Latest news with #QurAlis
Yahoo
18-06-2025
- Business
- Yahoo
QurAlis Announces New Global Headquarters
30,000 square foot state-of-the-art office suite, lab, and research facility doubles QurAlis' footprint in Cambridge's vibrant Alewife Biotech Park CAMBRIDGE, Mass., June 18, 2025--(BUSINESS WIRE)--QurAlis Corporation ("QurAlis"), a clinical-stage biotechnology company driving scientific breakthroughs into powerful precision medicines that have the potential to alter the trajectory of neurodegenerative and neurological diseases, today announced the opening of its new global corporate headquarters in Cambridge, MA. The company's new 30,000 square foot state-of-the-art research facility and office space located at 35 Cambridgepark Drive ("35 CPD"), doubles QurAlis' footprint in the heart of Cambridge's vibrant Alewife Biotech Park. "Maintaining our corporate headquarters in Cambridge positions us at the heart of a thriving biotechnology hub with world-class talent and leading research institutions," said Jason Brown, MBA, chief financial officer at QurAlis. "The facility at Alewife allowed us to expand our R&D footprint, lab capacity, and office space to support QurAlis' growth and enhance our leading precision medicine portfolio and platform capabilities. Our new headquarters provides our employees with a high-tech work environment conducive to collaboration and innovation with improved space and amenities. The space will continue to foster our mission-driven culture committed to discovering and developing medicines that have the potential to transform the lives of people living with neurodegenerative and neurological diseases." QurAlis and its landlord, Healthpeak, fully built out and furnished QurAlis' lab and office suite prior to occupancy. QurAlis' new headquarters offers a larger space with enhanced lab and discovery resources and a 5,000 square foot conferencing space for meeting and collaboration. More than one-third of the new facility is dedicated to a state-of-the-art 11,000 square foot lab with an expansive tissue culture suite and addition of an onsite antisense oligonucleotide (ASO) chemistry manufacturing lab to support programs utilizing QurAlis' FlexASO® technology platform. FlexASO® was developed to generate splice-switching ASOs with improved potency, increased therapeutic index and improved bio-distribution. This bespoke platform has the potential to tackle the spectrum of neurodegenerative and neurological diseases. "Healthpeak welcomes QurAlis to 35 Cambridgepark Drive," said Scott Bohn, chief development officer and head of lab at Healthpeak. "QurAlis' need for Class A lab space to progress their groundbreaking neuroscience work aligned seamlessly with our purpose-built life science campus, offering state-of-the-art amenities, a sustainable LEED Gold design, and unbeatable proximity to Alewife Station – an ideal environment for QurAlis' continued innovation and growth." Part of a 450,000 square foot RSF, three-building campus, 35 CPD is a LEED Gold 224,000 square foot RSF purpose-built life science building strategically located in West Cambridge directly across from the Alewife MBTA Station (Red line), at the end of Massachusetts Route 2, on Minuteman bike path and walkable to the Fresh Pond Reservation. 35 CPD, with attached garage, provides ample tenant amenities in the building including a Craft Food Hall, exclusive fitness center, private shower and locker rooms, secure bike room, client lounge, and activated outdoor spaces. QurAlis was represented in their search and lease negotiations by Carolyn Wheatley and Marc Consalvi at JLL. About QurAlis Corporation At QurAlis, we are neuro pioneers on a quest to cure, boldly seeking to translate scientific breakthroughs into powerful precision medicines. We work collaboratively with a relentless pursuit of knowledge, precise attention to craft, and compassion to discover and develop medicines that have the potential to transform the lives of people living with neurodegenerative and neurological diseases. QurAlis is the leader in development of precision therapies for amyotrophic lateral sclerosis (ALS). In addition to ALS, QurAlis is advancing a robust precision medicine pipeline to bring effective disease-modifying therapeutics to patients suffering from severe diseases defined by genetics and clinical biomarkers. For more information, please visit or follow us on X @QurAlisCo or LinkedIn. View source version on Contacts Kathy 310-403-8951
