Geoff McDonough, CEO (Generation Bio)

Gen­er­a­tion Bio snaps up Boston area plant as it rolls out a man­u­fac­tur­ing process it thinks will be a game chang­er

Gen­er­a­tion Bio is look­ing to move the field of gene ther­a­py in­to the fu­ture, and thinks it has a man­u­fac­tur­ing process in place that will al­low enough scale for broad dis­ease in­di­ca­tions. Now, all it needs is a plant to put those large-scale am­bi­tions in­to ac­tion.

Gen­er­a­tion signed a lease on a new 104,000-square-foot fa­cil­i­ty in Waltham, MA, set to be­come op­er­a­tional in 2023. The fa­cil­i­ty will be avail­able for pre­clin­i­cal and clin­i­cal de­vel­op­ment for its lead he­mo­phil­ia A pro­gram.

The shell is set up, and it has all the ad­van­tages that come with a large, in­dus­tri­al site that doesn’t al­ways fit in­side the crowd­ed in­fra­struc­ture of Cam­bridge, the fifth most dense­ly pop­u­lat­ed city in the US. But the Waltham site is just eight miles away from Gen­er­a­tion’s Kendall Square head­quar­ters, and the abil­i­ty to pro­vide greater con­nec­tiv­i­ty be­tween the two sites was im­por­tant to the team as it rolls out a brand-new man­u­fac­tur­ing process.

The lease will cost $104 mil­lion over a 12-year pe­ri­od, and Gen­er­a­tion will in­vest $45 mil­lion over the next two years in­to the site. The team will still use CD­MOs be­fore and af­ter the site is up and run­ning to en­sure re­dun­dan­cy and se­cure ad­di­tion­al ccD­NA sup­ply, but CEO Ge­off Mc­Do­nough said the team wants to re­ly on greater flex­i­bil­i­ty and greater fi­nan­cial lever­age that comes with its own sup­ply chain in house.

The holdup sur­round­ing tra­di­tion­al man­u­fac­tur­ing is scale. For ex­am­ple: there are 200 mil­lion peo­ple world­wide with he­mo­phil­ia A. “You just don’t have enough scale to get to (them),” Mc­Do­nough said. “This move al­lows us to con­sid­er ad­dress­ing those very large pop­u­la­tions.”

Gen­er­a­tion Bio is tout­ing what it’s call­ing a game-chang­ing man­u­fac­tur­ing process called rapid en­zy­mat­ic syn­the­sis — oth­er­wise known as RES — that can speed clin­i­cal man­u­fac­tur­ing of its non-vi­ral gene ther­a­pies from 28 days down to one.

With more flex­i­bil­i­ty on its man­u­fac­tur­ing runs, Gen­er­a­tion’s team is tar­get­ing in­fec­tious dis­eases and oth­er larg­er in­di­ca­tions, in ad­di­tion to its cur­rent work in rare ge­net­ic dis­eases. The com­pa­ny has been work­ing on two lead pro­grams for he­mo­phil­ia A and phenylke­tonuria (PKU). Wil­son dis­ease and Gauch­er dis­ease are al­so on the list

“For first gen­er­a­tion AAV man­u­fac­tur­ing, mak­ing that con­struct can on­ly be done in a cell sys­tem,” Mc­Do­nough said in a call with End­points News Tues­day. “Since you’re mak­ing it with cap­sid, you’re lim­it­ing your­self to a few thou­sand liters of prod­uct, and with that, very few pa­tients you can help, some­where around 10,000. We’ve al­ways had this idea of ‘look, our drug sub­stance is DNA, there are oth­er ways to make DNA in­clud­ing en­zy­mat­i­cal­ly, where you take the plas­mid in, and you un­der­go a fi­nite set of en­zy­mat­ic trans­for­ma­tions, and then you get that drug sub­stance, which is closed end­ed DNA com­ing out.”

Big Phar­ma's Twit­ter ex­o­dus; Mer­ck wa­gers $1.35B on buy­out; $3.5M gene ther­a­py; and more

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Paul Perreault, CSL Behring CEO

CSL lands FDA ap­proval for he­mo­phil­ia B gene ther­a­py, sets $3.5M list price

The FDA has approved the world’s first gene therapy for hemophilia B, ushering into the market a treatment that’s historic in both what it promises to do and how much it will cost.

CSL will be marketing the drug, Hemgenix, at a list price of $3.5 million — which sets a new record for the most expensive single-use gene therapy in the US.

