Passage Bio Announces Positive Feedback from FDA on Expansion of upliFT-D Trial of PBFT02 to Include FTD-C9orf72 Patients
July 16, 2024 07:00 ET
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Passage Bio
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...
Wave Life Sciences Announces Topline Results from Phase 1b/2a FOCUS-C9 Study of WVE-004 for C9orf72-associated Amyotrophic Lateral Sclerosis and Frontotemporal Dementia
May 23, 2023 07:30 ET
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Wave Life Sciences USA, Inc.
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...
Wave Life Sciences Announces Publication of Foundational Preclinical Data Supporting Development of WVE-004 for C9orf72-associated ALS and FTD
April 25, 2022 08:30 ET
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Wave Life Sciences USA, Inc.
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...
Wave Life Sciences Announces Positive Update to Ongoing Phase 1b/2a FOCUS-C9 Study Driven by Potent, Durable Reductions of Poly(GP) with Low, Single Doses of WVE-004
April 04, 2022 07:30 ET
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Wave Life Sciences USA, Inc.
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...
Amydis Announces Successful Completion of a Pre-IND Meeting with the FDA for the Development of a First-in-Class Retinal Tracer Targeting TDP43 for the Diagnosis of ALS
December 06, 2021 07:00 ET
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Amydis, Inc.
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...
Amydis Receives NIH Grant Award to Develop a Novel Tau Retinal Tracer for Alzheimer's Diseases and other Tauopathies
September 02, 2021 07:00 ET
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Amydis, Inc.
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,...
Wave Life Sciences Reports Second Quarter 2021 Financial Results and Provides Business Update
August 05, 2021 07:30 ET
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Wave Life Sciences USA, Inc.
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...
Wave Life Sciences Announces Initiation of Dosing in Phase 1b/2a FOCUS-C9 Clinical Trial of WVE-004 in Amyotrophic Lateral Sclerosis and Frontotemporal Dementia
July 20, 2021 08:00 ET
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Wave Life Sciences USA, Inc.
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...
Wave Life Sciences Reports First Quarter 2021 Financial Results and Provides Business Update
May 13, 2021 07:30 ET
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Wave Life Sciences USA, Inc.
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...
Wave Life Sciences Highlights Pipeline Progress and Expansion Leveraging New PN Backbone Chemistry Modifications
January 11, 2021 08:00 ET
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Wave Life Sciences USA, Inc.
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...