Boehringer or­ga­nizes a coali­tion in search of first CF gene ther­a­py, work­ing with re­search, man­u­fac­tur­ing part­ners

Join­ing hands with sev­er­al aca­d­e­m­ic and in­dus­try part­ners from the UK, Boehringer In­gel­heim is launch­ing a quest to de­vel­op a gene ther­a­py for cys­tic fi­bro­sis — one that, if suc­cess­ful, would shake up a field plagued by some re­cent fail­ures and dom­i­nat­ed by Ver­tex.

The back­bone of the col­lab­o­ra­tion is 17 years worth of work done by the UK Cys­tic Fi­bro­sis Gene Ther­a­py Con­sor­tium, pulling to­geth­er re­searchers from Im­pe­r­i­al Col­lege Lon­don and the Uni­ver­si­ties of Ox­ford and Ed­in­burgh. With help from Boehringer, the con­sor­tium will lead the de­vel­op­ment and com­ple­tion of pre­clin­i­cal stud­ies on a gene ther­a­py that it’s iden­ti­fied, to a point where the prod­uct will be ready for hu­man stud­ies.

To fa­cil­i­tate the tech trans­fer and man­u­fac­tur­ing process­es, Boehringer has al­so tapped Im­pe­r­i­al In­no­va­tions and Ox­ford Bio­Med­ica for a three-way part­ner­ship, such that the Ger­man drug­mak­er will have an op­tion to li­cense their vi­ral vec­tor and pro­duc­tion tech­nol­o­gy.

The big idea here is to de­vel­op a treat­ment that can ad­dress all the 2,000 known mu­ta­tions in the gene for the cys­tic fi­bro­sis trans­mem­brane con­duc­tance reg­u­la­tor (CFTR). Where­as ex­ist­ing ther­a­pies can on­ly slow the progress of the dis­ease and tend to have vary­ing ef­fects de­pend­ing on a pa­tient’s mu­ta­tion sta­tus, Boehringer says their nov­el ap­proach — in­tro­duc­ing a healthy copy of the CFTR gene in­to lung cells us­ing a lentivi­ral vec­tor via an in­haled for­mu­la­tion — has the po­ten­tial to be a uni­ver­sal op­tion.

Fi­nan­cial terms of the deal were not dis­closed and no time­lines were pro­vid­ed.

There’s been con­sid­er­able at­ten­tion on cys­tic fi­bro­sis as Ver­tex con­tin­ues to dom­i­nate the space and move for­ward with promis­ing com­bo stud­ies. A host of ri­vals have ap­peared, in­clud­ing Gala­pa­gos, but no one has pro­vid­ed any re­cent da­ta to sup­port one of these oth­er con­tenders.

In the past weeks, we’ve seen Pro­teosta­sis plunge on failed Phase IIb tri­als, with Celtaxsys join­ing the club last week clinch­ing shaky da­ta they claimed to be “clin­i­cal­ly mean­ing­ful.”

Ver­tex has scored big in the US with its new drugs, but the biotech has been build­ing up some ten­sion with sin­gle-pay­er groups in Eu­rope — which hap­pens to be Boehringer’s home turf.

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.

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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.

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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.

While No­var­tis ban­ish­es Zol­gens­ma scan­dal scars — Bio­gen goes on a Spin­raza 'of­fen­sive'

While Novartis painstakingly works to mop up the stench of the data manipulation scandal associated with its expensive gene therapy for spinal muscular atrophy (SMA) Zolgensma— rival Biogen is attempting to expand the use of its SMA therapy, Spinraza. 

The US drugmaker $BIIB secured US approval for Spinraza for use in the often fatal genetic disease in 2016. The approval covered a broad range of patients with infantile-onset (most likely to develop Type 1) SMA. 

Jason Kelly. Mike Blake/Reuters via Adobe

Eye­ing big ther­a­peu­tic push, Gink­go bags $290M to build a cell pro­gram­ming em­pire

Ginkgo Bioworks is on a roll. Days after publicizing a plan to nurture new startups via partnerships with accelerators Y Combinator and Petri, the Boston biotech says it has raised another $290 million for its cell programming platform to reach further and wider.

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UP­DAT­ED: Speak­er Nan­cy Pelosi to un­veil bill for fed­er­al­ly ne­go­ti­at­ed drug prices

After months of buzz from both sides of the aisle, Speaker Nancy Pelosi will today introduce her plan to allow the federal government to negotiate prices for 250 prescription drugs, setting up a showdown with a pharmaceutical industry working overtime to prevent it.

The need to limit drug prices is a rare point of agreement between President Trump and Democrats, although the president has yet to comment on the proposal and will likely face pressure to back a more conservative option or no bill at all. Republican Senator Chuck Grassley is reportedly lobbying his fellow party members on a more modest proposal he negotiated with Democratic Senator Ron Wyden in July.

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.

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