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.

Image courtesy of The Janssen Pharmaceutical Companies of Johnson & Johnson.

Pro­tect­ing the glob­al phar­ma­ceu­ti­cal in­no­va­tion ecosys­tem – what’s at stake?

We are living in a new era of healthcare that is rapidly advancing progress impacting patient outcomes and experiences. We’ve seen a remarkable pace of transformational innovation, applied research, and advanced clinical development over the last decade.

Despite this tremendous progress, there is much more work to be done, and patients are counting on us – now more than ever – to continue that momentum. At the heart of our industry is a focus on developing and delivering medicines for some of the world’s most challenging diseases, including those that have few or no effective treatments today.

Roger Perlmutter, Eikon Therapeutics CEO

Roger Perl­mut­ter builds Eikon's pipeline with deal-mak­ing flur­ry, rais­ing $106M more

Eikon Therapeutics announced three business development deals on Thursday, effectively dropping in a pipeline of cancer drugs alongside more than $100 million in fresh funding.

The Hayward, CA-based company has become one of biotech’s richest startups since its 2019 founding, having raised nearly $775 million. It’s developing a massive, automated research approach built around Nobel Prize-winning microscope science to peer inside cells and watch proteins in action. After its Series B last year, PitchBook reported a $3.02 billion valuation. And while CEO Roger Perlmutter declined to comment on that figure, he said its first tranche of nearly $106 million in Series C funding is a “meaningful step-up to our Series B valuation.”

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The Modulo Bio team with CEO Michael Horowitz (fourth from right in semicircle)

Ex­clu­sive: With $8M, neu­ro start­up Mod­u­lo Bio emerges to test small mol­e­cules for ALS, de­men­tia in CEO’s per­son­al mis­sion

Embarking on a personal mission after his best friend’s mother was diagnosed with a mutation-driven case of frontotemporal dementia, Michael Horowitz has pulled together $8 million in venture funding at Modulo Bio to create small molecules for neurodegenerative diseases.

The San Diego and Bay Area biotech will select its lead development candidate and some backup options within six months and then raise a Series A to investigate therapeutics for C9orf72 mutation-driven cases of ALS and frontotemporal dementia, Horowitz told Endpoints News.

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Eu­ro­pean Par­lia­ment calls mem­ber states to ac­tion on an­timi­cro­bial re­sis­tance

Members of the European Parliament have called on EU countries to develop national action plans against antimicrobial resistance (AMR), calling it a top-three priority health threat.

Parliament on Thursday announced recommendations for the fight against AMR, including national action plans that must be updated at least every two years, an EU-level database tracking AMR and antimicrobial use and increased partnership between the pharma industry, patient groups and academia.

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Roche plans to di­vest from lega­cy Genen­tech man­u­fac­tur­ing fa­cil­i­ty in Cal­i­for­nia

Roche is planning to make some changes to its subsidiary’s manufacturing network in California.

The Swiss pharma announced Wednesday that it plans to divest from Genentech’s manufacturing facility in Vacaville, CA, around 58 miles northeast of San Francisco. According to a statement from Roche, the move is part of a “broader strategy” to bring its manufacturing capabilities in line with its future pipeline. Roche is starting the process of finding a buyer for the site but has not named any candidates yet.

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Peter van de Sande, Synaffix CEO

Lon­za shells out $107M cash to snap up Synaf­fix and its ADC plat­form

After lining up a string of partnerships over the years, Dutch antibody-drug conjugate specialist Synaffix has found a new home: Lonza, the contract development and manufacturing giant.

Lonza is paying about $107 million (€100 million) in cash to acquire Synaffix, with up to $64 million (€60 million) in “additional performance-based consideration” on the table. Synaffix’s ADC tech platform will now become part of Lonza’s offering for biopharma clients, lending its bioconjugate technologies to not just ADCs but also targeted gene therapy, immune cell engagers and other applications.

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Who's con­fi­dent­ly in­vest­ing in biotech star­tups dur­ing these tense days? We've got some an­swers

We’ve got a changeup to our event schedule in Boston next week, where we’ll be doing a mix of live/streaming events at our base at The Seaport Hotel as part of a two-day lineup of webinars, virtual firesides and a cocktail hour Q&A with a veteran of the biotech financing scene.

The 9:30-10:30 am ET live slot on Tuesday, June 6, will now feature a panel conversation on the current state of affairs for VC investing in biotech, focusing on what startups are getting cash — and how. Alaa Halawa, head of US ventures at Mubadala, is confirmed, along with Brian Goodman at MPM and Geoff von Maltzahn, a general partner at Moderna-buoyed Flagship. I have a couple of other invites out and will let you know how that plays out.

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Albert Bourla, Pfizer CEO (Michel Euler/AP Images, Pool)

FDA ap­proves Pfiz­er’s RSV shot for old­er adults, tee­ing up a com­pet­i­tive $17B vac­cine mar­ket

The FDA approved Pfizer’s RSV vaccine called Abrysvo for older adults on Wednesday, placing another Big Pharma onto the commercial stage ahead of the next RSV season.

Pfizer’s approval comes weeks after GSK won approval for its rival shot, Arexvy. Those two vaccines are both approved for use in adults 60 years and older and will be reviewed by a CDC panel in June before they’re expected to commercially launch this fall. Wall Street analysts see RSV as the next multibillion-dollar vaccine market, with Jefferies analysts recently forecasting the RSV market will grow to $17 billion over the next decade.

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Richard Pazdur, FDA's OCE director (Flatiron Health via YouTube)

FDA's can­cer chief weighs in on com­mon chemo short­ages — re­port

Richard Pazdur, director of the FDA’s Oncology Center of Excellence, attributes the current shortage of two cancer drugs to drug companies that haven’t invested in building out their production capacity.

In an interview with The Cancer Letter, a weekly cancer publication, Pazdur said that the current shortages of cisplatin and carboplatin, a pair of drugs used to treat a wide range of cancer patients, are the result of two problems: manufacturers not investing in enhancing production capacity, and drug companies being dependent on one supplier of raw ingredients. The cisplatin shortage followed an inspection that revealed quality issues at a manufacturing facility, which then led to the shutdown of production. This led to a surge in carboplatin demand, creating a secondary shortage.

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