Mer­ck KGaA adds to gene ther­a­py man­u­fac­tur­ing boom with €100M fa­cil­i­ty in Cal­i­for­nia

Add a new, €100 mil­lion Cal­i­for­nia fa­cil­i­ty to the grow­ing gene ther­a­py in­fra­struc­ture.

Mer­ck KGaA an­nounced they are open­ing a sec­ond gene ther­a­py and vi­ral vec­tor fac­to­ry in Carls­bad, Cal­i­for­nia. The new cen­ter will be just one point in the glob­al sup­ply chain Big Phar­ma is rapid­ly erect­ing to keep man­u­fac­tur­ing ca­pac­i­ty for the new tech­nol­o­gy at pace with clin­i­cal de­vel­op­ment. Over the past year, No­var­tis, PTC Ther­a­peu­tics, Pfiz­er and Ver­tex each an­nounced or opened new fa­cil­i­ties in Switzer­land, North Car­oli­na and New Jer­sey that will help build gene ther­a­py.

In No­vem­ber, Reuters re­port­ed that 11 drug­mak­ers had set aside $2 bil­lion for the man­u­fac­tur­ing ef­fort. They were led by No­var­tis, the gi­ant be­hind the sec­ond FDA-ap­proved gene ther­a­py in Zol­gens­ma which planned to spend $500 mil­lion, and Pfiz­er, which has yet to get a gene ther­a­py ap­proved but will spend $600 mil­lion. Catal­ent and Ther­mo Fish­er, mean­while, each spent over a bil­lion dol­lars ac­quir­ing com­pa­nies in­volved in gene ther­a­py or vi­ral vec­tor man­u­fac­tur­ing.

Mer­ck KGaA last shored up its gene ther­a­py man­u­fac­tur­ing in 2016 — the year be­fore the first gene ther­a­py was ap­proved in the US — when they ex­pand­ed their orig­i­nal Carls­bad fa­cil­i­ty from 44,000 to 65,000 feet. At 140,000 feet, the new fa­cil­i­ty will be more than dou­ble the size of its old­er neigh­bor. Gene ther­a­pies and the vi­ral vec­tors used to de­liv­er them will be cooked up in 1,000 liter biore­ac­tors — met­al vats rough­ly the size of 260 gal­lons of milk. Mer­ck has not said when it is sched­uled for com­ple­tion.

Al­though Mer­ck KGaA lists no gene ther­a­pies in its most re­cent pipeline, the Ger­man drug­mak­er us­es its fa­cil­i­ties to help biotechs fo­cused on the emerg­ing modal­i­ty to man­u­fac­ture their prod­ucts. The list of ap­proved gene ther­a­pies can still be count­ed on one hand, but hun­dreds are now in clin­i­cal tri­als.

Di­ane Blu­men­thal

Man­u­fac­tur­ing emerged as a press­ing bot­tle­neck al­most as soon as Spark Ther­a­peu­tics got Lux­tur­na, a treat­ment for a form of in­her­it­ed blind­ness, ap­proved as the US’ first gene ther­a­py. In an op-ed in STAT last year, Spark’s head of tech­ni­cal op­er­a­tions,  Di­ane Blu­men­thal, de­scribed the process of build­ing in Philadel­phia one of the first in-house gene ther­a­py man­u­fac­tur­ing fa­cil­i­ties.  With reg­u­la­tors in­creas­ing­ly open to ap­prov­ing the treat­ments quick­ly, she en­cour­aged oth­er com­pa­nies to in­vest in man­u­fac­tur­ing in ad­vance of even know­ing if the ther­a­py works, par­tic­u­lar­ly be­cause gene ther­a­py re­quires cus­tomiza­tion in ways few oth­er modal­i­ties do.

And she warned the prob­lem will on­ly get more press­ing.

“There isn’t a gene ther­a­py man­u­fac­tur­ing play­book — yet — to guide the de­vel­op­ment of gene ther­a­pies,” Blu­men­thal wrote. “Man­u­fac­tur­ing a gene ther­a­py is on­ly half the bat­tle. The oth­er half is mak­ing enough of it, do­ing that as ef­fi­cient­ly as pos­si­ble, and get­ting it to the pa­tients who need it. These chal­lenges be­come even more ur­gent to tack­le as the in­dus­try shifts to the next chap­ter in gene ther­a­py de­vel­op­ment, from treat­ments made in small batch­es for small pa­tient pop­u­la­tions to big­ger vol­umes for larg­er rare-dis­ease pop­u­la­tions and com­mer­cial scale.”

