Geoff MacKay, Avrobio

Avro­bio is us­ing 'pod­s' to cre­ate gene ther­a­pies, and it be­lieves the ap­proach could change the game al­to­geth­er

Ge­off MacK­ay

Where oth­er com­pa­nies have gone big, Avro­bio is go­ing small. Its man­u­fac­tur­ing op­er­a­tion is no dif­fer­ent.

In­stead of try­ing to keep up with the val­i­da­tion of new fa­cil­i­ties — and spend­ing mon­ey and pre­cious time in the process — the com­pa­ny has opt­ed for a dif­fer­ent ap­proach: small, au­to­mat­ed pods that can be in­stalled in rent­ed clean rooms any­where. Though pro­duc­tion out­put isn’t as high as oth­er com­pa­nies, Avro­bio avoids brick-and-mor­tar and is able to stay flex­i­ble and nim­ble to re­spond to fluc­tu­a­tions.

“We be­lieve it’s the on­ly way to glob­al­ize,” said Ge­off MacK­ay, Avro’s founder and CEO. “We had no ap­petite to build big fa­cil­i­ties every­where in the world.”

The com­pa­ny based in Cam­bridge, MA is fo­cused on sin­gle-dose per­son­al­ized gene ther­a­py to cure Fab­ry dis­ease, Gauch­er dis­ease type 1 and cysti­nosis through the plat­form its dubbed pla­to. Un­like oth­er gene ther­a­pies that are de­liv­ered straight in­to pa­tients, Avro­Bio’s lentivi­ral-based ther­a­pies are de­liv­ered ex vi­vo in­to pa­tients’ stem cells be­fore pro­lif­er­a­tion and then in­fu­sion back in­to pa­tients.

The pods — typ­i­cal­ly used in hos­pi­tals for bone mar­row trans­plants — come from Mil­tenyi Biotec, and MacK­ay and his team re­pur­pose the tech for use in mod­i­fy­ing the pa­tients’ stem cells. The pods are housed in CMO clean rooms all over the world, from Hous­ton to Mel­bourne, San Jose and Maas­tricht, but the biotech even­tu­al­ly plans to scale up ca­pac­i­ty for its own in-house ver­sion of the tech­nol­o­gy.

MacK­ay be­lieves a lo­cal­ized man­u­fac­tur­ing chain is cru­cial be­cause it al­lows for each pa­tients’ stem cells to be stored in rel­a­tive prox­im­i­ty to the pa­tient.  The mod­el al­so al­lows a greater de­gree of flex­i­bil­i­ty — each dis­crete pod can be used for a wide va­ri­ety of ther­a­pies — and easy scal­a­bil­i­ty.

“The beau­ty here is that you val­i­date the pod, but reg­u­la­tors are ag­nos­tic about where you house the pod, be­cause the en­vi­ron­ment doesn’t change,” MacK­ay said. “It’s the ex­act same steps, the ex­act same en­vi­ron­ment, so you kind of just side­step all of the com­plex­i­ties of get­ting new man­u­fac­tur­ing fa­cil­i­ties on­line.”

It’s al­so a boon for Avro­Bio, which at the clin­i­cal stage is ap­proach­ing the point at which most gene ther­a­py com­pa­nies start think­ing of in­vest­ing heavy cap­i­tal in their own plants. With­out the need for a huge foot­print, that could Avro­Bio cap­i­tal flex­i­ble as it moves in­to the next stage of tri­als.

“When you’re a biotech com­pa­ny in ear­ly de­vel­op­ment, the cost of cap­i­tal is ob­vi­ous­ly pre­cious, and if you don’t have to spend hun­dreds of mil­lions of dol­lars with a VC type cost of cap­i­tal, that’s great … Nor­mal­ly, you don’t have that type of lux­u­ry,” MacK­ay said.

Avro­bio cur­rent­ly has 3 prod­ucts in clin­i­cal tri­als. Its gene ther­a­py for Fab­ry dis­ease is cur­rent­ly be­ing stud­ied in two clin­i­cal tri­als: 1 Phase I tri­al in Cana­da, and a Phase II tri­al in Aus­tralia, the US and Cana­da. It al­so has a ther­a­py for Gauch­er dis­ease type 1 that is in Phase I in Cana­da and Aus­tralia, and a cysti­nosis study in a Phase I/II tri­al at the Uni­ver­si­ty of Cal­i­for­nia, San Diego.

To­ward the end of 2018, the gene ther­a­py com­pa­ny saw its stock plum­met, drop­ping down 52% af­ter its da­ta sur­round­ing Fab­ry dis­ease treat­ment showed that vec­tor copy num­bers dropped in pe­riph­er­al blood in all of its pa­tients be­tween 1 and 18 months.

Re­cent­ly, the com­pa­ny an­nounced new da­ta that showed at least in a few pa­tients, the ther­a­py saw 100% re­duc­tion in kid­ney sub­strate, the end­point the FDA us­es for ap­proval. There has been strong safe­ty and ef­fi­ca­cy da­ta in all 14 pa­tients in the tri­al, and so far, a year from dos­ing, 10 pa­tients in clin­i­cal tri­als show no wan­ing ef­fi­ca­cy.

IDC: Life Sci­ences Firms Must Em­brace Dig­i­tal Trans­for­ma­tion Now

Pre-pandemic, the life sciences industry had settled into a pattern. The average drug took 12 years and $2.9 billion to bring to market, and it was an acceptable mode of operations, according to Nimita Limaye, Research Vice President for Life Sciences R&D Strategy and Technology at IDC.

COVID-19 changed that, and served as a proof-of-concept for how technology can truly help life sciences companies succeed and grow, Limaye said. She recently spoke about industry trends at Egnyte’s Life Sciences Summit 2022. You should watch the entire session, free and on-demand, but here’s a brief recap of why she’s urging life sciences companies to embrace digital transformation.

