Biotech vet MacK­ay grabs a $25M A round to back a gene ther­a­py up­start

Ge­off McK­ay

Af­ter com­plet­ing an 11-year stint as CEO of Organo­gen­e­sis, Ge­off MacK­ay gath­ered a small, ex­pe­ri­enced team to­geth­er and be­gan a glob­al search for new gene ther­a­pies at Avro­Bio. Ear­li­er this year, they land­ed seed fi­nanc­ing from At­las Ven­ture af­ter ze­ro­ing in on the work of a pair of sci­en­tists in Toron­to. And to­day he land­ed a $25 mil­lion A round to get his two lead pro­grams in­to the clin­ic lat­er this year.

MacK­ay be­lieves that this is ex­act­ly the right time to get in­to gene ther­a­py, af­ter pi­o­neers like Blue­bird have es­tab­lished how the tech­nol­o­gy can work safe­ly. And he’s putting that to the test with a new gene ther­a­py aimed at rare cas­es of Fab­ry dis­ease.

“We like that the con­cept has been val­i­dat­ed,” says the biotech vet, who al­so ex­pects to be able to move quick­ly now that the A round is in place. MacK­ay says that if all goes ac­cord­ing to plan, the first round of safe­ty and ef­fi­ca­cy da­ta from small stud­ies should show up next year. But he’s not try­ing to rush things.

For the Fab­ry pro­gram, which will look to sub­sti­tute en­zyme re­place­ment ther­a­pies with a once-and-done cu­ra­tive treat­ment, in­ves­ti­ga­tors are us­ing an ex vi­vo ap­proach: ex­tract­ing CD34+ hematopo­et­ic stem cells from pa­tients and then us­ing a lentivi­ral vec­tor to reengi­neer them to ex­press the nor­mal gene be­fore in­fus­ing them back in­to the pa­tient.

Avro­Bio al­so has an en­try in the fast-grow­ing im­munother­a­py are­na for acute myeloid leukemia. It’s ex­tract­ing can­cer cells and en­gi­neer­ing them to ex­press IL-12 in or­der to spur a durable T cell as­sault on the can­cer.

“IL-12 not a new con­cept or cy­tokine,” says the CEO. “It’s been stud­ied for 20 years.” It’s al­so been too tox­ic to use sys­tem­i­cal­ly, so Avro­Bio is us­ing its tech­nol­o­gy to get right in­to the tu­mor mi­croen­vi­ron­ment, where it can do all the dam­age it can af­ter cap­tur­ing all the neoanti­gens in the can­cer cell.

The Avro­Bio team is work­ing with two sci­en­tif­ic co-founders, Jef­frey Medin, who un­til re­cent­ly was a pro­fes­sor at the Uni­ver­si­ty of Toron­to along with Christo­pher Paige. Avro­Bio is based in Cam­bridge, home turf to MacK­ay, with a fa­cil­i­ty in Toron­to where it can re­main close to its sci­en­tif­ic home.

At­las Ven­ture, Clarus and SV Life Sci­ences co-led the A-round, con­tribut­ing their part­ners to the board. Bruce Booth at At­las is sign­ing on as chair­man while Scott Re­quadt, a man­ag­ing di­rec­tor at Clarus, and Josh Resnick at SV Life Sci­ences join the board.

Right now, the team con­sists of a core group of 8, which MacK­ay says is set to grow.

Grow­ing ac­cep­tance of ac­cel­er­at­ed path­ways for nov­el treat­ments: but does reg­u­la­to­ry ap­proval lead to com­mer­cial suc­cess?

By Mwango Kashoki, MD, MPH, Vice President-Technical, and Richard Macaulay, Senior Director, of Parexel Regulatory & Access

In recent years, we’ve seen a significant uptake in the use of regulatory options by companies looking to accelerate the journey of life-saving drugs to market. In 2018, 73% of the novel drugs approved by the U.S. Federal Drug Administration (FDA) were designated under one or more expedited development program categories (Fast Track, Breakthrough Therapy, Priority Review, and Accelerated Approval).ᶦ

Sanofi out­lines big API plans as coro­n­avirus out­break re­port­ed­ly threat­ens short­age of 150 drugs

As the world becomes increasingly dependant on Asia for the ingredients of its medicines, Sanofi sees business to be done in Europe.

