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.

Fangliang Zhang, AP Images

Leg­end fetch­es $424 mil­lion, emerges as biggest win­ner yet in pan­dem­ic IPO boom

Amid a flurry of splashy pandemic IPOs, a J&J-partnered Chinese biotech has emerged with one of the largest public raises in biotech history.

Legend Biotech, the Nanjing-based CAR-T developer, has raised $424 million on NASDAQ. The biotech had originally filed for a still-hefty $350 million, based on a range of $18-$20, but managed to fetch $23 per share, allowing them to well-eclipse the massive raises from companies like Allogene, Juno, Galapagos, though they’ll still fall a few dollars short of Moderna’s record-setting $600 million raise from 2018.

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As it hap­pened: A bid­ding war for an an­tibi­ot­ic mak­er in a mar­ket that has rav­aged its peers

In a bewildering twist to the long-suffering market for antibiotics — there has actually been a bidding war for an antibiotic company: Tetraphase.

It all started back in March, when the maker of Xerava (an FDA approved therapy for complicated intra-abdominal infections) said it had received an offer from AcelRx for an all-stock deal valued at $14.4 million.

The offer was well-timed. Xerava was approved in 2018, four years after Tetraphase posted its first batch of pivotal trial data, and sales were nowhere near where they needed to be in order for the company to keep its head above water.

Mer­ck wins a third FDA nod for an­tibi­ot­ic; Mereo tack­les TIG­IT with $70M raise in hand

Merck — one of the last big pharma bastions in the beleaguered field of antibiotic drug development — on Friday said the FDA had signed off on using its combination drug, Recarbrio, with hospital-acquired bacterial pneumonia and ventilator-associated bacterial pneumonia. The drug could come handy for use in hospitalized patients who are afflicted with Covid-19, who carry a higher risk of contracting secondary bacterial infections. Once SARS-CoV-2, the virus behind Covid-19, infects the airways, it engages the immune system, giving other pathogens free rein to pillage and plunder as they please — the issue is particularly pertinent in patients on ventilators, which in any case are breeding grounds for infectious bacteria.

RA Cap­i­tal, Hill­house join $310M rush to back Ever­est's climb to com­mer­cial heights in Chi­na

Money has never been an issue for Everest Medicines. With an essentially open tab from their founders at C-Bridge Capital, the biotech has gone two and a half years racking up drug after drug, bringing in top exec after top exec, and issuing clinical update after update.

But now other investors want in — and they’re betting big.

Everest is closing its Series C at $310 million. The first $50 million comes from the Jiashan National Economic and Technological Development Zone; the remaining C-2 tranche was led by Janchor Partners, with RA Capital Management and Hillhouse Capital as co-leaders. Decheng Capital, GT Fund, Janus Henderson Investors, Rock Springs Capital, Octagon Investments all joined.

President Donald Trump (left) and Moncef Slaoui, head of Operation Warp Speed (Alex Brandon, AP Images)

UP­DAT­ED: White House names fi­nal­ists for Op­er­a­tion Warp Speed — with 5 ex­pect­ed names and one no­table omis­sion

A month after word first broke of the Trump Administration’s plan to rapidly accelerate the development and production of a Covid-19 vaccine, the White House has selected the five vaccine candidates they consider most likely to succeed, The New York Times reported.

Most of the names in the plan, known as Operation Warp Speed, will come as little surprise to those who have watched the last four months of vaccine developments: Moderna, which was the first vaccine to reach humans and is now the furthest along of any US effort; J&J, which has not gone into trials but received around $500 million in funding from BARDA earlier this year; the joint AstraZeneca-Oxford venture which was granted $1.2 billion from BARDA two weeks ago; Pfizer, which has been working with the mRNA biotech BioNTech; and Merck, which just entered the race and expects to put their two vaccine candidates into humans later this year.

Is a pow­er­house Mer­ck team prepar­ing to leap past Roche — and leave Gilead and Bris­tol My­ers be­hind — in the race to TIG­IT dom­i­na­tion?

Roche caused quite a stir at ASCO with its first look at some positive — but not so impressive — data for their combination of Tecentriq with their anti-TIGIT drug tiragolumab. But some analysts believe that Merck is positioned to make a bid — soon — for the lead in the race to a second-wave combo immuno-oncology approach with its own ambitious early-stage program tied to a dominant Keytruda.

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Drug man­u­fac­tur­ing gi­ant Lon­za taps Roche/phar­ma ‘rein­ven­tion’ vet as its new CEO

Lonza chairman Albert Baehny took his time headhunting a new CEO for the company, making it absolutely clear he wanted a Big Pharma or biotech CEO with a good long track record in the business for the top spot. In the end, he went with the gold standard, turning to Roche’s ranks to recruit Pierre-Alain Ruffieux for the job.

Ruffieux, a member of the pharma leadership team at Roche, spent close to 5 years at the company. But like a small army of manufacturing execs, he gained much of his experience at the other Big Pharma in Basel, remaining at Novartis for 12 years before expanding his horizons.

Covid-19 roundup: Ab­b­Vie jumps in­to Covid-19 an­ti­body hunt; As­traZeneca shoots for 2B dos­es of Ox­ford vac­cine — with $750M from CEPI, Gavi

Another Big Pharma is entering the Covid-19 antibody hunt.

AbbVie has announced a collaboration with the Netherlands’ Utrecht University and Erasmus Medical Center and the Chinese-Dutch biotech Harbour Biomed to develop a neutralizing antibody that can treat Covid-19. The antibody, called 47D11, was discovered by AbbVie’s three partners, and AbbVie will support early preclinical work, while preparing for later preclinical and clinical development. Researchers described the antibody in Nature Communications last month.

David Meline (file photo)

Mod­er­na’s new CFO took a cut in salary to jump to the mR­NA rev­o­lu­tion­ary. But then there’s the rest of the com­pen­sa­tion pack­age

David Meline took a little off the top of his salary when he jumped from the CFO post at giant Amgen to become the numbers czar at the upstart vaccines revolutionary Moderna. But the SEC filing that goes with a major hire also illustrates how it puts him in line for a fortune — provided the biotech player makes good as a promising game changer.

To be sure, there’s nothing wrong with the base salary: $600,000. Or the up-to 50% annual cash bonus — an industry standard — that comes with it. True, the 62-year-old earned $999,000 at Amgen in 2019, but it’s the stock options that really count in the current market bliss for all things biopharma. And there Meline did well.

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