Geoff von Maltzahn, Tessera Therapeutics CEO

Flag­ship-found­ed Tessera fu­els up with $300M 'to go on a re­al­ly big ad­ven­ture' in gene edit­ing

A few years ago, the gene edit­ing ap­proach at Tessera was just a “twin­kle in our eye,” CEO and co-founder Ge­off von Maltzahn said.

Nu­cle­as­es — the cru­cial ma­chin­ery un­der­ly­ing the gene edit­ing field — do a pret­ty good job of cut­ting DNA, al­low­ing sci­en­tists to switch off a spe­cif­ic gene. But von Maltzahn and Ja­cob Rubens, Tessera’s co-founder and chief in­no­va­tion of­fi­cer, saw the op­por­tu­ni­ty in be­ing able to do more.

Since launch­ing in 2018, Tessera has cre­at­ed mul­ti­ple plat­forms for what von Maltzahn calls gene writ­ing, or the abil­i­ty to write a short or long se­quence in­to the genome au­tonomous­ly and with­out mak­ing dou­ble strand breaks.

“Each of those three plat­forms is able to do things that are quite dis­tinc­tive and im­por­tant, and of­fer the po­ten­tial for a whole port­fo­lio of fu­ture clin­i­cal in­di­ca­tions and med­i­cines to come,” he said.

But, as the CEO says: “You got­ta have fu­el in the tank to go on a re­al­ly big ad­ven­ture.”

On Tues­day, Tessera un­veiled a rough­ly $300 mil­lion Se­ries C round, bring­ing the com­pa­ny’s to­tal raise past $500 mil­lion. A glob­al syn­di­cate of in­vestors dug in­to their wal­lets for this round, in­clud­ing the biotech’s founder Flag­ship Pi­o­neer­ing.

Al­so chip­ping in were: a sub­sidiary of the Abu Dhabi In­vest­ment Au­thor­i­ty; the Alas­ka Per­ma­nent Fund Cor­po­ra­tion; Al­ti­tude Life Sci­ence Ven­tures; AR­TIS Ven­tures; Cor­morant As­set Man­age­ment; Han­wha Im­pact Part­ners; Longevi­ty Vi­sion Fund; March Cap­i­tal; the SALT Fund; Soft­Bank Vi­sion Fund 2; funds and ac­counts ad­vised by T. Rowe Price As­so­ci­ates, and oth­ers.

The com­pa­ny is de­ploy­ing a three-tiered plat­form — sans CRISPR — us­ing mo­bile ge­net­ic el­e­ments in pur­suit of some “re­al­ly sen­sa­tion­al med­i­cines,” von Maltzahn said.

The cat­e­go­ry of “gene writ­ers,” von Maltzahn said, are RNA gene writ­ers that can rewrite the genome, chang­ing any one base pair to an­oth­er or mak­ing small in­ser­tions or dele­tions.

The sec­ond ap­proach is “to give just two RNAs to hu­man cells, and have those cells wake up the next day with a DNA gene in their genome.”

“That’s pos­si­ble be­cause the ma­chin­ery that we bor­rowed from is able to re­verse tran­scribe an RNA se­quence in­to the form of DNA, and so we de­liv­er an mR­NA en­cod­ing and gene writer pro­tein, it grabs on­to the oth­er RNA, takes it to the genome and then one let­ter at a time it lit­er­al­ly writes new DNA in­to that lo­ca­tion on the genome,” he said.

Those two ap­proach­es would al­low sci­en­tists to make mod­i­fi­ca­tions to the genome with no vi­ral vec­tors, open­ing the door for “in­cred­i­ble” scal­a­bil­i­ty, cus­tomiza­tion and speed, the CEO ex­plained.

The third cat­e­go­ry is what von Maltzahn calls DNA gene writ­ers, in which they “de­liv­er an mR­NA that then codes for a pro­tein that grabs on­to the loop of DNA, takes it to the genome and au­tonomous­ly in­serts it, and that of­fers the po­ten­tial to al­low AAVs to be cures for ge­net­ic dis­eases in di­vid­ing cell types like the liv­er,” he said.

The Se­ries C funds will be used to “ag­gres­sive­ly in­vest in the plat­form,” move pro­grams to­ward the clin­ic, build up man­u­fac­tur­ing ca­pa­bil­i­ties, and ex­pand the rough­ly 200-per­son team. The first fo­cus will be on liv­er and rare ge­net­ic dis­ease, sick­le cell, and en­gi­neer­ing CAR-T cells “both out­side and in­side the body.”

Von Maltzahn de­clined to com­ment on how soon those pro­grams might reach the clin­ic. And when asked if he’s plan­ning a pub­lic de­but, he re­spond­ed: “No plans to go pub­lic in the near fu­ture.”

“[What] fi­nanc­ings with the splashy head­lines… of­ten miss is just how much sci­en­tif­ic un­cer­tain­ty and courage and team­work and re­solve it takes to climb these kinds of moun­tains,” he said. “We feel re­al­ly for­tu­nate to have the op­por­tu­ni­ty to build the com­pa­ny we’ve been dream­ing of.”

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.

Joshua Cohen (L) and Justin Klee, Amylyx co-CEOs

BREAK­ING: Af­ter long and wind­ing road, FDA ap­proves Amy­lyx's ALS drug in vic­to­ry for pa­tients and ad­vo­ca­cy groups

For just the third time in its 116-year history, the FDA has approved a new treatment for Lou Gehrig’s disease, or ALS.

US regulators gave the thumbs-up to the drug, known as Relyvrio, in a massive win for patients and their families. The approval, given to Boston-area biotech Amylyx Pharmaceuticals, comes after two years of long and contentious debates over the drug’s effectiveness between advocacy groups and FDA scientists, following the readout of a mid-stage clinical trial in September 2020.

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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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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.

FDA's ad­vanced ther­a­pies of­fice pro­vides more clar­i­ty on gene ther­a­py CMC con­sid­er­a­tions

As the Office of Tissue and Advanced Therapies (OTAT) transforms into the Office of Therapeutic Products (OTP), with new user fee funds and “super office” status, the department focused on cell and gene therapies also opened its doors to a town hall Thursday offering clarification on guidance and regulations for manufacturers.

Some of the major concerns from manufacturers were the CMC considerations between first-in-human studies and late-phase studies supporting a marketing approval.

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Some­one old, some­one new: Mod­er­na pro­motes CTO, raids No­var­tis for re­place­ment amid pipeline push

Moderna CEO Stéphane Bancel made clear on the last quarterly call that “now is not the time to slow down.” On Thursday, he made a bit more room in the cockpit.

The company unveiled a new executive role on Thursday, promoting former chief technical operations and quality officer Juan Andres to president of strategic partnerships and enterprise expansion, and poaching a former Novartis exec to take his place.

Cell and gene ther­a­pies from acad­e­mia: EMA to help 5 projects go­ing af­ter un­met clin­i­cal needs

The European Medicines Agency said Thursday that it’s launching a new pilot program to help academic and other nonprofit researchers developing advanced therapy medicinal products, which includes cell and gene therapies.

Academics have proven to be enormously useful in feeding new products, like chimeric antigen receptor (CAR)-T cell therapies first developed by Memorial Sloan Kettering, and ushered to the market by biopharma companies. Jean Bennett, formerly with the University of Pennsylvania, also saw her research lead to the approval of gene therapy Luxturna, which Roche now owns.

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.

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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