Haul­ing in a $100M mega-round, gene ther­a­py start­up Gen­er­a­tion Bio builds al­ter­na­tive to po­ten­tial­ly risky vi­ral vec­tors

A gene ther­a­py com­pa­ny head­ed up by big­wigs and backed ear­ly on by At­las has pulled in a mega-round of $100 mil­lion. The 2-year-old start­up cap­tured in­vestors’ at­ten­tion by sug­gest­ing it knows a bet­ter way to do gene trans­fer than the po­ten­tial­ly tox­ic vi­ral vec­tor strate­gies of late.

Gen­er­a­tion Bio is the Boston/Cam­bridge up­start, and it’s a com­pa­ny that’s strayed from the pack when it comes to the how of gene ther­a­py. While its ri­vals in the field are buck­ling down on vi­ral ve­hi­cles like ade­no as­so­ci­at­ed virus­es (AAV), Gen­er­a­tion Bio is tak­ing a dif­fer­ent route.

Robert Kotin

Found­ed in part by ex-NIH, ex-Voy­ager ex­ec­u­tive Robert Kotin, whom you may know as one of the world’s top ex­perts on vi­ral trans­port, the new com­pa­ny cen­ters on a tech they’re call­ing close-end­ed DNA (ceD­NA). This can move from the cy­to­plasm of the cell to the nu­cle­us with­out an AAV car­ry­ing it along. Once in­side the nu­cle­us, it can cre­ate high lev­els of gene ex­pres­sion ca­pa­ble of ad­dress­ing a broad range of ge­net­ic dis­eases.

Gen­er­a­tion Bio says the hope is that this tech can ad­dress the emerg­ing con­cern re­volv­ing around vi­ral vec­tors. Well-known gene ther­a­py com­pa­nies like Spark and Sol­id Bio­sciences are us­ing AAVs as vec­tors, a method that, when used in high dos­es, has re­cent­ly been called in­to ques­tion by a pi­o­neer in the field, Penn pro­fes­sor James Wil­son.

Wil­son sur­prised the gene ther­a­py com­mu­ni­ty in Jan­u­ary when he quit the ad­vi­sor board of Sol­id and raised alarms about the po­ten­tial tox­i­c­i­ty of high-dose AAV stud­ies.

Gen­er­a­tion Bio is cap­i­tal­iz­ing on the un­cer­tain­ty cloud­ing the lead­ing tech in the field by por­tray­ing their ther­a­pies as po­ten­tial­ly safer.

Last month, the com­pa­ny’s CEO Ge­off Mc­Do­nough told End­points the com­pa­ny would start by us­ing a lipid nanopar­ti­cle to de­liv­er ceD­NA to the liv­er, where it will make sys­temic pro­teins to fight dis­eases.

“These ther­a­pies are de­signed to over­come the im­muno­genic­i­ty as­so­ci­at­ed with vi­ral-vec­tor gene ther­a­pies, which has his­tor­i­cal­ly lim­it­ed the num­ber of pa­tients that can be treat­ed and pre­vent­ed re-dos­ing,” Gen­er­a­tion Bio stat­ed in a press re­lease. “Since its found­ing in 2016, Gen­er­a­tion Bio has demon­strat­ed durable, dose-de­pen­dent ex­pres­sion and the abil­i­ty to in­crease ex­pres­sion in the liv­er with re-dos­ing in vi­vo with the Ge­neWave plat­form.”

In­vestors ap­pear to be in­trigued. The $100 mil­lion fi­nanc­ing was a Se­ries B led by Fi­deli­ty Man­age­ment & Re­search Com­pa­ny, with par­tic­i­pa­tion from In­vus, Deer­field Man­age­ment Com­pa­ny, Cas­din Cap­i­tal, Fore­site Cap­i­tal, and Leerink Part­ners’ Af­fil­i­ates.

This round comes less than two months af­ter Gen­er­a­tion Bio an­nounced its $25 mil­lion Se­ries A backed by At­las.

The new mon­ey will be used to take two ther­a­peu­tic can­di­dates through IND-en­abling stud­ies.

“This will be a crit­i­cal­ly im­por­tant year for us as we de­vel­op the Ge­neWave plat­form and work to­wards our first de­vel­op­ment can­di­dates to ad­dress rare dis­eases of the liv­er,” Mc­Do­nough said in a state­ment. “The round will al­so sup­port the de­vel­op­ment of ap­proach­es to de­liv­er ceD­NA to oth­er tis­sues such as the eye, the cen­tral ner­vous sys­tem and the lungs.”

Im­age: Gen­er­a­tion Bio’s pres­i­dent and CEO Ge­off Mc­Do­nough. Gen­er­a­tion Bio

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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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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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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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.

Sulagna Bhattacharya, Nanoscope Therapeutics CEO

Nanoscope’s eye dis­ease gene ther­a­py shows mixed re­sults in PhII

Dallas-based biotech Nanoscope Therapeutics unveiled Phase II results on its gene therapy for a rare eye disease Thursday morning.

In the RESTORE trial, 18 patients with retinitis pigmentosa got a gene therapy called MCO-010 while nine got placebo. On a vision test called the MLYMT, the treatment group had a one-point greater change over one year in their score compared to the placebo group, the primary endpoint of the study. However, the 95% confidence interval was 0.0 to 3.0, meaning the result was not statistically significant. The p-value was not provided.

Luke Miels, GSK chief commercial officer

GSK picks up Scynex­is' FDA-ap­proved an­ti­fun­gal drug for $90M up­front

GSK is dishing out $90 million cash to add an antifungal drug to its commercial portfolio, in a deal spotlighting the pharma giant’s growing focus on infectious diseases.

The upfront will lock in an exclusive license to Scynexis’ Brexafemme, which was approved in 2021 to treat a yeast infection known as vulvovaginal candidiasis, except in China and certain other countries where Scynexis already out-licensed the drug.

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John Flavin, Portal Innovations CEO

Por­tal In­no­va­tions takes its Chica­go biotech in­vest­ing, lab space mod­el to Boston with Bob Langer start­up in tow

Life sciences investor and lab operator Portal Innovations is taking the blueprint it set in Chicago and expanding to Massachusetts, where it will deploy $25 million, and help house new startups in the former Boston Globe headquarters.

Portal CEO and founder John Flavin told Endpoints News the company will open its Boston-area operations in the coming weeks, and its first lab occupant will be Syntis Bio, an early-stage biotech out of Moderna co-founder Bob Langer, MIT professor Giovanni Traverso and Sherlock Biosciences co-founder Rahul Dhanda.

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