For sale: Boston Chil­dren’s team touts a new CRISPR de­liv­ery sys­tem for triple-neg­a­tive breast can­cer — plus — af­ter test­ing it in mice and cells

A team of in­ves­ti­ga­tors at Boston Chil­dren’s has de­vel­oped a new de­liv­ery ve­hi­cle for CRISPR that amps up its abil­i­ty to elim­i­nate a key tar­get in triple-neg­a­tive breast can­cer — which they’ve demon­strat­ed in mice and tu­mor cells. And they have it up for auc­tion now in the hot on­col­o­gy field.

En­gi­neer­ing their way around the car­go lim­i­ta­tions and cell pen­e­tra­tion chal­lenges of cur­rent­ly used CRISPR de­liv­ery tech, the re­searchers used a new soft par­ti­cle de­liv­ery plat­form that they em­ployed to hone in on tu­mor cells us­ing an­ti­bod­ies to guide them to ICAM-1 mol­e­cules.

Once at the site, the sci­en­tists say they knocked out the onco­gene Lipocalin 2 — which dri­ves metas­ta­sis in pa­tients — with an 81% rate of ef­fi­cien­cy in tu­mor cells. And they al­so scored high on mice.

“Us­ing a soft par­ti­cle al­lows us to pen­e­trate the tu­mor bet­ter, with­out side ef­fects, and with big­ger car­go,” says Peng Guo, the study’s first au­thor. “Our sys­tem can sub­stan­tial­ly in­crease tu­mor de­liv­ery of CRISPR.”

Mar­sha Moses Boston Chil­dren’s Hos­pi­tal

“Our sys­tem can de­liv­er sig­nif­i­cant­ly more drug to the tu­mor, in a pre­cise and safe way,” adds Mar­sha Moses who co-led the project. And bur­nish­ing the val­ue, the sci­en­tists say it can be par­tic­u­lar­ly use­ful in pe­di­atric cas­es and de­liv­er­ing oth­er drugs.

Vi­ral sys­tems now em­ployed can threat­en off-tar­get im­pact, which rais­es safe­ty is­sues. And they can’t go big on the car­go. So sci­en­tists have been hard at it look­ing for new de­liv­ery ve­hi­cles that can do it bet­ter.

Their work mar­ries two of the hottest fields in R&D: gene edit­ing and on­col­o­gy. And in case you missed the sub­tle sales mes­sage, their state­ment blunt­ly notes that they are al­ready in li­cens­ing talks “with a num­ber of com­pa­nies in­ter­est­ed in the tech­nol­o­gy.”

So­cial im­age: Shut­ter­stock

As­traZeneca trum­pets the 'mo­men­tous' da­ta they found for Tagris­so in an ad­ju­vant set­ting for NSCLC — but many of the ex­perts aren’t cheer­ing along

AstraZeneca is rolling out the big guns this evening to provide a salute to their ADAURA data on Tagrisso at ASCO.

Cancer R&D chief José Baselga calls the disease-free survival data for their drug in an adjuvant setting of early stage, epidermal growth factor receptor-mutated NSCLC patients following surgery “momentous.” Roy Herbst, the principal investigator out of Yale, calls it “transformative.”

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Paul Hudson, Sanofi CEO (Getty Images)

Sanofi CEO Paul Hud­son has $23B burn­ing a hole in his pock­et. And here are some hints on how he plans to spend that

Sanofi has reaped $11.1 billion after selling off a big chunk of its Regeneron stock at $515 a share. And now everyone on the M&A side of the business is focused on how CEO Paul Hudson plans to spend it.

After getting stung in France for some awkward politicking — suggesting the US was in the front of the line for Sanofi’s vaccines given American financial support for their work, versus little help from European powers — Hudson now has the much more popular task of managing a major cash cache to pull off something in the order of a big bolt-on. Or two.

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The Avance Clinical leadership team: CEO Yvonne Lungershausen, Sandrien Louwaars - Director Business Development Operations, Gabriel Kremmidiotis - Chief Scientific Officer, Ben Edwards - Chief Strategy Officer

How Aus­tralia De­liv­ers Rapid Start-up and 43.5% Re­bate for Ear­ly Phase On­col­o­gy Tri­als

About Avance Clinical

Avance Clinical is an Australian owned Contract Research Organisation that has been providing high-quality clinical research services to the local and international drug development industry for 20 years. They specialise in working with biotech companies to execute Phase 1 and Phase 2 clinical trials to deliver high-quality outcomes fit for global regulatory standards.

