A fast-mov­ing en­try in the gene edit­ing race is speed­ing to the clin­ic, fu­eled by an $83.5M round

The last time I talked to Arthur Tzian­a­bos a lit­tle more than a year ago, the CEO at Ho­mol­o­gy Med­i­cines had a team of about 10 and a new ap­proach to in vi­vo gene trans­fer and gene edit­ing that was still in pre­clin­i­cal liftoff mode. To­day, Tzian­a­bos is whip­ping the cov­er off a whop­ping $83.5 mil­lion ven­ture round with some A-lis­ter sup­port so his team of 40-plus can take their work in­to hu­mans for the first time.

“We re­al­ly have made a tremen­dous amount of progress,” Tzian­a­bos tells me by way of catch­ing up.

There’s a lead in­di­ca­tion now — an in­born er­ror of me­tab­o­lism in­volv­ing the liv­er is as spe­cif­ic as he wants to get right now — with a long list of pre­clin­i­cal projects in CD34 dis­eases, cys­tic fi­bro­sis, CNS dis­eases, Duchenne mus­cu­lar dy­s­tro­phy and so on. When you’re a pre­clin­i­cal biotech work­ing on the foun­da­tion for a plat­form tech that seeks to ed­it and cor­rect genes us­ing your own ade­no-as­so­ci­at­ed virus vec­tors, you can think about what you want to keep and what you want to part­ner.

Ho­mol­o­gy is one of the up­starts trav­el­ing one step be­hind the pi­o­neer­ing crews that boot­ed up gene edit­ing in a big way. That group in­cludes Ed­i­tas, CRISPR Ther­a­peu­tics and In­tel­lia — which all made vir­tu­al­ly overnight de­buts on­to the pub­lic mar­kets with a set of IPOs that at­tract­ed a tremen­dous amount of in­vestor at­ten­tion — and which sagged as the long clin­i­cal road to an ac­tu­al ther­a­py came in­to view.

These days, the pi­o­neers al­so are spend­ing a sig­nif­i­cant amount of time wran­gling over patents as they work to get in­to the clin­ic. It’s no easy task birthing a new tech­nol­o­gy, as Ed­i­tas found out when it had to de­lay its time­line re­cent­ly in or­der to work through a de­lay on the man­u­fac­tur­ing side.

Tzian­a­bos and his team are learn­ing from the lead­ers, and may al­so be mov­ing faster to­ward the clin­ic than the out-front ri­vals know.

“We want to be there in a short pe­ri­od of time,” says the CEO about hu­man tri­als. (No, he isn’t be­ing more spe­cif­ic than that.) He has plen­ty of cash now to get through a proof-of-con­cept tri­al, and a chance to sign up a part­ner or two to help ex­pand the pipeline work more quick­ly.

Tzian­a­bos gives his Bed­ford, MA-based biotech high scores for their pre­clin­i­cal ef­fort on fine tun­ing gene trans­fer and edit­ing, start­ing with a page out of the book of na­ture on the way DNA is re­paired to point to an in vi­vo ap­proach that can be a sim­pler and more di­rect means of po­ten­tial­ly cur­ing ge­net­ic dis­eases.

But he’s al­so start­ing to look at what needs to hap­pen on man­u­fac­tur­ing to pre­vent fu­ture foul-ups.

“I think peo­ple got a lit­tle over their skis on that point,” he says about the field. And he has built up in­ter­nal and ex­ter­nal man­u­fac­tur­ing ops.

“CMC, bi­o­log­ics man­u­fac­tur­ing is com­pli­cat­ed any­way,” says Tzian­a­bos, point­ing to the cri­sis that Gen­zyme went through be­fore Sanofi ac­quired the com­pa­ny. The new gene edit­ing com­pa­nies are com­ing straight out of acad­e­mia, he adds, and that kind of tech al­ways takes time to in­dus­tri­al­ize.

At Ho­mol­o­gy, says the CEO, “that was one of our main fo­cus­es right out of the gate.”

Add it all up, he says, and “I would put us not too far be­hind where these guys (the pi­o­neers) are. That’s what at­tract­ed this team to the tech­nol­o­gy.”

It is al­so what at­tract­ed Deer­field, which led the round. New in­vestors in­clude Fi­deli­ty Man­age­ment and Re­search Com­pa­ny, HBM Health­care In­vest­ments, Mav­er­ick Ven­tures, No­var­tis, Rock Springs Cap­i­tal, Vi­da Ven­tures, Vi­vo Cap­i­tal and Alexan­dria Ven­ture In­vest­ments. Ex­ist­ing in­vestors 5AM Ven­tures, ARCH Ven­ture Part­ners and Temasek al­so jumped back in.

As of now, Ho­mol­o­gy has raised a to­tal of $127 mil­lion in a lit­tle more than a year, of­fer­ing an­oth­er ex­am­ple of the kind of funds that are be­ing spent to pur­sue a rad­i­cal new tech­nol­o­gy. It’s still ear­ly stages, with years of clin­i­cal work ahead, but Ho­mol­o­gy is mov­ing fast.

Aerial view of Genentech's campus in South San Francisco [Credit: Getty]

Genen­tech sub­mits a big plan to ex­pand its South San Fran­cis­co foot­print

The sign is still there, a quaint reminder of whitewashed concrete not 5 miles from Genentech’s sprawling, chrome-and-glass campus: South Francisco The Industrial City. 

