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Driving Scientific Breakthroughs into Powerful Precision Medicines for ALS and Other Neurodegenerative Diseases

Scientific Presentations and Publications

QRL-201

ENCALS 2025 – ‘ANQUR, the First-in-Human Phase 1 Study of QRL-201 in ALS Advances to Dose-Range Finding Using a Novel Population Pharmacokinetic Analysis’ (poster)

ALS Nexus 2024 – ‘Stathmin-2, a New Target for ALS, Development of QRL-201’ (presentation)

Target ALS 2024 – ‘QRL-201-01 ANQUR: A multi-center, randomized, double-blind, placebo-controlled, multiple-ascending dose study to evaluate the safety and tolerability of QRL-201 in amyotrophic lateral sclerosis’ (presentation)

QRL-101

Target ALS 2025 – ‘Development of QRL-101 for ALS’ (presentation)

ENCALS 2025 – ‘QRL-101 – Phase 1 Studies Evaluating Safety and Pharmacodynamics of a KCNQ2/3 Modulator in Healthy Volunteers & Next Steps for Development in ALS’ (poster)

PACTALS 2023 – ‘Evaluating the Safety, Tolerability, and Pharmacokinetics of QRL-101 in Two Phase 1 Studies: QRL-101-01 in Healthy Adults and QRL-101-02 in Adults Living With ALS’ (poster)

MND Symposium 2023 – ‘Evaluating the Safety, Tolerability and Pharmacokinetics of QRL-101 in Two Phase 1 Studies: QRL-101-01 in Healthy Adults and QRL-101-02 in Adults Living With ALS’ (poster)

FlexASO® Splice Modulator Platform

RNA Therapeutics USA 2024 – ‘Recent Advances in the Development of Splice-Switching Oligonucleotides for CNS Diseases’  (presentation)

Oligonucleotide Therapeutics and Delivery 2024 – ‘FlexASO and Human Cellular Models to Streamline Oligonucleotide Development to the Clinic’ (presentation)

4th Annual Oligonucleotides for CNS Summit 2024 – ‘FlexASO and Human Cellular Models to Streamline Oligonucleotide Development to the Clinic’ (presentation)

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