Kevin Friedman, Kelonia president and CSO

Gene ther­a­py out­fit emerges from stealth with big am­bi­tions. First stop — CAR-T

There’s a new play­er in the gene ther­a­py space in Boston.

Backed by sci­ence out of Stan­ford and MIT, the small out­fit has big dreams, big am­bi­tions, and is cen­tered on try­ing to turn ex vi­vo gene ther­a­py in­to in vi­vo. How­ev­er, it’s start­ing with look­ing to re­de­fine CAR-T.

Kelo­nia Ther­a­peu­tics, backed by VCs such as Hori­zons Ven­tures and Ven­rock, of­fi­cial­ly launched Thurs­day af­ter be­ing in stealth for a lit­tle over a year, and has a bit of a cash stash thanks to $50 mil­lion from a Se­ries A. Pres­i­dent and CSO Kevin Fried­man told End­points News that the biotech has enough run­way to “progress us quite a ways to­ward the clin­ic” with­out giv­ing an ex­act time­frame.

Its sci­en­tif­ic co-founders in­clude pro­fes­sors Michael Birn­baum out of MIT, along with ge­neti­cist and chemist Michael Fis­chbach from Stan­ford. Some of Fis­chbach’s re­search has been uti­lized be­fore in biotech-build­ing — name­ly Fed­er­a­tion Bio, an out­fit in the mi­cro­bio­me are­na that Ven­rock in­vest­ed $50 mil­lion in­to two years ago.

Fried­man billed the com­pa­ny as a new play­er try­ing to ad­dress big hur­dles in the cell and gene ther­a­py game. In the realm of T cell im­munother­a­py, sci­en­tists take T cells from a pa­tient, mod­i­fy them and then in­ject those same cells back in­to the orig­i­nal pa­tient. CAR-T, a spe­cif­ic type of T cell im­munother­a­py, is usu­al­ly im­ple­ment­ed against cer­tain types of can­cers.

In re­cent years, a lot of com­pa­nies have been look­ing in­to al­lo­genic (or “off-the-shelf”) CAR-T ther­a­pies, look­ing to be able to use T cells from donors oth­er than the pa­tient. And with Kelo­nia, the biotech is look­ing to re­de­fine CAR-T by by­pass­ing the more in­di­vid­u­al­ized cell man­u­fac­tur­ing al­to­geth­er, es­sen­tial­ly be­ing able to de­liv­er a drug/gene ther­a­py that will mod­i­fy T cells in­side the pa­tient.

“And so, es­sen­tial­ly har­ness­ing all of the clin­i­cal ben­e­fits that we’ve seen with CAR and TCR ther­a­pies in terms of how the pa­tients have re­spond­ed to this type of med­i­cine, and elim­i­nat­ing the com­plex­i­ty of the man­u­fac­tur­ing process by tran­si­tion­ing the de­liv­ery di­rect­ly to the pa­tient it­self,” Fried­man said.

Kelo­nia’s pro­posed in vi­vo gene de­liv­ery tech­nol­o­gy will com­bine a few po­tent lentivi­ral vec­tor-like par­ti­cles with an “ad­justable tar­get­ing sys­tem” to more pre­cise­ly de­liv­er gene ther­a­py pay­loads to where the ther­a­py is need­ed — in or­der to treat a broad range of dis­eases. And while it’s big ideas, oth­er star­tups have made sim­i­lar promis­es, and many are look­ing to chart their own path. One such ex­am­ple is Umo­ja Bio­phar­ma, a Seat­tle com­pa­ny which launched in 2020 with $53 mil­lion to in­ves­ti­gate us­ing a lentivi­ral vec­tor to gen­er­ate CAR-T cells.

