Summary of Voyager Therapeutics Conference Call Company Overview - Voyager Therapeutics is a multimodality neurotherapeutics company focused on optimizing delivery systems for gene therapies targeting neurological diseases, particularly Alzheimer's disease [2][3] Core Programs and Partnerships - The company has two main platforms: a gene therapy platform that discovers capsids capable of crossing the blood-brain barrier (BBB) and a multimodality approach to optimize delivery [2] - Voyager is heavily focused on Alzheimer's disease, with multiple partner programs involving Neurocrine, Novartis, and AstraZeneca [3] - The company has a program in Phase 1 for an anti-TAU antibody, with expected readouts next year [3] Key Insights on TAU Antibody Strategy - Voyager's TAU antibody strategy is based on a unique animal model that expresses human TAU, which may predict the efficacy of antibodies in humans [4][5] - Previous failures of other TAU antibodies are acknowledged, but Voyager believes their approach, which includes a specific antibody for pathological forms of TAU, could yield better results [6][7] - The company plans to use TAU-PET imaging as a primary measurement for pharmacodynamics, as fluid-based biomarkers have shown inconsistent results [9][10] Gene Therapy Considerations - Voyager's gene therapy approach aims to deliver therapies with a lower risk of inflammatory side effects, using a capsid that detargets the liver and achieves significant knockdown of TAU [16][18] - Concerns about the potential risks of knocking down all forms of TAU are addressed, with references to animal studies showing viability despite TAU knockouts [19][20] Future Development and Partnerships - Voyager is looking for partnerships to advance their TAU antibody and gene therapy programs, particularly for Phase 3 trials [12][35] - The company is optimistic about the potential of their frataxin gene therapy program, which aims to address both neurological and cardiac effects [24][26] Broader Industry Context - The discussion touches on the regulatory landscape for gene therapies, particularly for rare diseases, and the importance of demonstrating significant effect sizes on hard endpoints for accelerated approvals [33][34] - Voyager emphasizes its commitment to addressing severe neurological diseases through various modalities, including gene therapy and small molecules [42] Conclusion - Voyager Therapeutics is positioned as a key player in the neurotherapeutics space, with a strong focus on Alzheimer's disease and innovative delivery mechanisms. The company is actively pursuing partnerships and clinical trials to advance its promising therapies [42]