Ray Tabibiazar, SalioGen CEO

Gene ther­a­py 3.0 ef­forts net Sali­o­Gen a nine-fig­ure Se­ries B, but much re­mains un­der wraps

Rough­ly 10 months ago, a fledg­ling biotech emerged from stealth with a mod­est Se­ries A and a big promise look­ing to de­vel­op gene ther­a­py 3.0. The promise inched clos­er to re­al­i­ty Wednes­day as in­vestors have now hopped on board thanks to a new, nine-fig­ure round.

Sali­o­Gen Ther­a­peu­tics closed its $115 mil­lion Se­ries B, the com­pa­ny an­nounced Wednes­day morn­ing, aim­ing to push for­ward its “gene cod­ing” plat­form and grow­ing pre­clin­i­cal pipeline. The biotech, which fo­cus­es on ac­ti­vat­ing dor­mant mam­malian en­zymes to ed­it genes in vi­vo, se­cured the new raise af­ter flesh­ing out some of the tech­nolo­gies’ ap­pli­ca­tions, CEO Ray Tabib­i­azar told End­points News.

Tout­ing the com­pa­ny’s abil­i­ty to char­ac­ter­ize such en­zymes, Tabib­i­azar said the plat­form “has the abil­i­ty to in­te­grate any size of ge­net­ic code in­to the genome. So that al­lows you not on­ly to pur­sue large genes, but mul­ti­ple genes.”

The method­ol­o­gy al­so doesn’t re­quire the need for the nu­cle­as­es and vi­ral vec­tors as­so­ci­at­ed with ear­li­er forms of gene edit­ing, such as CRISPR. Sali­o­Gen’s plat­form, Tabib­i­azar said, can ed­it the genome with­out caus­ing “any dou­ble strand DNA breaks, or even sin­gle strand breaks.”

A lot has gone on be­hind the scenes that Sali­o­Gen isn’t ready to share just yet, how­ev­er, as Tabib­i­azar de­clined to put a time­line on when his com­pa­ny’s pro­grams would hit the clin­ic. Sali­o­Gen’s web­site lists sev­er­al dis­ease ar­eas it’s ex­plor­ing, in­clud­ing fa­mil­ial hy­per­c­ho­les­terolemia and in­her­it­ed mac­u­lar de­gen­er­a­tion, but Tabib­i­azar fur­ther de­clined to say which, if any, rep­re­sent the biotech’s lead pro­gram.

“That’s one of the ad­van­tages of be­ing a pri­vate com­pa­ny,” Tabib­i­azar said, adding, “One of the ways that we run the com­pa­ny, we have mul­ti­ple hors­es in a race. So we are re­al­ly ad­vanc­ing a pipeline of pro­grams … that’s ex­act­ly one of the rea­sons we ac­tu­al­ly raised large cap­i­tal.”

Every­thing re­mains in the pre-IND phase, though the FH and eye pro­grams are among the clos­est to be­ing ready for hu­man test­ing. Sali­o­Gen is al­so work­ing on pro­grams for the heart, bone mar­row and kid­ney, as well as a cys­tic fi­bro­sis can­di­date that gar­nered in­vest­ment in­ter­est from the Cys­tic Fi­bro­sis Foun­da­tion in Wednes­day’s raise.

Sali­o­Gen has al­so ramped up work on dis­cov­er­ing more en­zymes that could po­ten­tial­ly be used to de­liv­er its treat­ments. Tabib­i­azar said any of the en­zymes are pro­gram­ma­ble re­gard­less of the af­fect­ed or dis­eased tis­sue, but he again de­murred when asked how many en­zymes Sali­o­Gen is work­ing to char­ac­ter­ize.

“I can tell you we’ve done more than one,” he said.

As the biotech IPO mar­ket slowed down late last year, in­vestors large­ly turned their fo­cus to­ward ear­li­er-stage in­vest­ments in com­pa­nies that had not yet pro­duced clin­i­cal da­ta. Sali­o­Gen is seem­ing­ly a part of this trend, now hav­ing raised $135 mil­lion in less than a year.

But Tabib­i­azar isn’t look­ing at mak­ing the pub­lic leap just yet, say­ing Sali­o­Gen has “plen­ty of cush­ion” to ad­vance its pipeline and plat­form right now. That be­ing said, he not­ed how every com­pa­ny has to leave its op­tions open and be op­por­tunis­tic if the right mo­ment comes along.

“It’s the tim­ing of when you raise more mon­ey, and how you do it needs to be tai­lored to the needs of the com­pa­ny,” he said. “Right now we have, you know, $115 mil­lion that we can sup­port our pro­grams, so we don’t have any im­me­di­ate need to raise more mon­ey.”

Wednes­day’s round was co-led by Gor­don­MD Glob­al In­vest­ments and EPIQ Cap­i­tal Group. In ad­di­tion to the Cys­tic Fi­bro­sis Foun­da­tion, new in­vestors in­clud­ed Fi­deli­ty Man­age­ment & Re­search Com­pa­ny, T. Rowe Price, D1 Cap­i­tal Part­ners, Sym­Bio­sis, the ven­ture arm of Foun­da­tion Fight­ing Blind­ness and oth­ers. The round al­so in­clud­ed con­tin­ued sup­port from PBM Cap­i­tal, which led the Se­ries A.

Alexander Lefterov/Endpoints News

A new can­cer im­munother­a­py brings cau­tious hope for a field long await­ing the next big break­through

Bob Seibert sat silent across from his daughter at their favorite Spanish restaurant near his home in Charleston County, SC, their paella growing cold as he read through all the places in his body doctors found tumors.

