Pre­ci­sion Bio­Sciences backs off-the-shelf can­cer cell ther­a­py ef­fort with new man­u­fac­tur­ing plant

The field of off-the-shelf cell ther­a­peu­tics is grow­ing, with the promise of quick­er, cheap­er, and eas­i­er to man­u­fac­ture ther­a­pies than the first gen­er­a­tion of au­tol­o­gous CAR-Ts that im­pressed with their po­ten­cy but fell short on adop­tion due to man­u­fac­tur­ing wob­bles. And Pre­ci­sion Bio­Sciences — which is us­ing its gene edit­ing tech­nol­o­gy to de­vel­op cell based im­munother­a­pies de­rived from healthy donors in­stead of the pa­tient — is plan­ning ahead. On Thurs­day, the com­pa­ny un­veiled a new Durham, NC fa­cil­i­ty ded­i­cat­ed to mak­ing its off-the-shelf CAR-T prod­ucts.

No­var­tis’ pi­o­neer­ing CAR-T drug — Kym­ri­ah — was ap­proved by the FDA amidst great fan­fare in 2017, but adop­tion was ham­pered by man­u­fac­tur­ing woes. No­var­tis lat­er added ca­pac­i­ty, hav­ing bought cell and gene ther­a­py man­u­fac­tur­er Cell­for­Cure. These prod­ucts re­quire enor­mous man­u­fac­tur­ing heft — mak­ers first iso­late cells from the pa­tient, ma­nip­u­late them in the lab by adding chimeric anti­gen re­cep­tors to di­rect T cells to snuff out can­cer cells and then re-in­fuse them back in­to the pa­tient.

Durham, North Car­oli­na-based Pre­ci­sion is hard­ly the first drug de­vel­op­er that has tak­en note of these chal­lenges. Take­da, for in­stance, which plans to take its off-the-shelf CAR-Ts in­to the clin­ic in 2021, is in the fi­nal stages of com­plet­ing its in-house man­u­fac­tur­ing fa­cil­i­ty in the Boston area, which is armed with the ca­pac­i­ty to sup­ply a range of clin­i­cal tri­als.

For Pre­ci­sion $DTIL, this new plant is part of a larg­er in­vest­ment in its can­cer im­munother­a­py plat­form and fa­cil­i­ties ex­pan­sions in North Car­oli­na this year.

“(W)e’ve known from the be­gin­ning that it was crit­i­cal for us to ad­dress the need for scal­able man­u­fac­tur­ing of cell-ther­a­py prod­ucts…” not­ed co-founder and chief Matt Kane in a state­ment.

“[The plant] al­so has the po­ten­tial to be a com­mer­cial launch fa­cil­i­ty with the ca­pac­i­ty to gen­er­ate up to 10,000 dos­es of CAR T cell ther­a­pies and 4,000 dos­es of gene ther­a­pies per year,” he added.

The drug de­vel­op­er is aim­ing to have its cer­ti­fi­ca­tions in place to be­gin man­u­fac­tur­ing in the fourth quar­ter — and plans to use the fa­cil­i­ty to cre­ate drug in­gre­di­ents for Phase I/II clin­i­cal tri­als sched­uled to be­gin next year.

Op­ti­miz­ing Cell and Gene Ther­a­py De­vel­op­ment and Pro­duc­tion: How Tech­nol­o­gy Providers Like Corn­ing Life Sci­ences are Spurring In­no­va­tion

Remarkable advances in cell and gene therapy over the last decade offer unprecedented therapeutic promise and bring new hope for many patients facing diseases once thought incurable. However, for cell and gene therapies to reach their full potential, researchers, manufacturers, life science companies, and academics will need to work together to solve the significant challenges facing the industry.

Amid mon­key­pox fears, biotechs spring to ac­tion; Mod­er­na’s CFO trou­ble; Cuts, cuts every­where; Craft­ing the right pro­teins; 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.

It’s always a bittersweet moment saying goodbye, but as Josh Sullivan goes off to new adventures we are grateful for the way he’s built up the Endpoints Manufacturing section — which the rest of the team will now carry forward. If you’re not already, this may be a good time to sign up for your weekly dose of drug manufacturing news. Thank you for reading and wish you a restful weekend.

