Striv­ing for high­er res imag­ing of cells, Har­vard team de­buts start­up with back­ing from ARCH, North­pond

As sci­en­tists race to find new ways to look in­to what’s go­ing on in­side cells, ARCH Ven­ture Part­ners and North­pond Ven­tures are in­ject­ing $14 mil­lion in­to a Har­vard team promis­ing to vi­su­al­ize ac­tiv­i­ty on a “sub­cel­lu­lar lev­el” — down to every RNA.

Xi­aowei Zhuang

Xi­aowei Zhuang and David Walt are two of the promi­nent sci­en­tists be­hind Viz­gen. Zhuang, a Howard Hugh­es Med­ical In­sti­tute In­ves­ti­ga­tor and Har­vard pro­fes­sor, was the in­ven­tor of an­oth­er pop­u­lar su­per-res­o­lu­tion mi­croscopy method while David Walt was a sci­en­tif­ic founder of the se­quenc­ing gi­ant Il­lu­mi­na.

Viz­gen was found­ed on MER­FISH, or mul­ti­plexed er­ror-ro­bust flu­o­res­cence in situ hy­bridiza­tion, which was in­vent­ed by Jef­frey Mof­fitt while he was a post­doc at Zhuang’s lab. MER­FISH is, in turn, built on FISH — a tech­nol­o­gy that’s been used for decades to de­tect DNA or RNA tar­gets by send­ing flu­o­res­cent probes to bind to them.

David Walt

The new imag­ing tech­nol­o­gy lever­ages new er­ror de­tec­tion and cor­rec­tion tech­niques, in­clud­ing bar­cod­ing schemes and com­bi­na­to­r­i­al la­bel­ing and imag­ing, to achieve high­er ac­cu­ra­cy and save time.

As a re­sult, it al­lows sci­en­tists to in­ves­ti­gate many more RNA species, ac­cord­ing to the com­pa­ny.

“Giv­en its abil­i­ty to quan­ti­fy RNAs across a wide range of abun­dances with­out am­pli­fi­ca­tion bias while pre­serv­ing na­tive con­text, we en­vi­sion that MER­FISH will en­able many ap­pli­ca­tions of in situ tran­scrip­tom­ic analy­ses of in­di­vid­ual cells in cul­ture or com­plex tis­sues,” Zhuang and Mof­fitt wrote with col­lab­o­ra­tors in a pa­per in­tro­duc­ing the tech in 2015.

Walt fur­ther ex­plained why that’s use­ful in an in­ter­view with Xcon­o­my:

Every im­age that’s tak­en as a con­se­quence of the way that this process is done is done with ap­prox­i­mate­ly 100 nanome­ters of res­o­lu­tion, so you can zoom out all the way to the whole tis­sue and see where the gene ex­pres­sion is oc­cur­ring, then you can zoom in to a sin­gle cell and look at the sub­cel­lu­lar ex­pres­sion of every one of those RNAs that are present in the sam­ple.

Jef­frey Mof­fitt

That can tell re­searchers a lot about cell type, state, or­ga­ni­za­tion, in­ter­ac­tions as well as func­tion with­in the tis­sue.

“The deep in­for­ma­tion un­locked by MER­FISH ex­pos­es bi­ol­o­gy at the net­work lev­el, dri­ving in­sights that will help us build the fu­ture of hu­man health,” Kei­th Cran­dell, man­ag­ing di­rec­tor at ARCH, said in a state­ment.

The 20 un­der 40: In­side the next gen­er­a­tion of bio­phar­ma lead­ers

“Each generation needs a new music,” Francis Crick wrote in 1988, reflecting back on his landmark discovery. Crick was 35, then, in 1953, when he began working with a 23-year-old named James Watson, and 37 when the pair unveiled the double helix. Rosalind Franklin, whose diffraction work undergirded their metal model, was 32.

The model would become the score for a new era in biology, one devoted to cracking the basic structures turning inside life. Subsequent years would bring new conductors and new rhythms: Robert Swanson, 29 when he convinced a 39-year-old Herb Boyer to build a company off his work and call it Genentech; Phillip Sharp, 29 when he discovered RNA splicing and 34 when he co-founded Biogen; Frances Arnold, 36 when she pioneered directed evolution; Feng Zhang, 31 when he published his CRISPR paper.

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FDA Commissioner Stephen Hahn and President Donald Trump at a press briefing on March 19, 2020. (AP Images)

Biotech ex­ecs warn that the FDA is fum­bling their re­sponse to the Covid-19 open-door promise, de­lay­ing progress

A few days ago the FDA touted a procedure for Covid-19 meds that committed the agency to immediate action for developers, formalizing a high-speed response that’s been promised for weeks.

Bioregnum Opinion Column by John Carroll

Decisions that once required months would be measured in hours under the Coronavirus Treatment Acceleration Program. “In many cases” trial protocols could be hammered out in less than a single day. If you had a potential solution to the crisis, the appropriate staffer would be in touch “to get studies underway quickly.”

It would be the ultimate high-speed regulatory pathway from Phase I to approval. Red tape was banished.

