With a po­ten­tial cure for lyso­so­mal stor­age dis­or­ders in hand, Avro­bio rais­es $60M to back gene ther­a­py pipeline

In gene ther­a­py, re­spons­es from just one or two pa­tients can have a huge in­flu­ence on an­a­lysts’ opin­ions. Just ask the ex­ec­u­tive team at blue­bird bio, which trum­pets each pa­tient’s progress with de­light — or cal­i­brates a new de­vel­op­ment strat­e­gy when smiles turn to frowns.

So it’s no won­der that Avro­bio is so proud of its very first pa­tient.

Ge­off MacK­ay

At a sci­en­tif­ic con­fer­ence in Japan last fall, re­searchers re­viewed the case of a pa­tient with Fab­ry dis­ease whose blood lev­els of the en­zyme a-Gal A were near ze­ro at the start of treat­ment. With­in 45 days of re­ceiv­ing their gene ther­a­py AVR-RD-01, the pa­tient’s plas­ma a-Gal A ac­tiv­i­ty in­creased in­to the nor­mal range, they said. And 6 months lat­er that hadn’t changed.

In oth­er words, they got the re­sponse they want­ed and Pa­tient 1 cleared the first hur­dle on dura­bil­i­ty. All sys­tems go for a young com­pa­ny that is out to cre­ate a once-and-done fix.

At­las seed­ed the com­pa­ny — Bruce Booth is the proud chair­man — and then launched it in the spring of 2016 with a $25 mil­lion A round that backed the work of 15 staffers. Over the last few months, its staff has dou­bled. And more hires are be­ing planned for lat­er this year.

That up­beat start has helped in­spire a hefty $60 mil­lion round to back the mid-stage work that will soon be­gin for a com­pa­ny that has laid out a broad plan to fol­low some of the lead­ers in lyso­so­mal stor­age dis­or­ders with the promise of a cure that could one day rel­e­gate the cur­rent stan­dard of care for these ail­ments to an his­toric foot­note.

Bruce Booth

The Se­ries B round was co-led by Cor­morant As­set Man­age­ment and Sur­vey­or Cap­i­tal (a Citadel com­pa­ny), and in­clud­ed Ais­ling, Brace Phar­ma Cap­i­tal, Even­tide, Morn­ing­side and Leerink Part­ners, along with ex­ist­ing in­vestors At­las Ven­ture, SV Health In­vestors and Clarus Ven­tures.

Get­ting an in­vest­ment group like Leerink in your round — not an every­day event in biotech — might make you think that there’s an IPO in the off­ing, es­pe­cial­ly with 2018 look­ing like an es­pe­cial­ly good year to take a biotech pub­lic. Gene ther­a­py pi­o­neers have al­so earned a lot of re­spect on Nas­daq, even with some tur­bu­lence for every set­back.

But Avro­bio CEO Ge­off MacK­ay is cir­cum­spect about any near-term of­fer­ings.

The com­pa­ny’s fo­cus this year is on kick­ing off a crit­i­cal Phase II tri­al for Fab­ry dis­ease, he says, to run par­al­lel to the Phase I.

There are no time­lines for the tri­als on pub­lic dis­play, though.

In gene ther­a­py, even af­ter the first US ap­proval, all com­pa­nies in the field tend to get striped with the same brush. MacK­ay’s de­sire now is to make sure that every­one knows that Avro­bio is in the lentivi­ral camp, where you’ll find lead­ers like blue­bird bio.

“AAV is a dif­fer­ent vec­tor,” he says. “It’s not fair to ex­trap­o­late to lentivi­ral.”

And at that point I hadn’t even men­tioned the James Wil­son study sound­ing the alarm around high-dose gene ther­a­pies us­ing AAV tech ear­li­er in the week.

A break­through with a gene ther­a­py would un­doubt­ed­ly have a huge im­pact on the pa­tients who de­pend on steady in­fu­sions of some high-priced ther­a­pies.

“We’re very re­spect­ful of the con­tri­bu­tion Gen­zyme and Shire’s made,” says MacK­ay. “Twen­ty years ago these pa­tients had no op­tions, then the pa­tients had an op­tion. When we look at it with fresh eyes, we see a sig­nif­i­cant op­por­tu­ni­ty to im­prove on the stan­dard of care.”

“Pa­tients now re­ly on bi­week­ly in­fu­sions of en­zymes, with se­vere chal­lenges to cost, com­pli­ance and qual­i­ty of life,” adds the CEO. “When ERT suc­ceeds it slows the pro­gres­sion of dis­ease but doesn’t halt it. The cen­tral premise is to de­liv­er via a one-time in­fu­sion for 24-7 pro­tec­tion, which we hope will halt the pro­gres­sion rather than just slow it down.”

Avro­bio has a long ways to go, but it has big plans for when it ar­rives.

Stephen Hahn, FDA commissioner (AP Images)

As FDA sets the stage for the first Covid-19 vac­cine EUAs, some big play­ers are ask­ing for a tweak of the guide­lines

Setting the stage for an extraordinary one-day meeting of the Vaccines and Related Biological Products Advisory Committee this Thursday, the FDA has cleared 2 experts of financial conflicts to help beef up the committee. And regulators went on to specify the safety, efficacy and CMC input they’re looking for on EUAs, before they move on to the full BLA approval process.

All of this has already been spelled out to the developers. But the devil is in the details, and it’s clear from the first round of posted responses that some of the top players — including J&J and Pfizer — would like some adjustments and added feedback. And on Thursday, the experts can offer their own thoughts on shaping the first OKs.

