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Sökning: WFRF:(Geissler Mary)

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1.
  • Coelho, Teresa, et al. (författare)
  • Safety and efficacy of RNAi therapy for transthyretin amyloidosis
  • 2013
  • Ingår i: The New England journal of medicine. - 1533-4406. ; 369:9, s. 819-29
  • Tidskriftsartikel (refereegranskat)abstract
    • BACKGROUND: Transthyretin amyloidosis is caused by the deposition of hepatocyte-derived transthyretin amyloid in peripheral nerves and the heart. A therapeutic approach mediated by RNA interference (RNAi) could reduce the production of transthyretin. METHODS: We identified a potent antitransthyretin small interfering RNA, which was encapsulated in two distinct first- and second-generation formulations of lipid nanoparticles, generating ALN-TTR01 and ALN-TTR02, respectively. Each formulation was studied in a single-dose, placebo-controlled phase 1 trial to assess safety and effect on transthyretin levels. We first evaluated ALN-TTR01 (at doses of 0.01 to 1.0 mg per kilogram of body weight) in 32 patients with transthyretin amyloidosis and then evaluated ALN-TTR02 (at doses of 0.01 to 0.5 mg per kilogram) in 17 healthy volunteers. RESULTS: Rapid, dose-dependent, and durable lowering of transthyretin levels was observed in the two trials. At a dose of 1.0 mg per kilogram, ALN-TTR01 suppressed transthyretin, with a mean reduction at day 7 of 38%, as compared with placebo (P=0.01); levels of mutant and nonmutant forms of transthyretin were lowered to a similar extent. For ALN-TTR02, the mean reductions in transthyretin levels at doses of 0.15 to 0.3 mg per kilogram ranged from 82.3 to 86.8%, with reductions of 56.6 to 67.1% at 28 days (P<0.001 for all comparisons). These reductions were shown to be RNAi-mediated. Mild-to-moderate infusion-related reactions occurred in 20.8% and 7.7% of participants receiving ALN-TTR01 and ALN-TTR02, respectively. CONCLUSIONS: ALN-TTR01 and ALN-TTR02 suppressed the production of both mutant and nonmutant forms of transthyretin, establishing proof of concept for RNAi therapy targeting messenger RNA transcribed from a disease-causing gene. (Funded by Alnylam Pharmaceuticals; ClinicalTrials.gov numbers, NCT01148953 and NCT01559077.).
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2.
  • Geissler, Jan, et al. (författare)
  • Improving Patient Involvement in Medicines Research and Development: : A Practical Roadmap
  • 2017
  • Ingår i: Therapeutic Innovation and Regulatory Science. - : Sage Publications. - 2168-4790 .- 2168-4804. ; 51:5, s. 612-619
  • Tidskriftsartikel (refereegranskat)abstract
    • The value of patient involvement (PI) in medicines research and development (R&D) is increasingly recognized by all health stakeholders. Despite numerous ongoing PI initiatives, PI so far lacks structure and consistency in approach. Limited formal documentation of PI activities further hampers the sharing of experience and learnings, preventing timely and systematic implementation. This article summarizes the outcomes of several multistakeholder discussions during 2013-2016 in a practical roadmap for PI in medicines R&D. The roadmap highlights specific opportunities for PI along the 4 key stages of the medicines R&D life cycle and is illustrated with concrete examples. This roadmap's aim is to provide a tool to facilitate PI during medicines research and development and is being shared to encourage implementation and further refinement.
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