As­traZeneca and Mod­er­na take a big, ear­ly step for­ward in their quest to prove mR­NA works in hu­mans

Biotech uni­corn Mod­er­na has re­vamped its de­vel­op­ment struc­ture, pulled back the veil on more of its R&D work and spot­light­ed a sig­nif­i­cant clin­i­cal step for­ward for one of its most ad­vanced — though still very ear­ly-stage — mes­sen­ger RNA ther­a­pies in the pipeline.

Tack­ling the very am­bi­tious work of re­gen­er­at­ing car­dio tis­sue — long one of the Holy Grails in stem cell ther­a­py — Mod­er­na’s close part­ners at As­traZeneca say they got the safe­ty and ef­fi­ca­cy da­ta they were look­ing for on AZD-8601 in Phase I. Work­ing with a bio­mark­er at this stage, in­ves­ti­ga­tors tracked ex­pres­sion of VEGF-A pro­tein in the skin — giv­ing them some ear­ly con­fi­dence that a tech­nol­o­gy ini­tial­ly de­vel­oped at Har­vard had some re­al proof-of-mech­a­nism ev­i­dence to back it up with. That in turn has in­spired As­traZeneca to take the next step on the mes­sen­ger RNA front as it jour­neys in­to Phase II, where they will look for more sol­id ev­i­dence of its re­gen­er­a­tive pow­ers among pa­tients un­der­go­ing coro­nary artery by­pass graft­ing.

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