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.”

MedTech clinical trials require a unique regulatory and study design approach and so engaging a highly experienced CRO to ensure compliance and accurate data across all stages is critical to development milestones.

In­no­v­a­tive MedTech De­mands Spe­cial­ist Clin­i­cal Tri­al Reg­u­la­to­ry Af­fairs and De­sign

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Mathai Mammen (Rob Tannenbaum, Endpoints News at BIO 2018)

Math­ai Mam­men makes an abrupt ex­it as head of the big R&D group at J&J

In an after-the-bell shocker, J&J announced Monday evening that Mathai Mammen has abruptly exited J&J as head of its top-10 R&D group.

Recruited from Merck five years ago, where the soft-spoken Mammen was being groomed as the successor to Roger Perlmutter, he had been one of the top-paid R&D chiefs in biopharma. His group spent $12 billion last year on drug development, putting it in the top 5 in the industry.

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Illustration: Kim Ryu for Endpoints News

Why non-opi­oid pain drugs keep fail­ing — and what's next for the field

In 1938, Rita Levi-Montalcini was forced to move her lab into her bedroom in Turin, as Mussolini’s facist government expelled Jewish people from studying or working in schools in Italy. Levi-Montalcini, then just a few years out of medical school and using sewing needles as scalpels in her makeshift lab, would soon discover nerve growth factor, or NGF, in chicken embryos.

Her discoveries formed the basis of our understanding of the peripheral nervous system and how cells talk to each other, and Levi-Montalcini went on to win the Nobel Prize in 1986. Much later, NGF was hailed as a promising target for new pain therapies, with some analysts quoting an $11 billion market. However, the latest anti-NGF candidate, Pfizer and Eli Lilly’s tanezumab, was rejected by the FDA last year because of a side effect that dissolved bone in some of its patients.

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Samantha Du, Zai Lab CEO

Any­one still look­ing for a CD47? Zai Lab shelves PhI pro­gram af­ter re­view­ing 'com­pet­i­tive land­scape'

Over the past few years, the promise of blocking CD47 — a “don’t eat me” signal co-opted by cancer cells — has sent drugmakers big and small into a frenzy. But one biotech is now bowing out.

Zai Lab is deprioritizing ZL-1201, its CD47 inhibitor, scrapping plans for a Phase II trial. It will now “pursue out-licensing opportunities,” the company said in its Q2 update. The decision was based on a review of the competitive landscape, it added, without going into further details.

Ted Love, Global Blood Therapeutics CEO

Up­dat­ed: Pfiz­er scoops up Glob­al Blood Ther­a­peu­tics and its sick­le cell ther­a­pies for $5.4B

Pfizer is dropping $5.4 billion to acquire Global Blood Therapeutics.

Just ahead of the weekend, word got out that Pfizer was close to clinching a $5 billion buyout — albeit with other potential buyers still at the table. The pharma giant, flush with cash from Covid-19 vaccine sales, apparently got out on top.

The deal immediately swells Pfizer’s previously tiny sickle cell disease portfolio from just a Phase I program to one with an approved drug, Oxbryta, plus a whole pipeline that, if all approved, the company believes could make for a $3 billion franchise at peak.

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Steve Paul, Karuna Therapeutics CEO (Third Rock)

Karuna's schiz­o­phre­nia drug pass­es a close­ly-watched PhI­II test, will head to FDA in mid-2023

An investigational pill that combines a former Eli Lilly CNS compound with an overactive bladder drug was better than placebo at reducing a scale of symptoms experienced by patients with schizophrenia in a Phase III trial.

Karuna Therapeutics’ drug passed the primary goal in EMERGENT-2, the Boston biotech said early Monday morning, alongside quarterly earnings. The study is the first of Karuna’s four Phase III clinical trials to read out in schizophrenia and will provide the backbone to the biotech’s first drug approval application, slated for mid-2023.

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Civi­ca, Mayo Clin­ic and oth­ers sound off on FDA draft guid­ance to mit­i­gate drug short­ages

Several pharma groups are laying out the positives and negatives of new FDA draft guidance on how best to handle drug shortages.

The draft is intended to help companies develop and implement risk management plans (RMPs) to assist with any shortages of drugs or biologics. The guidance recommends a framework and factors for stakeholders to develop RMPs for their establishments, API manufacturers and others.

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HHS Secretary Xavier Becerra (Patrick Semansky/AP Images)

US weighs new route of ad­min­is­tra­tion for mon­key­pox vac­cine as cas­es climb — re­port

Less than a week after HHS Secretary Xavier Becerra declared monkeypox a national health emergency, reports have emerged that the US plans to extend its vaccine supply by opting for a different route of administration.

Officials are expected to call for intradermal injection of Bavarian Nordic’s Jynneos vaccine — the only shot approved specifically for monkeypox in the US — as opposed to subcutaneous injection, unnamed sources told both the New York Times and Washington Post on Tuesday.

To avoid short­age of Mer­ck­'s di­a­betes drug, FDA al­lows high­er im­pu­ri­ty lev­els tem­porar­i­ly

The FDA said Tuesday that it recently became aware of a nitrosamine impurity, Nitroso-STG-19 or NTTP, in certain samples of Merck’s type 2 diabetes drug Januvia.

To avoid a shortage of the drug that pulled in more than $10.5 billion for Merck in 2020 and 2021, and to help ensure patients have access to an adequate supply, the FDA said it will not object to the temporary distribution of sitagliptin containing the impurity above the acceptable intake limit.

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