In a statement provided to Endpoints News, the Australian company noted that the current costs of treating people with moderate to severe hemophilia B can be significant over a lifetime. By some estimates, healthcare systems could spend more than $20 million per person.

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Image: Shutterstock

MIT re­searchers re­veal DNA "Paste" tech be­hind lat­est gene edit­ing start­up

MIT scientists have developed a tool that they say can insert large gene sequences where they want in the genome.

In a paper published Thursday in Nature Biotechnology, MIT fellows Omar Abudayyeh, Jonathan Gootenberg and colleagues detail a technology they call PASTE, which they say can potentially be used to insert long strands of DNA and treat genetic diseases caused by many different mutations, such as cystic fibrosis and Leber congenital amaurosis, a rare eye disorder that causes blindness.

Elon Musk (GDA via AP Images)

Biggest drug com­pa­nies halt­ed Twit­ter ad buys af­ter Lil­ly in­sulin spoof

Almost all of the drug industry’s biggest advertisers cut their spending on Twitter to zero or near-zero over the last two weeks amid worries about impersonation of their brands by pranksters and the future of the social media company.

Among 18 of the biggest pharmaceutical advertisers in the US market, 12 cut their Twitter ad spending to nothing for the week beginning Nov. 14, according to Pathmatics, which tracks data on prescription drug ad spending as well as general corporate advertising. The list of drugmakers cutting spending to zero includes Merck, AstraZeneca, Eli Lilly, Novartis, Pfizer and others.

Rob Davis, Merck CEO

Up­dat­ed: No Seagen here: 'Do more' means a small $1.35B pur­chase of Ima­go for Mer­ck

Merck is making an acquisition, the Big Pharma announced before Monday’s opening bell. No, Seagen is not entering the fold, as had been speculated for quarters.

Folding under Merck’s wings will be Pfizer-backed Imago BioSciences. For nearly a year, Merck CEO Rob Davis has been saying the pharma giant needs to “do more” on the business development front after its 2021 $11.5 billion acquisition of Acceleron.

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J&J's Spra­va­to pulls a PhI­II win against Sero­quel XR in treat­ment-re­sis­tant de­pres­sion

A day before Thanksgiving, J&J’s Janssen has a new cut of Phase III Spravato data to be grateful for.

The pharma giant announced on Wednesday that its nasal spray, also known as esketamine, beat extended-release quetiapine, previously sold by AstraZeneca as Seroquel XR, in treatment-resistant depression (TRD). Of 676 adults, a significantly higher number of patients on Spravato were able to achieve remission and avoid relapse after 32 weeks, according to J&J.

Isao Teshirogi, Shionogi president and CEO (Kyodo via AP Images)

Sh­ionogi's Covid an­tivi­ral lands first ap­proval in Japan's new emer­gency ap­proval path­way

Japanese regulators on Tuesday signed off on Shionogi’s homegrown antiviral for Covid-19, known as Xocova (ensitrelvir), making it the first approval under Japan’s emergency regulatory approval system.

The emergency approval, following a back-and-forth with regulators since last February, is based on a safety profile with more than 2,000 patients who have accessed the pill, and clinical symptomatic efficacy for five typical Omicron-related symptoms (primary endpoint) and antiviral efficacy (key secondary endpoint) in patients with mild to moderate SARS-CoV-2 infection, regardless of risk factors or vaccination status, and during the Omicron-dominant phase of the pandemic.

Fu­ji­film to build $188M man­u­fac­tur­ing plant in North Car­oli­na’s re­search tri­an­gle

As the Japanese conglomerate Fujifilm continues to invest heavily in its CDMO arm, one of its manufacturing divisions is teeing up a major investment.

Fujifilm Irvine Scientific announced on Tuesday that parent Fujifilm is making a $188 million investment to build a cell culture media manufacturing site in the Research Triangle Park in North Carolina. The new site will mark Fujifilm Irvine’s fifth manufacturing site globally and its second in the US.

Karen Aiach, Lysogene CEO (RE(ACT) Discovery Institute)

Gene ther­a­py flunks PhII/III study, but for­mer Sarep­ta part­ner sees a path for­ward — if it can find the cash

The development path for Lysogene’s gene therapy for MPS IIIA has been a rocky one. After the FDA slapped a partial clinical hold on a Phase II/III study, a patient already dosed in the trial died, although it was deemed unrelated to treatment. Then earlier this year, Sarepta pulled out of their three-year partnership due to disagreements on who will handle commercial supply.

And now, Lysogene reported the trial has failed its primary endpoint.

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