Emile Nuwaysir, BlueRock CEO

Look­ing to build new smart cells, Blue­Rock teams with Tim Lu's Sen­ti Bio in 'sec­ond-or­der' en­gi­neer­ing al­liance

Blue­Rock, the Ver­sant-backed and Bay­er-owned biotech best known for its Parkin­son’s cell ther­a­py, has been qui­et­ly lay­ing out much grander plans for their tech­nol­o­gy.

The com­pa­ny, CEO Emile Nuwaysir ex­plains, doesn’t just want to recre­ate the healthy hu­man cell — as they’ve done in Parkin­son’s — but al­so to ma­nip­u­late it or add whole new fea­tures: a so-called kill-switch that al­lows doc­tors to con­trol cells af­ter they’ve been im­plant­ed, for ex­am­ple, or mol­e­c­u­lar “sen­sors” that pre­vent the cell from func­tion­ing any­where but in the pre­cise tis­sue it’s need­ed.

“Some of that is pret­ty sim­ple,” Nuwaysir said. “But some of that is sec­ond-or­der en­gi­neer­ing.”

Tim Lu

For that sec­ond-or­der en­gi­neer­ing, Nuwaysir de­cid­ed Blue­Rock could use some help. On Wednes­day, they signed a col­lab­o­ra­tion with Sen­ti Bio, the syn­thet­ic bi­ol­o­gy and cell cir­cuit­ry biotech found­ed and led by MIT’s Tim Lu. The two groups will work on de­vel­op­ing mol­e­c­u­lar “sen­sors” and “di­als” Blue­Rock can pro­gram in­to their cell ther­a­pies.

Out­side of Parkin­son’s, Blue­Rock has yet to dis­close any of their pro­grams, ex­cept to say they are among the vast num­ber of dis­eases and cell types that fall un­der the cat­e­gories of im­munol­o­gy, neu­rol­o­gy, and car­di­ol­o­gy. So Nuwaysir was re­luc­tant to talk about specifics.

He said, how­ev­er, that the part­ners would fo­cus on feats of en­gi­neer­ing more dif­fi­cult than adding a kill switch or oth­er small mod­i­fi­ca­tions. In­stead, they’ll try to use Sen­ti’s knowl­edge of the in­tri­cate ge­net­ic cir­cuit­ry that gov­ern how cells func­tion to cre­ate cell ther­a­pies that on­ly turn “on” when they see a par­tic­u­lar mark­er of a dis­ease, in the area cen­tral to that dis­ease: for ex­am­ple, when the tis­sue is in­flamed and they’re next to a spe­cif­ic cell type.

Sen­ti has demon­strat­ed one of the ways that ap­proach can work in can­cer. In a process called log­ic-gat­ing, they equip a T cell with a re­cep­tor for a pro­tein ex­pressed on both tu­mors and healthy tis­sues. When the cell sees that pro­tein, it doesn’t at­tack, like stan­dard CAR-T would. In­stead, it ac­ti­vates a sec­ond re­cep­tor and, like nu­clear sub­marines that fire on­ly when both crew mem­bers turn their keys, on­ly at­tacks if the sec­ond re­cep­tor finds its tar­get. The method can in the­o­ry pre­vent CAR-T from in­ad­ver­tent­ly de­stroy­ing healthy cells.

The com­pa­nies al­so in­di­cat­ed in their re­lease that they would pro­gram cir­cuits that let doc­tors fine­ly tune the cells’ func­tion with small mol­e­cules — es­sen­tial­ly a bright­ness and dim­ming switch, rather than a kill switch.

Nuwaysir wouldn’t give time­lines for the col­lab­o­ra­tion, ex­cept to say that it was very ear­ly stage: Don’t ex­pect it to im­pact Parkin­son’s, which they hope to bring in­to the clin­ic soon, or any of the pro­grams in de­vel­op­ment be­hind it.

“It re­quires a lev­el of so­phis­ti­ca­tion that the field is just learn­ing,” he said.

Un­pack­ing the Aduhelm de­ci­sion, Ver­tex's half full glass, a $525M J&J breakup, and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

By now you have surely read about the FDA’s controversial approval of Biogen’s Alzheimer’s drug and all its reverberations. But I’d still recommend checking out the meaty recap below to make sure you didn’t miss all the angles that the Endpoints team has covered. If you’d rather look ahead, look no further than our three-day virtual panels next week at BIO, where we will discuss what the new normal means for every part of the industry.

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What does a clear ma­jor­i­ty of the bio­phar­ma in­dus­try think of the FDA ap­proval of ad­u­canum­ab? 'Hor­ri­fy­ing' 'Dan­ger­ous' 'Con­fus­ing' 'Dis­as­ter'

Over the years, we’ve become used to seeing a consensus emerge early in our industry polls at Endpoints News. And when we took the pulse of drug hunters on the heels of a controversial FDA approval for aducanumab this week, it became immediately apparent that the vast majority of our readers — heavily concentrated among biopharma staffers and execs — were incensed by what they had just witnessed.

