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.”

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Amid continued controversy around whether Biogen’s new flagship drug, Aduhelm, should have been approved at all — and swelling, heated debates surrounding its $56,000 price tag — the agency had no issue handing them and their Japanese partner Eisai a breakthrough therapy designation for a second anti-amyloid beta antibody, lecanemab, late Wednesday.

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Hervé Hoppenot, Incyte CEO (Jeff Rumans)

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After the FDA lambasted their PD-1 ahead of an adcomm earlier this week, Incyte ran into new trouble Thursday as ODAC panelists voted against an accelerated OK by a wide margin.

Members of the Oncologic Drugs Advisory Committee recommended with a 13-4 vote to defer a regulatory decision on Incyte’s retifanlimab until after more data can be collected from a placebo-controlled trial. The PD-1 therapy is due for a PDUFA date in late July after receiving priority review earlier this year.

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In a lengthy review document and a pair of memos from top officials, the FDA released on Tuesday night its most detailed argument yet for approving Biogen’s intensely controversial Alzheimer’s drug aducanumab.

The documents amount to an agency attempt to quench the firestorm their decision kindled, as outside advisors members resigned and experts warned that an unproven drug now could stretch Medicare’s budget to a breaking point. Ultimately, the documents show how CDER director Patrizia Cavazzoni and Office of New Drugs director Peter Stein both concurred with FDA neuroscience head Billy Dunn on the accelerated approval while the staff at FDA’s Office of Biostatistics did not think an approval was warranted.

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From left: Rajul Jain, Stefan Vitorovic, Arjun Goyal, Arie Belldegrun, Jean-Philippe (JP) Kouakou-Zebouah, Helen Kim

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Among the list of bright names in biopharma, few shine brighter than Kite founder and serial entrepreneur Arie Belldegrun, who has rattled off a remarkable run of success in recent years. Now, a Belldegrun investment team is locking up a massive third fund to keep chasing the cutting edge in therapeutics.

Vida Ventures closed its third investment fund at a whopping $825 million — its largest yet — as the ever-expanding VC firm hits 30 companies in its portfolio developing new routes to hard-to-treat diseases, the company said Thursday.

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Karen Flynn, Catalent

Q&A: When the pan­dem­ic struck, Catal­en­t's CCO had just joined the team

Karen Flynn came aboard Catalent’s team just in time.

The company was going through a surge of changes, and she had been brought over from her role as CCO of West Pharmaceutical Services to serve in the same capacity for the New Jersey-based CDMO. Then a few months later, the pandemic was in full-force.

Since then, Catalent’s been in hyper-expansion mode. In early May, it acquired Promethera’s Hepatic Cell Therapy Support SA subsidiary and its 32,40-square-foot facility in Gosselies, Belgium. Prior to that, the company acquired Belgian CDMO Delphi Genetics, wrapped up the expansion of an already-existing site in Madison, WI and added an ultra-low temperature freezer partner in Sterling. As Emergent has botched millions of doses of AstraZeneca’s vaccine, the company has swooped in to move that production to its Maryland plant as well.

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Richard Pazdur (vis AACR)

FDA en­cour­ages in­clud­ing in­cur­able can­cer pa­tients in tri­als, re­gard­less of pri­or ther­a­pies

The FDA on Thursday called to include those with incurable cancers (when there is no potential for cure or for prolonged/near normal survival) in appropriate clinical trials, regardless of whether they have received existing alternative treatments.

Historically, many cancer clinical trials have required that participating patients previously received multiple therapies, according to Richard Pazdur, director of the FDA’s Oncology Center of Excellence.

On heels of Aduhelm ap­proval, Bris­tol My­ers jumps back in­to Alzheimer's race

Bristol Myers Squibb last put major resources behind an Alzheimer’s drug nearly a decade ago, when their own attempt at targeting amyloid flamed out in mid-stage studies. They invented another molecule, a Tau-targeted antibody, but jettisoned it to Biogen in 2017 as they dropped out of neuroscience altogether.

But on Thursday, the New York pharma announced they were getting back in the game. Bristol Myers exercised an $80 million option to bring a tau-targeted antibody from Prothena into a Phase I study. The opt-in, which Bristol Myers triggered ahead of analyst expectations, opens the door for another $1.7 billion in milestones down the road.

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James Peyer, Cambrian

Can a cell ther­a­py treat mus­cu­lar dy­s­tro­phy? A Ger­man bil­lion­aire's an­ti-ag­ing start­up is try­ing to find out

Gene therapy companies have faced huge hurdles trying to deliver healthy genes into muscular dystrophy patients’ muscle cells, so here’s an idea: Why don’t we just replace the muscle cells themselves?

Over the last two years, Vita Therapeutics has been exploring that possibility, building on early stem cell work from Johns Hopkins professor Peter Andersen. And on Tuesday they announced a $32 million Series A to begin to move their first therapy into the clinic, where they hope it will help rebuild muscle in patients with a type of dystrophy that afflicts the arms and legs.