UNC13A

QurAlis Strengthens Global Presence With Opening of European Headquarters That Includes R&D Manufacturing

Retrieved on: 
Wednesday, January 3, 2024
Asos, ALS, FTD, UNC13A, Doctor of Philosophy, Pathology, Biomarker, Frontotemporal dementia, Patient, Pharmaceutical industry

CAMBRIDGE, Mass., Jan. 3, 2024 /PRNewswire/ -- QurAlis Corporation, a clinical-stage biotechnology company driving scientific breakthroughs into powerful precision medicines that will alter the trajectory of amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), and other neurodegenerative diseases, today announced the opening of its European Union (EU) headquarters in Leiden, The Netherlands. This new location will serve as the hub for the Company's European operations including the production of QurAlis' products for its clinical trials through commercialization.

Key Points: 
  • This new location will serve as the hub for the Company's European operations including the production of QurAlis' products for its clinical trials through commercialization.
  • "The expansion of our operations into Europe represents a new chapter for QurAlis, building upon the tremendous momentum of our organization," said Kasper Roet, Ph.D., CEO and co-founder of QurAlis.
  • "In a short period of time, QurAlis has made significant progress with regulatory approvals for our clinical programs in the EU, Canada, and the UK.
  • In addition to the new Leiden office, QurAlis' global corporate headquarters are in Cambridge, Massachusetts.

QurAlis to Present Data That Show TDP-43 Pathology Drives Loss of Synaptic UNC13A Function in Neurodegenerative Diseases Including ALS and Frontotemporal Dementia

Retrieved on: 
Wednesday, November 29, 2023
Therapeutic index, Pathology, ALS, Patient, Asos, FTD, Synapse, TAR, Frontotemporal dementia, HIV disease progression rates, UNC13A

CAMBRIDGE, Mass., Nov. 29, 2023 /PRNewswire/ -- QurAlis Corporation, a clinical-stage biotechnology company driving scientific breakthroughs into powerful precision medicines that will alter the trajectory of amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), and other neurodegenerative diseases, 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 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.

Key Points: 
  • 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.
  • UNC13A is an essential regulator of neurotransmitter release at synapses and is one of a number of pre-mRNAs that becomes mis-spliced due to loss of nuclear 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 or show TDP-43 pathology which greatly exacerbates UNC13A mis-splicing leading to loss of function of the UNC13A protein.
  • In addition to UNC13A, QurAlis is currently exploring this ASO technology for multiple other disease targets.

AcuraStem Enters into a License Agreement with Takeda to Advance PIKFYVE Therapeutics

Retrieved on: 
Monday, September 25, 2023
Frontotemporal dementia, Asos, BM, PIKFYVE, Human, Therapy, Head, Survival, Innovation, MBA, UNC13A, Doctor of Philosophy, DSM-IV codes, Science, Paul Auguste Hariot, Shi, Dementia, Patient, Amyotrophy, Master of Science, ASO, Government, IND, FTD, MRCP, Pharmaceutical industry, Takeda, ALS

MONROVIA, Calif., Sept. 25, 2023 /PRNewswire/ -- AcuraStem, a patient-based biotechnology company pioneering how treatments are developed for neurodegenerative diseases, announced today that it has entered into a license agreement with Takeda to develop and commercialize AcuraStem's PIKFYVE targeted therapeutics including AS-202, an innovative antisense oligonucleotide (ASO) for the treatment of Amyotrophic Lateral Sclerosis (ALS).

Key Points: 
  • AcuraStem's therapeutic strategy for PIKFYVE focuses on addressing neurodegeneration by expelling toxic protein aggregates and protecting healthy neuronal function.
  • Under the terms of the agreement, Takeda will receive an exclusive, worldwide license to AcuraStem's PIKFYVE program.
  • "I'm very pleased that AcuraStem is partnering with Takeda around their PIKFYVE program," said Justin Ichida, PhD.
  • Destum Partners acted as transaction advisor to AcuraStem, and Foley Hoag acted as legal counsel to AcuraStem.

QurAlis to Present Data Showing Link Between TDP-43 Pathology and Role of UNC13A in Neuronal Biology Related to ALS and Other Neurodegenerative Diseases

Retrieved on: 
Wednesday, September 6, 2023
Pathology, TAR, HIV disease progression rates, Patient, Frontotemporal dementia, Synapse, ASO, ALS, FTD, Conference, Agriculture, Vaccine, Disease, UNC13A

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.

Key Points: 
  • 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.
  • "UNC13A is a genetically validated target in ALS and FTD and our results showed that TDP-43 pathology drives loss of synaptic UNC13A function.
  • "We recently launched our newest program that targets UNC13A mis-splicing and look forward to advancing this program along with our other programs targeting neurodegenerative diseases so that we can make a real difference in patients' lives."
  • QurAlis will present these data in a poster presentation at the 1st Biennial Conference on TDP-43 Function and Dysfunction in Disease in Trieste, Italy on Thursday, September 7, 2023.

QurAlis Reveals Newest Program Targeting UNC13A RNA Mis-splicing Incorporating its FlexASO™ Platform

Retrieved on: 
Monday, March 20, 2023
Asos, DNA, FRCP, Gene, Therapeutic index, Episcopal conference, RNA, Gene therapy, Cytoplasm, HIV disease progression rates, Patient, Muscular Dystrophy Association, CST, Frontotemporal dementia, Synapse, ASO, MDA, ALS, FTD, Disease, Vaccine, UNC13A

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.

Key Points: 
  • 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.

Maze Therapeutics Announces Publication in Nature Describing a Novel Mechanism Linking Well-Established Genetic Drivers of ALS and FTD

Retrieved on: 
Wednesday, February 23, 2022
Research, Mental Health, Genetics, Clinical Trials, Other Health, Biotechnology, Pharmaceutical, Health, Science, Other Science, Risk, Lists of diseases, Human, Johns Hopkins University, HIV disease progression rates, Twitter, Compass, RNA, Stanford University, Gene, University, Data science, FTD, Therapy, Frontotemporal dementia, UCSF, Disease, Amyotrophic lateral sclerosis, Neuron, Nervous system, LinkedIn, ALS, Neurodegeneration, RNA splicing, Patient, Technology, Genetic variation, University of Pennsylvania, Cell nucleus, Maze, Mayo Clinic, Research, Vaccine, UNC13A

The research was led by Maze Therapeutics co-founder Aaron Gitler, Ph.D., at Stanford University in collaboration with researchers at Maze Therapeutics and other institutions, including Mayo Clinic, UCSF, University of Pennsylvania and Johns Hopkins University.

Key Points: 
  • The research was led by Maze Therapeutics co-founder Aaron Gitler, Ph.D., at Stanford University in collaboration with researchers at Maze Therapeutics and other institutions, including Mayo Clinic, UCSF, University of Pennsylvania and Johns Hopkins University.
  • The findings help explain how a genetic variant of UNC13A and dysfunctional TDP-43 can lead to the development of ALS or FTD.
  • UNC13A and TDP-43 are well-established contributors to ALS, but how they lead to disease had been a mystery.
  • Our Compass platform is built to find genetic insights like this, which is a key step in translating them into therapies for patients.