Brendan Frey (Deep Genomics)

Deep Ge­nomics, now flush with cash, plans to take dozens of RNA ther­a­pies to the clin­ic

It was 2002 when Bren­dan Frey no­ticed a huge gap in biotech. The hu­man genome had just been se­quenced, al­low­ing sci­en­tists to map ge­net­ic mu­ta­tions. But there weren’t enough da­ta to un­der­stand the con­se­quences of those mu­ta­tions, or re­al­ly do much about them.

Pre­dict­ing there would be an ex­plo­sion of new da­ta, Frey spent the next 13 years work­ing on a way to sift through it all. Now, thanks to ad­vances in RNA ther­a­peu­tics, med­i­cine is be­com­ing pro­gram­ma­ble, Frey said. And on Wednes­day, a slate of in­vestors bet $180 mil­lion that his com­pa­ny’s AI plat­form can make sense of it.

“What’s re­al­ly cool about RNA ther­a­peu­tics is that they’re ba­si­cal­ly a se­quence of let­ters,” Frey said. “Change the se­quence of let­ters one way, you tar­get a dif­fer­ent gene. Change it one way, you can in­crease the amount of pro­tein pro­duced by that gene. Change the se­quence of let­ters a dif­fer­ent way, you can de­crease the pro­tein.”

It’s all dig­i­tal in­for­ma­tion, Frey said. And thanks to AI and deep learn­ing tools, Deep Ge­nomics says it can do things like fig­ure out which mech­a­nisms of ac­tion will (or won’t) work against a spe­cif­ic gene, with­out per­form­ing a sin­gle ex­per­i­ment.

“We can take a gene where an­oth­er com­pa­ny would have spent two years on it and then failed and dropped it, and we can ac­tu­al­ly drug that gene, or we know to put it at the bot­tom of the list, just don’t do it now, it’s go­ing to be too hard,” he said.

In 2019, the com­pa­ny put for­ward its first pre­clin­i­cal can­di­date, a ther­a­py for Wil­son dis­ease that’s ex­pect­ed to hit the clin­ic along with three oth­er can­di­dates by 2023. Us­ing the AI sys­tem, the team says it was able to go from tar­get iden­ti­fi­ca­tion to de­clar­ing a win­ner in 18 months. Deep Ge­nomics has a to­tal of 10 can­di­dates hurtling to­ward the clin­ic, and Frey says he ex­pects to add 20 more in the near fu­ture.

The oth­er three can­di­dates ex­pect­ed to hit the clin­ic by 2023 are for fron­totem­po­ral de­men­tia, gout and Nie­mann-Pick type C dis­ease.

The AI space is teem­ing with play­ers, like Enve­da, which nabbed a $51 mil­lion Se­ries A round last month to pur­sue new ther­a­pies for Wil­son dis­ease, NASH and Parkin­son’s dis­ease. Up­on pulling in a $225 mil­lion Se­ries C round last month, In­sil­i­co CEO Alex Zha­voronkov laid out big plans to emerge as the Ama­zon or Google of the field. Around the same time, UK-based Ex­sci­en­tia splurged on the three-year-old mol­e­cule screen­ing biotech All­cyte in an at­tempt to edge out ri­vals.

What sep­a­rates Deep Ge­nomics from some of its peers — like Re­cur­sion, Ex­sci­en­tia or in­sitro — is its sole fo­cus on RNA bi­ol­o­gy, Frey said.

“We like RNA bi­ol­o­gy be­cause of that rock sol­id frame­work,” he said. “We have 100 petabytes of da­ta, so every­thing’s in place.”

Frey says the AI tech is less like a grand, all-know­ing com­put­er, and more like a work­bench, with dozens of tools that have de­fined func­tions and scopes. For ex­am­ple, one tool was built to go through data­bas­es of pa­tient mu­ta­tions and find drug tar­gets based on RNA bi­ol­o­gy — but re­searchers at the com­pa­ny re­al­ized it could al­so be used to an­a­lyze dif­fer­ent types of an­i­mal mod­els and fig­ure out which ones would re­ca­pit­u­late hu­man bi­ol­o­gy.

“That’s kind of the ad­van­tage of the work­bench metaphor is it sort of frees peo­ple up to be more cre­ative,” Frey said.

Soft­bank Vi­sion Fund 2 led the Se­ries C round, with a hand from Fi­deli­ty Man­age­ment & Re­search Com­pa­ny, Cana­di­an Pen­sion Plan In­vest­ment Board, True Ven­tures, Am­pli­tude Ven­tures, Khosla Ven­tures and Mag­net­ic Ven­tures. When asked if an IPO is in the near fu­ture, Frey said he doesn’t plan to take the com­pa­ny pub­lic at least un­til they reach the clin­ic.

“A lot of com­pa­nies have gone pub­lic pre­clin­i­cal­ly in the last year, and the prob­lem is that if they stum­ble in get­ting in­to the clin­ic, then the ex­is­tence of the com­pa­ny will be put in­to ques­tion,” he said. “We don’t want to be in that sit­u­a­tion.”

Health­care Dis­par­i­ties and Sick­le Cell Dis­ease

In the complicated U.S. healthcare system, navigating a serious illness such as cancer or heart disease can be remarkably challenging for patients and caregivers. When that illness is classified as a rare disease, those challenges can become even more acute. And when that rare disease occurs in a population that experiences health disparities, such as people with sickle cell disease (SCD) who are primarily Black and Latino, challenges can become almost insurmountable.