Business Wire
18-06-2025
- Business
- Business Wire
QurAlis Announces New Global Headquarters
CAMBRIDGE, Mass.--(BUSINESS WIRE)-- QurAlis Corporation ('QurAlis'), a clinical-stage biotechnology company driving scientific breakthroughs into powerful precision medicines that have the potential to alter the trajectory of neurodegenerative and neurological diseases, today announced the opening of its new global corporate headquarters in Cambridge, MA. The company's new 30,000 square foot state-of-the-art research facility and office space located at 35 Cambridgepark Drive ('35 CPD'), doubles QurAlis' footprint in the heart of Cambridge's vibrant Alewife Biotech Park. 'Maintaining our corporate headquarters in Cambridge positions us at the heart of a thriving biotechnology hub with world-class talent and leading research institutions,' said Jason Brown, MBA, chief financial officer at QurAlis. 'The facility at Alewife allowed us to expand our R&D footprint, lab capacity, and office space to support QurAlis' growth and enhance our leading precision medicine portfolio and platform capabilities. Our new headquarters provides our employees with a high-tech work environment conducive to collaboration and innovation with improved space and amenities. The space will continue to foster our mission-driven culture committed to discovering and developing medicines that have the potential to transform the lives of people living with neurodegenerative and neurological diseases.' QurAlis and its landlord, Healthpeak, fully built out and furnished QurAlis' lab and office suite prior to occupancy. QurAlis' new headquarters offers a larger space with enhanced lab and discovery resources and a 5,000 square foot conferencing space for meeting and collaboration. More than one-third of the new facility is dedicated to a state-of-the-art 11,000 square foot lab with an expansive tissue culture suite and addition of an onsite antisense oligonucleotide (ASO) chemistry manufacturing lab to support programs utilizing QurAlis' FlexASO® technology platform. FlexASO® was developed to generate splice-switching ASOs with improved potency, increased therapeutic index and improved bio-distribution. This bespoke platform has the potential to tackle the spectrum of neurodegenerative and neurological diseases. 'Healthpeak welcomes QurAlis to 35 Cambridgepark Drive,' said Scott Bohn, chief development officer and head of lab at Healthpeak. 'QurAlis' need for Class A lab space to progress their groundbreaking neuroscience work aligned seamlessly with our purpose-built life science campus, offering state-of-the-art amenities, a sustainable LEED Gold design, and unbeatable proximity to Alewife Station – an ideal environment for QurAlis' continued innovation and growth.' Part of a 450,000 square foot RSF, three-building campus, 35 CPD is a LEED Gold 224,000 square foot RSF purpose-built life science building strategically located in West Cambridge directly across from the Alewife MBTA Station (Red line), at the end of Massachusetts Route 2, on Minuteman bike path and walkable to the Fresh Pond Reservation. 35 CPD, with attached garage, provides ample tenant amenities in the building including a Craft Food Hall, exclusive fitness center, private shower and locker rooms, secure bike room, client lounge, and activated outdoor spaces. QurAlis was represented in their search and lease negotiations by Carolyn Wheatley and Marc Consalvi at JLL. About QurAlis Corporation At QurAlis, we are neuro pioneers on a quest to cure, boldly seeking to translate scientific breakthroughs into powerful precision medicines. We work collaboratively with a relentless pursuit of knowledge, precise attention to craft, and compassion to discover and develop medicines that have the potential to transform the lives of people living with neurodegenerative and neurological diseases. QurAlis is the leader in development of precision therapies for amyotrophic lateral sclerosis (ALS). In addition to ALS, QurAlis is advancing a robust precision medicine pipeline to bring effective disease-modifying therapeutics to patients suffering from severe diseases defined by genetics and clinical biomarkers. For more information, please visit or follow us on X @QurAlisCo or LinkedIn.
