Reck­on­ing the po­ten­tial of CRISPR/Cas9 tech, NIH launch­es $190M genome edit­ing re­search ini­tia­tive

Fran­cis Collins, the di­rec­tor of the Na­tion­al In­sti­tutes of Health CQ/Roll Call Get­ty

As the first hu­man tri­al in­volv­ing CRISPR comes in­to view in the Unit­ed States, the NIH has stepped up with a $190 mil­lion re­search ini­tia­tive to help float all the boats rid­ing on the wave of new genome edit­ing tech­nolo­gies.

Named So­mat­ic Cell Genome Edit­ing, the pro­gram will award funds over six years to re­searchers col­lab­o­rat­ing to ex­pand the toolk­it for the whole sci­en­tif­ic com­mu­ni­ty.  im­prov­ing the de­liv­ery mech­a­nisms, de­vel­op­ing bet­ter ed­i­tors, de­sign­ing as­says to eval­u­ate those edit­ing tools, and as­sem­bling a pack­age of knowl­edge and meth­ods. Ul­ti­mate­ly, the hope is to re­move bar­ri­ers to broad adop­tion of the tech in the clin­ic.

“Genome edit­ing tech­nolo­gies such as CRISPR/Cas9 are rev­o­lu­tion­iz­ing bio­med­ical re­search,” said di­rec­tor Fran­cis Collins in a state­ment. “The fo­cus of the So­mat­ic Cell Genome Edit­ing pro­gram is to dra­mat­i­cal­ly ac­cel­er­ate the trans­la­tion of these tech­nolo­gies to the clin­ic for treat­ment of as many ge­net­ic dis­eases as pos­si­ble.”

As its name sug­gests, the pro­gram fo­cus­es on so­mat­ic cells — non-re­pro­duc­tive cells that do not pass DNA down to the next gen­er­a­tion. In oth­er words, any changes to the DNA in­tro­duced by these kinds of ther­a­peu­tics would not be in­her­it­ed. That avoids the stick­i­est eth­i­cal con­cerns tied to the use of US tax­pay­er funds.

This new in­vest­ment by the NIH comes at a piv­otal mo­ment for gene edit­ing.

The pi­o­neers have had their patent fight, and star-stud­ded new play­ers such as Ex­on­ics and Tmu­ni­ty are start­ing to come on the scene. With Chi­na rac­ing ahead in the clin­ic — 86 pa­tients have had their genes edit­ed, per the Wall Street Jour­nal — de­vel­op­ing the in­fra­struc­ture need­ed to ad­vance next-gen tech ben­e­fits all play­ers.

Mary Ellen Per­ry

In fact, the spe­cif­ic di­rec­tions of the pro­gram are the prod­uct of a brain­storm­ing work­shop with in­dus­try ex­perts, pa­tient ad­vo­cates and aca­d­e­m­ic re­searchers. That’s ac­cord­ing to Mary Ellen Per­ry, who first pre­sent­ed the con­cept to a group of NIH in­sid­ers in Sep­tem­ber. What comes out of this pro­gram, she said, could im­pact every­thing from com­mon dis­or­ders to rare dis­eases.

“It’s al­most dif­fi­cult to think of a con­di­tion that this might not be ap­plic­a­ble to,” Per­ry said at the meet­ing.

ZS Per­spec­tive: 3 Pre­dic­tions on the Fu­ture of Cell & Gene Ther­a­pies

The field of cell and gene therapies (C&GTs) has seen a renaissance, with first generation commercial therapies such as Kymriah, Yescarta, and Luxturna laying the groundwork for an incoming wave of potentially transformative C&GTs that aim to address diverse disease areas. With this renaissance comes several potential opportunities, of which we discuss three predictions below.

Allogenic Natural Killer (NK) Cells have the potential to displace current Cell Therapies in oncology if proven durable.

Despite being early in development, Allogenic NKs are proving to be an attractive new treatment paradigm in oncology. The question of durability of response with allogenic therapies is still an unknown. Fate Therapeutics’ recent phase 1 data for FT516 showed relatively quicker relapses vs already approved autologous CAR-Ts. However, other manufacturers, like Allogene for their allogenic CAR-T therapy ALLO-501A, are exploring novel lymphodepletion approaches to improve persistence of allogenic cells. Nevertheless, allogenic NKs demonstrate a strong value proposition relative to their T cell counterparts due to comparable response rates (so far) combined with the added advantage of a significantly safer AE profile. Specifically, little to no risk of graft versus host disease (GvHD), cytotoxic release syndrome (CRS), and neurotoxicity (NT) have been seen so far with allogenic NK cells (Fig. 1). In addition, being able to harness an allogenic cell source gives way to operational advantages as “off-the-shelf” products provide improved turnaround time (TAT), scalability, and potentially reduced cost. NKs are currently in development for a variety of overlapping hematological indications with chimeric antigen receptor T cells (CAR-Ts) today, and the question remains to what extent they will disrupt the current cell therapy landscape. Click for more details.

