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ADDF and AFTD Partner to Support Wave Life Sciences’ FTD and ALS Clinical Program

NEW YORK, January 25, 2022 /PRNewswire/ — The Alzheimer’s Drug Discovery Foundation (ADDF) and the Association for Frontotemporal Degeneration (AFTD) today announced their partnership to support the FOCUS-C9 phase of Wave Life Sciences 1b/2a clinical trial investigating WVE-004 as a potential treatment for C9orf72– associated frontotemporal degeneration (C9-FTD), as well as amyotrophic lateral sclerosis (C9-ALS). The partnership provides an investment from the ADDF and AFTD that will support the assessment of fluid biomarkers, functional assessments and digital biomarkers in FOCUS-C9, potentially leading to clinically meaningful results to inform the development of treatments for DFT.

The ADDF and AFTD made the decision to support the FOCUS-C9 trial following a review of Wave’s clinical research application for the Treat FTD Fund, which supports the development of new drugs to treat FTD. Specifically, members of the Treat FTD Fund Joint Steering Committee, an expert panel convened by the ADDF in conjunction with the AFTD, and the ADDF Scientific Review Committee reviewed and commented on the phase 1b/2a study design, preclinical data supporting the program, and study team references.

“This investment exemplifies many of our priorities: collaboration, innovative science and the development of more rigorous methods for conducting clinical trials,” said Howard Fillit, MD, Co-Founder and Scientific Director of ADDF. “We must work together – as the ADDF and AFTD have done for years – to expand our scientific knowledge of all neurodegenerative diseases so that we can help provide meaningful treatments for people with FTD, Alzheimer’s and other related dementias.”

“The AFTD is proud to support, through the Treat FTD Fund, this innovative and potentially important clinical trial,” said Susan LJ Dickinson, CEO of AFTD. “For so many people living with FTD, this trial represents hope for effective treatments and to ease the journey of the next family facing this disease. Our ongoing collaborations with ADDF and Wave Life Sciences portend a future without this disease, and we are grateful to all clinical investigators and those diagnosed with FTD who will participate in this important research.”

The FOCUS-C9 trial is original in that it is a “basket” type study designed to evaluate the effects of genetically targeted treatment in patients with different disease phenotypes (FTD, FTD with ALS or ALS) that share a common molecular etiology, as has been used in oncology trials but has not yet been applied in neurology and C9orf72 population specifically. Wave’s focus on C9-FTD makes it a unique program in the C9orf72 clinical research landscape. It is also unique in the use of novel oligonucleotide chemistry which has shown enhanced cellular and nuclear uptake.

“We are grateful to the ADDF and AFTD panel of experts for their support and recognition of the innovative approach we have taken to rapidly advancing our clinical program,” said Michael Panzara, MD, MPH, Chief Medical Officer and Head of Therapeutic Discovery and Development at Wave Life Sciences. “In addition to advancing WVE-004 as a new genetically targeted treatment for FTD and ALS, we look forward to sharing the many learnings that will emerge from this trial with the wider medical and scientific communities.”

WVE-004 is a stereopure antisense oligonucleotide designed to selectively target transcriptional variants containing a hexanucleotide repeat expansion (G4VS2) associated with the C9orf72 gene, thus sparing C9orf72 protein. g4VS2 extensions in C9orf72 are one of the most common genetic causes of sporadic and hereditary forms of ALS and FTD.

Founded in 1998 by Leonard A. and Ronald S. Lauder, the Alzheimer’s Drug Discovery Foundation is dedicated to rapidly accelerating drug discovery to prevent, treat and cure Alzheimer’s disease. The ADDF is the only public charity focused exclusively on funding drug development for Alzheimer’s disease, employing a venture philanthropy model to support research in universities and the biotech industry.

Thanks to the generosity of its donors, the ADDF has awarded over $209 million to fund more than 690 Alzheimer’s disease drug discovery programs, biomarker programs and clinical trials in 19 countries. To learn more, please visit:

Founded in 2002, the Association for Frontotemporal Degeneration (AFTD) is the leading US nonprofit organization working to improve the lives of people with FTD, their care partners, and loved ones. The AFTD promotes and funds research into the diagnosis, treatment and cure of FTD; stimulates greater public awareness; provides information and support to those directly affected; promotes the education of health professionals; and advocates for appropriate and affordable services. To learn more, visit

Wave Life Sciences (Nasdaq: WVE) is a clinical-stage genetic medicine company committed to providing life-changing treatments for people struggling with devastating diseases. Wave aspires to develop best-in-class drugs across multiple therapeutic modalities using PRISM, the company’s proprietary drug discovery and development platform that enables the precise design, optimization and production of oligonucleotides stereopure. Driven by a resolute sense of urgency, the Wave team targets a wide range of genetically defined diseases so that patients and families can achieve a better future. To learn more, visit and follow Wave on Twitter @WaveLifeSci.

SOURCE Alzheimer’s Drug Discovery Foundation

Rodney N.

The author Rodney N.