Yahoo
15-05-2025
- Business
- Yahoo
QurAlis Announces Exclusive License on Novel Mechanism for Fragile X Syndrome (FXS) to Enable Development of First Potential Disease-Modifying Therapy
Collaboration with UMass Chan Medical School confirmed FMR1-217 as well as an RNA-targeted mechanism to restore functional FMRP protein to develop potential treatments for FXS QurAlis' preliminary data suggest feasibility of biomarker to detect mis-splicing of FMR1 in FXS; company advancing FMR1-217 as precision medicine target in up to 80 percent of FXS patients Company applying its FlexASO® platform and expertise of splicing targets toward having a candidate nominated for IND-enabling studies in the near future CAMBRIDGE, Mass., May 15, 2025--(BUSINESS WIRE)--QurAlis Corporation ("QurAlis"), a clinical-stage biotechnology company driving scientific breakthroughs into powerful precision medicines that have the potential to alter the trajectory of neurodegenerative and neurological diseases, today announced it has entered into an exclusive license agreement with UMass Chan Medical School ("UMass Chan") on a novel RNA-targeted mechanism confirmed to restore functional protein for Fragile X syndrome (FXS). Fragile X syndrome is the leading inherited form of intellectual disability and the most common single genetic cause of autism. It is a genetic condition caused by a mutation of a single gene – Fragile X messenger ribonucleoprotein 1 (FMR1) – on the X chromosome. This mutation of FMR1 causes a range of developmental problems including learning disabilities, behavioral challenges, and cognitive impairment. QurAlis' exclusive license agreement is a result of its 2024 partnership and collaboration with UMass Chan to explore the biology of FXS to determine and confirm relevant targets that could enable antisense oligonucleotide (ASO)-mediated correction for FXS. QurAlis leveraged its deep understanding, knowledge and expertise in developing ASOs as part of the collaboration. QurAlis confirmed the findings from the original publication of the UMass Chan researchers and is advancing FMR1 as a precision medicine target in up to 80 percent of FXS patients. The mis-spliced form of FMR1, designated as FMR1-217, is widely expressed throughout cortical brain areas affected in FXS and can be measured in blood and cerebrospinal fluid. Preliminary data suggest biomarker feasibility to detect mis-splicing of FMR1 in patients with FXS. "FXS is a devastating neurodevelopmental disorder with no effective disease-modifying therapies available. Our initial partnership with UMass Chan confirmed that FMR1-217 is a validated genetic target for FXS," said Kasper Roet, Ph.D., chief executive officer and co-founder of QurAlis. "This groundbreaking discovery of a novel RNA-targeted mechanism to restore functional protein for FXS and the feasibility of a biomarker to detect mis-splicing of FMR1 in FXS patients opens up a completely new type of therapeutic approach through splice correction. We look forward to applying QurAlis' FlexASO® platform and deep knowledge and expertise of ASO splicing targets toward having a candidate nominated for IND-enabling studies in the near future, so that we can bring a potential new precision medicine option to patients." Joel Richter, Ph.D., the Arthur F. Koskinas Chair in Neuroscience and professor of molecular medicine at UMass Chan, and colleagues Sneha Shah, Ph.D., and Jonathan K. Watts, Ph.D., together with Elizabeth Berry-Kravis, M.D., Ph.D., at Rush University Medical Center, have shown that aberrant alternative splicing, or mis-splicing, of messenger RNA (mRNA) plays a fundamental role in FXS. In a seminal publication by the group, it was revealed that in FXS patients, FMR1 mRNA is still being expressed, but is mis-spliced, comprising a short, truncated alternative mRNA variant called FMR1-217 which results in non-functional FMRP protein expression. Working with patient-derived cells, Dr. Richter's lab and Dr. Berry-Kravis initially demonstrated that ASOs can successfully inhibit the mis-splicing, reduce expression of FMR1-217, rescue proper FMR1 mRNA, and restore FMRP protein expression. "This is a meaningful step in the process of taking basic biological discoveries and turning them into practical therapies that can benefit patients in the clinic," said Dr. Richter. "QurAlis' platform and expertise in neurodegenerative disorders are industry leading and well positioned to address the mis-splicing of FMR1 RNA and restore functional FMRP protein expression. This partnership has not only validated our years-long research but also has resulted