Dan Siegwart (UT Southwestern)

Cou­pling mR­NA with tR­NA and new de­liv­ery tech, MPM and Or­biMed help birth new $80M biotech in­to a slowed down world

Part­ners at MPM and Or­biMed were hop­ing David Lock­hart could help them with due dili­gence when they brought him and his team at Tran­scrip­Tx in to look at a tiny up­start spun out of the Uni­ver­si­ty of Texas South­west­ern Med­ical Cen­ter in Dal­las. Dubbed Re­Code Ther­a­peu­tics, it was de­vel­op­ing a lipid nanopar­ti­cle (LNP) de­liv­ery plat­form for var­i­ous ge­net­ic med­i­cines, with a lead pro­gram in cys­tic fi­bro­sis backed by the CF Foun­da­tion.

But Lock­hart, the for­mer CSO of Am­i­cus Ther­a­peu­tics and a one-time biotech en­tre­pre­neur, saw an im­me­di­ate match with the mR­NA and res­pi­ra­to­ry dis­ease work that Tran­scrip­Tx was do­ing. Since his com­pa­ny was al­so look­ing to raise mon­ey, wouldn’t it make sense to merge the two?

So in­stead of two Se­ries B rounds, MPM and Or­biMed have now put their mon­ey be­hind a brand new biotech that’s tak­ing the Re­Code name and $80 mil­lion in Se­ries A cash to hus­tle to­ward the clin­ic and get se­ri­ous about BD — al­beit at a slight­ly slow­er pace amid a pan­dem­ic.

“We closed the merg­er and the fi­nanc­ing on March 6,” Lock­hart told End­points News. “Even though in some ways that wasn’t very long ago, the world was kind of in a dif­fer­ent place even three weeks ago.”

Colt Ven­tures co-led the round with Or­biMed, with par­tic­i­pa­tion from Vi­da Ven­tures, Hunt Tech­nol­o­gy Ven­tures and Os­age Uni­ver­si­ty Part­ners.

The aca­d­e­m­ic labs at UT South­west­ern, where founders Dan Sieg­wart and Philip Thomas are based, have been shut down. With most of the staff work­ing from home, the CEO of­ten finds him­self work­ing in an emp­ty of­fice in Men­lo Park by him­self. Re­Code doesn’t plan to pull the trig­ger on any new hires in the next few weeks.

What they are do­ing, though, is lay­ing the ground­work so that they have every­thing lined up to go at full speed once they can, from pre­clin­i­cal ex­per­i­ments to job can­di­dates. The sci­en­tists who are tend­ing to cru­cial cell lines are al­so still at work while keep­ing their dis­tance, which is rel­a­tive­ly easy with a team of 12 in Cal­i­for­nia and just 3 in Texas.

The orig­i­nal lead pro­gram at Tran­scrip­Tx was de­signed to treat the rare ge­net­ic dis­or­der pri­ma­ry cil­iary dysk­i­ne­sia (PCD) us­ing an mR­NA drug de­liv­ered by an LNP li­censed ear­li­er. While Re­Code is mov­ing for­ward with it, the team will al­so test oth­er can­di­dates us­ing the pro­pri­etary plat­form in par­al­lel. If the re­sults pan out, Re­Code will like­ly swap it out, Lock­hart said.

Not on­ly is the class of LNPs in­vent­ed by Sieg­wart — who stud­ied un­der MIT whiz Robert Langer — eas­i­er to make and ver­sa­tile in the pay­load they can car­ry, they can al­so be de­liv­ered sys­tem­at­i­cal­ly then dis­trib­uted to dif­fer­ent tis­sue types.

Philip Thomas

There are no ap­proved treat­ments for PCD, and as with CF be­fore Ver­tex’s ther­a­pies came along, pa­tients can die of res­pi­ra­to­ry fail­ure in their 30s or 40s even on the best sup­port­ive care. And sev­er­al ge­net­ic mu­ta­tions are known to cause the dis­ease.

“PCD alone is a pipeline that will be big­ger than the Gen­zyme pipeline that made Gen­zyme what it was,” Lock­hart said, cit­ing the rare dis­ease gi­ant that Am­i­cus was of­ten com­pared to.