As oncology sponsors look internationally to speed-up trials after unprecedented COVID-19 suspensions and delays, Australia, which has led the world in minimizing the pandemic’s impact, stands out as an attractive destination for early phase trials. This in combination with the streamlined regulatory system and the financial benefits including a very favourable exchange rate and the R & D cash rebate makes Australia the perfect location for accelerating biotech clinical programs.

Ear­ly sur­vival da­ta boost Zio­phar­m's 'con­trolled IL-12' im­munother­a­py for glioblas­toma

An unconventional pairing of a gene therapy and an oral drug that promises to attack recurrent or progressive glioblastoma with controlled release of IL-12 has turned up more promising — if early — overall survival data. On top of boosting its case as a monotherapy, the data can also bode well for a combination with Regeneron’s PD-1 inhibitor, Libtayo.

Both the treatment and its developer, Ziopharm Oncology, have come a long way. The stock price peaked in 2015 but cratered in 2016 following a patient death in a Phase I.

As tislelizum­ab gains trac­tion in Chi­na, BeiGene pulls the cur­tain on NSCLC da­ta sup­port­ing the PD-1 drug

In a world now brimming with checkpoint inhibitors, companies often struggle to make a mark given a raft of therapies have already captured a considerable portion of the vast oncology market.

BeiGene’s tislelizumab was the fourth PD(L)-1 inhibitor to secure approval in China — and as it works on expanding its share the company has put out detailed data on the use of the drug in certain patients with lung cancer.

David Chang, Allogene CEO (Jeff Rumans)

Head­ed to PhII: Al­lo­gene CEO David Chang com­pletes a pos­i­tive ear­ly snap­shot of their off-the-shelf CAR-T pi­o­neer

Allogene CEO David Chang has completed the upbeat first portrait of the biotech’s off-the-shelf CAR-T contender ALLO-501 at virtual ASCO today, keeping all eyes on a drug that will now try to go on to replace the first-wave personalized pioneers he helped create.

The overall response rate outlined in Allogene’s abstract for treatment-resistant patients with non-Hodgkin lymphoma slipped a little from the leadup, but if you narrow the patient profile to treatment-naïve patients — removing the 3 who had previous CAR-T therapy who didn’t respond, leaving 16 — the ORR lands at 75% with a 44% complete response rate. And 9 of the 12 responders remained in response at the data cutoff, offering a glimpse on durability that still has a long way to go before it can be completely nailed down.

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Pablo Legorreta, founder and CEO of Royalty Pharma AG, speaks at the annual Milken Institute Global Conference in Beverly Hills, California (Patrick T. Fallon/Bloomberg via Getty Images)

Cap­i­tal­iz­ing Pablo: The world’s biggest drug roy­al­ty buy­er is go­ing pub­lic. And the low-key CEO di­vulges a few se­crets along the way

Pablo Legorreta is one of the most influential players in biopharma you likely never heard of.

Over the last 24 years, Legorreta’s Royalty Pharma group has become, by its own reckoning, the biggest buyer of drug royalties in the world. The CEO and founder has bought up a stake in a lengthy list of the world’s biggest drug franchises, spending $18 billion in the process — $2.2 billion last year alone. And he’s become one of the best-paid execs in the industry, reaping $28 million from the cash flow last year while reserving 20% of the cash flow, less expenses, for himself.

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Ku­ra flash­es pos­i­tive HRAS da­ta on once-failed J&J drug

Troy Wilson was working with J&J on their KRAS inhibitor and periodically thumbing through their publications when he spotted an old drug called tipifarnib that looked promising. So promising, in fact, that the large pharma had run it through over 5,000 patients across 70 trials, hoping they would at some point be able to nail down who were the small slice of patients who responded in some studies.

Af­ter star­ring at ASH last fall, Gilead’s new Forty Sev­en crew col­ors in more promis­ing da­ta for ma­grolimab at AS­CO

We now know the full, early-stage story behind the drug that inspired Gilead CEO Dan O’Day’s recent $5 billion acquisition of Forty Seven.

Following up on their ASCO abstract from a couple of weeks ago, the team at Forty Seven is making their return appearance this week holding clearly promising early-stage data on their lead drug magrolimab as they ponder whether they should roll on a quest to obtain an accelerated approval.