UNC13A is an essential regulator of neurotransmitter release at synapses; genetic alteration of UNC13A caused by mis-splicing occurs in 58 percent of all ALS patients and up to half of all frontotemporal dementia cases
QurAlis’ therapeutic approach utilizing the FlexASO™ Splice Modulator Platform corrects UNC13A mis-splicing to restore synapse function and improve disease outcomes
CAMBRIDGE, Mass., March 20, 2023 – QurAlis Corporation, a clinical-stage biotechnology company developing breakthrough precision medicines for amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases with genetically validated targets, today announced the launch of its newest program that targets UNC13A mis-splicing, a critical genetic alteration in neurodegenerative diseases like ALS and frontotemporal dementia (FTD). Incorporating its proprietary FlexASO™ Splice Modulator Platform, QurAlis’ antisense oligonucleotides (ASOs) correct this mis-splicing, restore UNC13A protein production, and reduce cryptic exons that may contribute to disease progression. An exon is a segment of a DNA or RNA molecule containing information coding for a protein or peptide sequence.
“We are excited to bring our UNC13A program out of stealth. UNC13A is the third QurAlis program focused on a genetic target for the development of much-needed precision medicines for the sporadic ALS and FTD populations,” said Kasper Roet, Ph.D., chief executive officer of QurAlis. “This critical genetic alteration of UNC13A leading to mis-splicing is believed to occur in 58 percent of all ALS patients and up to half of all FTD cases. We look forward to advancing our UNC13A program along with our two lead clinical programs in ALS and robust pipeline so that we can make a real difference in patients’ lives.”
Angela Genge, M.D., FRCP(C), chief medical officer of QurAlis will introduce the Company’s UNC13A program during an oral presentation at the 2023 Muscular Dystrophy Association (MDA) Clinical and Scientific Congress. Dr. Genge’s oral presentation entitled, “Gene Directed Therapy for Sporadic ALS,” is scheduled for Tuesday, March 21, 2023, during the Gene Therapy in ALS section being held from 2:00-4:00pm CST.
UNC13A is an essential regulator of neurotransmitter release at synapses. In ALS and FTD, TDP-43 accumulates in the cytoplasm and no longer maintains its normal function controlling RNA metabolism in the nucleus. Due to its loss, certain pre-mRNA transcripts are mis-spliced resulting in expression of a cryptic exon-containing transcript that interferes with appropriate protein generation. UNC13A is a pre-mRNA that is mis-spliced due to loss of TDP-43 in disease. Fifty-eight percent of ALS patients and up to half of FTD patients carry a single nuclear polymorphism in the UNC13A gene which greatly exacerbates UNC13A mis-splicing leading to loss of function of the UNC13A protein.
The QurAlis FlexASO Splice Modulator Platform was developed to generate splice-switching ASOs with improved potency and an increased therapeutic index. In addition to UNC13A, the Company is currently exploring this ASO technology for multiple disease targets.
About QurAlis Corporation
QurAlis is trailblazing the path to conquering amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases with genetically validated targets with next-generation precision medicines. QurAlis’ proprietary platforms and unique biomarkers enable the design and development of drugs that act directly on disease-causing genetic alterations. Founded by an internationally recognized team of neurodegenerative biologists from Harvard Medical School and Harvard University, QurAlis is advancing a deep pipeline of antisense oligonucleotides and small molecule programs including addressing sub-forms of ALS that account for the majority of ALS patients. For more information, please visit www.quralis.com or follow us on Twitter @QurAlisCo.