Park­er In­sti­tute sci­en­tists her­ald a cell ther­a­py break­through in the lab, us­ing next-gen CRISPR tech to en­gi­neer T cells

The two pi­o­neer­ing CAR-T ther­a­pies on the mar­ket — Kym­ri­ah and Yescar­ta — re­ly on vi­ral vec­tors to com­plete the en­gi­neer­ing work need­ed to make a can­cer pa­tient’s T cells in­to a ther­a­py. But a group of Park­er In­sti­tute for Can­cer Im­munother­a­py-backed sci­en­tists at UCSF and UCLA say they have per­fect­ed a new ap­proach us­ing CRISPR gene edit­ing tech that they be­lieve will trans­form the field — open­ing the door to a range of cell ther­a­pies that can be built and man­u­fac­tured far more eas­i­ly than the first-gen crowd.

An­toni Ribas

There’s noth­ing new about CRISPR, of course. The sim­ple, some­what blunt gene edit­ing tech­nol­o­gy — gen­er­al­ly fo­cused on cut­ting in­to genes while strug­gling with past­ing DNA — has tak­en aca­d­e­m­ic labs, and quite a few com­mer­cial ones, by storm in re­cent years. But the sci­en­tists in­volved in this project, in­clud­ing not­ed UCLA re­searcher An­toni Ribas, say they’ve come up with some new tech­niques that could make it a wide­ly used al­ter­na­tive to vi­ral vec­tors — which have some­times been in short sup­ply.

Fred Rams­dell

And they say it fits all the hall­marks of a tech ad­vance: Faster, bet­ter, cheap­er.

“What takes months or even a year may now take a cou­ple weeks us­ing this new tech­nol­o­gy,” not­ed Fred Rams­dell, vice pres­i­dent of re­search at PI­CI. “If you are a can­cer pa­tient, weeks ver­sus months could make a huge dif­fer­ence.”

The same goes for the com­pa­nies in­volved.

The re­searchers say this new ap­proach makes it pos­si­ble to in­sert long stretch­es of DNA in­to genes, open­ing the door to more pre­cise ther­a­pies that could be far more ef­fec­tive in tack­ling can­cer or some oth­er T cell tar­get­ed dis­ease.

Alex Mar­son

Up to now, says Alex Mar­son, as­so­ciate pro­fes­sor of mi­cro­bi­ol­o­gy and im­munol­o­gy at UCSF, CRISPR has been ef­fec­tive in knock­ing genes out — by cut­ting and in­ter­rupt­ing. “You could change a few nu­cleotides at a time,” he not­ed, “but if you put in big­ger da­ta, it would kill the cells.”

This new de­vel­op­ment of theirs us­es elec­tro­po­ra­tion to con­di­tion cell mem­branes and has been used in the lab to go much fur­ther — not just past­ing in­to ran­dom places, but be­ing far more pre­cise. 

“This is sort of a flex­i­ble thing to change the way T cells work,” Mar­son adds, and the teams in­volved used it in the lab to work ex vi­vo on a rare and se­vere form of pe­di­atric au­toim­mune dis­ease, cor­rect­ing mu­ta­tions. A sec­ond project used the tech­nol­o­gy to cre­ate a new T cell ther­a­py for melanoma, suc­cess­ful­ly test­ing it in mice.

Theo Roth

Now the fo­cus will shift to mov­ing this in­to hu­mans.

“This is some­thing we’re very ac­tive­ly work­ing to­ward now,” says Mar­son, who’s pur­su­ing con­ver­sa­tions with the FDA as well as com­mer­cial part­ners to lay out the next steps of get­ting this moved to­ward the clin­ic.

“There has been thir­ty years of work try­ing to get new genes in­to T cells,” said UCSF stu­dent and lead au­thor Theo Roth, who’s been do­ing much of the fine tun­ing work in the lab. “Now there should no longer be a need to have six or sev­en peo­ple in a lab work­ing with virus­es just to en­gi­neer T cells, and if we be­gin to see hun­dreds of labs en­gi­neer­ing these cells in­stead of just a few, and work­ing with in­creas­ing­ly more com­plex DNA se­quences, we’ll be try­ing so many more pos­si­bil­i­ties that it will sig­nif­i­cant­ly speed up the de­vel­op­ment of fu­ture gen­er­a­tions of cell ther­a­py.”

Aerial view of Genentech's campus in South San Francisco [Credit: Getty]

Genen­tech sub­mits a big plan to ex­pand its South San Fran­cis­co foot­print

The sign is still there, a quaint reminder of whitewashed concrete not 5 miles from Genentech’s sprawling, chrome-and-glass campus: South Francisco The Industrial City. 

The city keeps the old sign, first erected in 1923, as a tourist site and a kind of civic memento to the days it packed meat, milled lumber and burned enough steel to earn the moniker “Smokestack of the Peninsula.” But the real indication of where you are and how much has changed both in San Francisco and in the global economy since a couple researchers and investors rented out an empty warehouse 40 years ago comes in a far smaller blue sign, resembling a Rotary Club post, off the highway: South San Francisco, The Birthplace of Biotech.

