last two years.

Fund­ed with $46 mil­lion pro­vid­ed by At­las as well as Or­biMed, Abing­worth and Sanofi Ven­tures, Q32 wants to bring the first tis­sue-tar­get­ed com­ple­ment drugs in­to the clin­ic by the end of 2021. The CU Health­care In­no­va­tion Fund and Chil­dren’s Hos­pi­tal Col­orado are al­so in­vestors.

Michael Holers

Over his years of re­search and col­lab­o­ra­tion with col­league Joshua Thur­man and Steven Tom­lin­son at the Med­ical Uni­ver­si­ty of South Car­oli­na, Holers told End­points News, the com­ple­ment field has evolved to re­veal tar­gets that can help sci­en­tists ze­ro in on dis­eased tis­sue, rather than block­ing com­ple­ment ac­ti­va­tion sys­tem­i­cal­ly.

When you think through how the com­ple­ment sys­tem func­tions, you see “a lot of high lev­els of sys­temic pro­teins, but its re­al tis­sue in­jury, its re­al dam­age is lo­cal­ized to cells and tis­sues on the sur­face of cells, in the in­ter­sti­tium,” he said.

Yet know­ing how to di­rect drugs to the right places wasn’t enough, as his ex­pe­ri­ence with a pre­vi­ous start­up dubbed Tal­i­gen — which had an ear­ly tis­sue tar­get­ing tech­nol­o­gy that was ac­quired but ul­ti­mate­ly re­tired by Alex­ion — showed. You al­so need deep knowl­edge in pro­tein en­gi­neer­ing to make sure you’re rein­ing in the com­ple­ment sys­tem prop­er­ly.

That’s where At­las and She­lia Vi­o­lette, Q32’s co-founder, CSO and pres­i­dent of re­search, comes in.

Rather than an­ti­bod­ies, they are work­ing on fu­sion pro­teins that grab the tis­sue-spe­cif­ic tar­gets on one end and car­ries a nat­u­ral­ly oc­cur­ring pro­tein that nor­mal­ly keeps the sys­tem in check.

“One of the things that is now bet­ter un­der­stood is that across pret­ty much any dis­ease where com­ple­ment ac­ti­va­tion be­comes dys­reg­u­lat­ed, it’s be­cause you’re los­ing the op­ti­mal func­tion of those neg­a­tive reg­u­la­tor pro­teins,” Vi­o­lette, who left a lengthy ca­reer at Bio­gen to be­come At­las’ en­tre­pre­neur-in-res­i­dence in 2016, said.

It’s so cen­tral to what they do that the start­up re­named it­self from Ad­mirx to Q32, draw­ing in­spi­ra­tion from the re­gion of the chro­mo­some where these mol­e­cules re­side, she added; the num­ber 32 al­so is a shoutout to the im­por­tance of strat­e­gy, em­bod­ied by chess.

Michael Brox­son

The lead can­di­date blocks both C3 and C5 con­ver­tases, Holers said, play­ing in­to mul­ti­ple parts of the sys­tem. Their ini­tial fo­cus will be in kid­ney and skin dis­eases.

On top of the plat­form play, Q32 al­so in-li­censed an un­re­lat­ed an­ti­body that blocks IL-7 re­cep­tors from Bris­tol My­ers Squibb. The deal closed in Oc­to­ber, as Bris­tol My­ers Squibb was auc­tion­ing off as­sets on the back of its Cel­gene buy­out. At that point, it was al­so close to nom­i­nat­ing its first de­vel­op­ment can­di­date on the com­ple­ment side.

“In the spir­it of full trans­paren­cy,” CEO Mike Brox­son, a Take­da vet and for­mer chief of Goldfinch Bio who of­fi­cial­ly came on board in Feb­ru­ary, said, “but for Covid we might have launched the com­pa­ny sev­er­al months ago.”

While the com­pound, ADX-914, re­sides in the realm of adap­tive im­mu­ni­ty rather than in­nate im­mu­ni­ty where its tech plat­form op­er­ates, it pur­sues a sim­i­lar strat­e­gy not to com­plete­ly knock out a prob­lem­at­ic path­way but fine tune it. (Just don’t ex­pect any more such deals; af­ter this the 12-per­son team will fo­cus en­tire­ly on the com­ple­ment plat­form.)

“We’re re­al­ly about restor­ing home­osta­sis,” Brox­son said.

Qual­i­ty Con­trol in Cell and Gene Ther­a­py – What’s Re­al­ly at Stake?

In early 2021, Bluebird Bio was forced to suspend clinical trials of its gene therapy for sickle cell disease after two patients in the trial developed cancer. As company scientists rushed to assess whether there was any causal link between the therapy and the cancer cases, Bluebird’s stock value plummeted – as did those of multiple other biopharma companies developing similar therapies.

While investigations concluded that the gene therapy was unlikely to have caused cancer, investors and the public may be more skittish regarding the safety of gene and cell therapies after this episode. This recent example highlights how delicate the fields of cell and gene therapy remain today, even as they show great promise.