in the confirmation of a novel target for FXS, which we hope will lead to much-needed treatment options for FXS patients and their families." Dr. Berry-Kravis added, "I am very excited that we will be able to continue development of this potential genetically based disease-modifying FMRP-restoring therapy that is expected to have a major impact on the FXS field and the spectrum of treatment options available to improve function in people with FXS." An orphan disease, FXS affects approximately 87,000 individuals in the U.S. alone – one in 4,000 men and one in 6,000 women. Though FXS occurs in both genders, males are more frequently affected than females, and generally with greater severity. In addition to intellectual disability, FXS patients endure a wide range of disabling symptoms including severe anxiety, social aversion, hyperactivity and attention deficit, sensory hypersensitivity, aggression, developmental seizures, and others. There are no effective disease-modifying therapies currently available for FXS. ASOs are short, engineered single-stranded DNA/RNA molecules that can selectively bind RNA to regulate its expression in the cell. ASO technology has been leading in the field of protein regulation and has since allowed us to develop treatments for neurodegenerative disease by changing the expression of genes connected to the disease. QurAlis' FlexASO® platform was developed to generate splice-switching ASOs with improved potency, increased therapeutic index and improved bio-distribution. This bespoke platform has the potential to tackle the spectrum of neurodegenerative and neurological diseases. About QurAlis Corporation At QurAlis, we are neuro pioneers on a quest to cure, boldly seeking to translate scientific breakthroughs into powerful precision medicines. We work collaboratively with a relentless pursuit of knowledge, precise attention to craft, and compassion to discover and develop medicines that have the potential to transform the lives of people living with neurodegenerative and neurological diseases. QurAlis is the leader in development of precision therapies for amyotrophic lateral sclerosis (ALS). In addition to ALS, QurAlis is advancing a robust precision medicine pipeline to bring effective disease-modifying therapeutics to patients suffering from severe diseases defined by genetics and clinical biomarkers. For more information, please visit or follow us on X @QurAlisCo or LinkedIn. View source version on Contacts Kathy 310-403-8951 Sign in to access your portfolio
Business Wire
15-05-2025
- Business
- Business Wire
Quiver Bioscience and QurAlis Announce Research Collaboration to Advance Novel Therapeutic Approach for Fragile X Syndrome
CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Quiver Bioscience ('Quiver') and QurAlis Corporation ('QurAlis'), today announced that the companies have entered into a research collaboration to advance a novel gene-targeted therapeutic approach for the treatment of Fragile X syndrome (FXS). The goal of the collaboration is to combine Quiver's unique 'Genomic Positioning System' (GPS) drug discovery platform with QurAlis' expertise in developing next-generation precision medicines for neurodegenerative and neurological diseases to build a foundational data package in support of advancing a potentially transformative therapeutic for FXS. Quiver's GPS platform integrates unique-in-world, scalable, human neuronal electrophysiology data (the 'language' of the brain) with artificial intelligence and machine learning (AI/ML) to drive novel insights into disease biology and enable optimized drug discovery. Quiver has successfully applied its GPS approach to a variety of central nervous system (CNS) disorders and recently published modeling and drug discovery efforts in FXS. 'Our platform technology is uniquely suited to improving understanding of the molecular and cellular basis of neurogenetic disorders such as FXS. We are excited to embark on this partnership with QurAlis which aspires to bring about groundbreaking therapies for the FXS community,' said Graham Dempsey, Ph.D., co-founder and CEO of Quiver Bioscience. 'FXS is a devastating neurodevelopmental disease. It is the leading inherited form of intellectual disability and known cause of autism for which there are no disease-modifying therapies,' said Kasper Roet, Ph.D., CEO and co-founder of QurAlis. 