Here comes the oral GLP-1 drug for di­a­betes — but No­vo Nordisk is­n't dis­clos­ing Ry­bel­sus price just yet

Novo Nordisk’s priority review voucher on oral semaglutide has paid off. The FDA approval for the GLP-1 drug hit late Friday morning, around six months after the NDA filing.

Rybelsus will be the first GLP-1 pill to enter the type 2 diabetes market — a compelling offering that analysts have pegged as a blockbuster drug with sales estimates ranging from $2 billion to $5 billion.

Ozempic, the once-weekly injectable formulation of semaglutide, brought in around $552 million (DKK 3.75 billion) in the first half of 2019.

As Nas­daq en­rolls the fi­nal batch of 2019 IPOs, how have the num­bers com­pared to past years?

IGM Biosciences’ upsized IPO haul, coming after SpringWorks’ sizable public debut, has revved up some momentum for the last rush of biotech IPOs in 2019.

With 39 new listings on the books and roughly two more months to go before winding down, Nasdaq’s head of healthcare listings Jordan Saxe sees the exchange marking 50 to 60 biopharma IPOs for the year.

“December 15 is usually the last possible day that companies will price,” he said, as companies get ready for business talks at the annual JP Morgan Healthcare Conference in January.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 60,200+ biopharma pros reading Endpoints daily — and it's free.

Oxitec biologist releases genetically modified mosquitoes in Piracicaba, Brazil in 2016 [credit: Getty Images]

In­trex­on unit push­es back against claims its GM mos­qui­toes are mak­ing dis­ease-friend­ly mu­tants

When the hysteria of Zika transmission sprang into the American zeitgeist a few years ago, UK-based Oxitec was already field-testing its male Aedes aegypti mosquito, crafted to possess a gene engineered to obliterate its progeny long before maturation.

But when a group of independent scientists evaluated the impact of the release of these genetically-modified mosquitoes in a trial conducted by Oxitec in Brazil between 2013 and 2015, they found that some of the offspring had managed to survive — prompting them to speculate what impact the survivors could have on disease transmission and/or insecticide resistance.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 60,200+ biopharma pros reading Endpoints daily — and it's free.

[via AP Images]

Pur­due threat­ens to walk away from set­tle­ment, asks to pay em­ploy­ees mil­lions in bonus­es

There are two updates on the lawsuit against Purdue Pharma over its role in fueling the opioid epidemic, as the Sackler family threatens to walk away from their pledge to pay out $3 billion if a bankruptcy judge does not stop outstanding state lawsuits against them. At the same time, the company has asked permission to pay millions in bonuses to select employees.

Purdue filed for chapter 11 bankruptcy this week as part of its signed resolution to over 2,000 lawsuits. The deal would see the Sackler family that owns Purdue give $3 billion from their personal wealth and the company turned into a trust committed to curbing and reversing overdoses.

David Grainger [file photo]

'Dis­con­nect the bas­tard­s' — one biotech's plan to break can­cer cell­s' uni­fied de­fens­es

Chemotherapy and radiotherapy are the current gladiators of cancer treatment, but they come with well-known limitations and side-effects. The emergence of immunotherapy — a ferocious new titan in oncologist’s toolbox — takes the brakes off the immune system to kill cancer cells with remarkable success in some cases, but the approach is not always effective. What makes certain forms of cancer so resilient? Scientists may have finally pieced together a tantalizing piece of the puzzle, and a new biotech is banking on a new approach to fill the gap.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 60,200+ biopharma pros reading Endpoints daily — and it's free.

A fa­vorite in Alex­ion’s C-suite is leav­ing, and some mighty sur­prised an­a­lysts aren’t the least bit hap­py about it

Analysts hate to lose a biotech CFO they’ve come to trust and admire — especially if they’re being blindsided by a surprise exit.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 60,200+ biopharma pros reading Endpoints daily — and it's free.

Scott Gottlieb, AP Images

Scott Got­tlieb is once again join­ing a team that en­joyed good times at the FDA un­der his high-en­er­gy stint at the helm

Right after jumping on Michael Milken’s FasterCures board on Monday, the newly departed FDA commissioner is back today with news about another life sciences board post that gives him a ringside chair to cheer on a lead player in the real-world evidence movement — one with very close ties to the FDA.

Aetion is reporting this morning that Gottlieb is joining their board, a group that includes Mohamad Makhzoumi, a general partner at New Enterprise Associates, where Gottlieb returned after stepping out of his role at the FDA 2 years after he started.

Gottlieb — one of the best connected execs in biopharma — knows this company well. As head of FDA he championed the use of real-world evidence to help guide drug developers and the agency in gaining greater efficiencies, which helped set up Aetion as a high-profile player in the game.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 60,200+ biopharma pros reading Endpoints daily — and it's free.

Tower Bridge in London [Shutterstock]

#UK­BIO19: Join GSK’s Hal Bar­ron and a group of top biotech ex­ecs for our 2nd an­nu­al biotech sum­mit in Lon­don

Over the past 10 years I’ve made a point of getting to know the Golden Triangle and the special role the UK biopharma industry plays there in drug development. The concentration of world class research institutes, some of the most accomplished scientists I’ve ever seen at work and a rising tide of global investment cash leaves an impression that there’s much, much more to come as biotech hubs are birthed and nurtured.