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.

Da­ta Lit­er­a­cy: The Foun­da­tion for Mod­ern Tri­al Ex­e­cu­tion

In 2016, the International Council for Harmonisation (ICH) updated their “Guidelines for Good Clinical Practice.” One key shift was a mandate to implement a risk-based quality management system throughout all stages of a clinical trial, and to take a systematic, prioritized, risk-based approach to clinical trial monitoring—on-site monitoring, remote monitoring, or any combination thereof.

Mer­ck scraps Covid-19 vac­cine pro­grams af­ter they fail to mea­sure up on ef­fi­ca­cy in an­oth­er ma­jor set­back in the glob­al fight

After turning up late to the vaccine development game in the global fight against Covid-19, Merck is now making a quick exit.

The pharma giant is reporting this morning that it’s decided to drop development of 2 vaccines — V590 and V591 — after taking a look at Phase I data that simply don’t measure up to either the natural immune response seen in people exposed to the virus or the vaccines already on or near the market.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 98,500+ biopharma pros reading Endpoints daily — and it's free.

Jean-Christophe-Hyvert, Lonza

Lon­za look­ing to build on 'd­if­fer­en­ti­at­ed ad­van­tage' in Covid-19, CD­MO mar­ket­place in 2021

It’s not new for Lonza, the Swiss CDMO nearing its quasquicentennial anniversary, to be in the upper echelon of the biotech manufacturing industry.

But 2020 — as it was for many CDMOs — was a special year even by Lonza’s standards. The company inked a deal to produce 1 billion worldwide doses of Moderna’s Covid-19 vaccine and tapped pharma vet Pierre-Alain Ruffieux to lead its operations, moves which have allowed Lonza to make a myriad of other deals that will continue to ramp up its global production capacity.

Matt Gline (L) and Vivek Ramaswamy

Scoop: Vivek Ra­maswamy is hand­ing the CEO job to a top lieu­tenant at Roivant — but he’s not ex­act­ly leav­ing the biotech scene

Over the past 7 years since founding Roivant, Vivek Ramaswamy has been a constant blur of biotech building motion.

He launched his first biotech with an Alzheimer’s drug he picked up cheap, and watched the experiment implode in one of the highest profile pivotal disasters seen in the last decade. But it didn’t slow the 30-something exec down; if anything, he hit the accelerator. Ramaswamy blazed global paths and went on to raise billions to spur the creation of a large lineup of little Vants promising big things at a fast pace. He sold off a section of the Vant brigade to Sumitomo Dainippon for $3 billion. And more recently the relentless dealmaker has been building a computational discovery arm to add an AI-driven approach to kicking up new programs and companies, supplementing the in-licensing drive while pursuing advances that have created more than 700 jobs at Roivant, with $2 billion in reserves.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

José Baselga, AstraZeneca cancer chief (Brent N. Clarke/FilmMagic via Getty Images)

As­traZeneca's Calquence nabs an­oth­er win against Im­bru­vi­ca, but Eli Lil­ly is on its heels

Three years after first launching Calquence as a second generation BTK inhibitor, AstraZeneca continues to tout new data to compete with J&J and AbbVie’s first generation blockbuster Imbruvica.

The British pharma announced on Monday that Calquence passed a head-to-head Phase III study against Imbruvica in chronic lymphocytic leukemia, proving non-inferior — i.e. just as good — as the older drug. Although AstraZeneca did not break down any of the numbers, they said the drug proved superior on safety, triggering fewer cases of atrial fibrillation, an irregular heartbeat that can lead to stroke or heart failure.

Can strug­gling Iterum turn the cor­ner to an an­tibi­ot­ic suc­cess sto­ry? They will know in six months

More than five years after Corey Fishman and Michael Dunne dusted sulopenem off Pfizer’s shelves — the second castoff antibiotic they’ve brought out of the pharma giant — and founded Iterum Therapeutics around that single drug, they have lined up a quick shot at approval with priority review from the FDA.

The decision, six months from now, will mark a make-or-break moment for a struggling biotech that has just enough cash to keep the lights on until the third quarter.

Bahija Jallal, Immunocore

Buried in Im­muno­core's IPO fil­ings? A kick­back scheme from a now for­mer em­ploy­ee

Immunocore spent much of 2019 dealing with the fallout of the Neil Woodford scandal, as the former star investor’s fall crashed the biotech’s valuation out of unicorn range. Now it turns out that the company spent 2020 dealing with another internal scandal.

The longtime UK biotech darling disclosed in their IPO filing last week that they had fallen victim to an alleged kickback scheme involving one of their employees. After a whistleblower came forward, they said in their F-1, they spent the summer and spring investigating, finding fraud on the part of an employee and two outside vendors.

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

Covid-19 roundup: Mod­er­na dou­bles down on Covid-19 with new boost­er tri­als; Aus­tralia plans do­mes­tic pro­duc­tion of As­traZeneca vac­cine amid dis­tri­b­u­tion lag

As Merck bows out of the global race to develop vaccines for Covid-19, Moderna is doubling down to make sure they can quell new variants that have recently emerged and quickly spread.

The Cambridge, MA-based biotech put out word on Monday that in vivo studies indicate their mRNA vaccine works well enough against two strains first detected in the UK and South Africa. But with a six-fold reduction in neutralizing titers observed against the latter strain, the company is launching a new study of a booster version to make sure it can do the job.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 98,500+ biopharma pros reading Endpoints daily — and it's free.

News brief­ing: Jef­frey Lei­den to chair Tmu­ni­ty board of di­rec­tors; Op­di­vo wins new ap­proval in ad­vanced RCC

Longtime Vertex CEO Jeffrey Leiden is taking on a new role.

Leiden has been appointed chairman of Tmunity’s board of directors, the company announced Monday. The move comes about a year and a half after Leiden announced he’d be stepping down from his position at Vertex.

Vertex saw immense growth under Leiden, leading the company from its exit out of hepatitis C, when cures were moving in, and into cystic fibrosis. The company’s cystic fibrosis triple combo therapy Trikafta is already its best-seller, reaching the distinction just six weeks after launch and recording the strongest first quarter of sales for any drug, per some estimates.