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.

M&A: a crit­i­cal dri­ver for sus­tain­able top-line growth in health­care

2021 saw a record $600B in healthcare M&A activity. In 2022, there is an anticipated slowdown in activity, however, M&A prospects remain strong in the medium to long-term. What are future growth drivers for the healthcare sector? Where might we see innovations that drive M&A? RBC’s Andrew Callaway, Global Head, Healthcare Investment Banking discusses with Vito Sperduto, Global Co-Head, M&A.

15 LGBTQ lead­ers in bio­phar­ma; Paul Stof­fels’ Gala­pa­gos re­vamp; As­traZeneca catch­es up in AT­TR; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

A return to in-person conferences also marks a return to on-the-ground reporting. My colleagues Beth Synder Bulik and Nicole DeFeudis were on-site at Cannes Lions, bringing live coverage of pharma’s presence at the ad festival — accompanied by photos from Clara Bui, our virtual producer, that bring you right to the scene. You can find a recap (and links to all the stories) below.

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Abortion-rights protesters regroup and protest following Supreme Court's decision to overturn Roe v. Wade. (AP Photo/Gemunu Amarasinghe)

Fol­low­ing SCO­TUS de­ci­sion to over­turn abor­tion pro­tec­tions, AG Gar­land says states can't ban the abor­tion pill

Following the Supreme Court’s historic decision on Friday to overturn Americans’ constitutional right to an abortion after almost 50 years, Attorney General Merrick Garland sought to somewhat reassure women that states will not be able to ban the prescription drug sometimes used for abortions.

Following the decision, the New England Journal of Medicine also published an editorial strongly condemning the reversal, saying it “serves American families poorly, putting their health, safety, finances, and futures at risk.”

AstraZeneca's new Evusheld direct to consumer campaign aims to reach more immunocompromised patients.

As­traZeneca de­buts first con­sumer cam­paign for its Covid-19 pro­phy­lac­tic Evusheld — and a first for EUA drugs

AstraZeneca’s first consumer ad for Evusheld is also a first for drugs that have been granted emergency use authorizations during the pandemic.

The first DTC ad for a medicine under emergency approval, the Evusheld campaign launching this week aims to raise awareness among immunocompromised patients — and spur more use.

Evusheld nabbed emergency authorization in December, however, despite millions of immunocompromised people looking for a solution and now more widespread availability of the drug.

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Yong Dai, Frontera Therapeutics CEO

Scoop: Lit­tle-known Or­biMed-backed biotech clos­es $160M round to start gene ther­a­py tri­al

Frontera Therapeutics, a China and US biotech, has closed a $160 million Series B and received regulatory clearance to test its first gene therapy stateside, Endpoints News has learned.

Led by the largest shareholder, OrbiMed, the biotech has secured $195 million total since its September 2019 founding, according to an email reviewed by Endpoints. The lead AAV gene therapy program is for an undisclosed rare eye disease, according to the source.

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DEM BioPharma CEO David Donabedian (L) and executive chair Jan Skvarka

Long­wood sets an­oth­er 'don't eat me' biotech in­to gear with help of for­mer Tril­li­um CEO Jan Skvar­ka

Jonathan Weissman and team are out with a cancer-fighting biotech riding the appetite for those so-called “don’t eat me” and “eat me” signals.

The scientific co-founder — alongside fellow Whitehead Institute colleague Kipp Weiskopf and Stanford biologist Michael Bassik — has launched DEM BioPharma with incubator Longwood Fund and a crop of other investors.

In all, the nascent, 10-employee biotech has $70 million to bankroll hematology- and solid tumor-based programs, including a lead asset that could enter human trials in two to three years, CEO David Donabedian told Endpoints News.

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GSK says its drug for chron­ic hep B could ‘lead to a func­tion­al cure’ — but will it be alone or in com­bi­na­tion?

GSK, newly branded and soon-to-be demerged, shared interim results from its Phase II trial on its chronic hepatitis B treatment, one that it says has the “potential to lead to a functional cure.”

At a presentation at the EASL International Liver Congress, GSK shared that in around 450 patients who received its hep B drug bepirovirsen for 24 weeks, just under 30% had hepatitis B surface antigen and viral DNA levels that were too low to detect.

De­spite a slow start to the year for deals, PwC pre­dicts a flur­ry of ac­tiv­i­ty com­ing up

Despite whispers of a busy year for M&A, deal activity in the pharma space is actually down 30% on a semi-annualized basis, according to PwC’s latest report on deal activity. But don’t rule out larger deals in the second half of the year, the consultants said.

PwC pharmaceutical and life sciences consulting solutions leader Glenn Hunzinger expects to see Big Pharma companies picking up earlier stage companies to try and fill pipeline gaps ahead of a slew of big patent cliffs. Though a bear market continues to maul the biotech sector, Hunzinger said recent deals indicate that pharma companies are still paying above current trading prices.

Joe Wiley, Amryt Pharma CEO

Am­ryt Phar­ma sub­mits a for­mal dis­pute res­o­lu­tion to the FDA over re­ject­ed skin dis­ease drug

The story of Amryt Pharma’s candidate for the genetic skin condition epidermolysis bullosa, or EB, will soon enter another chapter.

After the Irish drugmaker’s candidate, dubbed Oleogel-S10 and marketed as Filsuvez, was handed a CRL earlier this year, the company announced in a press release that it plans to submit a formal dispute resolution request for the company’s NDA for Oleogel-S10.