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A new chap­ter in the de­cen­tral­ized clin­i­cal tri­al ap­proach

Despite the promised decentralized trial revolution, we haven’t yet moved the needle in a significant way, although we are seeing far bolder commitments to this as we continue to experience the pandemic restrictions for some time to come. The vision of grandeur is one thing, but operationalizing and execution are another and recognising that change, particularly mid-flight on studies, is worthy of thorough evaluation and consideration in order to achieve success. Here we will discuss one of the critical building blocks of a Decentralized and Remote Trial strategy: TeleConsent; more than paper under glass, it is a paradigm change and key digital enabler.

RBC's Bri­an Abra­hams holds a mock ad­comm on Bio­gen's iffy ad­u­canum­ab da­ta — and most of these ex­perts don't see a path to an ap­proval

As catalysts go, few loom larger than the aducanumab adcomm slated for Nov. 6.

With its big franchise under assault, Biogen is betting the ranch that its mixed late-stage Alzheimer’s data can squeak past the experts and regulators and get onto the market. And the topic — after a decade of Alzheimer’s R&D disasters in what still represents the El Dorado of drug markets — remains in the center ring of discussions around late-stage pipeline prospects.

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Spar­ingVi­sion rais­es $52M to kick off long jour­ney for a next-gen gene ther­a­py that goes much, much broad­er than Lux­tur­na

Until Spark Therapeutics’ pioneering gene therapy, Luxturna, came along, patients with retinitis pigmentosa had few treatment options. Even after it was approved, though, the majority were left with the exact same options.

Because it’s targeting mutations in a specific gene known as RPE65, Luxturna can only address 2 to 3% of the entire RP population, Stephane Boissel told Endpoints News.

Boissel is the newly-minted CEO of SparingVision, a French biotech co-founded by José-Alain Sahel and Thierry Léveillard at the Institut de la Vision. They have their sight set on a new kind of AAV construct — a next-generation gene therapy if you will — that can treat all patients of RP independent of genetic mutations.

Giovanni Caforio, Bristol Myers Squibb CEO (Christopher Goodney/Bloomberg via Getty Images)

Here's how Bris­tol My­er­s' CEO Gio­van­ni Caforio com­plet­ed a $13B buy­out: He moved fast, upped the bid quick­ly and de­mand­ed every­one to keep up

Bristol Myers Squibb CEO Giovanni Caforio does not waste time. He also likes everyone around him to keep up.

Anyone reading over the insider account filed with the SEC of the back-and-forth over his $13 billion buyout of MyoKardia $MYOK could reach only one conclusion: The CEO who had willingly crafted a $74 billion Celgene acquisition had found something else he liked — and he was willing to pay a nice premium to get it.

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Sean McClain, AbSci

UP­DAT­ED: Months af­ter strik­ing a deal with Sanofi, Ab­Sci nails its largest fundraise yet to sup­port its pro­tein print­ing tech

Months after nailing down a $10.4 million Series D and a partnership with French pharma giant Sanofi, AbSci is pulling in its largest fundraise yet — $65 million to advance what it calls its protein printing tech.

The Vancouver, WA-based biotech is working on a more efficient way to manufacture proteins — from full-length antibodies to insulin — using E. coli. The company says it’s on a quest to make protein production “as simple as DNA synthesis,” allowing for swift production of difficult-to-produce biotherapeutics. It’s tagging the Series E for expansion of its production capacity, which includes a new 60,000-square-foot facility in Vancouver.

Roche finds a home for a new, $500M man­u­fac­tur­ing lo­gis­tics hub, promis­ing 500 jobs

Roche is pouring $500 million into its Canadian headquarters in Mississauga, Ontario to set up a new hub that will coordinate logistics for its global supply chain.

Over the 5-year investment, the Swiss pharma giant expects to add 200 jobs over next year and another 300 by the end of 2023.

Introduced as a $190 million global pharmaceutical development site in 2011, the campus currently houses Roche’s Canadian commercial unit as well as product development, global procurement and pharma informatics. The new expansion will see it organize manufacturing across 13 plants and 11 sites, according to FiercePharma.

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MD An­der­son es­tab­lish­es new $50M+ biotech fund to choose the next promis­ing can­cer ther­a­pies

Researchers at MD Anderson have a new partner to help them get early-stage programs into the clinic.

In conjunction with The Focus Fund, MD Anderson is launching the Cancer Focus Fund to advance investigational cancer therapies from late preclinical development through Phase I and Phase Ib/II clinical trials. The fund will start off with more than $50 million of initial capital.

“Without appropriate support, we know that some therapies with great potential may be delayed, may not be developed properly in the clinical setting or may never make it into clinical studies,” MD Anderson senior VP Ferran Prat said in a statement. “Through investment from the Cancer Focus Fund and the support of MD Anderson, we hope to advance worthwhile new treatments past the traditional hurdles in the drug development process.”

CAR-plus: Irish biotech re­cruits Kite alum Chris Now­ers to prep dual-tar­get­ing NK cell ther­a­py for the clin­ic

Soon after Chris Nowers left Cell Medica — freshly rebranded Kuur Therapeutics — in February, the Kite Pharma alum was introduced to another cell therapy player.

The basic idea of building an off-the-shelf allogeneic platform with a CAR-NK approach was familiar to him, riding on the same wave as Takeda, J&J-backed Fate, Nkarta and others. But then there was something else that stood out: a membrane-bound TNF related apoptosis inducing ligand variant, or TRAIL variant, that’s also engineered onto the NK cell for a dual-targeted attack.