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.

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Biogen’s controversial new Alzheimer’s drug Aduhelm has been approved on the basis of removing amyloid plaque from the brain, but the new neuro-focused pharma and biotechs have much loftier aims. Significantly curbing or even curing the most notorious disorders would prove the Holy Grail for a complex system that has tied the world’s best drug developers in knots for decades.

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Ugur Sahin, BioNTech CEO (Bernd von Jutrczenka/dpa via AP Images)

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Art Levinson (Calico)

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