Matthew Porteus (Credit: Timothy Archibald for Stanford Children's)

Ver­sant teams up with Stan­ford gene edit­ing ex­perts on a $45M next-gen play — mar­ry­ing CRISPR and AAV to fix sick­le cell

When a re­searchers talk about gene edit­ing, they’re usu­al­ly think­ing about sev­er­al steps. First you need to ze­ro in on the de­fec­tive gene; then, de­pend­ing on the need, you’d want to knock out, re­place or in­sert ge­net­ic ma­te­r­i­al.

CRISPR/Cas9 tech­nolo­gies have trans­formed the field by mak­ing a break­through for the first prob­lem. In­duc­ing dou­ble-strand­ed DNA breaks, or achiev­ing sin­gle-let­ter changes as base edit­ing al­lows, have promis­ing ap­pli­ca­tions in mul­ti­ple dis­eases that are start­ing to get test­ed in hu­mans.

Jer­el Davis

But Ver­sant Ven­tures, one of the pi­o­neer­ing in­vestors in the space, still sees a gap. And they’re team­ing up with a group of promi­nent Stan­ford re­searchers — plus a sea­soned biotech ex­ec — to fill it.

“Achiev­ing high-ef­fi­cien­cy tar­get­ed gene in­te­gra­tion has been a crit­i­cal ob­jec­tive of gene edit­ing for more than 15 years, but on­ly now is this tech­no­log­i­cal­ly pos­si­ble,” Jer­el Davis, Ver­sant’s Van­cou­ver-based man­ag­ing di­rec­tor, said.

Graphite Bio has $45 mil­lion to start things off, but the mon­ey mat­ters lit­tle when com­pared to the rich brain bank it’s draw­ing from.

Matthew Por­teus, an aca­d­e­m­ic founder of CRISPR Ther­a­peu­tics, is lend­ing the tech plat­form to cre­ate this next-gen play along­side gene ther­a­py ex­pert Maria Grazia Ron­car­o­lo.

Draw­ing from re­search work led by Dan­ny De­v­er while a post­doc at Por­teus’ lab, Graphite’s big promise is to in­crease in­te­gra­tion ef­fi­cien­cy from less than 1% to greater than 50% “across di­verse ge­net­ic le­sions in a wide range of cell types.”

Daniel De­v­er

Specif­i­cal­ly, De­v­er and Por­teus showed that they could cor­rect the sick­le β glo­bin gene in pa­tient-de­rived hematopoi­et­ic stem cells ex vi­vo by com­bin­ing Cas9 ri­bonu­cle­o­pro­teins with a donor mol­e­cule that serves as a tem­plate, de­liv­ered in re­com­bi­nant ade­no-as­so­ci­at­ed vi­ral vec­tors of serotype 6 (rAAV6).

That could make for a much more scal­able re­place­ment for trans­plants, they wrote in a 2017 pa­per de­scrib­ing mouse stud­ies.

“No­tably, we de­vise an en­rich­ment par­a­digm to pu­ri­fy a pop­u­la­tion of HSPCs with >90% tar­get­ed in­te­gra­tion,” they wrote. “We al­so show ef­fi­cient cor­rec­tion of the SCD-caus­ing E6V mu­ta­tion in pa­tient-de­rived HSPCs that af­ter dif­fer­en­ti­a­tion in­to ery­thro­cytes, ex­press adult β-glo­bin (HbA) mR­NA, con­firm­ing in­tact tran­scrip­tion­al reg­u­la­tion of edit­ed HBB al­le­les.”

Josh Lehrer

Chief ex­ec­u­tive Josh Lehrer is lead­ing the charge to start Phase I for this pro­gram in ear­ly 2021, bring­ing all the sick­le cell knowl­edge and ex­pe­ri­ence from a six-year run as Glob­al Blood Ther­a­peu­tics, most re­cent­ly as CMO. More pre­clin­i­cal ther­a­pies are in the works for un­named se­ri­ous dis­eases. Sam­sara Bio­Cap­i­tal is join­ing Ver­sant for the launch round and send­ing Abe Bas­san to the board, which al­so fea­tures Davis and Car­lo Riz­zu­to from Ver­sant.

Biogen CEO Michel Vounatsos (via Getty Images)

With ad­u­canum­ab caught on a cliff, Bio­gen’s Michel Vounatsos bets bil­lions on an­oth­er high-risk neu­ro play

With its FDA pitch on the Alzheimer’s drug aducanumab hanging perilously close to disaster, Biogen is rolling the dice on a $3.1 billion deal that brings in commercial rights to one of the other spotlight neuro drugs in late-stage development — after it already failed its first Phase III.

The big biotech has turned to Sage Therapeutics for its latest deal, close to a year after the crushing failure of Sage-217, now dubbed zuranolone, in the MOUNTAIN study.

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Pascal Soriot (AP Images)

As­traZeneca, Ox­ford on the de­fen­sive as skep­tics dis­miss 70% av­er­age ef­fi­ca­cy for Covid-19 vac­cine

On the third straight Monday that the world wakes up to positive vaccine news, AstraZeneca and Oxford are declaring a new Phase III milestone in the fight against the pandemic. Not everyone is convinced they will play a big part, though.

