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

Ray Tabibiazar, SalioGen CEO

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, $150 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.

Their Staying Power Lies in their Patient-Centricity

Decentralized clinical trials (DCTs) were traditionally utilized in an isolated fashion prior to the COVID-19 pandemic. To continue their research within the constraints of the pandemic, sponsors and clinical investigators pivoted to a decentralized model out of necessity. At the onset, regulatory agencies offered some guidance on the digital approaches that are acceptable to ensure DCT approaches are applied in a way that maintains patient safety, as well as data quality and integrity.

Patrick Collison, co-founder of Stripe, has become one of Silicon Valley’s biggest advocates for new forms of funding and conducting science (Matt Winkelmeyer/Getty Images for WIRED)

It’s big days for biology.

The pandemic has seen a series of very public scientific breakthroughs: mRNA vaccine, Covid antibodies, CRISPR as therapy. The minds behind these advancements have graced magazine covers and received prestigious awards.

But the last two years have also, far more quietly, seen a series of new experiments in how to fund the next generation of scientific breakthroughs.

Since March 2020, investors, academics, a significant number of Silicon Valley types, at least one Russian billionaire and two crypto billionaires and, most recently, a few West Coast universities have launched a series of grant programs, institutes, NGOs and companies hoping to change how life science research is done. Though unaffiliated and varying greatly in both size and form, they have broadly promised to evade bureaucracy and misaligned incentives and advance both basic and not-so-basic research in ways they say can’t be done in either conventional academia or profit-focused biotech.

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Michael Heffernan, Avenge Bio CEO

Michael Heffernan was already the head of a biotech company focused on chronic pain, called Collegium Pharmaceutical, which he successfully brought public in 2015. But when his wife was diagnosed with ovarian cancer, he knew that was his next mission.

Heffernan’s wife is doing well — but unfortunately, that isn’t the case for most patients, he said. In 2018, he stepped down from Collegium to look for a better option for women with recurrent refractory ovarian cancer. That’s when he met Rice University’s Omid Veiseh, who was working on an immunotherapy platform with a physician over at The University of Texas MD Anderson Cancer Center.

BioNTech CEO Ugur Sahin (Photo by ddp images/Sipa USA)

One of the biggest storylines of biotech’s next half-decade will be where Moderna, BioNTech and their growing list of rivals push the mRNA technology that’s already changed the world.

BioNTech and Pfizer gave part of the answer Wednesday, announcing a new partnership to co-develop an mRNA-based shingles vaccine. The candidate will be ready for early-stage trials in the second half of 2022, the companies said.

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The FDA quietly announced on Tuesday that just before New Year’s eve, the agency again paused all of its non-mission-critical inspections in the US, likely leading to an even larger backlog of inspections worldwide.

The pause, which will last at least two weeks, is meant to ensure the safety of FDA employees and the companies it regulates as the agency further adapts to the spread of Omicron.

For anyone who’s been following how the US government has been allocating and shipping supplies of its Covid-19 treatments over the past year, the news has shifted so many times that it can be difficult to keep track of what’s still being shipped and where.

More change is coming this week too, as HHS has now decided to re-start shipments of both Eli Lilly (bamlanivimab plus etesevimab) and Regeneron (casirivimab plus imdevimab) monoclonal antibody products after a short pause because neither product works against the new variant Omicron. Lilly’s combo also was halted last June due to the presence of other variants.

Kicking off 2022, hundreds of pharmaceuticals, including some blockbusters, saw their list prices rise by about 5% on average. But overall, net drug prices (cost after rebates) declined for the fourth year in a row, potentially complicating already stalled drug price reform efforts.

Among the drugs seeing new increases as of Jan. 1 are Gilead’s bevy of blockbuster HIV drugs.

Biktarvy, which pulled in more than $7 billion in worldwide sales in 2020, saw a 4.8% price increase in 2021, and now, another 5.6% increase in 2022, according to a new report from the nonprofit 46brooklyn Research.

All the big R&D trends are on display in this new list of drug approvals for 2021. Plus one.

Add up everything OK’d from CDER and CBER, and you have 60 new drug approvals for last year, topping the 59 in 2020. That’s a close second to the 64 OKs that came out of the FDA in 2018. The dark days of the early 2000s are a distant memory now, with a host of hungry upstarts promising to make their own entries one day as Big Pharmas double down on innovation.

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Shoshana Shendelman, Applied Therapeutics CEO/founder

When the FDA lifted a clinical hold on Applied Therapeutics’ lead program in galactosemia last February, the New York biotech signaled that they were then on a smooth road toward an accelerated approval, with plans to file an NDA in the third quarter of 2021.

Regulators, though, apparently changed their mind.

Applied has decided to hold on submitting an NDA for AT-007 as a treatment for galactosemia, the company disclosed, following discussions with the FDA in which the agency indicated that ‘clinical outcomes data will likely be required for approval.’
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