Graphic: Kathy Wong for Endpoints News

What kind of biotech start­up wins a $3B syn­di­cate, woos a gallery of mar­quee sci­en­tists and re­cruits GSK's Hal Bar­ron as CEO in a stun­ner? Let Rick Klaus­ner ex­plain

It started with a question about a lifetime’s dream on a walk with tech investor Yuri Milner.

At the beginning of the great pandemic, former NCI chief and inveterate biotech entrepreneur Rick Klausner and the Facebook billionaire would traipse Los Altos Hills in Silicon Valley Saturday mornings and talk about ideas.

Milner’s question on one of those mornings on foot: “What do you want to do?”

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Hal Barron, Endpoints UKBIO20 (Jeff Rumans)

'Al­tos was re­al­ly a once-in-a-life­time op­por­tu­ni­ty': Hal Bar­ron re­flects on his big move

By all accounts, Hal Barron had one of the best jobs in Big Pharma R&D. He made more than $11 million in 2020, once again reaping more than his boss, Emma Walmsley, who always championed him at every opportunity. And he oversaw a global R&D effort that struck a variety of big-dollar deals for oncology, neurodegeneration and more.

Sure, the critics never let up about what they saw as a rather uninspiring late-stage pipeline, where the rubber hits the road in the Big Pharma world’s hunt for the next big near-term blockbuster, but the in-house reviews were stellar. And Barron was firmly focused on bringing up the success rate in clinical trials, holding out for the big rewards of moving the dial from an average 10% success rate to 20%.

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Executive Director of the EMA Emer Cooke (AP Photo/Geert Vanden Wijngaert)

Eu­ro­pean Par­lia­ment signs off on strength­en­ing drug reg­u­la­tor's abil­i­ty to tack­le short­ages

The European Parliament on Thursday endorsed a plan to increase the powers of the European Medicines Agency, which will be better equipped to monitor and mitigate shortages of drugs and medical devices.

By a vote of 655 to 31, parliament signed off on a provisional agreement reached with the European Council from last October, in which the EMA will create two shortage steering groups (one for drugs, the other for devices), a new European Shortages Monitoring Platform to facilitate data collection and increase transparency, and on funding for the work of the steering groups, task force, working parties and expert panels that are to be established.

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FDA+ roundup: FDA's neu­ro­science deputy de­parts amid on­go­ing Aduhelm in­ves­ti­ga­tions; Califf on the ropes?

Amid increased scrutiny into the close ties between FDA and Biogen prior to the controversial accelerated approval of Aduhelm, the deputy director of the FDA’s office of neuroscience has called it quits after more than two decades at the agency.

Eric Bastings will now take over as VP of development strategy at Ionis Pharmaceuticals, the company said Wednesday, where he will provide senior clinical and regulatory leadership in support of Ionis’ pipeline.

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Sec­ondary patents prove to be key in biosim­i­lar block­ing strate­gies, re­searchers find

While the US biosimilars industry has generally been a disappointment since its inception, with FDA approving 33 biosimilars since 2015, just a fraction of those have immediately followed their approvals with launches. And more than a handful of biosimilars for two of the biggest blockbusters of all time — AbbVie’s Humira and Amgen’s Enbrel — remain approved by FDA but still have not launched because of legal settlements.

Hal Barron (GSK via YouTube)

GSK R&D chief Hal Bar­ron jumps ship to run a $3B biotech start­up, Tony Wood tapped to re­place him

In a stunning switch, GlaxoSmithKline put out word early Wednesday that R&D chief Hal Barron is exiting the company after 4 years — a relatively brief run for the man chosen by CEO Emma Walmsley in late 2017 to turn around the slow-footed pharma giant.

Barron is being replaced by Tony Wood, a close associate of Barron’s who’s taking one of the top jobs in Big Pharma R&D. He’ll be closer to home, though, for GSK. Barron has been running a UK and Philadelphia-based research organization from his perch in San Francisco.

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Kenneth Galbraith, incoming Zymeworks CEO

Zymeworks re­places half its C-suite, aims to lay off 25% of to­tal work­force as new CEO takes over

New Zymeworks CEO Kenneth Galbraith is aiming to hit the ground running when his tenure officially begins next month, but he’ll be doing so with a much different looking team.

In a lengthy press release outlining the biotech’s 2022 goals, Galbraith said Zymeworks will be laying off at least 25% of its staff over the course of the year. Half of its C-suite will also be replaced immediately as Galbraith looks to remake the company in his image after Ali Tehrani, Zymeworks’ founder and CEO since 2003, stepped down two weeks ago.

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CBO: Medicare ne­go­ti­a­tions will ham­per drug de­vel­op­ment more than pre­vi­ous­ly thought

As President Biden’s Build Back Better Act — and, with it, potentially the Democrats’ last shot at major drug pricing reforms in the foreseeable future — remains on life support, the Congressional Budget Office isn’t helping their case.

The CBO last week released a new slide deck, outlining an update to its model on how Medicare negotiations might take a bite out of new drugs making it to market. The new model estimates a 10% long-term reduction in the number of new drugs, whereas a previous CBO report from August estimated that 8% fewer new drugs will enter the market over 30 years.