CEO Ray Tabibiazar (SalioGen)

Look­ing to take ad­van­tage of 'si­lenced' en­zymes, Sali­o­Gen emerges from stealth with eyes set on gene ther­a­py 3.0

Gene ther­a­py has made big strides over the years, from the first gen­er­a­tion AAV-based ther­a­pies to the CRISPR/Cas9 tech­nol­o­gy that has now tak­en the sci­en­tif­ic world — and No­bel Prize com­mit­tee — by storm. But where ex­act­ly does the fu­ture, or gene ther­a­py 3.0, lie?

That’s the ques­tion a new biotech is aim­ing to an­swer, as Sali­o­Gen Ther­a­peu­tics emerges from stealth Mon­day morn­ing with a $20 mil­lion Se­ries A. And they be­lieve they’ve found a new de­liv­ery sys­tem that can more pre­cise­ly de­liv­er genes in vi­vo than the rel­a­tive­ly large ade­no-as­so­ci­at­ed virus or CRISPR sys­tem: mam­malian-de­rived en­zymes.

Mon­day’s round was led by PBM Cap­i­tal and in­clud­ed oth­er undis­closed in­vestors.

Based in Burling­ton, MA, Sali­o­Gen came about when CEO Ray Tabib­i­azar and co-founder Joseph Hig­gins, who worked on the Hu­man Genome Pro­ject in the 1990s, set out rough­ly a year and a half ago look­ing to find what the nat­ur­al evo­lu­tion of gene ther­a­py might be. They be­lieved that while CRISPR and oth­er tech­nolo­gies like Pre­ci­sion Bio’s AR­CUS were promis­ing, re­searchers could re­al­ly on­ly go af­ter dis­or­ders that in­volve big­ger genes with spe­cif­ic mu­ta­tions, as those plat­forms in­volve cut­ting, knock­ing out or re­plac­ing spe­cif­ic DNA se­quences.

The pair iden­ti­fied three is­sues that need­ed solv­ing in the cur­rent gene ther­a­py field in or­der to get the com­pa­ny off the ground. First was the gene edit­ing it­self, or how Sali­o­Gen could take a dif­fer­ent ap­proach than what’s al­ready out there. Sec­ond is the de­liv­ery method: Tabib­i­azar and Hig­gins felt they couldn’t re­ly on the AAVs. And last was how to make man­u­fac­tur­ing cheap­er in or­der to make the ther­a­pies, which of­ten run up sev­er­al hun­dred thou­sands of dol­lars in costs, more ac­ces­si­ble.

Through these steps came Sali­o­Gen’s pro­pri­etary Ex­act DNA In­te­gra­tion Tech­nol­o­gy plat­form, or ED­IT. The goal, Tabib­i­azar told End­points News, is to es­sen­tial­ly pluck these mam­malian en­zymes that have been “si­lenced” over the course of evo­lu­tion and re­pur­pose them in­to de­liv­er­ing the gene ther­a­py. And the whole pro­ce­dure of tak­ing genes and putting them in­to new cells takes less than a month, sharply cut­ting down on pro­duc­tion costs.

“It puts the ge­net­ic code in­to the genome, and then it’s done, it’s gone,” Tabib­i­azar told End­points.

Not on­ly does that ad­dress the three is­sues they’d hoped to fix, it al­lows for a genome to be rewrit­ten in­side the body with­out the wor­ry AAVs or Cas9s will con­tin­ue mak­ing changes af­ter the tar­get­ed gene has been fixed. Tabib­i­azar is call­ing this process “gene cod­ing,” in that it puts new ge­net­ic code back in­to one’s genome through the en­zyme de­liv­ery.

He likened it to a soft­ware and hard­ware up­grade anal­o­gy — the en­zymes, or the soft­ware, con­tains the up­date and fix­es the is­sues in the body’s hard­ware.

“If you have an Ap­ple [prod­uct] and you get Ap­ple soft­ware, Ap­ple hard­ware, you’re not go­ing to use the app on An­droid,” Tabib­i­azar said. “It’s the same thing here, if you have a mam­malian en­zyme, which rewrites the soft­ware in­to your mam­malian hard dri­ve, you want it to be a fit be­tween the soft­ware and the hard­ware.”

Though Tabib­i­azar says the ap­pli­ca­tion of the plat­form is quite wide, Sali­o­Gen will be fo­cus­ing on fa­mil­ial hy­per­c­ho­les­terolemia and in­her­it­ed mac­u­lar de­gen­er­a­tion first. In FH, the com­pa­ny is tak­ing what it sees as a new ap­proach, aim­ing to re­place the en­tire re­cep­tor gene in the liv­er re­spon­si­ble for the dan­ger­ous­ly high cho­les­terol and LDL lev­els as­so­ci­at­ed with the dis­ease. It’s a field with a few play­ers al­ready, with Verve and Pre­ci­sion Bio both go­ing af­ter HoFH.

Both pro­grams are still in the pre­clin­i­cal stage, but Tabib­i­azar hopes they can be in the clin­ic with­in the next two to two-and-a-half years. The key now is en­sur­ing the ther­a­pies can be safe and build­ing out the plat­form with the fi­nanc­ing. Tabib­i­azar said hav­ing PBM Cap­i­tal on their side, who al­so made ear­ly-stage in­vest­ments in the biotechs that cre­at­ed Lux­tur­na and Zol­gens­ma, pro­vides a cru­cial val­i­da­tion for Sali­o­Gen.

