Ram Aiyar, Korro CEO

Can RNA edit­ing take At­las start­up where Ver­tex could­n't go? Ko­r­ro lands $116M to find out

As the CEO of RNA edit­ing start­up Ko­r­ro Bio, Ram Ai­yar of­ten gets the same ques­tion.

“I get al­ways asked, you know, if you can fix DNA, why both­er with RNA?” he told End­points News. “And it’s like ask­ing, which child do you pre­fer — your old­er one or your younger one?”

But in­vestors are ap­pre­ci­at­ing the dif­fer­ence. More than a year af­ter clos­ing its Se­ries A just shy of the megaround mark, Ko­r­ro Bio is back in the spot­light with $116 mil­lion in fresh cash and a lead can­di­date — tar­get­ing al­pha-1 an­tit­rypsin de­fi­cien­cy, or AATD.

Where­as gene edit­ing on the DNA lev­el promis­es a one-and-done ap­proach to ge­net­ic dis­or­ders com­pared to the tra­di­tion­al ap­proach of block­ing prob­lem­at­ic pro­teins, RNA edit­ing oc­cu­pies a space some­where in be­tween: a tran­sient yet pre­cise fix that gets to the root of a dis­ease.

And un­like the oth­er RNA modal­i­ties, from an­ti­sense oligonu­cleotides to RNA in­ter­fer­ence, Ko­r­ro is not in­ter­est­ed in knock­ing down pro­duc­tion of path­o­gen­ic pro­teins.

Nes­san Berming­ham

In­spired by a nat­ur­al mech­a­nism in squid and oc­to­pus, the biotech’s sci­en­tists use oligonu­cleotide guides to re­cruit an en­doge­nous en­zyme known as ADAR to a spe­cif­ic RNA site where they want to con­vert an A to a G — the kind of change that can make a world of dif­fer­ence for dis­or­ders like Rett syn­drome, co-founder and chair­man Nes­san Berming­ham pre­vi­ous­ly not­ed.

“All the tar­gets that we look at, we’re look­ing at it from a gain of func­tion stand­point,” Ai­yar said. “So ei­ther in­creas­ing the half life of a pro­tein, or up­reg­u­lat­ing the pro­tein, or re­pair­ing the pro­tein that is at a low lev­el, is re­al­ly go­ing to be the fo­cus. It’s a modal­i­ty that on­ly small mol­e­cules cur­rent­ly re­al­ly play in, but they haven’t been able to do that suc­cess­ful­ly be­cause they’re not high­ly spe­cif­ic.”

Ai­yar be­lieves those unique char­ac­ter­is­tics would al­low Ko­r­ro to tar­get preva­lent in­di­ca­tions, set­ting them apart from peers like Shape Ther­a­peu­tics, which he notes is fo­cus­ing on per­ma­nent edit­ing. And AATD, with some­where be­tween 100,000 and 150,000 pa­tients, is just one of them.

First brought on­to the broad­er biotech stage by Ver­tex, AATD is marked by mis­fold­ed pro­teins that go from the liv­er to the lung. Ver­tex tried to fix it by bind­ing to the pro­tein in a way that it be­lieves is re­spon­si­ble for the mis­fold­ing, but culled its ini­tial small mol­e­cule can­di­dates af­ter de­cid­ing they’re un­like­ly to show clin­i­cal ben­e­fit.

“It’s a very, very dif­fer­en­ti­at­ed pro­gram rel­a­tive to small mol­e­cule cor­rec­tors be­cause they’re just sit­ting in the pock­et and try­ing to pre­vent the mis­fold­ing, where­as we are fix­ing the amino acid se­quence on the RNA side to en­sure prop­er fold­ing,” Ai­yar said.

De­liv­ery, he reck­ons, will be the main chal­lenge here. But since Ko­r­ro is work­ing with mech­a­nisms like GalNAc and LNPs, Ai­yar notes that “we are stand­ing on shoul­ders of gi­ants” like Al­ny­lam and Io­n­is.