Brad Bolzon (Versant)

Ver­sant pulls the wraps off of near­ly $1B in 3 new funds out to build the next fleet of biotech star­tups. And this new gen­er­a­tion is built for speed

Brad Bolzon has an apology to offer by way of introducing a set of 3 new funds that together pack a $950 million wallop in new biotech creation and growth.

“I want to apologize,” says the Versant chairman and managing partner, laughing a little in the intro, “that we don’t have anything fancy or flashy to tell you about our new fund. Same team, around the same amount of capital, same investment strategy. If it ain’t broke, don’t fix it.”

But then there’s the flip side, where everything has changed. Or at least speeded into a relative blur. Here’s Bolzon:

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Sen. Patty Murray (D-WA) (Graeme Sloan/Sipa USA/Sipa via AP Images)

Sen­a­tors to NIH: Do more to pro­tect US bio­med­ical re­search from for­eign in­flu­ence

Although Thursday’s Senate health committee hearing was focused on how foreign countries and adversaries might be trying to steal or negatively influence biomedical research in the US, the only country mentioned by the senators and expert witnesses was China.

Committee chair Patty Murray (D-WA) made clear in her opening remarks that the US cannot “let the few instances of bad actors” overshadow the hard work of the many immigrant researchers in the US, many of which have won Nobel prizes for their work. But she also said, “There is more the NIH can be doing here.”

JP Gabriel, Ocugen

JP Gabriel watched from the bleach­ers as the pan­dem­ic raged. Now head of sup­ply chain at Ocu­gen, he's ready to bat

The world was in the middle of the most pressing public health risk his generation had ever seen, and JP Gabriel felt like he was sitting on the sidelines. As a VP of biologics and mRNA manufacturing at Ultragenyx, Gabriel watched from the sidelines as players like Pfizer/BioNTech and Moderna used mRNA tech to chase their own Covid-19 vaccines.

This month, Gabriel got the chance to get his hands dirty against the pandemic — but it won’t be with mRNA.

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Jenny Rooke (Genoa Ventures)

Ear­ly Zymer­gen in­vestor Jen­ny Rooke re­flects on 'chimeras' in biotech, what it takes to spot a $500M gem

When Jenny Rooke first heard of Zymergen back in 2014, she knew she was looking at something different and exciting. The Emeryville, CA biotech held the promise of blending biology and technology to solve a huge unmet need for cost-effective chemicals — of all things — and a stellar founding team to boot.

But back then, West Coast venture capitalists didn’t see in Zymergen the one thing they were looking for in a winning biotech: therapeutic potential. Rooke, however, saw an opportunity and made her bets. Seven years later, that bet is paying off in a big way.

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Saurabh Saha at Endpoints News' #BIO19

On the heels of $250M launch, Centes­sa barges ahead with an IPO to fu­el its 10-in-1 Medicxi pipeline

Francesco De Rubertis made no secret of IPO plans for Centessa, his 10-in-1 legacy play. Barely two months later, the S-1 is in.

The hot-off-the-press filing depicts the same grand vision that the longtime VC touted when he did the rounds in February: Take the asset-centric mindset that he’s been preaching at Medicxi over the years, and roll up a bunch of biotech upstarts, with unrelated risk profiles, into 1 pharma company that can carry on the development at scale.

Law pro­fes­sors call for FDA to dis­close all safe­ty and ef­fi­ca­cy da­ta for drugs

Back in early 2018 when Scott Gottlieb led the FDA, there was a moment when the agency seemed poised to release redacted complete response letters and other previously undisclosed data. But that initiative never gained steam.

Now, a growing chorus of researchers are finding that a dearth of public data on clinical trials and pharmaceuticals means industry and the FDA cannot be held accountable, two law professors from Yale and New York University write in an article published Wednesday in the California Law Review.

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Novavax CEO Stanley Erck at the White House in 2020 (Andrew Harnik, AP Images)

As fears mount over J&J and As­traZeneca, No­vavax en­ters a shaky spot­light

As concerns rise around the J&J and AstraZeneca vaccines, global attention is increasingly turning to the little, 33-year-old, productless, bankruptcy-flirting biotech that could: Novavax.

In the now 16-month race to develop and deploy Covid-19 vaccines, Novavax has at times seemed like the pandemic’s most unsuspecting frontrunner and at times like an overhyped also-ran. Although they started the pandemic with only enough cash to last 6 months, they leveraged old connections and believers into $2 billion and emerged last summer with data experts said surpassed Pfizer and Moderna. They unveiled plans to quickly scale to 2 billion doses. Then they couldn’t even make enough material to run their US trial and watched four other companies beat them to the finish line.

FDA of­fers scathing re­view of Emer­gent plan­t's san­i­tary con­di­tions, em­ploy­ee train­ing af­ter halt­ing pro­duc­tion

The FDA wrapped up its inspection of Emergent’s troubled vaccine manufacturing plant in Baltimore on Tuesday, after halting production there on Monday. By Wednesday morning, the agency already released a series of scathing observations on the cross contamination, sanitary issues and lack of staff training that caused the contract manufacturer to dispose of millions of AstraZeneca and J&J vaccine doses.