“All our fo­cus is to do it very spe­cif­ic,” Tabib­i­azar said. “Not on­ly do we know how to tar­get it to a spe­cif­ic cell, we know how to tar­get it to a spe­cif­ic lo­ca­tion with­in the genome.”

Health­care Dis­par­i­ties and Sick­le Cell Dis­ease

In the complicated U.S. healthcare system, navigating a serious illness such as cancer or heart disease can be remarkably challenging for patients and caregivers. When that illness is classified as a rare disease, those challenges can become even more acute. And when that rare disease occurs in a population that experiences health disparities, such as people with sickle cell disease (SCD) who are primarily Black and Latino, challenges can become almost insurmountable.

The End­points 11: They've got mad mon­ey and huge am­bi­tions. It's time to go big or go home

These days, selecting a group of private biotechs for the Endpoints 11 spotlight begins with a sprint to get ahead of IPOs and the M&A teams at Big Pharma. I’ve had a couple of faceplants earlier this year, watching some of the biotechs on my short list choose a quick leap onto Nasdaq or into the arms of a buyer.

Vividion, you would have been a great pick for the Endpoints 11. I’m sorry I missed you.

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Dave Lennon, former president of Novartis Gene Therapies

So what hap­pened with No­var­tis Gene Ther­a­pies? Here's your an­swer

Over the last couple of days it’s become clear that the gene therapy division at Novartis has quietly undergone a major reorganization. We learned on Monday that Dave Lennon, who had pursued a high-profile role as president of the unit with 1,500 people, had left the pharma giant to take over as CEO of a startup.

Like a lot of the majors, Novartis is an open highway for head hunters, or anyone looking to staff a startup. So that was news but not completely unexpected.

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Who are the women su­per­charg­ing bio­phar­ma R&D? Nom­i­nate them for this year's spe­cial re­port

The biotech industry has faced repeated calls to diversify its workforce — and in the last year, those calls got a lot louder. Though women account for just under half of all biotech employees around the world, they occupy very few places in C-suites, and even fewer make it to the helm.

Some companies are listening, according to a recent BIO survey which showed that this year’s companies were 2.5 times more likely to have a diversity and inclusion program compared to last year’s sample. But we still have a long way to go. Women represent just 31% of biotech executives, BIO reported. And those numbers are even more stark for women of color.

FDA au­tho­rizes Pfiz­er's vac­cine boost­er for se­niors, those at high risk for se­vere Covid-19

The Biden administration’s goal of kicking off its booster shot drive for the entire US population this week is not quite going as planned.

First, Pfizer applied for approval of a supplemental application for the booster shots, but since last Friday’s adcomm reviewing them, the plan has devolved into an EUA, which the FDA issued late Thursday evening.

The population that is now eligible for the booster, six months after receiving the first pair of Pfizer-BioNTech vaccines, also narrowed from what Pfizer applied for (everyone who’s eligible for the initial Pfizer shots) to just those who are 65 or older, or at high-risk of a Covid infection, including health care workers and others with occupational hazards.

Stéphane Bancel, AP Images

Fi­nal analy­sis of US-fund­ed Mod­er­na Covid vac­cine tri­al shows 98% ef­fi­ca­cy against se­vere dis­ease

A final look at the results of the placebo-controlled Moderna trial in the New England Journal of Medicine, published Thursday afternoon, shows how the vaccine continues to prevent Covid-19 and severe cases after more than five months following the second shot.

Of the more than 30,000 enrolled in the trial that ultimately led to the vaccine’s EUA, only two people in the vaccine group got a severe form of the disease, compared to 106 in the placebo group — leading to an efficacy of 98%.

Emma Walmsley, GlaxoSmithKline CEO (Credit: Fang Zhe/Xinhua/Alamy Live News)

The fire un­der Glax­o­SmithK­line's Em­ma Walm­s­ley grows as an­oth­er well-known ac­tivist in­vestor grabs its pitch­fork — re­port

Bluebell Capital Partners, a proxy brawler fresh off a campaign to oust global food giant Danone’s CEO and most of its board of directors, has bought a stake in UK drugmaker GlaxoSmithKline with its eyes trained directly on Emma Walmsley, the Financial Times reported Wednesday.

The London-based hedge fund joins another notorious activist firm in Paul Singer’s Elliott Management, which earlier this year called for a shakeup in leadership at GSK to handle what the company described as a wealth of riches across the drug giant’s portfolio hindered by limited vision from top staff.

Maureen Hillenmeyer, Hexagon Bio CEO

Hexa­gon Bio rais­es $61M to con­tin­ue ef­forts to turn fun­gi in­to drugs

A year after raising a $47 million launch round, the fungi-loving drug hunters at Hexagon Bio have more than doubled their coffers.

Hexagon announced today that it raised another $61 million for its efforts to design cancer and infectious disease drugs based on insights mined from the DNA in millions of species of fungi. The new financing brings Hexagon’s committed funding to over $108 million.

FDA+ roundup: Bs­U­FA III ready for show­time, court tells FDA to re-work com­pound­ing plan, new guid­ance up­dates and more

The FDA has now spelled out what exactly will be included in the third iteration of Biosimilar User Fee Act (BsUFA) from 2023 through 2027, which similarly to the prescription drug deal, sets fees that industry has to pay for submitting applications, in exchange for firm timelines that the agency must meet.

This latest deal includes several sweeteners for the biosimilar industry, which has yet to make great strides in the US market, with shorter review timelines for safety labeling updates and updates to add or remove an indication that does not contain efficacy data.

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