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.

A new era of treat­ment: How bio­mark­ers are chang­ing the way we think about can­cer

AJ Patel was recovering from a complicated brain surgery when his oncologist burst into the hospital room yelling, “I’ve got some really great news for you!”

For two years, Patel had been going from doctor to doctor trying to diagnose his wheezing, only to be dealt the devastating news that he had stage IV lung cancer and only six months to live. And then they found the brain tumors.

“What are you talking about?” Patel asked. He had never seen an oncologist so happy.

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David Ricks, Eli Lilly CEO (David Paul Morris/Bloomberg via Getty Images)

Eli Lil­ly set to in­vest $2.1B in home state man­u­fac­tur­ing boost

Eli Lilly is looking to expand its footprint in its home Hoosier State by making a major investment in manufacturing.

The pharma is investing $2.1 billion in two new manufacturing sites at Indiana’s LEAP Lebanon Innovation and Research District in Boone County, northwest of Lilly’s headquarters in Indianapolis.

The two new facilities will expand Lilly’s manufacturing network for active ingredients and new therapeutic modalities, including genetic medicines, according to a press release.

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US sees spike in Paxlovid us­age as Mer­ck­'s mol­nupi­ravir and As­traZeneca's Evusheld are slow­er off the shelf

New data from HHS show that more than 162,000 courses of Pfizer’s Covid-19 antiviral Paxlovid were administered across the US over the past week, continuing a streak of increased usage of the pill, and signaling not only rising case numbers but more awareness of how to access it.

In comparison to this week, about 670,000 courses of the Pfizer pill have been administered across the first five months since Paxlovid has been on the US market, averaging about 33,000 courses administered per week in that time.

Pfiz­er and CD­MOs ramp up Paxlovid man­u­fac­tur­ing with Kala­ma­zoo plant ex­pan­sion lead­ing the way

As the Covid-19 pandemic continues to evolve, pharma companies and manufacturers are exploring how to step up production on antivirals.

Pfizer is planning to expand its Kalamazoo-area facility to increase manufacturing capabilities for the oral Covid-19 antiviral Paxlovid, according to a report from Michigan-based news site MLive. The expansion of the facility, which serves as Pfizer’s largest manufacturing location, is expected to create hundreds of “high-skilled” STEM jobs, MLive reported. No details about the project’s cost and timeline have been released, but according to MLive, Pfizer will announce the details of the expansion at some point in early June.

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FDA spells out the rules and re­stric­tions for states seek­ing to im­port drugs from Cana­da

The FDA is offering more of an explanation of the guardrails around its program that may soon allow states to import prescription drugs in some select circumstances from Canada, but only if such imports will result in significant cost reductions for consumers.

While the agency has yet to sign off on any of the 5 state plans in the works so far, and PhRMA’s suit to block the Trump-era rule allowing such imports is stalled, the new Q&A guidance spells out the various restrictions that states will have to abide by, potentially signaling that a state approval is coming.

Peter Thompson, Terremoto Biosciences interim CEO

For­mer Prin­cip­ia team looks to shake up co­va­lent small mol­e­cules again, this time at 'earthquake' scale

Terremoto Biosciences goes back a long ways, in a sense, to about a dozen years ago when Principia Biopharma was founded by UCSF professor Jack Taunton. Peter Thompson initially helmed the biotech.

The company helped expand covalent small molecule inhibitors beyond oncology and into autoimmune disease by targeting cystine. But that amino acid is uncommon in a lot of proteins, offering fewer drug targets than, say, lysine, which is present in most proteins of interest. So, over the years, Taunton went back to the drawing board to check out that second amino acid.

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Simba Gill, CEO of Evelo Biosciences

While down 87% YOY, Evelo gets Flag­ship and oth­ers to in­fuse new cap­i­tal for come­back hope

Just four years after Flagship spinout Evelo Biosciences went public in an IPO worth $85 million, the biotech has seen its share price tank from $13 a share this time last year (ultimately reaching a peak of over $17) to now under $1.50. And today, it looks like Flagship still thinks the fledging biotech, in a down market, is still worth something after initial pre-IPO backing from the likes of Google’s GV, Celgene, Mayo Clinic and Alexandria Venture.

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Pfizer CEO Albert Bourla at the World Economic Forum (Gian Ehrenzeller/Keystone via AP Images)

All about ac­cess: Pfiz­er moves to a non-prof­it mod­el for drug sales in 45 low­er-in­come coun­tries

Leading the way to increase access to cheaper drugs worldwide, Pfizer said Wednesday it will provide all current and future patent-protected medicines and vaccines available in the US or EU on a not-for-profit basis to about 1.2 billion people in 45 lower-income countries.

Rwanda, Ghana, Malawi, Senegal and Uganda are the first five countries to sign on to this accord, which will also seek to blaze new paths for quick and efficient regulatory and procurement processes to reduce the usual delays in making new medicines and vaccines available in these countries.

Almirall is tapping artificial intelligence on behalf of its sales force for insights and efficiencies. (via Shutterstock)

Almi­rall rolls out sales rep ar­ti­fi­cial in­tel­li­gence sys­tem, cut­ting pre-call prep and 'wind­shield time'

Dermatology specialty pharma Almirall is making its sales reps smarter. Not with extra training or educational courses, but instead with artificial intelligence tools.

It began a soft launch of a sales rep AI and machine learning platform it calls Polaris last August in one of its 7 US coverage regions. The platform from Aktana gathers information from across Almirall internal sources and external ones – such as claims and prescribing data – to generate insights for reps. Now, instead of spending hours prepping for a sales call, Polaris can generate details about a physician’s preferences, past behaviors and prescription habits for reps in minutes, said Almirall head of commercial operations Vincent Cerio.

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