At the In­flec­tion Point for the Next Gen­er­a­tion of Can­cer Im­munother­a­py

While oncology researchers have long pursued the potential of cellular immunotherapies for the treatment of cancer, it was unclear whether these therapies would ever reach patients due to the complexity of manufacturing and costs of development. Fortunately, the recent successful development and regulatory approval of chimeric antigen receptor-engineered T (CAR-T) cells have demonstrated the significant benefit of these therapies to patients.

All about Omi­cron; We need more Covid an­tivi­rals; GSK snags Pfiz­er’s vac­cine ex­ec; Janet Wood­cock’s fu­ture at FDA; and more

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Biospec­i­men M&A: Dis­cov­ery ac­quires Al­bert Li's he­pa­to­cyte project; PhI­II tri­al on Bay­er's Nube­qa reached pri­ma­ry end­point

Discovery Life Sciences has acquired what claims to be the Maryland-based host of the world’s largest hepatocyte inventory, known as IVAL, to help researchers select more effective and safer drug candidates in the future.

The combined companies will now serve a wider range of drug research and development scientists, according to Albert Li, who founded IVAL in 2004 and is set to join the Discovery leadership team as the CSO of pharmacology and toxicology.

Usama Malik

Ex-Im­munomedics CFO charged with in­sid­er trad­ing, faces up to 20 years in prison af­ter al­leged­ly tip­ping off girl­friend and rel­a­tives of a PhI­II suc­cess

The former CFO of Immunomedics, who helped steer the company to its $21 billion buyout by Gilead last year, has been charged with insider trading, the Department of Justice announced Thursday.

Usama Malik tipped off his then-girlfriend and four others that a Phase III study for Trodelvy would be stopped early four days before Immunomedics publicly announced the result in April 2020, DoJ alleged in its complaint. The individuals then purchased Immunomedics shares, selling them after the news broke and Immunomedics’ stock price doubled.

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Merck's new antiviral molnupiravir (Quality Stock Arts / Shutterstock)

As Omi­cron spread looms, oral an­tivi­rals ap­pear to be one of the best de­fens­es — now we just need more

After South African scientists reported a new Covid-19 variant — dubbed Omicron by the WHO — scientists became concerned about how effective vaccines and monoclonal antibodies might be against it, which has more than 30 mutations in the spike protein.

“I think it is super worrisome,” Dartmouth professor and Adagio co-founder and CEO Tillman Gerngross told Endpoints News this weekend. Moderna CEO Stéphane Bancel echoed similar concerns, telling the Financial Times that experts warned him, “This is not going to be good.”

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Ab­b­Vie tacks on a new warn­ing to Rin­voq la­bel as safe­ty frets crimp JAK class

The safety problems that continue to plague the JAK class as new data highlight some severe side effects are casting a large shadow over AbbVie’s Rinvoq.

As a result of a recent readout highlighting major adverse cardiac events (MACE), malignancy, mortality and thrombosis with Xeljanz a couple of months ago, AbbVie put out a notice late Friday afternoon that it is adding the new class risks to its label for their rival drug.

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Pfiz­er, Am­gen and Janssen seek fur­ther clar­i­ty on FDA's new ben­e­fit-risk guid­ance

Three top biopharma companies are seeking more details from the FDA on how the agency conducts its benefit-risk assessments for new drugs and biologics.

While Pfizer, Amgen and Janssen praised the agency for further spelling out its thinking on the subject in a new draft guidance, including a discussion of patient experience data as part of the assessment, the companies said the FDA could’ve included more specifics in the 20-page draft document.

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Re­searchers move clos­er to de­ci­pher­ing blood clots from As­traZeneca, J&J's Covid-19 vac­cines

Researchers may be nearing an answer for the mysterious and life-threatening blood clots that appeared on very rare occasions in people who received the J&J or AstraZeneca Covid-19 vaccine.

The new work builds on an early hypothesis researchers in Norway put forward last spring, when the cases first cropped up. They proposed the events were similar to blood clots that can occur in a small subset of patients who receive heparin, one of the most commonly used blood thinners.

Janet Woodcock (AP Images)

Janet Wood­cock plots her fu­ture at FDA, with se­nior ad­vi­sor role to fall back on if Califf wins con­fir­ma­tion

Acting FDA commissioner Janet Woodcock has been the face of just about every drug approval decision at the agency since the turn of the century. Since the pandemic began, she’s moved between the top of the drugs center to the head of therapeutics at Operation Warp Speed, leading the drive for work on Covid-targeted mAbs and antivirals.

Looking forward — and pending a quick Senate confirmation to cement Rob Califf’s return to the top of FDA early next year — Woodcock’s role at the agency will again be in flux.

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