CRISPR had no soon­er start­ed to shake the very foun­da­tions of drug de­vel­op­ment be­fore its lim­i­ta­tions be­gan to loom large. Gene edit­ing could change the world — if on­ly you could get around the hur­dles that threat­ened to trip up every pro­gram.

Blake By­ers

So it’s on­ly nat­ur­al to see CRISPR 2.0 tak­ing shape be­fore the pi­o­neers can get the lead ther­a­pies through de­vel­op­ment. And who bet­ter than Google’s GV ven­ture arm to take the lead spot in a small syn­di­cate back­ing some sci­en­tists with their own unique twist on a so­lu­tion?

GV gen­er­al part­ner Blake By­ers took point on this one, join­ing with some in­di­vid­ual in­vestors who are stay­ing in the back­ground. They were all at­tract­ed by the idea that the small team at Spot­light Ther­a­peu­tics could by­pass the de­liv­ery is­sues em­bed­ded in every gene edit­ing pro­gram by us­ing pro­gram­ma­ble CRISPR ri­bonu­cle­o­pro­teins de­signed for in vi­vo cell-tar­get­ed de­liv­ery.

And now the Spot­light team is jump­ing in­to the in­dus­try spot­light with a $30 mil­lion launch round to go af­ter lead pro­grams in he­mo­glo­binopathies and im­muno-on­col­o­gy.

Right now, de­spite all the promise of CRISPR, the pi­o­neers are un­able to ad­dress spe­cif­ic cell types in vi­vo, says CEO Mary Haak-Frend­scho. This new ap­proach of­fers the op­por­tu­ni­ty “to re­al­ly open up, de­moc­ra­tize gene edit­ing.”

Mary Haak-Frend­scho

The tech used to­day de­pends a lot on us­ing an AAV or LNP to ex­press the ed­i­tor in the tar­get cell, says By­ers. Those have bet­ter up­take in liv­er cells, so that’s where the ear­ly play­ers have start­ed. And as we’ve seen at Sol­id and oth­er places, there are lin­ger­ing con­cerns about safe­ty and ef­fi­ca­cy.

Spot­light, he says, is tak­ing a dif­fer­ent, com­plete­ly non-vi­ral ap­proach. And it starts with a ques­tion.

“Why don’t we bind the RNP (ri­bonu­cle­o­pro­tein) ed­i­tor to oth­er things?” asks By­ers. “That could be oth­er pro­teins, let’s say an an­ti­body. Get an­ti­bod­ies to a tar­get on the cell sur­face, it’s in­ter­nal­ized at some rate, then hitch­hikes in­to the cell and from there ed­it the tar­get site in the genome.”

“There’s a lot of things you can latch your gene ed­i­tors to,” says By­ers. The goal is to ex­plore the space, search­ing for 5 to 10 port­fo­lio prod­ucts bound to dif­fer­ent hitch­hik­er mol­e­cules that you can use to get your ed­i­tor of choice in­to the ex­act cell type you want, with high speci­fici­ty and low cost of goods.

Alex Mar­son

In the process, you have to es­cape de­struc­tion by the lyso­some and go on to the nu­cle­o­some to do the edit­ing, so there’s plen­ty of de­bug­ging along the way to get it to work right.

Much of the in­spi­ra­tion for this work came from 3 key sci­en­tists who are ad­vis­ing the start­up.

Alex Mar­son and Patrick Hsu thought a lot of this out. Mar­son is at UC San Fran­cis­co and Hsu is now close by at UC Berke­ley. Ja­cob Corn at ETH Zürich rounds out the line­up of sci­en­tif­ic founders with pres­ti­gious re­sumes.

Patrick Hsu

The longterm plan is to stick with the non-vi­ral ap­proach and build a pipeline.

As A rounds go these days, $30 mil­lion isn’t a lot of mon­ey, par­tic­u­lar­ly when you’re talk­ing about a tech plat­form ap­proach like this. I point­ed that out to By­ers, who wasn’t feel­ing apolo­getic about the $30 mil­lion.

“How old school of us,” he re­spond­ed, adding that there are dif­fer­ent strate­gies for dif­fer­ent play­ers. Some should be laser fo­cused on prod­uct de­vel­op­ment. Be­sides, he adds, “small mounts of cap­i­tal are very fo­cus­ing for a com­pa­ny.”

Ja­cob Corn

The mon­ey “gets us to 2022,” says the CEO. At that point they ex­pect to have their first de­vel­op­ment can­di­date, with­in a year of the clin­ic for ei­ther or both their cho­sen in­di­ca­tions.

And then they can see about start­ing their own gene edit­ing rev­o­lu­tion.

As Congress considers whether to adopt sweeping new legislation to lower prescription drug prices across the board, the Kaiser Family Foundation is out with a new report on Monday showing how a more targeted approach on a subset of drugs might be a more efficient way to save government funds.

“This analysis shows that Medicare Part D and Part B spending is highly concentrated among a relatively small share of covered drugs, mainly those without generic or biosimilar competitors,” wrote Juliette Cubanski, deputy director of the program on Medicare policy at KFF, and Tricia Neuman, SVP of KFF. “Focusing drug price negotiation or reference pricing on a subset of drugs that account for a disproportionate share of spending would be an efficient use of administrative resources, though it would also leave some potential savings on the table.”

Editor’s note: Interested in following biopharma’s fast-paced IPO market? You can bookmark our IPO Tracker here.

It was another busy week in the biotech IPO market as the second quarter continues to churn out significant investment into the sector.

Recursion led the way with a $436 million raise on Friday, pricing its IPO at $18, the high end of its range. Our own John Carroll went in depth on that raise over the weekend. Also on Friday, preclinical cancer biotech Biomea Fusion debuted with a $153 million raise priced at its own high end of $17 per share. The two companies helped push the combined IPO raise for 2021 to nearly $5.5 billion.

It’s always a surprise when a court has to step in to tell the FDA that it erred in performing one of its main duties: classifying whether a medical product is drug or a device.

But that’s what the US Court of Appeals for the District of Columbia did on Friday, making clear to the world’s top drug regulator that Genus Medical Technologies’ contrast agent barium sulfate (also known as Vanilla SilQ) should not be considered a drug, as the FDA had said, but a medical device.

Seagen is riding the wave of two new priority reviews straight to the FDA.

The Bothell, WA-based biotech and their partners at Astellas announced Monday that two supplemental BLAs for Padcev had been accepted by US regulators. FDA has set Aug. 17 as the PDUFA date for the reviews.

“With our recent regulatory submissions, we intend to provide the highest level of clinical evidence supporting Padcev use — overall survival data from a randomized Phase III trial — and expand availability in multiple countries where there is unmet medical need,” said Astellas oncology chief Andrew Krivoshik.