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.

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.

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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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.

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.

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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Work taking place in the clean rooms at Vor (Credit: Vor)

Vor Bio opts to keep man­u­fac­tur­ing op­er­a­tions in-house for de­vel­op­ing stem cell, CAR-T ther­a­pies

While it is not uncommon for a biotech to go down the route of having the product manufactured by a contract organization, one small biotech is looking to keep its card close to its chest.

Vor Biopharma has started manufacturing operations at an in-house facility at its HQ in Cambridge, MA after beginning construction last summer.

According to the biotech, the facility aims to develop Vor’s hematopoietic stem cells (eHSCs) and CAR-T therapies for patients with blood cancers. The site will initially manufacture a clinical supply of its candidate VCAR33allo to support its IND, which is slated to be submitted in the first half of next year. It also plans to transfer the production of VOR33 to the facility. Vor is getting to work quickly as engineering runs for VCAR33allo has started this week.

Aim­ing for fourth nod, Sarep­ta files an­oth­er DMD gene ther­a­py to FDA; Ax­some head­ed to­ward mi­graine re­sub­mis­sion

Sarepta Therapeutics has filed the data needed for an FDA accelerated approval, which would be the biotech’s fourth if granted by the agency.

The biotech has yet to complete confirmatory trials for those first three conditional nods. The filing for its fourth Duchenne muscular dystrophy treatment, disclosed Thursday, is not a surprise. Sarepta said in late-July it would do so after releasing positive results for the Roche-partnered gene therapy.

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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