Bay­er sounds re­treat from a $670 mil­lion CAR-T pact in the wake of a pa­tient death

Two months after Atara Biotherapeutics hit the hold button on its lead CAR-T 2.0 therapy following a patient death, putting the company under the watchful eye of the FDA, its Big Pharma partners at Bayer are bowing out of a $670 million global alliance. And the move is forcing a revamp of Atara’s pipeline plans, even as research execs vow to continue work on the two drugs allied with Bayer 18 months ago, which delivered a $60 million cash upfront.

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Try­ing to shake up the Parkin­son's par­a­digm, Ab­b­Vie sub­mits NDA for con­tin­u­ous, 24-hour in­fu­sion ther­a­py

AbbVie is approaching the FDA with a new therapy to potentially treat Parkinson’s disease, using prodrugs of two medications commonly used for the condition.

The Big Pharma submitted its NDA for ABBV-951, a solution of levodopa and carbidopa prodrugs being evaluated in advanced Parkinson’s patients who don’t respond well to oral therapy, AbbVie announced Friday morning. Researchers are hoping a positive Phase III study that reads out in late October will help move things along quickly at the agency.

Sanofi and Re­gen­eron clear the fin­ish line in an in­flam­ma­to­ry esoph­a­gus dis­ease, leav­ing Take­da in the dust

With atopic dermatitis rivals breathing down Dupixent’s neck, Sanofi and Regeneron on Friday secured a first win in new territory in what Sanofi’s head of immunology and inflammation Naimish Patel called the fastest approval he’s ever seen.

The FDA approved Dupixent on Friday to treat patients 12 years and older with eosinophilic esophagitis (EoE), an inflammatory condition that causes swelling and scarring of the esophagus. The approval came just a couple months after regulators granted Dupixent priority review, and months ahead of its PDUFA date on Aug. 3.

Fu­ji­film con­tin­ues its biotech build­ing spree with new fa­cil­i­ty in Chi­na

A Japanese conglomerate is making a big play in China with the opening of a new facility, as it continues to expand.

Fujifilm Irvine Scientific has opened its new Innovation and Collaboration Center in Suzhou New District, China, an area in Jiangsu province specifically designated for technological and industrial development.

According to Fujifilm, the 12,000-square-foot site will be responsible for the company’s cell culture media optimization, analysis and design services. Cell culture media itself often requires customization of formulas and protocols to achieve the desired quantity and quality of therapeutic desired. Fujifilm Irvine Scientific is offering these services from its headquarters in California and Japan to its customers globally, as well as in China now.

Emer Cooke, EMA director (AP Photo/Geert Vanden Wijngaert)

Ahead of FDA, EMA rec­om­mends au­tho­riz­ing new gene ther­a­py treat­ment for ul­tra-rare dis­ease

Aromatic amino acid decarboxylase (AADC) deficiency is an ultra-rare genetic disease that leaves patients unable to produce certain hormones in the brain, such as dopamine and serotonin, usually leading to developmental delays, weak muscle tone and inability to control the movement of the limbs. It can also lead to multiple organ failure.

To date, there have been no treatments approved for AADC deficiency, which has been identified in less than 150 patients.

Ather­sys tries to post-hoc-an­a­lyze its way out of an­oth­er tri­al fail for stroke stem cell ther­a­py

Athersys’ stem cell therapy has failed yet again.

In a 206-person trial conducted in Japan, Athersys’ stem cell therapy for stroke failed its primary endpoint of “excellent outcome,” a combined measure of three stroke recovery scores.

While a greater percentage of patients in the treatment group reached the primary endpoint compared to placebo, that difference was not statistically significant.

Siddhartha Mukherjee (Brian Ach/Getty Images for The New Yorker)

All Blue's $733M bid to ac­quire Zymeworks turns hos­tile as board bat­tles back — af­ter a biotech celebri­ty jumps in

Yesterday, the team at All Blue Capital — bent on the takeover of a badly battered Zymeworks — brought in celebrated oncologist, Pulitzer prize-winning writer and biotech exec Siddhartha Mukherjee to add some glitz to their proposed board. But they’re still not winning over any converts.

This morning, Zymeworks’ board officially turned this acquisition offer into a hostile showdown, rejecting the unsolicited offer and marshaling its forces to prevent a buyout at $10.50 per share.

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