The city keeps the old sign, first erected in 1923, as a tourist site and a kind of civic memento to the days it packed meat, milled lumber and burned enough steel to earn the moniker “Smokestack of the Peninsula.” But the real indication of where you are and how much has changed both in San Francisco and in the global economy since a couple researchers and investors rented out an empty warehouse 40 years ago comes in a far smaller blue sign, resembling a Rotary Club post, off the highway: South San Francisco, The Birthplace of Biotech.

Here comes the oral GLP-1 drug for di­a­betes — but No­vo Nordisk is­n't dis­clos­ing Ry­bel­sus price just yet

Novo Nordisk’s priority review voucher on oral semaglutide has paid off. The FDA approval for the GLP-1 drug hit late Friday morning, around six months after the NDA filing.

Rybelsus will be the first GLP-1 pill to enter the type 2 diabetes market — a compelling offering that analysts have pegged as a blockbuster drug with sales estimates ranging from $2 billion to $5 billion.

Ozempic, the once-weekly injectable formulation of semaglutide, brought in around $552 million (DKK 3.75 billion) in the first half of 2019.

As Nas­daq en­rolls the fi­nal batch of 2019 IPOs, how have the num­bers com­pared to past years?

IGM Biosciences’ upsized IPO haul, coming after SpringWorks’ sizable public debut, has revved up some momentum for the last rush of biotech IPOs in 2019.

With 39 new listings on the books and roughly two more months to go before winding down, Nasdaq’s head of healthcare listings Jordan Saxe sees the exchange marking 50 to 60 biopharma IPOs for the year.

“December 15 is usually the last possible day that companies will price,” he said, as companies get ready for business talks at the annual JP Morgan Healthcare Conference in January.

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Oxitec biologist releases genetically modified mosquitoes in Piracicaba, Brazil in 2016 [credit: Getty Images]

In­trex­on unit push­es back against claims its GM mos­qui­toes are mak­ing dis­ease-friend­ly mu­tants

When the hysteria of Zika transmission sprang into the American zeitgeist a few years ago, UK-based Oxitec was already field-testing its male Aedes aegypti mosquito, crafted to possess a gene engineered to obliterate its progeny long before maturation.

But when a group of independent scientists evaluated the impact of the release of these genetically-modified mosquitoes in a trial conducted by Oxitec in Brazil between 2013 and 2015, they found that some of the offspring had managed to survive — prompting them to speculate what impact the survivors could have on disease transmission and/or insecticide resistance.

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Pur­due threat­ens to walk away from set­tle­ment, asks to pay em­ploy­ees mil­lions in bonus­es

There are two updates on the lawsuit against Purdue Pharma over its role in fueling the opioid epidemic, as the Sackler family threatens to walk away from their pledge to pay out $3 billion if a bankruptcy judge does not stop outstanding state lawsuits against them. At the same time, the company has asked permission to pay millions in bonuses to select employees.

Purdue filed for chapter 11 bankruptcy this week as part of its signed resolution to over 2,000 lawsuits. The deal would see the Sackler family that owns Purdue give $3 billion from their personal wealth and the company turned into a trust committed to curbing and reversing overdoses.

While No­var­tis ban­ish­es Zol­gens­ma scan­dal scars — Bio­gen goes on a Spin­raza 'of­fen­sive'

While Novartis painstakingly works to mop up the stench of the data manipulation scandal associated with its expensive gene therapy for spinal muscular atrophy (SMA) Zolgensma— rival Biogen is attempting to expand the use of its SMA therapy, Spinraza. 

The US drugmaker $BIIB secured US approval for Spinraza for use in the often fatal genetic disease in 2016. The approval covered a broad range of patients with infantile-onset (most likely to develop Type 1) SMA. 

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Eye­ing big ther­a­peu­tic push, Gink­go bags $290M to build a cell pro­gram­ming em­pire

Ginkgo Bioworks is on a roll. Days after publicizing a plan to nurture new startups via partnerships with accelerators Y Combinator and Petri, the Boston biotech says it has raised another $290 million for its cell programming platform to reach further and wider.

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UP­DAT­ED: Speak­er Nan­cy Pelosi to un­veil bill for fed­er­al­ly ne­go­ti­at­ed drug prices

After months of buzz from both sides of the aisle, Speaker Nancy Pelosi will today introduce her plan to allow the federal government to negotiate prices for 250 prescription drugs, setting up a showdown with a pharmaceutical industry working overtime to prevent it.

The need to limit drug prices is a rare point of agreement between President Trump and Democrats, although the president has yet to comment on the proposal and will likely face pressure to back a more conservative option or no bill at all. Republican Senator Chuck Grassley is reportedly lobbying his fellow party members on a more modest proposal he negotiated with Democratic Senator Ron Wyden in July.

Jeff Kindler's Cen­trex­ion re­news bid to make pub­lic de­but

Jeffrey Kindler’s plan to take his biotech — which is developing a slate of non-opioid painkillers — public, is back on.

The Boston based company, led by former Pfizer $PFE chief Kindler, originally contemplated a $70 million to $80 million IPO last year— but eventually postponed that strategy. On Wednesday, the company revived its bid to make a public debut in a filing with the SEC — although no pricing details were disclosed.