In CF, Ver­tex has made dra­mat­ic break­throughs with its ar­se­nal of drugs tar­get­ing mu­tant CFTR pro­teins, cov­er­ing 90% of pa­tients. Re­Code’s goal is to catch the oth­er 10%: those with a non­sense mu­ta­tion that pre­vents a full length pro­tein from be­ing pro­duced in the first place. With the LNP tech, its ther­a­py ush­ers in a piece of trans­fer RNA — a bridge be­tween mes­sen­ger RNA and pro­tein syn­the­sis — to the pre­ma­ture stop codon and al­ters the cell ma­chin­ery, coax­ing it to make a func­tion­al ver­sion of CFTR.

PTC Ther­a­peu­tics has tak­en a sim­i­lar read-through ap­proach with its small mol­e­cule drug, but while ataluren has demon­strat­ed (con­tro­ver­sial) ef­fect in Duchenne mus­cu­lar dy­s­tro­phy, it failed in CF.

“They’re ac­tu­al­ly sort of sis­ter dis­eases in some ways,” he added. “The same doc­tors that treat PCD treat peo­ple with CF.”

Be­yond these ini­tial pro­grams, for which clin­i­cal tri­als are still ex­pect­ed in 2021, there’s plen­ty of room for li­cens­ing and col­lab­o­ra­tions. It was a key top­ic for the new board of di­rec­tors, now com­pris­ing Or­biMed’s Pe­ter Thomp­son, MPM’s Ed Hur­witz, Vi­da Ven­tures’ He­len Kim, R.A. Ses­sion from Bridge­Bio and Michael Tor­res, Re­Code founder and new VP of R&D.

“We had our first board meet­ing with the new board just on Fri­day,” Lock­hart said. “Every­one was in a room by them­selves talk­ing in­to a phone.”

Tesla and SpaceX founder Elon Musk gestures to the audience after being recognized by President Trump following the successful launch of a Falcon 9 rocket at the Kennedy Space Center. (via Getty Images)

Tes­la chief Elon Musk teams up with Covid-19 play­er Cure­Vac to build 'R­NA mi­cro­fac­to­ries'

Elon Musk has joined the global tech crusade now underway to revolutionize vaccine manufacturing — now aimed at delivering billions of doses of a new mRNA vaccine to fight Covid-19. And he’s cutting right to the front.

In a late-night tweet Wednesday, the Tesla chief announced:

Tesla, as a side project, is building RNA microfactories for CureVac & possibly others.

That’s not a lot to go on. But the tweet comes a year after Tesla’s German division in Grohmann and CureVac filed a patent on a “bioreactor for RNA in vitro transcription, a method for RNA in vitro transcription, a module for transcribing DNA into RNA and an automated apparatus for RNA manufacturing.” CureVac, in the meantime, has discussed a variety of plans to build microfactories that can speed up the whole process for a global supply chain.

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George Yancopoulos (Regeneron)

UP­DAT­ED: Re­gen­eron co-founder George Yan­copou­los of­fers a com­bat­ive de­fense of the po­lice at a high school com­mence­ment. It didn’t go well

Typically, the commencement speech at Yorktown Central School District in Westchester — like most high schools — is an opportunity to encourage students to face the future with confidence and hope. Regeneron president and co-founder George Yancopoulos, though, went a different route.

In a fiery speech, the outspoken billionaire defended the police against the “prejudice and bias against law enforcement” that has erupted around the country in street protests from coast to coast. And for many who attended the commencement, Yancopoulos struck the wrong note at the wrong time, especially when he combatively challenged someone for interrupting his speech with a honk for “another act of cowardness.”

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Elias Zerhouni (Photo by Vincent Isore/IP3/Getty Images)

Elias Zer­houni dis­cuss­es ‘am­a­teur hour’ in DC, the de­struc­tion of in­fec­tious dis­ease R&D and how we need to prep for the next time

Elias Zerhouni favors blunt talk, and in a recent discussion with NPR, the ex-Sanofi R&D and ex-NIH chief had some tough points to make regarding the pandemic response.

Rather than interpret them, I thought it would be best to provide snippets straight from the interview.

On the Trump administration response:

It was basically amateur hour. There is no central concept of operations for preparedness, for pandemics, period. This administration doesn’t want to or has no concept of what it takes to protect the American people and the world because it is codependent. You can’t close your borders and say, “OK, we’re going to be safe.” You’re not going to be able to do that in this world. So it’s a lack of vision, basically just a lack of understanding, of what it takes to protect the American people.