Even­tide As­set Man­age­ment led the Se­ries B, with par­tic­i­pa­tion from new in­vestors Fi­deli­ty Man­age­ment & Re­search Com­pa­ny, In­vus, Point72, Veri­tion Fund Man­age­ment, Monashee In­vest­ment Man­age­ment, Six­ty De­gree Cap­i­tal and an ad­di­tion­al health­care spe­cial­ist fund. All ex­ist­ing in­vestors joined, bring­ing to the syn­di­cate At­las Ven­ture, NEA, Wu Cap­i­tal, Qim­ing Ven­ture Part­ners USA, Sur­vey­or Cap­i­tal (a Citadel com­pa­ny), Cor­morant As­set Man­age­ment, MP Health­care Ven­ture Man­age­ment and Alexan­dria Ven­ture In­vest­ments.

Hav­ing grown the team from 27 to 59 in just around eight months, Ai­yar ex­pects the fund­ing to fu­el a con­tin­ued hir­ing dri­ve so Ko­r­ro has enough staffers to con­struct a siz­able pipeline. They’re de­vel­op­ing a CNS pro­gram to bal­ance out the lead liv­er in­di­ca­tion, with oth­er chron­ic in­di­ca­tions in mind, be­fore get­ting in­to the clin­ic, ide­al­ly in the next 18 months.

“The bi­ol­o­gy here is very nov­el,” he said. “I think it’s go­ing to be hard for peo­ple to dab­ble rather than, you know, take a very con­cert­ed ef­fort in terms of clin­i­cal de­vel­op­ment, be­cause the last thing you want to do is get to the clin­ic with a com­pound that looks good and then fall off the cliff very fast.”

Scoop: Boehringer qui­et­ly shut­ters a PhII for one of its top drugs — now un­der re­view

Boehringer Ingelheim has quietly shut down a small Phase II study for one of its lead drugs.

The private pharma player confirmed to Endpoints News that it had shuttered a study testing spesolimab as a therapy for Crohn’s patients suffering from bowel obstructions.

A spokesperson for the company tells Endpoints:

Taking into consideration the current therapeutic landscape and ongoing clinical development programs, Boehringer Ingelheim decided to discontinue our program in Crohn’s disease. It is important to note that this decision is not based on any safety findings in the clinical trials.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Vas Narasimhan (Photographer: Jason Alden/Bloomberg via Getty Images)

No­var­tis de­tails plans to axe 8,000 staffers as Narasimhan be­gins sec­ond phase of a glob­al re­org

We now know the number of jobs coming under the axe at Novartis, and it isn’t small.

The pharma giant is confirming a report from Swiss newspaper Tages-Anzeiger that it is chopping 8,000 jobs out of its 108,000 global staffers. A large segment will hit right at company headquarters in Basel, as CEO Vas Narasimhan axes some 1,400 of a little more than 11,000  jobs in Switzerland.

The first phase of the work is almost done, the company says in a statement to Endpoints News. Now it’s on to phase two. In the statement, Novartis says:

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 144,500+ biopharma pros reading Endpoints daily — and it's free.

Lina Gugucheva, NewAmsterdam Pharma CBO

Phar­ma group bets up to $1B-plus on the PhI­II res­ur­rec­tion of a once dead-and-buried LDL drug

Close to 5 years after then-Amgen R&D chief Sean Harper tamped the last spade of dirt on the last broadly focused CETP cholesterol drug — burying their $300 million upfront and the few remaining hopes for the class with it — the therapy has been fully resurrected. And today, the NewAmsterdam Pharma crew that did the Lazarus treatment on obicetrapib is taking another big step on the comeback trail with a €1 billion-plus regional licensing deal, complete with close to $150 million in upfront cash.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 144,500+ biopharma pros reading Endpoints daily — and it's free.

How pre­pared is bio­phar­ma for the cy­ber dooms­day?

One of the largest cyberattacks in history happened on a Friday, Eric Perakslis distinctly remembers.

