Ed­i­tas and Cel­gene sub Juno are tack­ling hottest im­munother­a­py cells

As the first CRISPR-edit­ed can­cer pa­tients watch their treat­ments un­fold, one of the first CRISPR com­pa­nies is re­jig­ging a ma­jor on­col­o­gy deal.

Ed­i­tas Med­i­cine is amend­ing its long-run­ning col­lab­o­ra­tion with Cel­gene and their sub­sidiary Juno Ther­a­peu­tics. The new deal will ex­pand the fo­cus of their work to cov­er a sub­set of im­mune cells that have be­come an in­creas­ing­ly hot tar­get for im­munother­a­py: gam­ma-delta cells.

The deal will make Ed­i­tas el­i­gi­ble for a $70 mil­lion pay­ment along with oth­er pos­si­ble mile­stones and roy­al­ties.

“It’s a sig­nif­i­cant ex­pan­sion” of the deal, Ed­i­tas CSO Char­lie Al­bright told End­points News. These cells are part of the im­mune sys­tem and have sig­nif­i­cant po­ten­tial to treat sol­id tu­mors.

Char­lie Al­bright

Since it be­gan in 2015, the Juno-Ed­i­tas col­lab­o­ra­tion has fo­cused large­ly on al­pha-be­ta cells, the ones out­fit­ted with the spe­cial re­cep­tors in cur­rent CAR-T treat­ments. Sci­en­tists at those com­pa­nies and else­where have most pub­licly tried to ap­ply CRISPR to im­prove CAR-T, which now work sole­ly through vi­ral gene trans­fer.

But they have al­so worked on ex­pand­ing the ap­proach to oth­er im­mune cell types in hopes of mak­ing the treat­ment more ef­fec­tive, more ac­ces­si­ble or — as is the case with some of the gam­ma delta re­search — ex­pand it in­to oth­er can­cer types, es­pe­cial­ly sol­id tu­mors.

Ed­i­tas has been slow­ly build­ing their gam­ma-delta base through­out the year, Al­bright said. In April, they signed an agree­ment with Blue­Rock, in part to ac­cess pluripo­tent stem cells they hope to make in­to en­gi­neered gam­ma-delta cells that can be de­liv­ered to a pa­tient. (Es­sen­tial­ly a form of off-the-shelf CAR–T).

Sev­er­al com­pa­nies are now pur­su­ing gam­ma-delta im­munother­a­pies, in­clud­ing Gam­maDelta Ther­a­peu­tics and its new spin­off Adap­tate and Re­gen­eron-backed Adicet Bio. They’re bet­ting chiefly on these cells’ abil­i­ty to pen­e­trate the sol­id tu­mors that have been so re­sis­tant to the first wave of CAR-T treat­ments.

Al­bright ar­gued, though, that for these tech­niques to work you need gene edit­ing. In­nate abil­i­ties in the cells have to be tuned up, he said. You have to in­crease cells’ per­sis­tence and en­hance their abil­i­ty to sur­vive in a tu­mors’ mi­cro-en­vi­ron­ment. Ide­al­ly, he said, you even give it new abil­i­ties, such as the pow­er to cat­alyze the body’s in­nate im­mune sys­tem.

“You can’t do that with vi­ral trans­duc­tion,” Al­bright said. “You need gene edit­ing.”

Tal Zaks, Moderna CMO (Moderna via YouTube)

UP­DAT­ED: NI­AID and Mod­er­na spell out a 'ro­bust' im­mune re­sponse in PhI coro­n­avirus vac­cine test — but big ques­tions re­main to be an­swered

The NIAID and Moderna have spelled out positive Phase I safety and efficacy data for their Covid-19 vaccine mRNA-1273 — highlighting the first full, clear sketch of evidence that back-to-back jabs at the dose selected for Phase III routinely produced a swarm of antibodies to the virus that exceeded levels seen in convalescent patients — typically in multiples indicating a protective response.

Moderna execs say plainly that this first stage of research produced exactly the kind of efficacy they hoped to see in humans, with a manageable safety profile.

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Trans­port Sim­u­la­tion Test­ing for Your Ther­a­py is the Best Way to As­sure FDA Ex­pe­dit­ed Pro­gram Ap­proval

Modality Solutions is an ISO:9001-registered biopharmaceutical cold chain engineering firm with unique transport simulation capabilities that support accelerated regulatory approval for biologics and advanced therapeutic medicinal products (ATMP). Our expertise combines traditional validation engineering approaches with regulatory knowledge into a methodology tailored for the life sciences industry. We provide insight and execution for the challenges faced in your cold chain logistics network.