Tom Barnes, Orna Therapeutics CEO

UP­DAT­ED: 'We have failed to fail': Mer­ck gam­bles $250M cash on a next-gen ap­proach to mR­NA — af­ter punt­ing its big al­liance with Mod­er­na

Merck went in deep on its collaboration with Moderna on new mRNA programs, and dropped them all over time, including their RSV partnership. But after writing off what turned out as one of the most successful infectious disease players in the business, Merck is coming in this morning with a new preclinical alliance — this time embracing a biotech that hopes to eventually outdo the famously successful mRNA in a new run at vaccines and therapeutics.

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Bayer's first DTC ad campaign for chronic kidney disease drug Kerendia spells out its benefits

Bay­er aims to sim­pli­fy the com­plex­i­ties of CKD with an ABC-themed ad cam­paign

Do you know the ABCs of CKD in T2D? Bayer’s first ad campaign for Kerendia tackles the complexity of chronic kidney disease with a play on the acronym (CKD) and its connection to type 2 diabetes (T2D).

Kerendia was approved last year as the first and only non-steroidal mineralocorticoid receptor antagonist to treat CKD in people with type 2 diabetes.

In the TV commercial launched this week, A is for awareness, B is for belief and C is for cardiovascular, explained in the ad as awareness of the connection between type 2 and kidney disease, belief that something can be done about it, and cardiovascular events that may be reduced with treatment.

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James Mock, incoming CFO at Moderna

Mod­er­na taps new CFO from PerkinElmer af­ter for­mer one-day CFO oust­ed

When Moderna hired a new CFO last year,  it didn’t expect to see him gone after only one day. Today the biotech named his — likely much more vetted — replacement.

The mRNA company put out word early Wednesday that after the untimely departure of then brand-new CFO Jorge Gomez, it has now found a replacement in James Mock, the soon-to-be former CFO at diagnostics and analytics company PerkinElmer.

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Etleva Kadilli, director of UNICEF’s supply division

GSK lands first-ever UNICEF con­tract for malar­ia vac­cine worth $170M

GSK has landed a new first from UNICEF the first-ever contract for malaria vaccines, worth up to $170 million for 18 million vaccine doses distributed over the next three years.

The vaccine, known as Mosquirix or RTS,S, won WHO’s backing last October after a controversial start, but UNICEF said these doses will potentially save thousands of lives every year.

“We hope this is just the beginning,” Etleva Kadilli, director of UNICEF’s supply division, said. “Continued innovation is needed to develop new and next-generation vaccines to increase available supply, and enable a healthier vaccine market. This is a giant step forward in our collective efforts to save children’s lives and reduce the burden of malaria as part of wider malaria prevention and control programmes.”

Governor John Carney, Lieutenant Governor Bethany Hall-Long, Mayor Kenneth Branner, Jr., Delaware Prosperity Partnership President Kurt Foreman, WuXi AppTec Chairman and CEO Ge Li, WuXi AppTec Co-CEO and WuXi STA CEO Minzhang Chen, and others celebrate the groundbreaking for the WuXi STA Middletown campus.

WuXi breaks ground on Delaware fa­cil­i­ty, boost­ing its US pres­ence

While Middletown, Delaware’s main claim to fame was the site location for the film the Dead Poets Society, the city will soon play host to a massive manufacturing outfit.

WuXi AppTec’s contract research and manufacturing subsidiary WuXi STA, has broken ground on a 190-acre manufacturing campus in Middletown.

According to the company, this site will be WuXi’s second facility in the US, and it will create around 500 full-time jobs by 2026, but there are plans to kick off operations in 2025.

Joe Jonas (Photo by Anthony Behar/Sipa USA)(Sipa via AP Images)

So­lo Jonas broth­er car­ries Merz's new tune in Botox ri­val cam­paign

As the lyrics of his band’s 2019 pop-rock single suggest, Joe Jonas is only human — and that means even he gets frown lines. The 33-year-old singer-songwriter is Merz’s newest celebrity brand partner for its Botox rival Xeomin, as medical aesthetics brands target a younger audience.

Merz kicked off its “Beauty on Your Terms” campaign on Tuesday, featuring the Jonas brother in a video ad for its double-filtered anti-wrinkle injection Xeomin.

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Kate Haviland, Blueprint Medicines CEO

Blue­print met all its end­points in bid for ex­pand­ed Ay­vak­it la­bel — but stock trends low­er any­way

Blueprint Medicines announced this morning that the second part of its study on Ayvakit in non-advanced systemic mastocytosis (SM) — a rare disease in which a type of white blood cells known as mast cells builds up — met all endpoints, but the biopharma left key questions unanswered.

In 212 patients, with 141 in the treatment arm and 71 in the control arm, patients who got Ayvakit saw an average 15.6-point decrease in their symptom scores compared to a 9.2-point decrease in the placebo arm at 24 weeks. In an extension study, those on Ayvakit saw their symptom scores drop by 20.2 points by week 48.

Paul Perreault, CSL Behring CEO

CSL CEO Paul Per­reault de­ter­mined to grow plas­ma col­lec­tion af­ter full-year sales dip

As the ink dries on CSL’s $11.7 billion Vifor buyout, the company posted a dip in profits, due in part to a drop in plasma donations amid the pandemic.

However, CEO Paul Perreault assured investors and analysts on the full-year call that the team has left “no stone unturned” when assessing options to grow plasma volumes. The chief executive also spelled out positive results for the company’s monoclonal antibody garadacimab in hereditary angioedema (HAE), though he isn’t revealing the exact numbers just yet.