But it’s clear that for some — and quite likely many — biopharma execs, the actual agency response has not measured up to the promise. Beyond the front ranks of advanced companies in the field, like Gilead, or for drugs endorsed by President Trump, it may not even come close.

“The first response is this form letter everyone gets,” says one biotech CEO who’s reached out to the FDA on Covid-19. And when you try to cut through that, the ball gets dropped as it is passed from top officials to the frontline staff actually charged with getting things done.

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GSK's Hal Bar­ron buys a $250M stake in George Scan­gos' Vir and makes a bee­line to the clin­ic with Covid-19 an­ti­bod­ies

GlaxoSmithKline is diving straight into the swirling waters of Covid-19 R&D work, and investing $250 million to grab a chunk of equity in one of the emerging stars in infectious disease research to make it official.

GSK put out word this morning that it is partnering with Vir Biotechnology $VIR, the infectious disease startup founded in the Bay Area by former Biogen CEO George Scangos. They’re planning a leap into Phase II studies for 2 preclinical antibody candidates — VIR-7831 and VIR-7832 — that have been engineered to target the SARS-CoV-2 spike protein.

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Ready to de­clare a de­fin­i­tive come­back in two months, Im­munomedics stops PhI­II ear­ly, re­cruits new CEO

More than a year ago, hit by a surprise complete response letter from the FDA, Immunomedics bid its then-CEO, Michael Pehl, adieu and began a 15-month quest to resolve the manufacturing issues cited in the CRL and seek a new leader — all the while moving forward with a Phase III study on its lead drug for metastatic triple-negative breast cancer.

Today the biotech said their stars are finally aligning. Not only is Novartis Oncology vet Harout Semerjian coming on board as CEO to steer what they believe will be a smooth sail to a new PDUFA date in June, Immunomedics has also been informed that their late-stage trial can be stopped early due to “compelling evidence of efficacy.”

Gilead CEO Daniel O'Day attends a meeting with the President and other biopharma leaders at the White House on March 2, 2020 (AP Photo)

Ramp­ing up glob­al pro­duc­tion of remde­sivir, Gilead CEO Dan O’Day de­tails plans to dis­trib­ute 1.5M dos­es to fight Covid-19 — for free

Gilead is still some days away from turning the card on its first round of data on remdesivir’s ability to fight severe cases of Covid-19, but the big biotech is ramping up an emergency supply of a million courses of therapy as it starts free distribution of the drug to tens of thousands of patients under their compassionate use and expanded access program as well as clinical trials.

In his latest open letter posted over the weekend, Gilead CEO Dan O’Day outlined how the company has been successful in cutting production time on remdesivir while repurposing some of their own facilities and turning to contract manufacturers to build a near-term supply of 1.5 million doses. They are still working on efficacy and dosing, but that supply could cover 140,000 courses of treatment. That supply, he added, would be more widely available following a potential approval.

An­oth­er day, an­oth­er boat­load for biotech. Deer­field adds $840M to rush of ven­ture dol­lars

The biotech dollars just keep rolling in.

Even as the world economy faces an economic contraction unprecedented in nature, biotech venture capital firms are announcing huge new investment pots. The latest? Deerfield Management Co.

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Small mol­e­cules, bi­o­log­ics and now gene ther­a­pies: Ger­many's Evotec adds an­oth­er feath­er to its R&D cap

German drug discovery company Evotec — which has a thriving rolodex of biopharma partners such as Bayer, Boehringer Ingelheim, Novartis, Novo Nordisk, Pfizer, Sanofi, and Takeda — is now venturing into gene therapies.

The company swallowed Seattle-based Just Biotherapeutics, a company focused on reducing the cost of manufacturing protein therapies last year. It is now setting up a dedicated R&D site for gene therapies in Austria, in an effort to achieve a “modality-agnostic” repertoire — small molecules, biologics and now gene therapies.

A pair of PhI­II fail­ures spells last rites for Men­lo’s once-promis­ing Mer­ck drug

Four months after an intercontinental merger, Menlo Therapeutics is counting yet another pair of trial failures — ones with significant consequences for the companies, their shareholders and the drug.

In two pivotal Phase III trials, Menlo’s lead drug serlopitant failed to treat pruritus associated with prurigo nodularis — basically itchiness from a particular skin disease that causes red lesions on a person’s arms or legs. Serlopitant has long been the company’s only drug and as recently as 2018, it looked promising enough to support a stock price of $37. In April of that year, a Phase II failure demolished the stock price overnight: $35 to $9. Other subsequent stumbles trickled the ticker down to just above $2.

Af­ter putting aside a bit­ter le­gal feud, Al­ny­lam and Dicer­na chiefs make nice with an RNAi col­lab­o­ra­tion

John Maraganore and Douglas Fambrough used to be at each other’s throats as Alnylam pursued claims that its RNAi rivals at Dicerna had improperly purloined the IP it had picked up from Merck in a bargain basement fire sale.

But that was all settled up close to 2 years ago with a settlement from Dicerna’s Fambrough. And now the two are moving ahead in a close R&D partnership that makes them collaborators on a couple of key disease targets.