The po­ten­tial op­por­tu­ni­ties for gene ther­a­py 2.0 have even at­tract­ed the at­ten­tion of Big Phar­ma when Bris­tol My­ers, Astel­las, NI­BR and Gilead’s Kite all backed Or­na Ther­a­peu­tics’ Se­ries A last year, af­ter the biotech launched to ex­plore next-gen gene ther­a­py via oR­NA. Last Au­gust, Mus­tang Bio made a deal with Lar­ry Pease’s lab out of the Mayo Clin­ic for its CAR-T tech, ex­plic­it­ly de­signed to form CAR-T cells in­side the pa­tient via two steps: a pep­tide to ramp up T cell pro­duc­tion, then di­rect­ly in­tro­duc­ing a vi­ral CAR con­struct in­to a pa­tient’s lymph nodes.

Kelo­nia has around 20 em­ploy­ees so far, but will ex­pand in due time, Fried­man said. In the in­ter­im, the com­pa­ny has a straight­for­ward goal of a mad dash to­wards the clin­ic, and they have no plans on stop­ping just with CAR-T, or their ini­tial fo­cus on on­col­o­gy. But as to what in­di­ca­tions, de­tails re­main few — but Kelo­nia did say in a state­ment that it hopes to use its tech­nol­o­gy to en­able gene ther­a­py de­liv­ery in­to tis­sues pre­vi­ous­ly known as hard to reach, such as re­nal and neu­ro­log­i­cal tis­sues.

Kelo­nia al­so has two strate­gic part­ner­ships right out of the gate. One is with Till­man Gern­gross’ Adimab, and the oth­er is the Cam­bridge, MA-based biotech El­e­vate­Bio. Fried­man said that these two part­ner­ships will pro­vide im­por­tant ca­pa­bil­i­ties that Kelo­nia does not want to build in­ter­nal­ly just yet — an­ti­body dis­cov­ery in Adimab’s case and man­u­fac­tur­ing for El­e­vate­Bio.

As of now, the com­pa­ny is in the ear­ly phas­es of eval­u­at­ing the tech­nol­o­gy and the pre­clin­i­cal work nec­es­sary to bring a drug for­ward. Fried­man al­so said that any fu­ture fi­nanc­ing will come along nat­u­ral­ly and so far, is re­main­ing more fo­cused on the clin­i­cal end.

Pi­o­neer­ing Click Chem­istry in Hu­mans

Reimagining cancer treatments

Cancer is a leading cause of death worldwide, accounting for nearly 10 million deaths in 2020, which is nearly one in six deaths. Recently, we have seen incredible advances in novel cancer therapies such as immune checkpoint inhibitors, cell therapies, and antibody-drug conjugates that have revamped cancer care and improved survival rates for patients.

Despite this significant progress in therapeutic targeting, why are we still seeing such a high mortality rate? The reason is that promising therapies are often limited by their therapeutic index, which is a measure of the effective dose of a drug, relative to its safety. If we could broaden the therapeutic indices of currently available medicines, it would revolutionize cancer treatments. We are still on the quest to find the ultimate cancer medicine – highly effective in several cancer types, safe, and precisely targeted to the tumor site.

Joshua Cohen (L) and Justin Klee, Amylyx co-CEOs

Up­dat­ed: Af­ter long and wind­ing road, FDA ap­proves Amy­lyx's ALS drug in vic­to­ry for pa­tients and ad­vo­ca­cy groups

For just the third time in its 116-year history, the FDA has approved a new treatment for Lou Gehrig’s disease, or ALS.

US regulators gave the thumbs-up to the drug, known as Relyvrio, in a massive win for patients and their families. The approval, given to Boston-area biotech Amylyx Pharmaceuticals, comes after two years of long and contentious debates over the drug’s effectiveness between advocacy groups and FDA scientists, following the readout of a mid-stage clinical trial in September 2020.

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Ivan Cheung, Eisai US chairman and CEO

Bio­gen, Ei­sai re­fresh amy­loid hy­poth­e­sis with PhI­II show­ing Alzheimer's med slows cog­ni­tive de­cline

In the first look at Phase III data for lecanemab, Eisai and Biogen’s follow-up Alzheimer’s drug to the embattled Aduhelm launch, results show the drug passed with flying colors on a test looking at memory, problem solving and other dementia metrics.