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Aaron Kesselheim (Scott Eisen/AP Images for AIDS Healthcare Foundation)

Har­vard’s Aaron Kessel­heim re­signs from ex­pert pan­el in wake of ad­u­canum­ab OK, blast­ing FDA for ‘worst drug ap­proval de­ci­sion in re­cent U.S. his­to­ry'

A third member of the FDA’s Peripheral and Central Nervous System Drugs Advisory Committee has resigned in the wake of Biogen’s controversial Aduhelm approval, slamming the agency as he left and further deepening the controversy surrounding the decision.

Harvard University professor Aaron Kesselheim quit in protest Thursday afternoon, calling the Aduhelm OK “probably the worst drug approval decision in recent U.S. history.” Kesselheim follows both Joel Perlmutter, a neurologist from Washington University in St. Louis, and David Knopman, a neurologist from the Mayo Clinic, out the door.

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David Knopman (Mayo Clinic via YouTube)

A sec­ond ad­comm mem­ber aban­dons his post in af­ter­math of con­tro­ver­sial ad­u­canum­ab de­ci­sion

As the fallout from the FDA’s approval of Alzheimer’s med aducanumab grows, a second member of the adcomm overseeing that drug’s review has walked away. But even with two experts now having resigned from that committee in protest, is there enough broad-level outrage to prevent another aducanumab from getting approved?

The FDA on Wednesday lost another member of its Peripheral and Central Nervous System Drugs Advisory Committee as Mayo Clinic neurologist David Knopman hit the exit over the agency’s decision to approve Biogen’s Alzheimer’s drug Aduhelm despite the committee’s near-unanimous vote against it.

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FDA au­tho­rizes about 10M J&J vac­cine dos­es, trash­es 60M more from trou­bled Emer­gent plant

The FDA on Friday released about 10 million doses of J&J’s vaccine for use, and disposed of another 60 million doses that were manufactured at the now-shuttered Emergent BioSolutions facility in Baltimore where cross-contamination occurred.

The agency said it’s not yet ready to allow the Emergent plant to be included in the J&J EUA, but that may occur soon. FDA came to the decision to authorize some of the doses after reviewing facility records and quality testing results.

Ver­tex and CRISPR Ther­a­peu­tics un­veil more pos­i­tive gene ther­a­py da­ta, but busul­fan again casts a shad­ow over the field

Less than 12 hours after revealing a flop on its second shot for alpha-1 antitrypsin deficiency, Vertex plowed ahead with another data drop from its partnership with CRISPR Therapeutics. And though the topline proved positive, concerns over conditioning agents continue to linger over the collaboration, as well as the entire gene therapy space.

Presenting data from two trials at the European Hematology Association annual meeting, the pair announced that follow-up data of at least three months for 22 patients with genetic blood disorders indicated a “consistent and sustained” response to the experimental drug CTX001. All 15 patients with transfusion-dependent beta thalassemia did not need further blood transfusions and all seven with severe sickle cell disease were pain free, the biotechs announced.

Janet Woodcock, acting FDA commissioner, at Thursday's Senate Appropriations hearing (Bill Clark/CQ Roll Call via AP Images)

Sen­a­tors lam­bast new Alzheimer’s drug’s price but give Janet Wood­cock a free pass on the ap­proval de­ci­sion

Senate Finance Democrats took aim at Biogen’s pricey new Alzheimer’s drug on Thursday, but members on both sides of the aisle at a separate appropriations hearing didn’t question acting FDA commissioner Janet Woodcock on the approval.

“I was appalled that Biogen priced their Alzheimer’s drug approved by the FDA at $56,000 per year — I’m not going to debate whether this is effective or not, but it’s double the household median income for Michiganders over the age of 65,” Sen. Debbie Stabenow (D-MI) said at the finance hearing.

Reshma Kewalramani, Vertex CEO (BIO via YouTube)

UP­DAT­ED: Ver­tex strikes out on its lat­est big shot at a rare ge­net­ic dis­ease. But they're go­ing to keep on swing­ing

It’s been several months since Vertex culled one of its small molecules for alpha-1 antitrypsin deficiency (AATD), taking a big hit after evidence of liver damage surfaced in a key Phase II trial. Now we learned that the company has whiffed on its second shot, and there’s nothing left in the clinic to treat the rare genetic disease — but that won’t stop it from trying.

Despite avoiding the safety issues that plagued the last candidate, Vertex $VRTX is taking the axe to VX-864 after Phase II results revealed the magnitude of the drug’s response is “unlikely to translate into substantial clinical benefit.” As a result of the news, the company’s stock fell 12.5% after hours.

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Bris­tol My­er­s' CAR-T Breyanzi busts out a win in ear­li­er-line lym­phoma, po­ten­tial­ly crack­ing open an ex­pand­ed mar­ket

Despite being third to the field in B cell lymphoma, Bristol Myers Squibb has repeatedly argued its CAR-T Breyanzi could have the juice to overtake its older competitors. Going into earlier lines of therapy may be the golden ticket on that front, and now Breyanzi has a late-stage win to back up that effort.

Bristol Myers’s Breyanzi beat out physicians’-choice salvage therapy followed by high-dose chemo and a stem cell transplant — what the drugmaker called a “gold standard treatment” — in second-line patients with relapsed or refractory large B cell lymphoma, according to topline data from the Phase III TRANSFORM study released Thursday.