Perakslis, who was head of Takeda’s R&D Data Sciences Institute and visiting faculty at Harvard Medical School at the time, had spent that morning completing a review on cybersecurity for the British Medical Journal. Moments after he turned it in, he heard back from the editor: “Have you heard what’s going on right now?”

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

(AP Photo/Gemunu Amarasinghe)

Some phar­ma com­pa­nies promise to cov­er abor­tion-re­lat­ed trav­el costs — while oth­ers won't go that far yet

As the US Department of Health and Human Services promises to support the millions of women who would now need to cross state lines to receive a legal abortion, a handful of pharma companies have said they will pick up employees’ travel expenses.

GSK, Sanofi, Johnson & Johnson, BeiGene, Alnylam and Gilead have all committed to covering abortion-related travel expenses just four days after the Supreme Court overturned Roe v. Wade and revoked women’s constitutional right to an abortion.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 144,500+ biopharma pros reading Endpoints daily — and it's free.

Aurobindo Pharma co-founders P. V. Ram Prasad Reddy (L) and K. Nityananda Reddy

Au­robindo Phar­ma re­ceives warn­ing let­ter from In­di­a's SEC fol­low­ing more FDA ques­tion marks

Indian-based generics manufacturer Aurobindo Pharma has been in the crosshairs of the FDA for several years now, but the company is also attracting attention from regulators within the subcontinent.

According to the Indian business news site Business Standard, a warning letter was sent to the company from the Securities Exchange Board of India, or SEBI.

The letter is related to disclosures made by the company on an ongoing FDA audit of the company’s Unit-1 API facility in Hyderabad, India as well as observations made by the US regulator between 2019 and 2022.

New Charles River Laboratories High Quality (HQ) Plasmid DNA Centre of Excellence at Bruntwood SciTech’s Alderley Park in Cheshire, United Kingdom. (Charles River)

Charles Riv­er Lab­o­ra­to­ries to start cell and gene ther­a­py man­u­fac­tur­ing at UK site in Sep­tem­ber

While Massachusetts-based Charles River Laboratories has been on an acquisition spree, they are not against planting their flag. The latest move by the company sees them crossing the pond to establish a manufacturing site in the UK.

The company on Tuesday opened its cell and gene therapy manufacturing center at Bruntwood SciTech’s Alderley Park in Cheshire, United Kingdom. The expansion follows Charles River’s acquisition of Cognate BioServices and Cobra Biologics in 2021 for $875 million. Cognate is a plasmid DNA, viral vector and cell therapy CDMO.

Bristol Myers Squibb (Alamy)

CVS re­sumes cov­er­age of block­buster blood thin­ner af­ter price drop fol­lows Jan­u­ary ex­clu­sion

Following some backlash from the American College of Cardiology and patients, Bristol Myers Squibb and Pfizer lowered the price of their blockbuster blood thinner Eliquis, thus ensuring that CVS Caremark would cover the drug after 6 months of it being off the major PBM’s formulary.

“Because we secured lower net costs for patients from negotiations with the drug manufacturer, Eliquis will be added back to our template formularies for the commercial segment effective July 1, 2022, and patient choices will be expanded,” CVS Health said in an emailed statement. “Anti-coagulant therapies are among the non-specialty products where we are seeing the fastest cost increases from drug manufacturers and we will continue to push back on unwarranted price increases.”

#Can­nes­Lions2022: Con­sumer health ex­ecs call on agen­cies to in­volve pa­tients in cre­ative process

CANNES — When Tamara Rogers joined GSK back in 2018, “science was king and R&D were the gods.” Now the global chief marketing officer of consumer healthcare wants to make room for another supreme being: the consumer.

As health and wellness becomes more relevant to consumers amid the pandemic, four health-focused executives called on marketers to involve patients in their creative process in a panel discussion at the Cannes Lions advertising creativity festival.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 144,500+ biopharma pros reading Endpoints daily — and it's free.