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Who are the women blaz­ing trails in bio­phar­ma R&D and lead­ing the fight against Covid-19? Nom­i­nate them for End­points' spe­cial re­port

One of the many inequalities the pandemic has laid bare is the gender imbalance in biomedical research. A paper examining Covid-19 research authorship wondered out loud: Where are the women?

It’s a question that echoes beyond our current times. In the biopharma world, not only are women under-represented in R&D roles (particularly at higher levels), their achievements and talents could also be undermined by stereotypes and norms of leadership styles. The problem is even more dire for women of color.

Sanjiv Patel, Relay CEO

Re­lay reaps $400M IPO wind­fall af­ter draw­ing the cur­tain on mo­tion-based drug de­sign pipeline

A red-hot venture market back in 2018 propelled Relay Therapeutics to a whopping $400 million crossover round. And now, the biotech is riding on a red-hot IPO market to a public debut that brought in the same exact amount.

The $400 million raise breaks down to 20 million shares sold at $20 each — above even the amended proposed range of $16 to $18.

It’s also double what Relay had penciled in with the initial S-1 filing, in yet another illustration that those figures are often nothing more than a placeholder these days.

BioAt­la rakes in $72.5M Se­ries D, ad­vanc­ing re­search for pH-de­tect­ing can­cer treat­ments

A little over a year after agreeing to a worldwide collaboration with hefty Chinese biotech BeiGene worth up to $270 million, BioAtla is ready for another haul.

The San Diego-based biotech pulled in a $72.5 million haul for their Series D financing, which the company announced Wednesday. Funds will go toward their four main clinical programs developing cancer treatments, two of which are currently in Phase II, BioAtla president and former Celgene COO Scott Smith said.

Mene Pangalos, AstraZeneca R&D chief (AstraZeneca via YouTube)

A day af­ter Mod­er­na vac­cine re­sults, ru­mors swirl of pend­ing As­traZeneca da­ta

A day after Moderna and the NIH published much-anticipated data from their Phase I Covid-19 vaccine trial, attention is turning to AstraZeneca which, according to a UK report, is expected to publish its own early data tomorrow.

ITV’s Robert Peston reported that AstraZeneca will publish the Phase I data in The Lancet. 

AstraZeneca and Moderna represent the two most ambitious Covid-19 vaccine efforts, having set the quickest timelines for approval (though they were recently joined in that regard by the Pfizer-BioNTech partnership) and some of the loftiest goals in total doses. Yet there is even less known about AstraZeneca’s vaccine’s effect on humans than there was about Moderna’s before yesterday. Although, in a controversial move, Moderna released some statistics from its Phase I in May, AstraZeneca has yet to say anything about what it saw in its Phase I trial — a move consistent with the scientific convention to withhold data until it can be published in a peer-reviewed journal.

Ludwig Hantson, Alexion CEO

Why pay $4B for a steady di­et of dis­ap­point­ment? Porges turns thumbs down on Alex­ion’s M&A strat­e­gy, of­fers some point­ers

When Alexion announced recently that it was paying $1.4 billion to bag Portola and its underperforming Factor Xa inhibitor reversal agent, you could hear the head-scratching going on around virtual Wall Street.

Why was Alexion going down the discount lane for new products? And why something like this? Analysts have been urging Alexion to get serious about M&A for years if it was serious about diversifying the company beyond Soliris and its successor drug. But this wasn’t the kind of heavy-impact deal they were looking for.

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Mil­li­pore­Sig­ma to build new $20 mil­lion, 12,000-square-foot lab in Switzer­land

On the heels of opening a new laboratory in Shanghai last week, MilliporeSigma is continuing its construction push.

The Merck KGgA life science subsidiary announced Wednesday its intentions to build a new $20 million lab in Buchs, Switzerland to support its reference materials business. It’s estimated that the new facility will be completed in December 2021 and open in early 2022 and is expected to be 12,000 square feet.

Stéphane Bancel, Moderna CEO (Steven Ferdman/Getty Images)

‘Plan­ning to vac­ci­nate every­one in the US,’ Mod­er­na out­lines ef­forts to sup­ply their Covid-19 vac­cine as man­u­fac­tur­ing ramps up ahead of PhI­II

Twelve days from the planned start of their Phase III pivotal trial, the executive crew at Moderna has set up the manufacturing base needed to begin production of the first 500,000 doses of their Covid-19 vaccine with plans to feed it into a global supply chain. But the initial batches will likely be ready in the US first, where company CEO Stéphane Bancel plans to be able to vaccinate everyone.

“We have started making commercial product at-risk, and will continue to do so every day and every week of the month,” Bancel told analysts during their morning call on the Phase I data just published in the New England Journal of Medicine.

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