Joseph Klim
Director of Neuroscience NuCyRNA Therapeutics
Seminars
Thursday 4th June 2026
Panel Discussion: How Can We Treat Underlying TDP43 Pathology Outside of Clinical Presentation to Develop Effective Treatments in ALS, FTD & Beyond?
10:00 am
- What is the current understanding of TDP‑43 pathology across ALS, FTD, and related neurodegenerative diseases, and how does it inform therapeutic strategies?
- How can therapies be designed to target upstream TDP‑43 mislocalization, aggregation, or loss-of-function, independent of clinical symptom presentation?
- The role of biomarkers and imaging in detecting subclinical TDP‑43 pathology and enabling earlier intervention in patients
- Challenges and opportunities in translating preclinical findings into human studies, including target engagement, dosing strategies, and safety considerations
- Potential combinatorial approaches, such as pairing TDP‑43-targeted therapies with neuroprotective or gene-modulating modalities to enhance efficacy
Thursday 4th June 2026
Panel Discussion: Blending Modalities for Complex Neurodegeneration: What Will It Take to Treat ALS?
3:00 pm
- How regulatory expectations differ across modalities (oral small molecules, ASOs, viral gene therapies, RNA medicines, cell therapies) and how developers can prepare for emerging FDA/EMA frameworks in ALS
- Which modalities are best suited for distinct ALS biological subtypes, including TDP-43 proteinopathy, C9orf72 repeat expansion, inflammatory/neuroimmune signatures, and mitochondrial/metabolic dysfunction
- Opportunities and challenges for combining modalities—for example coupling gene modulation with neuroprotective small molecules, pairing oligos with smallmolecule chaperones, or integrating metabolic stabilizers with anti-inflammatory approaches
- How modality mechanisms intersect (protein homeostasis, RNA regulation, axonal transport, synaptic resilience) and how cross-mechanistic synergy could inform trial design or biomarker development
- What is needed to run smarter, modality-agnostic clinical trials—harmonized biomarkers (NfL, speech, digital mobility), shared control arms, adaptive designs, and platform/basket approaches