'We look forward to this research collaboration with Quiver. The combination of enabling technologies and drug development experience built through this partnership holds great promise for progressing novel therapeutics for FXS, for which there exists a significant unmet medical need.' FXS, the leading genetic form of intellectual disability and autism spectrum disorder, is caused by loss of the FMR1 encoded protein Fragile X Messenger Ribonucleoprotein (FMRP). It currently affects approximately 87,000 individuals in the U.S. alone – occurring at an incidence of 1 in 4,000 males and 1 in 6,000 females. In addition to intellectual disability, FXS symptoms include delays in development, seizures, speech difficulties, hyperactivity and attention deficit, severe anxiety, and others. There are no disease-modifying therapies currently available for FXS. Destum Partners acted as transaction advisor to Quiver Bioscience. About Quiver Bioscience Quiver Bioscience is a technology-driven company established to create transformational medicines for the brain while simultaneously uncovering new biology and novel, effective drug targets. Using advanced single-cell imaging and multi-omics, we are building the world's most information-rich neuronal insight map via our "Genomic Positioning System." Our approach integrates cutting-edge scalable human models, state-of-the-art technology and proprietary engineering, and learning and surrogate AI/ML models to identify novel therapeutic targets and the best candidate molecules to deliver new and meaningful therapeutics to patients. For information, including additional publications describing application of Quiver's GPS to drug discovery, visit or follow us on LinkedIn. About QurAlis Corporation At QurAlis, we are neuro pioneers on a quest to cure, boldly seeking to translate scientific breakthroughs into powerful precision medicines. We work collaboratively with a relentless pursuit of knowledge, precise attention to craft, and compassion to discover and develop medicines that have the potential to transform the lives of people living with neurodegenerative and neurological diseases. QurAlis is the leader in development of precision therapies for amyotrophic lateral sclerosis (ALS). In addition to ALS, QurAlis is advancing a robust precision medicine pipeline to bring effective disease-modifying therapeutics to patients suffering from severe diseases defined by genetics and clinical biomarkers. For more information, please visit or follow us on X @QurAlisCo or LinkedIn.
Associated Press
15-05-2025
- Business
- Associated Press
QurAlis Announces Exclusive License on Novel Mechanism for Fragile X Syndrome (FXS) to Enable Development of First Potential Disease-Modifying Therapy
CAMBRIDGE, Mass.--(BUSINESS WIRE)--May 15, 2025-- QurAlis Corporation ('QurAlis'), a clinical-stage biotechnology company driving scientific breakthroughs into powerful precision medicines that have the potential to alter the trajectory of neurodegenerative and neurological diseases, today announced it has entered into an exclusive license agreement with UMass Chan Medical School ('UMass Chan') on a novel RNA-targeted mechanism confirmed to restore functional protein for Fragile X syndrome (FXS). Fragile X syndrome is the leading inherited form of intellectual disability and the most common single genetic cause of autism. It is a genetic condition caused by a mutation of a single gene – Fragile X messenger ribonucleoprotein 1 (FMR1) – on the X chromosome. This mutation of FMR1 causes a range of developmental problems including learning disabilities, behavioral challenges, and cognitive impairment. QurAlis' exclusive license agreement is a result of its 2024 partnership and collaboration with UMass Chan to explore the biology of FXS to determine and confirm relevant targets that could enable antisense oligonucleotide (ASO)-mediated correction for FXS. QurAlis leveraged its deep understanding, knowledge and expertise in developing ASOs as part of the collaboration. QurAlis confirmed the findings from the original publication of the UMass Chan researchers and is advancing FMR1 as a precision medicine target in up to 80 percent of FXS patients. The mis-spliced form of FMR1, designated as FMR1-217, is widely expressed throughout cortical brain areas affected in FXS and can be measured in blood and cerebrospinal fluid. Preliminary data suggest biomarker feasibility to detect mis-splicing of FMR1 in patients with FXS. 'FXS is a devastating neurodevelopmental disorder with no effective disease-modifying therapies available. Our initial partnership with UMass Chan confirmed that FMR1-217 is a validated genetic target for FXS,' said Kasper Roet, Ph.D., chief executive officer and co-founder of QurAlis. 