PTC Confident About Commercial Prospects For Gene Therapy Upstaza
One-Time Injection Gets CHMP Nod For AADC Deficiency
PTC CEO Stuart Peltz tells Scrip that cost watchdogs are likely to look favorably on the firm’s transformational gene therapy for a distressing rare brain disorder in children who could not even lift their heads before treatment.
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In this week's podcast version of Five Must-Know Things: etrasimod comes through for Pfizer; PTC confident on gene therapy; could CAR-Ts roll into the community setting; the attractions of Boston for Korean bioventures; and Ukraine war may claim Sputnik casualties.
Embattled gene therapy companies will take comfort from desire of FDA and EMA help smooth the process of bringing the novel treatments to market.
The company will prioritize US market for gene therapies; FDA also placed eli-cel for CALD on clinical hold following a case of MDS.