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Gene ther­a­py R&D deals turn red hot as Big Phar­ma steps up to play

This Sep­tem­ber will mark the 20th an­niver­sary of the death of Jesse Gelsinger, a young man suf­fer­ing from X-linked ge­net­ic dis­ease of the liv­er. He was killed in a gene ther­a­py study con­duct­ed by Penn’s James Wil­son, and the en­tire field en­dured a lengthy deep freeze as the field grap­pled with the safe­ty is­sues in­her­ent in the work.

Some thought gene ther­a­py R&D would nev­er sur­vive. But it did. And this year marked a land­mark ap­proval for Zol­gens­ma, a new gene ther­a­py for spinal mus­cu­lar at­ro­phy No­var­tis priced at $2.1 mil­lion.

“Gene ther­a­py is the hottest item on the block now. But there was a time when we first got in­to this tri­al, where there wasn’t a per­son in the world who be­lieved that gene ther­a­py would work. We have to re­mem­ber that,” not­ed gene ther­a­py in­ves­ti­ga­tor Jer­ry Mendell told SMA News To­day.

We’re still right on the pi­o­neer­ing fron­tier when it comes to get­ting ap­provals for gene ther­a­pies and launch­ing mar­ket­ing cam­paigns with the Eu­ro­pean green light for blue­bird’s lead­ing pro­gram last Fri­day un­der­scor­ing the nascent na­ture of the field. But gene ther­a­py R&D is boom­ing, and has been for sev­er­al years now.

The rapid growth of gene ther­a­py clin­i­cal de­vel­op­ment is well known, but we de­cid­ed to put some num­bers on it, to quan­ti­fy what’s go­ing on. Deal­For­ma chief Chris Doko­ma­ji­lar took a lot over the past 10 years, as the num­ber of deals, R&D part­ner­ships and buy­outs steadi­ly gained steam, spik­ing last year and on track to main­tain the surge in 2019.

The up­fronts and to­tals for the dol­lars on deals so far in 2019 is al­ready close to the 2018 mark, un­der­scor­ing a new phase of ne­go­ti­a­tions as the ma­jor play­ers step up to gain a piece of the late-stage and com­mer­cial ac­tion.

Once again, we’re look­ing at an “overnight” biotech suc­cess sto­ry, decades in the mak­ing.

At some point, that may start to brake the num­bers we’re see­ing. But for now, as ri­vals line up to com­pete for front­line promi­nence across a range of dis­eases, the ar­rows are all point­ed north.

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