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

The top 10 block­buster drugs in the late-stage pipeline — Eval­u­ate adds 6 new ther­a­pies to heavy-hit­ter list

Vertex comes in for a substantial amount of criticism for its no-holds-barred tactical approach toward wresting the price it wants for its commercial drugs in Europe. But the flip side of that coin is a highly admired R&D and commercial operation that regularly wins kudos from analysts for their ability to engineer greater cash flow from the breakthrough drugs they create.

Both aspects needed for success in this business are on display in the program backing Vertex’s triple for cystic fibrosis. VX-659/VX-445 + Tezacaftor + Ivacaftor — it’s been whittled down to 445 now — was singled out by Evaluate Pharma as the late-stage therapy most likely to win the crown for drug sales in 5 years, with a projected peak revenue forecast of $4.3 billion.

The latest annual list, which you can see here in their latest world preview, includes a roster of some of the most closely watched development programs in biopharma. And Evaluate has added 6 must-watch experimental drugs to the top 10 as drugs fail or go on to a first approval. With apologies to the list maker, I revamped this to rank the top 10 by projected 2024 sales, instead of Evaluate's net present value rankings.

It's how we roll at Endpoints News.

Here is a quick summary of the rest of the top 10:

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How small- to mid-sized biotechs can adopt pa­tient cen­tric­i­ty in their on­col­o­gy tri­als

By Lucy Clos­sick Thom­son, Se­nior Di­rec­tor of On­col­o­gy Pro­ject Man­age­ment, Icon

Clin­i­cal tri­als in on­col­o­gy can be cost­ly and chal­leng­ing to man­age. One fac­tor that could re­duce costs and re­duce bar­ri­ers is har­ness­ing the pa­tient voice in tri­al de­sign to help ac­cel­er­ate pa­tient en­roll­ment. Now is the time to adopt pa­tient-cen­tric strate­gies that not on­ly fo­cus on pa­tient needs, but al­so can main­tain cost ef­fi­cien­cy.

John Reed at JPM 2019. Jeff Rumans for Endpoints News

Sanofi's John Reed con­tin­ues to re­or­ga­nize R&D, cut­ting 466 jobs while boost­ing can­cer, gene ther­a­py re­search

The R&D reorganization inside Sanofi is continuing, more than a year after the pharma giant brought in John Reed to head the research arm of the Paris-based company.
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Partners Innovation Fund

David de Graaf now has his $28.5M launch round in place, build­ing a coen­zyme A plat­form in his lat­est start­up

Long­time biotech ex­ec David de Graaf has the cash he needs to set up the pre­clin­i­cal foun­da­tion for his coen­zyme A me­tab­o­lism com­pa­ny Comet. A few high-pro­file in­vestors joined the ven­ture syn­di­cate to sup­ply Comet with $28.5 mil­lion in launch mon­ey — enough to get it two years in­to the plat­form-build­ing game, with­in knock­ing dis­tance of the clin­ic.

Canaan jumped in along­side ex­ist­ing in­vestor Sofinno­va Part­ners to co-lead the round, with par­tic­i­pa­tion by ex­ist­ing in­vestor INKEF Cap­i­tal and new in­vestor BioIn­no­va­tion Cap­i­tal.

Neil Woodford. Woodford Investment Management via YouTube

Wood­ford braces po­lit­i­cal storm as UK fi­nan­cial reg­u­la­tors scru­ti­nize fund sus­pen­sion

The shock of Neil Wood­ford’s de­ci­sion to block with­drawals for his flag­ship fund is still rip­pling through the rest of his port­fo­lio — and be­yond. Un­der po­lit­i­cal pres­sure, UK fi­nan­cial reg­u­la­tors are now tak­ing a hard look while in­vestors con­tin­ue to flee.

In a re­sponse let­ter to an MP, the Fi­nan­cial Con­duct Au­thor­i­ty re­vealed that it’s opened an in­ves­ti­ga­tion in­to the sus­pen­sion fol­low­ing months of en­gage­ment with Link Fund So­lu­tions, which tech­ni­cal­ly del­e­gat­ed Wood­ford’s firm to man­age its funds.