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Sec­ond death trig­gers hold on Astel­las' $3B gene ther­a­py biotech's lead pro­gram, rais­ing fresh con­cerns about AAV

Seven months after Astellas shelled out $3 billion to acquire the gene therapy player Audentes, the biotech company’s lead program has been put on hold following the death of 2 patients taking a high dose of their treatment. And there was another serious adverse event recorded in the study as well, with a total of 3 “older” patients in the study affected.

The incidents are derailing plans to file for a near-term approval, which had been expected right about now.

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Douglas Love, Annexon CEO (Annexon)

IPO bound? A Bay Area biotech grabs a mega-round on the road to a piv­otal neu­rode­gen­er­a­tion pro­gram

South San Francisco-based Annexon has added $100 million to its cash reserves, along with a new roster of marquee investors backing their play on the classical complement pathway involved in neurodegeneration. And that may well fit the profile for an IPO — though right now everything seems to be working on that score.

Eighteen months after Bain and their syndicate partners put up $75 million to fuel clinical work, Annexon is back at the trough. And this time they’re adding Redmile Group for the lead role, with supporting investments from these new arrivals: BlackRock, Deerfield Management Company, Eventide Asset Management, Farallon Capital Management, Janus Henderson Investors and Logos Capital.

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Pfiz­er shares surge on pos­i­tive im­pact of their mR­NA Covid-19 vac­cine — part­nered with BioN­Tech — in an ear­ly-stage study

Pfizer and their partners at the mRNA specialist BioNTech have published the first glimpse of biomarker data from an early-stage study spotlighting the “robust immunogenicity” triggered by their Covid-19 vaccine, which is one of the leaders in the race to vanquish the global pandemic.

Researchers selected 45 healthy volunteers 18-55 years of age for the study. They were randomized to receive 2 doses, separated by 21 days, of 10 µg, 30 µg, or 100 µg of BNT162b1, “a lipid nanoparticle-formulated, nucleoside-modified, mRNA vaccine that encodes trimerized SARS-CoV-2 spike glycoprotein RBD.” Their responses were compared against the effect of a natural, presumably protective defense offered by a regular infection.

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Randy Schatzman, Bolt CEO (Bolt Biotherapeutics)

Bolt Bio­ther­a­peu­tics nabs $93.5M to push Provenge in­ven­tor's new idea deep­er in the clin­ic

A cancer-fighting concept from the inventor of the first cancer vaccine is nearing prime time, and its biotech developer has received a significant new infusion of cash to get it there.

Bolt Biotherapeutics announced a $93.5 million Series C round led by Sofinnova Investments and joined by more than 9 others, including Pfizer Ventures and RA Capital Management. That money will go toward pushing the San Francisco biotech’s platform of innate immune-boosting warheads through its first trial on metastatic solid tumors and into several more.

Josh Cohen, Justin Klee

Armed with pos­i­tive ALS da­ta, Amy­lyx scores $30M in fresh fund­ing to com­plete Alzheimer's PhII

Four years after announcing themselves to the biotech world with a new idea for drugging neurodegeneration, backing by the late Henri Termeer and $5 million from Morningside Venture, the young entrepreneurs at Amylyx are back for round 2.

Morningside continued to lead the $30 million Series B, with participation from Termeer’s widow, Belinda, and other unnamed investors. Having celebrated a topline Phase II win for its lead program in amyotrophic lateral sclerosis, Amylyx expects the cash to fund talks with regulators as well as a separate trial for the same drug in Alzheimer’s — for which they had just finished enrolling.

An ex­pe­ri­enced biotech is stitched to­geth­er from transpa­cif­ic parts, with 265 staffers and a fo­cus on ‘new bi­ol­o­gy’

Over the past few years, different teams at a pair of US-based biotechs and in labs in Japan have labored to piece together a group of cancer drug programs, sharing a single corporate umbrella with research colleagues in Japan. But now their far-flung operations have been knit together into a single unit, creating a pipeline with 10 cancer drug development programs — going from early-stage right into Phase III — and a host of discovery projects managed by a collective staff of some 265 people.

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