Third Rock, GV back Broad spin­out Cel­sius in bid to de­vel­op pre­ci­sion meds for au­toim­mune dis­ease

Third Rock is team­ing up with Google’s ven­ture arm to back a gene ther­a­py start­up step­ping out to­day with $65 mil­lion in new mon­ey. As you might ex­pect with GV’s in­volve­ment, the new ven­ture has a tech­nol­o­gy twist, and it might have its hands on some­thing big for the au­toim­mune world.

Vi­jay Kuchroo

Are you ready for a game of buzz­word bin­go? The start­up, called Cel­sius Ther­a­peu­tics, is us­ing new tech in ge­net­ic se­quenc­ing (check) — and a pro­pri­etary ma­chine learn­ing (check) al­go­rithm (check) — to tack­le pre­ci­sion meds (check) for can­cer (check) and au­toim­mune dis­ease. I’ll break that down for you lat­er.

The tech­nol­o­gy the com­pa­ny is built on was li­censed from the Broad In­sti­tute based on the work of Aviv Regev and Vi­jay Kuchroo (both found­ing sci­en­tists at Cel­sius), in­clud­ing non-ex­clu­sive li­cens­es to sin­gle-cell se­quenc­ing tech and ex­clu­sive li­cens­es to ear­ly-stage drug pro­grams.

The com­pa­ny’s co-founder and pres­i­dent, Christoph Lengauer (a Third Rock ven­ture part­ner), tells me Cel­sius has a few things that give it an edge: a mas­sive amount of an­i­mal and hu­man da­ta to work with, a ma­chine-learn­ing al­go­rithm to make sense of that da­ta, and a new tech­nol­o­gy to se­quence sin­gle cells rather than whole genomes.

Our body has tril­lions of cells, each with ge­net­ic in­for­ma­tion stored in­side. The sets of genes vary in dif­fer­ent cell types, de­ter­min­ing a cell’s func­tion — and some­times — the code be­hind dis­ease. Hom­ing in on sin­gle cells could help re­searchers bet­ter un­der­stand the in­di­vid­ual cells and their in­ter­ac­tions that cause dis­ease, per­haps lead­ing to bet­ter ther­a­pies down the road.

When com­par­ing sin­gle cell se­quenc­ing with whole genome se­quenc­ing, Lenguaer used an anal­o­gy about mak­ing smooth­ies with fruit.

“If you put straw­ber­ries and ki­wis in a blender, the col­or of your smooth­ie will be pink be­cause it’s the dom­i­nant cell pop­u­la­tion,” Lengauer said. “Any­thing re­lat­ed to the ki­wi would be lost. That’s whole genome se­quenc­ing. With this anal­o­gy, sin­gle cell se­quenc­ing would al­low you to see the in­di­vid­ual fruits in the smooth­ie. And if some­thing was rot­ten, you could pin­point the in­di­vid­ual fruit that’s gone bad.”

Of course, we’re not talk­ing of straw­ber­ries and ki­wis, but of cells that are caus­ing dis­ease and the genes that trig­ger their mal­func­tion. Cel­sius be­lieves this tech could be the key to bring pre­ci­sion med­i­cines to au­toim­mune dis­eases for the first time.

“Many dis­eases are dri­ven by the com­bined dys­func­tion of sev­er­al spe­cif­ic cell types, and the in­ter­ac­tions be­tween them,” said Regev, the MIT pro­fes­sor who co-found­ed Cel­sius, in a state­ment. “With tra­di­tion­al ge­nom­ic se­quenc­ing, we can­not iden­ti­fy these in­di­vid­ual con­tri­bu­tions — we on­ly see the av­er­age and can miss out key crit­i­cal caus­es. But for the first time, with the ap­proach­es dis­cov­ered by our team, where we com­bine mas­sive datasets of un­prece­dent­ed size and com­plex­i­ty with so­phis­ti­cat­ed ma­chine learn­ing al­go­rithms, we are able to dis­tin­guish the spe­cif­ic cells, among many oth­ers, that play a key role in dis­ease and iden­ti­fy the genes that are trig­ger­ing their mal­func­tion. We be­lieve our ap­proach will al­low us to more ef­fi­cient­ly iden­ti­fy spe­cif­ic tar­gets for treat­ing dis­eases in spe­cif­ic pa­tients and ul­ti­mate­ly de­vel­op med­i­cines for those tar­gets.”

Us­ing this ap­proach, the Broad In­sti­tute has un­cov­ered “a hand­ful” of tar­gets in au­toim­mune con­di­tions and can­cer. Lengauer said Cel­sius hopes to reach proof of con­cept in the next five years, and the Se­ries A should take them at least part­way to­ward that goal.

While Third Rock led the re­cent round, GV par­tic­i­pat­ed along with Her­itage Provider Net­work, Cas­din Cap­i­tal, Alexan­dria Ven­ture In­vest­ments and oth­ers.

The com­pa­ny, which has been op­er­at­ing in stealth mode for the past two years, em­ploys 15 peo­ple at its Cam­bridge head­quar­ters in Kendall Square.

Im­age: Aviv Regev, Third Rock part­ner Alex­is Borisy, Christoph Lengauer. Cel­sius Ther­a­peu­tics

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

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Lilly in-licensed global rights to sintilimab a year ago, building on the China alliance they have with Innovent. That cost the pharma giant $200 million in cash upfront, which they plan to capitalize on now with a long-awaited plan to bust up the high-price market in lung cancer and other cancers that have created a market worth tens of billions of dollars.

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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.

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Div­ing deep­er in­to in­her­it­ed reti­nal dis­or­ders, No­var­tis gob­bles up an­oth­er bite-sized op­to­ge­net­ics biotech

Right about a year ago, a Novartis team led by Jay Bradner and Cynthia Grosskreutz at NIBR swooped in to scoop up a Cambridge, MA-based opthalmology gene therapy company called Vedere. Their focus was on a rather narrow market niche: inherited retinal dystrophies that include a wide range of genetic retinal disorders marked by the loss of photoreceptor cells and progressive vision loss.

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The FDA on Monday handed an accelerated approval to Seagen and Genmab’s Tivdak (tisotumab vedotin-tftv, or “TV”) in second-line patients with recurrent or metastatic cervical cancer who previously progressed after chemotherapy rather than PD-(L)1 systemic therapy, the companies said in a release.

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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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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.

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

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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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