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.”

Forge Bi­o­log­ics’ cGMP Com­pli­ant and Com­mer­cial­ly Vi­able Be­spoke Affin­i­ty Chro­matog­ra­phy Plat­form

Forge Biologics has developed a bespoke affinity chromatography platform approach that factors in unique vector combinations to streamline development timelines and assist our clients in efficiently entering the clinic. By leveraging our experience with natural and novel serotypes and transgene conformations, we are able to accelerate affinity chromatography development by nearly 3-fold. Many downstream purification models are serotype-dependent, demanding unique and time-consuming development strategies for each AAV gene therapy product1. With the increasing demand to propel AAV gene therapies to market, platform purification methods that support commercial-scale manufacturing of high-quality vectors with excellent safety and efficacy profiles are essential.

Mathai Mammen, FogPharma's next CEO

Math­ai Mam­men hands in J&J's R&D keys to lead Greg Ver­dine’s Fog­Phar­ma 

In the early 1990s, Mathai Mammen was a teaching assistant in Greg Verdine’s Science B46 course at Harvard. In June, the former R&D head at Johnson & Johnson will succeed Verdine as CEO, president and chair of FogPharma, the same month the seven-year-old biotech kickstarts its first clinical trial.

After leading R&D at one of the largest drugmakers in the world, taking the company through more than half a dozen drug approvals in the past few years, not to mention a Covid-19 vaccine race, Mammen departed J&J last month and will take the helm of a Cambridge, MA biotech attempting to go after what Verdine calls the “true emperor of all oncogenes” — beta-catenin.

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Stéphane Bancel, Moderna CEO (AP Photo/Markus Schreiber)

Mod­er­na so­lid­i­fies deal with Kenya to build mR­NA man­u­fac­tur­ing fa­cil­i­ty

The mRNA player Moderna is further cementing its presence on the African continent.

Moderna announced on Thursday that it has finalized an agreement with Kenya’s government to partner up and bring an mRNA manufacturing facility to the east African nation. The new facility aims to manufacture up to 500 million doses of vaccines annually. Moderna also said the new facility will have the ability to spike its production capabilities to respond to public health emergencies on the continent or globally.

Sen­ate Fi­nance Com­mit­tee lobs more bi­par­ti­san pres­sure on­to PBMs

Congress is honing in on how it wants to overhaul the rules of the road for pharmacy benefit managers, with a Senate Finance Committee hearing Thursday serving as the latest example of the Hill’s readiness to make changes to how pharma middlemen operate.

While pledging to ensure patients and pharmacies “don’t get a raw deal,” Finance Committee Chair Ron Wyden (D-OR) laid out the beginning of what looks like a major bipartisan effort — moves the PBM industry is likely to challenge vigorously.

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Deborah Waterhouse, ViiV Healthcare CEO

MPP re­cruits three gener­ic man­u­fac­tur­ers to ex­pand use of Vi­iV's in­jectable PrEP drug

ViiV Healthcare has teamed up with the UN-backed Medicines Patent Pool and three generic manufacturers to expand access to medicine that can prevent HIV.

ViiV and the Medicines Patent Pool jointly announced Thursday that the MPP signed sub-licensing agreements with Aurobindo, Cipla and Viatris to manufacture generics of a long-acting form of cabotegravir for HIV pre-exposure prophylaxis (PrEP). As a result of the agreement, the manufacturers will be able to develop, manufacture and supply generic versions of cabotegravir LA in 90 countries — subject to regulatory approvals.

Nicklas Westerholm, Egetis Therapeutics CEO

Ac­qui­si­tion talks on­go­ing for Swedish rare dis­ease biotech Egetis, shares up al­most 40%

Shares of the Sweden-based rare disease biotech Egetis Therapeutics skyrocketed on Thursday afternoon as the company said it’s engaged in “ongoing discussion” with external parties regarding a “potential acquisition.”

Egetis confirmed rumors with a statement on Thursday while noting that there is no certainty that a takeover offer will be made.

Nonetheless, the possibility of an acquisition has shot up Egetis’ share price. By the afternoon on Thursday, its stock price was {$EGTX.ST} up over 38%. An Egetis spokesperson told Endpoints News in an email that it has no further comments.

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Lu­pus drug de­vel­op­ment mar­ket heat­ing up, while FDA links with ad­vo­ca­cy group to fur­ther ac­cel­er­ate re­search

The long-underserved systemic lupus erythematosus (SLE) market is suddenly buzzing with treatment possibilities. Less than two years after AstraZeneca’s approval for Saphnelo — the first new SLE drug in a decade and joining just one other approved in GSK’s Benlysta – the pipeline of potential drugs numbers in the dozens.

Although most are very early stage — Spherix Global Insights estimates five in Phase II/III — the pharma R&D enthusiasm is catching on among doctors, patients and advocacy groups. On Wednesday, the Lupus Research Alliance and the FDA formed a novel private-public partnership called Lupus Accelerating Breakthroughs Consortium (Lupus ABC) to help advance lupus clinical trial success.

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Af­ter safe­ty re­view, EMA mir­rors FDA with up­dat­ed rec­om­men­da­tions for JAK in­hibitors

The EMA released updated recommendations today for the use of JAK inhibitors (JAKi) after reviewing data from several clinical trials that showed increased incidents of issues in certain patients who have rheumatoid arthritis and other risk factors.

The EMA noted malignancy, major adverse cardiovascular events (MACE), serious infections, venous thromboembolism (VTE) and mortality in some patients.

Feng Zhang (Susan Walsh/AP Images)

In search of new way to de­liv­er gene ed­i­tors, CRISPR pi­o­neer turns to mol­e­c­u­lar sy­ringes

Bug bacteria are ruthless.

Some soil bacteria have evolved tiny, but deadly injection systems that attach to insect cells, perforate them and release toxins inside — killing a bug in just a few days’ time. Scientists, on the other hand, want to leverage that system to deliver medicines.

In a paper published Wednesday in Nature, MIT CRISPR researcher Feng Zhang and his lab describe how they engineered these syringes made by bacteria to deliver potential therapies like toxins that kill cancer cells and gene editors. With the help of an AI program, they developed syringes that can load proteins of their choice and selectively target human cells.

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