He had texted his wife, a pediatric intensive care nurse, when he got the alert that his online chart was ready. Although he saw immediately it was bad, many of the terms — peritoneal, right iliac — were inscrutable. But she was five hours downstate, at a loud group dinner the night before another daughter’s cheer competition.

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In a set­back, FDA or­ders Gilead to hit the brakes on their late-stage, $5B can­cer play

Gilead’s $5 billion drug magrolimab has run into a serious setback.

The FDA ordered Gilead to halt enrollment on their studies of the drug in combination with azacitidine after investigators reports revealed an “apparent imbalance” in the suspected unexpected serious adverse reactions between study arms. And the halt is raising questions about Gilead’s plans for a quick pitch to regulators.

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Graphic: Alexander Lefterov for Endpoints News

Small biotechs with big drug am­bi­tions threat­en to up­end the tra­di­tion­al drug launch play­book

Of the countless decisions Vlad Coric had to make as Biohaven’s CEO over the past seven years, there was one that felt particularly nerve-wracking: Instead of selling to a Big Pharma, the company decided it would commercialize its migraine drug itself.

“I remember some investors yelling and pounding on the table like, you can’t do this. What are you thinking? You’re going to get crushed by AbbVie,” he recalled.

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Mar­ket­ingRx roundup: Pfiz­er de­buts Pre­vnar 20 TV ads; Lil­ly gets first FDA 2022 pro­mo slap down let­ter

Pfizer debuted its first TV ad for its Prevnar 20 next-generation pneumococcal pneumonia vaccine. In the 60-second spot, several people (actor portrayals) with their ages listed as 65 or older are shown walking into a clinic as they turn to say they’re getting vaccinated with Prevnar 20 because they’re at risk.

The update to Pfizer’s blockbuster Prevnar 13 vaccine was approved in June, and as its name suggests is a vaccine for 20 serotypes — the original 13 plus seven more that cause pneumococcal disease. Pfizer used to spend heavily on TV ads to promote Prevnar 13 in 2018 and 2019 but cut back its TV budgets in the past two fall and winter seasonal spending cycles. Prevnar had been Pfizer’s top-selling drug, notching sales of just under $6 billion in 2020, and was the world’s top-selling vaccine before the Covid-19 vaccines came to market last year.

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Roy Baynes, Merck

FDA bats back Mer­ck’s ‘pipeline in a prod­uct,’ de­mands more ef­fi­ca­cy da­ta

Despite some heavy blowback from analysts, Merck execs maintained an upbeat attitude about the market potential of its chronic cough drug gefapixant. But the confidence may be fading somewhat today as Merck puts out news that the FDA is handing back its application with a CRL.

Dubbed by Merck’s development chief Roy Baynes as a “pipeline in a product” with a variety of potential uses, Merck had fielded positive late-stage data demonstrating the drug’s ability to combat chronic cough. The drug dramatically reduced chronic cough in Phase III, but so did placebo, leaving Merck’s research team with a marginal success on the p-value side of the equation.

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Albert Bourla (Photo by Steven Ferdman/Getty Images)

UP­DAT­ED: Pfiz­er fields a CRL for a $295M rare dis­ease play, giv­ing ri­val a big head start

Pfizer won’t be adding a new rare disease drug to the franchise club — for now, anyway.

The pharma giant put out word that their FDA application for the growth hormone therapy somatrogon got the regulatory heave-ho, though they didn’t even hint at a reason for the CRL. Following standard operating procedure, Pfizer said in a terse missive that they would be working with regulators on a followup.

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FDA slams door to piv­otal tri­al for bub­ble boy dis­ease gene ther­a­py as Mus­tang Bio runs in­to an­oth­er hold

Mustang Bio is in familiar territory, but that isn’t a place it necessarily wants to be.

The FDA has placed a hold on Mustang Bio’s pivitol trial for its gene therapy to treat patients with bubble boy disease, citing issues surrounding chemistry, manufacturing and controls clearance. It’s the second hold due to CMC issues the company has received in roughly 18 months.

An investigational new drug application was submitted in December 2021. If granted an IND, a Phase II study will then assess safety, tolerability and efficacy of MB-207. If approved by the FDA, the therapy would one day be eligible for a rare pediatric disease voucher.

Brian Thomas, Metagenomi CEO

Gen 2: Berke­ley spin­out lands $175M megaround to keep it on the cut­ting edge of the boom­ing gene-edit­ing field

The big bucks keep pumping into the gene-editing field.

This morning Metagenomi, allied with one of the biggest names in the mRNA field with a company DNA that includes the ubiquitous Jennifer Doudna, is showing off a $175 million B round that will pay for a rapid swelling of its staff in pursuit of some of the cutting-edge tech that keeps this field in the spotlight. And they’re aligning themselves with some major industry players with an eye on the clinic while getting behind some startups to help expand the work into new fields.

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Michael Egholm, Standard BioTools president and CEO (IsoPlexis)

Eli Cas­din co-leads $250M in­fu­sion in­to mi­croflu­idics play­er that land­ed NIH fund­ing for Covid-19 test­ing

In about 17 months, Fluidigm has gone from working with sharks to Vikings.

The South San Francisco-based company, which landed NIH money in a Shark Tank-style program for Covid-19 testing, announced that it will take on an investment worth $250 million from Casdin Capital and Viking Global Investors. It will also rebrand, and call itself Standard BioTools. The investment will help the company focus on the highest growth areas of discovery and development and expand its CRO and CMO service providers. Right now, the company’s customer reach is limited to basic research, it said.

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