'This groundbreaking discovery of a novel RNA-targeted mechanism to restore functional protein for FXS and the feasibility of a biomarker to detect mis-splicing of FMR1 in FXS patients opens up a completely new type of therapeutic approach through splice correction. We look forward to applying QurAlis' FlexASO® platform and deep knowledge and expertise of ASO splicing targets toward having a candidate nominated for IND-enabling studies in the near future, so that we can bring a potential new precision medicine option to patients.' Joel Richter, Ph.D., the Arthur F. Koskinas Chair in Neuroscience and professor of molecular medicine at UMass Chan, and colleagues Sneha Shah, Ph.D., and Jonathan K. Watts, Ph.D., together with Elizabeth Berry-Kravis, M.D., Ph.D., at Rush University Medical Center, have shown that aberrant alternative splicing, or mis-splicing, of messenger RNA (mRNA) plays a fundamental role in FXS. In a seminal publication by the group, it was revealed that in FXS patients, FMR1 mRNA is still being expressed, but is mis-spliced, comprising a short, truncated alternative mRNA variant called FMR1-217 which results in non-functional FMRP protein expression. Working with patient-derived cells, Dr. Richter's lab and Dr. Berry-Kravis initially demonstrated that ASOs can successfully inhibit the mis-splicing, reduce expression of FMR1-217, rescue proper FMR1 mRNA, and restore FMRP protein expression. 'This is a meaningful step in the process of taking basic biological discoveries and turning them into practical therapies that can benefit patients in the clinic,' said Dr. Richter. 'QurAlis' platform and expertise in neurodegenerative disorders are industry leading and well positioned to address the mis-splicing of FMR1 RNA and restore functional FMRP protein expression. This partnership has not only validated our years-long research but also has resulted in the confirmation of a novel target for FXS, which we hope will lead to much-needed treatment options for FXS patients and their families.' Dr. Berry-Kravis added, 'I am very excited that we will be able to continue development of this potential genetically based disease-modifying FMRP-restoring therapy that is expected to have a major impact on the FXS field and the spectrum of treatment options available to improve function in people with FXS.' An orphan disease, FXS affects approximately 87,000 individuals in the U.S. alone – one in 4,000 men and one in 6,000 women. Though FXS occurs in both genders, males are more frequently affected than females, and generally with greater severity. In addition to intellectual disability, FXS patients endure a wide range of disabling symptoms including severe anxiety, social aversion, hyperactivity and attention deficit, sensory hypersensitivity, aggression, developmental seizures, and others. There are no effective disease-modifying therapies currently available for FXS. ASOs are short, engineered single-stranded DNA/RNA molecules that can selectively bind RNA to regulate its expression in the cell. ASO technology has been leading in the field of protein regulation and has since allowed us to develop treatments for neurodegenerative disease by changing the expression of genes connected to the disease. QurAlis' FlexASO® platform was developed to generate splice-switching ASOs with improved potency, increased therapeutic index and improved bio-distribution. This bespoke platform has the potential to tackle the spectrum of neurodegenerative and neurological diseases. About QurAlis Corporation At QurAlis, we are neuro pioneers on a quest to cure, boldly seeking to translate scientific breakthroughs into powerful precision medicines. We work collaboratively with a relentless pursuit of knowledge, precise attention to craft, and compassion to discover and develop medicines that have the potential to transform the lives of people living with neurodegenerative and neurological diseases. QurAlis is the leader in development of precision therapies for amyotrophic lateral sclerosis (ALS). In addition to ALS, QurAlis is advancing a robust precision medicine pipeline to bring effective disease-modifying therapeutics to patients suffering from severe diseases defined by genetics and clinical biomarkers. For more information, please visit or follow us on X @QurAlisCo or LinkedIn. View source version on CONTACT: Kathy Vincent [email protected] 310-403-8951 KEYWORD: UNITED STATES NORTH AMERICA MASSACHUSETTS INDUSTRY KEYWORD: RESEARCH NEUROLOGY GENETICS BIOTECHNOLOGY HEALTH UNIVERSITY PHARMACEUTICAL SCIENCE EDUCATION SOURCE: QurAlis Corporation Copyright Business Wire 2025. PUB: 05/15/2025 07:45 AM/DISC: 05/15/2025 07:44 AM
