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Agency feedback from Type C meeting process supports the company's proposed clinical development plans to expand the upliFT-D trial to assess PBFT02 in FTD patients with C9orf72 gene mutations Plan...
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Potent and durable target engagement observed across cohorts, including with 10 mg doses administered every 12 weeks which were also generally safe and well-tolerated WVE-004 did not show clinical...
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Preclinical data published in Molecular Therapy Nucleic Acids demonstrate that WVE-004 potently reduces C9orf72 transcriptional variants and poly(GP) dipeptide repeat proteins in mice for at least six...
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Reductions in poly(GP), a key disease biomarker indicating target engagement, observed across all treatment groups after single doses Extending dose observation period from three months (day 85) to...
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SAN DIEGO, Dec. 06, 2021 (GLOBE NEWSWIRE) -- Amydis Inc., a biotechnology company developing novel ocular contrast agents (“tracers”) targeting CNS biomarkers in the eye, today announced the...
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SAN DIEGO, Sept. 02, 2021 (GLOBE NEWSWIRE) -- Amydis Inc., a leading ocular tracer pharmaceutical company with a broad portfolio of diagnostic drug candidates targeting CNS biomarkers in the eye,...
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Dosing underway in FOCUS-C9 clinical trial of WVE-004 in C9-ALS / C9-FTD – first oligonucleotide using PN backbone chemistry modifications in clinical study Recruitment ongoing for clinical trials in...
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First clinical dosing of a compound using PN backbone chemistry modifications Enrolling participants with C9-ALS, C9-FTD or mixed phenotype FOCUS-C9 is adaptive to enable rapid optimization of...
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Clinical trials underway with next-generation candidates incorporating PN chemistry Clinical data from PN chemistry programs expected in 2022 In vivo ADAR editing data for AATD program on track for...
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Three clinical trials to begin in 2021 with compounds containing Wave’s novel PN backbone chemistry modifications Data from ongoing PRECISION-HD and OLE clinical trials for Huntington’s disease...