QurAlis

QurAlis' CEO and founder, Kasper Roet, recently spoke with?Deborah Westphal, host of Research Renaissance, to explore the evolution of #ALS drug development, the power of genetic insights, and the future of #personalizedmedicine for #neurodegenerativediseases and #neurologicaldiseases. Kasper shares his journey from spinal cord injury research to ALS and how genetic discoveries reshaped his focus. He also talks about the pivotal role of genomics, AI, and stem cell technology in accelerating ALS treatments, the challenges being a biotech entrepreneur, QurAlis' founding and the pioneering #precisionmedicines QurAlis is developing to target ALS, epilepsy, and other neurodegenerative and neurological diseases at the molecular level, and the future of neurological disease research. Presented by The Karen Toffler Charitable Trust, Research Renaissance delves into the frontiers of cutting-edge brain science, exploring the complexities of neurological diseases, their root causes, potential treatments, and the pursuit of cures. You can listen to the episode at https://bit.ly/4253Blk

Recent research published in Nature explores how G-quadruplex (G4) RNA structures affect the phase behavior of FMRP, which plays a key role in mRNA trafficking. The research shows that G-rich RNA sequences significantly influence the dynamics and material properties of protein condensates, even without altering protein binding affinity. Increasing G-content modulates the viscosity and dynamics of these condensates. In the case of FMRP, post-translational methylation reduces RNA binding while having no effect on the properties of the condensates. This work offers new insights into how RNA structure shapes cellular organization, particularly in neurological diseases like #FragileXsyndrome. ? Congratulations to Alfredo Vidal Ceballos, Anna Geissmann, Denize C. Favaro, Priyasha Deshpande and Shana Elbaum-Garfinkle for their published research.

QurAlis was honored to sponsor the 10th Annual OPT Congress 2025 that was held last week in Boston. OPT Congress brings together leading chemists, biologists, toxicologists, CMC experts, regulatory specialists and technology providers to discuss advances in next-generation #oligonucleotides and #mRNA therapeutics. QurAlis' CTO, Hagen Cramer, was a featured speaker and presented, "How to Work with Your CMO for Successful Oligonucleotide Manufacturing." #precisionmedicine #neurodegenerativediseases #neurologicaldiseases

Our CEO and founder, Kasper Roet, spoke with Ben Comer and Matthew Pillar on the #BusinessofBiotech podcast to discuss how his diverse entrepreneurial and hands-on background including work at the Dutch Brain Bank informed his leadership at QurAlis. On this episode, Kasper talks about QurAlis' clinical programs targeting specific #geneticmechanisms in #ALS, #epilepsy, and other #neurodegenerativediseases and #neurologicaldiseases currently underway. He also talks about the regulatory challenges of bringing genetic medicines for rare diseases to market in the U.S., and how biotech leaders are working together to influence change so that American patients benefit from American innovation. Tune in to the full discussion here: https://bit.ly/4hFGQKw

QurAlis is excited to announce positive topline data from our Phase 1 clinical trial of QRL-101 evaluating biomarkers of #ALS and #epilepsy in healthy volunteers (QRL-101-05; NCT06681441). Results demonstrated that QRL-101 has the potential to provide a therapeutic effect in both diseases. #Kv7.2/7.3 is a voltage-gated potassium channel whose role is crucial for the regulation of both neuronal excitability and membrane potential. QRL-101 is a potentially best-in-class selective Kv7.2/7.3 ion channel opener for the treatment of hyperexcitability-induced disease progression in ALS, which occurs in both sporadic and genetic forms of ALS, with the majority caused by the mis-splicing of the KCNQ2 gene in the pre-mRNA. Kv7 is also a clinically validated target to regulate the hyperexcitable state in epilepsy. Read more in our press release at https://bit.ly/3FznUzH #precisionmedicine #neurodegenerativediseases #neurologicaldiseases

QurAlis joins the more than 7,300 partners in 120 countries to celebrate #BrainAwarenessWeek (#BAW25) which kicked off Monday, 3/10 and continues through Sunday, 3/16. Coordinated by the Dana Foundation, BAW25 is a global celebration of #neuroscience to educate about the wonders of the brain and raise awareness about brain health and the progress and benefits of brain research. #BrainWeek #BAW25

QurAlis is excited to announce positive topline data from our Phase 1 clinical trial of QRL-101 evaluating biomarkers of #ALS and #epilepsy in healthy volunteers (QRL-101-05; NCT06681441). Results demonstrated that QRL-101 has the potential to provide a therapeutic effect in both diseases. #Kv7.2/7.3 is a voltage-gated potassium channel whose role is crucial for the regulation of both neuronal excitability and membrane potential. QRL-101 is a potentially best-in-class selective Kv7.2/7.3 ion channel opener for the treatment of hyperexcitability-induced disease progression in ALS, which occurs in both sporadic and genetic forms of ALS, with the majority caused by the mis-splicing of the KCNQ2 gene in the pre-mRNA. Kv7 is also a clinically validated target to regulate the hyperexcitable state in epilepsy. Read more in our press release at https://bit.ly/3FznUzH #precisionmedicine #neurodegenerativediseases #neurologicaldiseases

QurAlis is excited to announce positive topline data from our Phase 1 clinical trial of QRL-101 evaluating biomarkers of #ALS and #epilepsy in healthy volunteers (QRL-101-05; NCT06681441). Results demonstrated that QRL-101 has the potential to provide a therapeutic effect in both diseases. #Kv7.2/7.3 is a voltage-gated potassium channel whose role is crucial for the regulation of both neuronal excitability and membrane potential. QRL-101 is a potentially best-in-class selective Kv7.2/7.3 ion channel opener for the treatment of hyperexcitability-induced disease progression in ALS, which occurs in both sporadic and genetic forms of ALS, with the majority caused by the mis-splicing of the KCNQ2 gene in the pre-mRNA. Kv7 is also a clinically validated target to regulate the hyperexcitable state in epilepsy. Read more in our press release at https://bit.ly/3FznUzH #precisionmedicine #neurodegenerativediseases #neurologicaldiseases

QurAlis was proud to sponsor the 2025 ALS ONE Snow Ball Gala that took place earlier this month. It was the tenth annual gala and another magical evening at which record funds were raised to help advance critical #ALS research and care. #precisionmedicine #neurodegenerativediseases

QurAlis was proud to sponsor the 2025 ALS ONE Snow Ball Gala that took place earlier this month. It was the tenth annual gala and another magical evening at which record funds were raised to help advance critical #ALS research and care. #precisionmedicine #neurodegenerativediseases