In­censed by Chi­na CRISPR scan­dal, US con­sor­tium am­pli­fy glob­al call to sus­pend hu­man em­bryo edit­ing

In­spired by his bi­ol­o­gist broth­er, Al­dous Hux­ley laid out his dystopi­an vi­sion — re­plete with ge­net­i­cal­ly mod­i­fied cit­i­zens strat­i­fied by an in­tel­li­gence-based so­cial hi­er­ar­chy — in his 1932 book Brave New World. The emer­gence of mi­to­chon­dr­i­al re­place­ment ther­a­py or ‘three-par­ent IVF’ and one rogue Chi­nese sci­en­tist lat­er, fears that un­bri­dled hu­man em­bryo edit­ing could be dan­ger­ous from a sci­en­tif­ic, eth­i­cal, and so­ci­etal per­spec­tive have reignit­ed. On Wednes­day, a con­sor­tium of sci­en­tists, bioethi­cists, and biotech ex­ec­u­tives wrote to US HHS sec­re­tary Alex Azar, call­ing for a col­lab­o­ra­tion on a bind­ing glob­al mora­to­ri­um on hu­man clin­i­cal germline ex­per­i­men­ta­tion.

Alex Azar

Glob­al­ly, guide­lines vary wide­ly about the ex­tent (or lack there­of) of germline re­search — in­tro­duc­ing her­i­ta­ble changes to sperm, eggs or em­bryos — is per­mit­ted. Some re­gions ban it al­to­geth­er; some al­low lab re­search but not preg­nan­cies (like in the UK); while oth­ers have no poli­cies. In the US, the NIH does not fund germline re­search, but pri­vate fund­ing is sanc­tioned.

Last month, an in­ter­na­tion­al group of ethi­cists and re­searchers, in­clud­ing ex­perts who orig­i­nal­ly de­vel­oped CRISPR–Cas9 as a gene-edit­ing tool, called for a mora­to­ri­um on clin­i­cal use of hu­man germline edit­ing un­til the safe­ty of the tech­nique has been es­tab­lished and a con­sen­sus on ac­cept­able use has been reached.

(W)e call for the es­tab­lish­ment of an in­ter­na­tion­al frame­work in which na­tions, while re­tain­ing the right to make their own de­ci­sions, vol­un­tar­i­ly com­mit to not ap­prove any use of clin­i­cal germline edit­ing un­less cer­tain con­di­tions are met.

The NIH sup­port­ed the call.

The move fol­lowed last year’s news that con­tro­ver­sial Shen­zhen-based re­searcher Jiankui He ge­net­i­cal­ly edit­ed em­bryos us­ing CRISPR/Cas9 tools — with in­ten­tion of in­duc­ing HIV re­sis­tance —  which were im­plant­ed and re­sult­ed in the birth of twin in­fant girls, trig­ger­ing a tem­pest in sci­en­tif­ic cir­cles the world over. In the wake of the scan­dal, the Chi­nese gov­ern­ment tight­ened its over­sight of gene edit­ing and oth­er “high-risk bio­med­ical tech­nolo­gies” in March.

“He pro­ceed­ed with­out clear med­ical need, in a sur­rep­ti­tious man­ner lack­ing any mean­ing­ful pub­lic or sci­en­tif­ic com­mu­ni­ty dis­cus­sion or con­sen­sus, and with­out any reg­u­la­to­ry ap­proval,” the group of 62 de­cried in the let­ter, con­demn­ing such hu­man germline clin­i­cal ex­per­i­men­ta­tion as ir­re­spon­si­ble.

Feng Zhang

“Such hu­man ge­net­ic ma­nip­u­la­tion should be con­sid­ered un­ac­cept­able and sup­port a bind­ing glob­al mora­to­ri­um un­til se­ri­ous sci­en­tif­ic, so­ci­etal, and eth­i­cal con­cerns are ful­ly ad­dressed,” the ex­perts, in­clud­ing CRISPR/Cas9 co-in­ven­tor Feng Zhang, said.

They un­der­scored a myr­i­ad of is­sues such as off-tar­get ef­fects and mu­ta­tions, which apart from the med­ical im­pact could pose ma­jor eth­i­cal con­cerns, be­cause re­search sub­jects would in­clude, and not on­ly be lim­it­ed, to em­bryos and chil­dren, but al­so fu­ture gen­er­a­tions of de­scen­dants.

Clin­i­cal germline gene edit­ing is ap­pro­pri­ate­ly pro­hib­it­ed in the Unit­ed States, across much of Eu­rope, in Chi­na, and in many oth­er coun­tries…Be­fore this sta­tus quo is re­vis­it­ed, it is vi­tal that ex­ten­sive dis­cus­sions and en­gage­ment take place among all ma­jor stake­hold­ers, in­clud­ing mem­bers of the sci­en­tif­ic, med­ical, pa­tient, care­giv­er, pol­i­cy, le­gal, eth­i­cal, and faith com­mu­ni­ties.

But the group was clear that the mora­to­ri­um should be lim­it­ed to germline edit­ing, not so­mat­ic (cells in the body ex­cept sperm and egg cells) cells, which have sig­nif­i­cant reper­cus­sions in the un­der­stand­ing and treat­ment of hu­man dis­ease.

Daniel O'Day [via AP Images]

UP­DAT­ED: Look­ing to re­solve lin­ger­ing doubts, Gilead un­leash­es a $5B late-stage cash al­liance with Gala­pa­gos

Daniel O’Day is executing his first major development deal since taking over as CEO of Gilead $GILD. And he’s going in deep to ally himself with a longstanding partner.