With an average efficacy of 70%, the headline number struck analysts as less impressive than the 95% and 94.5% protection that Pfizer/BioNTech and Moderna have boasted in the past two weeks, respectively. But the British partners say they have several other bright spots going for their candidate. One of the two dosing regimens tested in Phase III showed a better profile, bringing efficacy up to 90%; the adenovirus vector-based vaccine requires minimal refrigeration, which may mean easier distribution; and AstraZeneca has pledged to sell it at a fraction of the price that the other two vaccine developers are charging.

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Bob Nelsen (Photo by Michael Kovac/Getty Images)

Bob Nelsen rais­es $800M and re­cruits a star-stud­ded board to build the 'Fox­con­n' of biotech

Bob Nelsen spent his pandemic spring in his Seattle home, talking on the phone with Luciana Borio, the scientist who used to run pandemic preparedness on the National Security Council, and fuming with her about the dire state of American manufacturing.

Companies were rushing to develop vaccines and antibodies for the new virus, but even if they succeeded, there was no immediate supply chain or infrastructure to mass-produce them in a way that could make a dent in the outbreak.

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Jason Kelly, Ginkgo Bioworks CEO (Kyle Grillot/Bloomberg via Getty Images)

Af­ter Ko­dak de­ba­cle, US lends $1.1B to a syn­thet­ic bi­ol­o­gy com­pa­ny and their big Covid-19, mR­NA plans

In mid-August, as Kodak’s $765 million government-backed push into drug manufacturing slowly fell apart in national headlines, Ginkgo Bioworks CEO Jason Kelly got a message from his company’s government liaison: HHS wanted to know if they, too, might want a loan.

The government’s decision to lend Kodak three quarters of a billion dollars raised eyebrows because Kodak had never made drugs before. But Ginkgo, while not a manufacturing company, had spent the last decade refining new ways to produce materials inside cells and building automated facilities across Boston.

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Feng Tian, Ambrx CEO (Ambrx)

Af­ter 5 qui­et years, a for­mer Scripps spin­out rais­es $200M and an­nounces plans to try again at an IPO

The first time San Diego biotech Ambrx tried to go public in 2014, they failed and the company’s board switched to a radically different strategy: They sold themselves for an undisclosed amount to a syndicate of Chinese investors and pharma companies.

Now, after 5 quiet years, that syndicate has raised a mountain of cash and indicated they’ll soon make another bid to go public.

Earlier this month, Ambrx raised $200 million in what they billed as a crossover round financed by Fidelity, BlackRock, Cormorant Asset Management, HBM Healthcare Investments, Invus, Adage Capital Partners and Suvretta Capital Management. It’s the largest amount they’ve ever raised and, according to Crunchbase figures, more than doubles the total amount of VC capital collected since their launch 17 years ago.

Michelle Longmire, Medable CEO (Jeff Rumans)

Med­able gets $91M for vir­tu­al clin­i­cal tri­als, bring­ing to­tal raise to $136M

As biotechs look to get clinical studies back on track amid the pandemic, Medable returned to the venture well for the second time this year, bagging a $91 million Series C to build out its virtual trial platform.

The software provider recently launched three new apps for decentralizing clinical trials, and saw a 500% revenue spike this year. And it isn’t alone. Back in August, Science 37 secured a $40 million round for its virtual trial tech, with support from Novartis, Sanofi Ventures and Amgen. Patients and researchers are taking a liking to the online approach, suggesting regulators could allow it to become a new normal even after the pandemic is over.

The ad­u­canum­ab co­nun­drum: The PhI­II failed a clear reg­u­la­to­ry stan­dard, but no one is cer­tain what that means any­more at the FDA

Eighteen days ago, virtually all of the outside experts on an FDA adcomm got together to mug the agency’s Billy Dunn and the Biogen team when they presented their upbeat assessment on aducanumab. But here we are, more than 2 weeks later, and the ongoing debate over that Alzheimer’s drug’s fate continues unabated.

Instead of simply ruling out any chance of an approval, the logical conclusion based on what we heard during that session, a series of questionable approvals that preceded the controversy over the agency’s recent EUA decisions has come back to haunt the FDA, where the power of precedent is leaving an opening some experts believe can still be exploited by the big biotech.

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John Maraganore, Alnylam CEO (Scott Eisen/Bloomberg via Getty Images)

Al­ny­lam gets the green light from the FDA for drug #3 — and CEO John Maraganore is ready to roll

Score another early win at the FDA for Alnylam.

The FDA put out word today that the agency has approved its third drug, lumasiran, for primary hyperoxaluria type 1, better known as PH1. The news comes just 4 days after the European Commission took the lead in offering a green light.

An ultra rare genetic condition, Alnylam CEO John Maraganore says there are only some 1,000 to 1,700 patients in the US and Europe at any particular point. The patients, mostly kids, suffer from an overproduction of oxalate in the liver that spurs the development of kidney stones, right through to end stage kidney disease.

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Vipin Suri, Catamaran Bio CSO

Cata­ma­ran Bio sails in­to the CAR-NK wa­ters with a $42M launch round

Catamaran Bio’s founding members decided to jump into the CAR-NK game last December over drinks at a trendy bar in Boston.

They were sitting around a table, discussing an MD Anderson study which provided some of the first clinical proof that natural killer (NK) cells can be reengineered to attack tumors, much like CAR-T therapies. It was a “long and lively” discussion, COO Mark Boshar recalls. And by the time it was over, they had a starting point to launch a company.