One of the most-watched Alzheimer’s therapies in the clinic, lecanemab met the study’s primary goal on the CDR-SB — Clinical Dementia Rating-Sum of Boxes — giving the biotech the confidence to ask for full approval in the US, EU and Japan by next March 31. The experimental drug reduced clinical decline on the scale by 27% compared to placebo at 18 months, the companies said Tuesday night Eastern time and Wednesday morning in Japan.

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Nooman Haque, head of life sciences and healthcare at Silicon Valley Bank, and John Carroll

I’m head­ed to Lon­don soon for #EU­BIO22. Care to join me?

It was great getting back to a live ESMO conference/webinar in Paris followed by a live pop-up event for the Endpoints 11 in Boston. We’re staying on the road in October with our return for a live/streaming EUBIO22 in London.

Silicon Valley Bank’s Nooman Haque and I are once again jumping back into the thick of it with a slate of virtual and live events on October 12. I’ll get the ball rolling with a virtual fireside chat with Novo Nordisk R&D chief Marcus Schindler, covering their pipeline plans and BD work.

Some­one old, some­one new: Mod­er­na pro­motes CTO, raids No­var­tis for re­place­ment amid pipeline push

Moderna CEO Stéphane Bancel made clear on the last quarterly call that “now is not the time to slow down.” On Thursday, he made a bit more room in the cockpit.

The company unveiled a new executive role on Thursday, promoting former chief technical operations and quality officer Juan Andres to president of strategic partnerships and enterprise expansion, and poaching a former Novartis exec to take his place.

Gilead names 'k­ing­pin­s' in coun­ter­feit HIV med law­suit

Gilead is mounting its counterfeit drug lawsuit, naming two “kingpins” and a complex network of conspirators who allegedly sold imitation bottles of its HIV meds, some of which ended up in US pharmacies.

The pharma giant on Wednesday provided an update on what it called a “large-scale, sophisticated counterfeiting conspiracy,” accusing two new defendants of “leading and orchestrating” a scheme to sell hundreds of millions of dollars in illegitimate drugs posing as meds such as Biktarvy and Descovy.

Vlad Coric, Biohaven CEO (Photo Credit: Andrew Venditti)

As Amy­lyx de­ci­sion waits in the wings, Bio­haven’s ALS drug sinks (again) in plat­form tri­al

The FDA’s decision on Amylyx’s ALS drug is set to come out sometime Thursday. In a space with few drugs, any approval would be a major landmark.

But elsewhere in the ALS field, things are a bit more tepid.

Thursday morning, Biohaven announced that its drug verdiperstat failed its arm of an ALS platform trial led by Massachusetts General Hospital. According to a press release, the drug did not meet its primary endpoint — improvement on an ALS functional status test — or any key secondary endpoints at 24 weeks. The trial had enrolled 167 patients, giving them either verdiperstat or placebo twice a day.

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Tar­sus looks to raise aware­ness of eye­lid mite dis­ease in cam­paign aimed at eye­care spe­cial­ists

Eyelid mite disease may be “gross” but it’s also fairly common, affecting about 25 million people in the US.

Called demodex blepharitis, it’s a well-known condition among eyecare professionals, but they often don’t always realize how common it is. Tarsus Pharmaceuticals wants to change that with a new awareness campaign called “Look at the Lids.”

The campaign and website debut Thursday — just three weeks after Tarsus filed for FDA approval for a drug that treats the disease.

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Marcelo Bigal, Ventus Therapeutics CEO

No­vo Nordisk joins No­var­tis, Roche in NL­RP3 are­na, bet­ting $70M cash on NASH, car­diometa­bol­ic us­es

As a drug target, the NLRP3 inflammasome has drawn serious interest from Big Pharma, inspiring a series of M&A deals from Novartis and Roche on top of venture investments by others. Now Novo Nordisk is jumping on the bandwagon — and the Danish pharma giant is taking the target where it knows best.

Novo Nordisk is getting its NLRP3 inhibitors from Ventus Therapeutics, a Versant-backed startup that set out to make some of the best NLRP3 drugs out there by incorporating new insights into the structure of the target complex.

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