O’Day announced today that he is spending $5 billion in cash to add new late-stage drugs to Gilead’s pipeline, picking up rights to Galapagos’ $GLPG Phase III IPF drug GLPG1690 alongside adoption of the biotech’s Phase IIb drug GLPG1972 for osteoarthritis. And Gilead is also putting billions more on the table for milestones, gaining options for everything else in Galapagos’ pipeline, with a shot at all rights outside of Europe.

UP­DAT­ED: Am­gen, No­var­tis scrap Alzheimer's stud­ies -- is BACE fi­nal­ly dead or will Bio­gen and Ei­sai car­ry on?

The BACE theory of controlling Alzheimer’s died with failed pivotal projects at Merck, Eli Lilly and their partners at AstraZeneca. Now Amgen and Novartis have come along to bulldoze it under a mound of safety threats — leaving only Biogen and Eisai to carry on with a less than zero chance of success — with the notable addition that they may actually be doing harm to patients.

After the market closed Thursday, Amgen and Novartis announced that they were dumping two pivotal programs underway with the Banner Alzheimer’s Institute on their BACE drug CNP520 (umibecestat) after an independent review of the data indicated that patients’ cognitive abilities were actually worsening at a faster pace than the placebo arm.

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Christi Shaw at JP Morgan 2019. Jeff Rumans for Endpoints News

Fresh out of Eli Lil­ly, Christi Shaw sur­faces as Daniel O'­Day's new CEO at CAR-T pi­o­neer Kite

Well, that didn’t take long. 

We found out Thursday evening that Christi Shaw has given up her top post as the head of the Bio-Medicines group at Eli Lilly for the helm at CAR-T pioneer Kite. New Gilead CEO Daniel O’Day, a Roche veteran, had made finding a Kite CEO a top priority on his arrival at Gilead. And he went right for a headliner.

O’Day was clearly excited about the coup.

“We conducted an extensive search for a new leader at Kite and we believe that Christi’s unique set of skills will allow us to continue to build on our leadership position in cell therapy,” he said in a prepared statement. “Christi’s vast experience across complex therapeutic areas, and particularly in oncology, will serve Kite very well. She is clearly a leader who will bring teams and individuals together and I am confident she will build upon the entrepreneurial spirit at Kite as we seek to help more people with cancer around the world.”

Christi Shaw at JP Morgan 2019. Jeff Rumans for Endpoints News

Eli Lil­ly's Christi Shaw bows out of top post at the Bio-Med­i­cines unit

Less than 3 years after Eli Lilly CEO David Ricks recruited Novartis vet Christi Shaw to run their big Bio-Medicines business, she’s out.

In a statement put out Thursday morning, Lilly said that Shaw’s last day will come at the end of August. Patrik Jonsson, currently president and general manager of Lilly Japan, will succeed Shaw once he gets the paperwork sorted out.

Lilly’s shares dropped 4% on the news.

Jeff Poulton

Al­ny­lam’s Maraganore switch­es ‘per­haps the best CFO in mid-cap biotech’ with Shire vet Jeff Poul­ton

There’s a new CFO taking charge of the numbers at RNAi pioneer Alnylam.

Alnylam chief John Maraganore says that CFO Manmeet S. Soni is leaving in the proverbial pursuit of new opportunities. And he’s being replaced by ex-Shire CFO Jeff Poulton, not long after the Takeda takeover obliterated that position.

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Analy­sis: In most of the Big Phar­ma world, R&D spend­ing tow­ers over rev­enue from new drugs. Guess who beat the odds

It’s always been the case that there’s no medicine less useful than one that doesn’t make it to patients (unless you regard the task of R&D to be perpetually learning about swimming, while under water…). Yet, launching new medicines that physicians want, that payers will cover, and that patients will take, is a discipline that is unevenly distributed among the big players.

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President Donald Trump at State of the Union. AP Images

White House changes course to kill re­bate re­form

So what exactly is the White House’s plan to tackle rising drug prices? It doesn’t look like we will get definitive answers anytime soon. On the heels of President Donald Trump’s surprise revelation that an executive order is in the works to implement a “favored nations clause,” his administration is now putting out word that it has abandoned an earlier proposal to overhaul rebates.

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The FDA's Janet Wood­cock talks about some big changes she's push­ing for in drug de­vel­op­ment, and agency re­views

Janet Woodcock is perhaps the most influential regulator at the FDA. And when the head of CDER talks about the changes being made at the agency when it comes to clinical trial designs, or the need to reorganize for a specific disease arena, an assessment of the expansion of gene therapy or I/O, common development mistakes, and so on, you can be sure the industry pays attention to every word.

So it was with some eagerness that I opened up Geoffrey Porges’ summary of their recent conversation about the FDA. And I wasn’t disappointed. In a wide-ranging exchange with the SVB Leerink analyst, Woodcock discussed the growing importance of patient-reported outcomes in clinical trials, a campaign underway now to see if CROs would help spur more basket studies to compare drugs head-to-head, and much, much more.

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Ex-DARPA di­rec­tor pur­sues all-in-one can­cer pill as NED CEO; Karyopharm los­es com­mer­cial chief ahead of drug roll­out

“Why not try?”

That’s what Geoffrey Ling told me over the phone when asked about what led him to his journey to the position of CEO at NED Biosciences — a company with a lofty goal of creating an all-in-one oral drug to treat all types of cancer and making this drug available to not only developed nations, but also the developing world. 

Ling comes from an extensive background in medicine and the government. He is the co-leader of The Brain Health Project, a professor of neurology and an attending neurocritical care physician at John Hopkins University and Hospital, as well as the assistant director for Medical Innovation of the Science Division in president Obama’s White House Office of Science and Technology Policy (OSTP).