David Meek, new Mirati CEO (Marlene Awaad/Bloomberg via Getty Images)

Fresh off Fer­Gene's melt­down, David Meek takes over at Mi­rati with lead KRAS drug rac­ing to an ap­proval

In the insular world of biotech, a spectacular failure can sometimes stay on any executive’s record for a long time. But for David Meek, the man at the helm of FerGene’s recent implosion, two questionable exits made way for what could be an excellent rebound.

Meek, most recently FerGene’s CEO and a past head at Ipsen, has become CEO at Mirati Therapeutics, taking the reins from founding CEO Charles Baum, who will step over into the role of president and head of R&D, according to a release.

Dave Lennon, former president of Novartis Gene Therapies

Zol­gens­ma patent spat brews be­tween No­var­tis and Re­genxbio as top No­var­tis gene ther­a­py ex­ec de­parts

Regenxbio, a small licensor of gene therapy viral vectors spun out from the University of Pennsylvania, is now finding itself in the middle of some major league patent fights.

In addition to a patent suit with Sarepta Therapeutics from last September, Novartis, is now trying to push its smaller partner out of the way. The Swiss biopharma licensed Regenxbio’s AAV9 vector for its $2.1 million spinal muscular atrophy therapy Zolgensma.

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Rafaèle Tordjman (Jeito Capital)

Con­ti­nu­ity and di­ver­si­ty: Rafaèle Tord­j­man's women-led VC firm tops out first fund at $630M

For a first-time fund, Jeito Capital talks a lot about continuity.

Rafaèle Tordjman had spotlighted that concept ever since she started building the firm in 2018, promising to go the extra mile(s) with biotech entrepreneurs while pushing them to reach patients faster.

Coincidentally, the lack of continuity was one of the sore spots listed in a report about the European healthcare sector published that same year by the European Investment Bank — whose fund is one of the LPs, alongside the American pension fund Teacher Retirement System of Texas and Singapore’s Temasek, to help Jeito close its first fund at $630 million (€534 million). As previously reported, Sanofi had chimed in €50 million, marking its first investment in a French life sciences fund.

Time for round 2: Il­lu­mi­na-backed VC snags $325M for its next fund

Illumina Ventures closed off its second investment fund with a total commitment of $325 million, offering fresh fuel to back a slate of startups that have already included a smorgasbord of companies, covering everything from diagnostics to biotech drug development and genomics.

Fund II brings the total investment under Illumina Ventures’ oversight to $560 million, which has been focused on early-stage companies. And it has a transatlantic portfolio that includes SQZ, Twist and Encoded Therapeutics.

Volker Wagner (L) and Jeff Legos

As Bay­er, No­var­tis stack up their ra­dio­phar­ma­ceu­ti­cal da­ta at #ES­MO21, a key de­bate takes shape

Ten years ago, a small Norwegian biotech by the name of Algeta showed up at ESMO — then the European Multidisciplinary Cancer Conference 2011 — and declared that its Bayer-partnered targeted radionuclide therapy, radium-223 chloride, boosted the overall survival of castration-resistant prostate cancer patients with symptomatic bone metastases.

In a Phase III study dubbed ALSYMPCA, patients who were treated with radium-223 chloride lived a median of 14 months compared to 11.2 months. The FDA would stamp an approval on it based on those data two years later, after Bayer snapped up Algeta and christened the drug Xofigo.

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Ex­elix­is pulls a sur­prise win in thy­roid can­cer just days ahead of fi­nal Cabome­tyx read­out

Exelixis added a thyroid cancer indication to its super-seller Cabometyx’s label on Friday — months before the FDA was expected to make a decision, and days before the company was set to unveil the final data at #ESMO21.

At a median follow-up of 10.1 months, differentiated thyroid cancer patients treated with Cabometyx (cabozantinib) lived a median of 11 months without their disease worsening, compared to just 1.9 months for patients given a placebo, Exelixis said on Monday.

Den­mark's Gubra to col­lab­o­rate with Bay­er on pep­tides; Sam­sung and Bio­gen re­ceive FDA ap­proval for Lu­cen­tis biosim­i­lar

Danish biotech Gubra announced a research collaboration and license agreement with Bayer to develop peptide therapeutics to treat cardiorenal diseases. The collaboration will utilize Gubra’s peptide drug discovery platform to identify potential candidates.

This is not the first time Gubra has partnered with a company on peptide therapeutics — they partnered with Boehringer Ingelheim back in 2017 to create peptide therapeutics to treat obesity.

Raju Mohan, Ventyx Biosciences CEO

Months af­ter a mam­moth raise, Ven­tyx Bio­sciences dips back in­to ven­ture well

Several months after emerging from what CEO Raju Mohan called “quiet mode” with a mammoth $114 million raise, Ventyx Biosciences is now making its plans for the clinic loud and clear.

The California-based immune modulation player kicked the week off with a $51 million Series B, while also naming some key hires ahead of its big clinical push.

The CMO slot is going to Jörn Drappa, former CMO at Viela Bio before it was bought out by Horizon Therapeutics earlier this year. The AstraZeneca vet stayed on at Horizon for a while as executive VP of R&D before making the jump to Ventyx.