CAMBRIDGE, Mass., Sept. 6, 2023 /PRNewswire/ -- 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 it will present preclinical data showing that TDP-43 (TAR DNA-binding protein 43) pathology drives loss of synaptic UNC13A function in neurodegenerative diseases including ALS and frontotemporal dementia (FTD). Data also showed that an UNC13A splice-switching antisense oligonucleotide (ASO) prevented cryptic exon inclusion in UNC13A transcripts, increased UNC13A protein levels, and normalized localization of UNC13A protein at the synapse.
QurAlis, a biotech focused on developing treatments for amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases, inked a collaboration deal with Unlearn to leverage its AI digital technology for clinical trials.
CAMBRIDGE, Mass., June 8, 2023 /PRNewswire/ -- 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 it will present new preclinical data showing the potential of the company's antisense oligonucleotides (ASOs) generated from its proprietary FlexASO™ Splice Modulator Platform. Data from two of QurAlis' FlexASO programs targeting rescue of STATHMIN-2 (STMN2) and UNC13A showed up to three-fold improvement over standard ASOs in both potency and biodistribution, and significantly reduced off-target effects, often from approximately 50 to 0, in human motor neurons. In addition, QurAlis' FlexASOs were not associated with cytokine or chemokine production. Observations from both programs therefore indicate that FlexASO™ technology results in a higher therapeutic index than observed with traditional ASO.
QurAlis has received clinical trial authorisation (CTA) in the European Union (EU) to initiate a first-in-human, Phase I ANQUR clinical trial of QRL-201 for the treatment of amyotrophic lateral sclerosis (ALS).
CAMBRIDGE, Mass., June 6, 2023 /PRNewswire/ -- 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 that the European review has been completed for the Clinical Trial Regulation (CTR) and each participating country has issued a notice of acceptance for QRL-201 for the potential treatment of ALS. QurAlis expects to initiate the Phase 1 ANQUR clinical trial of QRL-201 in participating European Union (EU) countries by the fourth quarter of 2023.
CAMBRIDGE, Mass., June 1, 2023 /PRNewswire/ -- 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 that Kasper Roet, Ph.D., CEO and co-founder, will participate in the following conferences in June.
QurAlis became the latest biotech to join a steadily teeming amyotrophic lateral sclerosis (ALS) space with the a Phase I trial for a therapy designed to restore motor neuron function. nnOn April 6, Cambridge, Massachusetts-based QurAlis announced it had dosed the first patient in the Phase I ANQUR trial (NCT05633459) of its lead candidate QRL-201, antisense oligonucleotide (ASO). nnQRL-201 is designed to restore the expression of the protein STATHMIN-2, which is implicated in neuronal repair and axonal stability. STATHMIN-2 is highly expressed in motor neurons, but patients with ALS often experience significant decreases in the protein.
CAMBRIDGE, Mass., March 20, 2023 /PRNewswire/ -- 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.
CAMBRIDGE, Mass., March 9, 2023 /PRNewswire/ -- 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 it has closed an oversubscribed $88 million Series B financing, bringing the total funds raised to $143.5 million. The financing was led by EQT Life Sciences, investing from the LSP Dementia Fund, Sanofi Ventures, and Droia Ventures, with participation from the ALS Investment Fund and existing investors LS Polaris Innovation Fund, Mission BioCapital, INKEF Capital, Dementia Discovery Fund, Amgen Ventures, MP Healthcare Venture Management, Mitsui Global Investment, Dolby Family Ventures, Mission Bay Capital, and Sanford Biosciences.
QurAlis picked up $88 million for two Phase I studies, hoping to ride regulatory waves driven by the recent FDA approval of Amylyx Pharmaceuticals’ ALS therapy.