The French drugmaker said it’s creating the world’s second largest active pharmaceutical ingredients (API) manufacturer by spinning out its six current sites into a standalone company: Brindisi (Italy), Frankfurt Chemistry (Germany), Haverhill (UK), St Aubin les Elbeuf (France), Újpest (Hungary) and Vertolaye (France). They have mapped out €1 billion in expected sales by 2022 and 3,100 employees for the new operations headquartered in France.

Bio­gen touts new ev­i­dence from the gene ther­a­py com­pa­ny it wa­gered $800M on

A year ago, Biogen made a big bet on a small gene therapy company. Now they have new evidence one of their therapies could work.

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Anthony Fauci (AP Images)

UP­DAT­ED: NIH-part­nered Mod­er­na ships off its PhI-ready coro­n­avirus vac­cine can­di­date to a sea of un­cer­tain­ty

Off it goes.

Moderna has shipped the first batch of its mRNA vaccine against SARS-CoV-2 from its manufacturing facility in Norwood, Massachusetts, to the National Institute of Allergy and Infectious Diseases in Bethesda, Maryland, for a pioneering Phase I study.

It’s a hectic race against time. In the 42 days since Moderna selected the sequence they would use to develop their vaccine — a record time, no less — the number of confirmed cases around the world has surged astronomically from a few dozen to over 80,000, per WHO and Johns Hopkins estimates.

The candidate that they came up with, mRNA-1273, encodes for a prefusion stabilized form of the spike protein, which gives the virus its crown shape and plays a key role in transmission. The Coalition for Epidemic Preparedness Innovations, the Oslo-based group better known as CEPI, funded the manufacture of this batch.

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In fi­nal re­port, ICER ap­pears to have a change of heart on new acute mi­graine ther­a­pies

ICER appears to have reversed course on the fresh crop of acute migraine therapies.

The cost-effectiveness watchdog in November issued a draft report suggesting that existing generic medicines are more effective and cheaper than Allergan’s December-approved CGRP ubrogepant, Biohaven rival molecule, rimegepant (which is under FDA review), and Lilly’s October-sanctioned lasmiditan, which binds to 5-HT1F receptors.

Bi­cy­cle Ther­a­peu­tics takes Roche's Genen­tech on an up to $2B im­muno-on­col­o­gy ride

Bicycle Therapeutics — which is developing a new class of chemically synthesized drugs designed to be pharmacologically as active as biologics, yet manufactured as small molecules —  has scored another big partner: Roche’s Genentech.

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When drug val­ue as­sess­ment meets re­al-world ev­i­dence: ICER en­lists Ae­tion in pric­ing eval­u­a­tion

In a union of two of the hottest trends in the US biopharma world, ICER is teaming up with a high-profile company to integrate real-world evidence in their assessment of treatment value.

The drug pricing watchdog — formally the Institute for Clinical and Economic Review — said it will utilize Aetion’s evidence platform in “select upcoming assessments” and their new 24-month re-evaluations of drugs granted accelerated approval by the FDA.

Anthony Fauci, AP Images

First US Covid-19 tri­als set to get un­der­way in Ne­bras­ka and Wash­ing­ton, backed by NIH

The first US clinical trials on the novel coronavirus are scheduled to get underway next month at the University of Nebraska Medical Center, where American passengers were taken after being evacuated from the Diamond Princess cruise ship, and at the Kaiser Permanente Washington Health Research Institute. Both trials are sponsored by the NIH’s National Institute for Allergy and Infectious Diseases, which has led the US’s medical response to the outbreak.

UP­DAT­ED: NGM Bio takes leap for­ward in crowd­ed NASH field

South San Francisco-based NGM Bio may have underwhelmed with its interim analysis of a key cohort from a mid-stage NASH study last fall — but stellar topline data unveiled on Monday showed the compound induced significant signs of antifibrotic activity, NASH resolution and liver fat reduction, sending the company’s stock soaring.

There are an estimated 50+ companies focused on developing drugs for non-alcoholic steatohepatitis, or NASH, a common liver disease that has long flummoxed researchers. The first wave of NASH drug developers struggled with efficacy as well as safety — and companies big and small have crashed and burned.

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