Dave Barrett, Brian Chee, Amir Nashat, Amy Schulman. Polaris

Bob Langer's first port of call — Po­laris Part­ners — maps $400M for ninth fund

Health and tech ven­ture group Po­laris Part­ners, which counts Alec­tor, Al­ny­lam and Ed­i­tas Med­i­cine as part of its port­fo­lio, is set­ting up its ninth fund, rough­ly two years af­ter it closed Po­laris VI­II with $435 mil­lion in the bank, sur­pass­ing its tar­get by $35 mil­lion.

The Boston-based firm, in an SEC fil­ing, said it in­tends to raise $400 mil­lion for the fund. Po­laris — which rou­tine­ly backs com­pa­nies mold­ed out of the work done in the lab of pro­lif­ic sci­en­tist Bob Langer of MIT  — typ­i­cal­ly in­vests ear­ly, and sticks around till com­pa­nies are in the green. Like its peers at Flag­ship and Third Rock, Po­laris is all about cham­pi­oning the lo­cal biotech scene with a steady flow of start­up cash.

John Chiminski, Catalent CEO - File Photo

'It's a growth play': Catal­ent ac­quires Bris­tol-My­er­s' Eu­ro­pean launch pad, ex­pand­ing glob­al CD­MO ops

Catalent is staying on the growth track.

Just two months after committing $1.2 billion to pick up Paragon and take a deep dive into the sizzling hot gene therapy manufacturing sector, the CDMO is bouncing right back with a deal to buy out Bristol-Myers’ central launchpad for new therapies in Europe, acquiring a complex in Anagni, Italy, southwest of Rome, that will significantly expand its capacity on the continent.

There are no terms being offered, but this is no small deal. The Anagni campus employs some 700 staffers, and Catalent is planning to go right in — once the deal closes late this year — with a blueprint to build up the operations further as they expand on oral solid, biologics, and sterile product manufacturing and packaging.

This is an uncommon deal, Catalent CEO John Chiminski tells me. But it offers a shortcut for rapid growth that cuts years out of developing a green fields project. That’s time Catalent doesn’t have as the industry undergoes unprecedented expansion around the world.

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Arc­turus ex­pands col­lab­o­ra­tion, adding $30M cash; Ku­ra shoots for $100M raise

→  Rare dis­ease play­er Ul­tragenyx $RARE is ex­pand­ing its al­liance with Arc­turus $ARCT, pay­ing $24 mil­lion for eq­ui­ty and an­oth­er $6 mil­lion in an up­front as the two part­ners ex­pand their col­lab­o­ra­tion to in­clude up to 12 tar­gets. “This ex­pand­ed col­lab­o­ra­tion fur­ther so­lid­i­fies our mR­NA plat­form by adding ad­di­tion­al tar­gets and ex­pand­ing our abil­i­ty to po­ten­tial­ly treat more dis­eases,” said Emil Kakkis, the CEO at Ul­tragenyx. “We are pleased with the progress of our on­go­ing col­lab­o­ra­tion. Our most ad­vanced mR­NA pro­gram, UX053 for the treat­ment of Glyco­gen Stor­age Dis­ease Type III, is ex­pect­ed to move in­to the clin­ic next year, and we look for­ward to fur­ther build­ing up­on the ini­tial suc­cess of this part­ner­ship.”

UP­DAT­ED: Chica­go biotech ar­gues blue­bird, Third Rock 'killed' its ri­val, pi­o­neer­ing tha­lassemia gene ther­a­py in law­suit

Blue­bird bio $BLUE chief Nick Leschly court­ed con­tro­ver­sy last week when he re­vealed the com­pa­ny’s be­ta tha­lassemia treat­ment will car­ry a jaw-drop­ping $1.8 mil­lion price tag over a 5-year pe­ri­od in Eu­rope — mak­ing it the plan­et’s sec­ond most ex­pen­sive ther­a­py be­hind No­var­tis’ $NVS fresh­ly ap­proved spinal mus­cu­lar at­ro­phy ther­a­py, Zol­gens­ma, at $2.1 mil­lion. A Chica­go biotech, mean­while, has been fum­ing at the side­lines. In a law­suit filed ear­li­er this month, Er­rant Gene Ther­a­peu­tics al­leged that blue­bird and ven­ture cap­i­tal group Third Rock un­law­ful­ly prised a vi­ral vec­tor, de­vel­oped in part­ner­ship with the Memo­r­i­al Sloan Ket­ter­ing Can­cer Cen­ter (MSK), from its grasp, and thwart­ed the de­vel­op­ment of its sem­i­nal gene ther­a­py.