QurAlis Announces Exclusive License on Novel Mechanism for Fragile X Syndrome (FXS) to Enable Development of First Potential Disease-Modifying Therapy
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 www.quralis.com or follow us on X @QurAlisCo or LinkedIn.
View source version on businesswire.com:https://www.businesswire.com/news/home/20250515683206/en/
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
http://www.businesswire.com/news/home/20250515683206/en
Hashtags
Try Our AI Features
Explore what Daily8 AI can do for you:
Comments
No comments yet...
Related Articles
Yahoo
31-07-2025
- Yahoo
Asos appoints Natasja Laheij as chair in strategic board overhaul
UK-based online fashion retailer Asos has unveiled significant changes to its executive structure, confirming that Natasja Laheij will assume the role of chair and William Barker of deputy chair. Laheij's appointment will be effective from the end of the financial year 2025, while Barker's appointment is effective immediately. Jørgen Lindemann, who has served as chair of Asos since August 2022, will relinquish his board duties following a structured transition period that coincides with the end of his current term. Lindemann stated:"It has been a privilege to serve on the Asos board over the last four years, throughout an incredibly important period for the business. I have valued the Asos board and Natasja, William and José's insights and oversight during this time, and I am confident that we have the right leadership to deliver sustainable, profitable growth." Natasja joined the Asos board as an independent non-executive director in April 2023. She has three decades of experience in international commercial and financial management. Her expertise covers sectors including e-commerce and technology, with roles at Deloitte Australia, Sony Ericsson, Apple and as chief financial officer for Amazon Fashion Europe and for Google EMEA [Europe, Middle East and Africa]'s platforms and devices. Asos has initiated the process of appointing a new chair for the Audit Committee. An announcement regarding this appointment will be made in due course. The introduction of the deputy chair position aims to bolster the chair and management committee in strategy development, cultural enhancement and operational projects as needed. William joined the Asos board as a non-executive director in September 2023. As the founder and CEO of Camelot Partners, William brings experience in founding, building and revitalising digitally-enabled businesses. His portfolio includes leadership roles at Slate Auto, Re:Build Manufacturing and Tapi Carpets & Floors. Jose Manuel Martínez Gutiérrez, who joined the Asos board as an independent non-executive director in April 2023, will succeed Natasja as senior independent director. In June 2025, Asos collaborated with global digital payments provider to enhance the online shopping experience for its customers. "Asos appoints Natasja Laheij as chair in strategic board overhaul" was originally created and published by Retail Insight Network, a GlobalData owned brand. The information on this site has been included in good faith for general informational purposes only. It is not intended to amount to advice on which you should rely, and we give no representation, warranty or guarantee, whether express or implied as to its accuracy or completeness. You must obtain professional or specialist advice before taking, or refraining from, any action on the basis of the content on our site. Error while retrieving data Sign in to access your portfolio Error while retrieving data Error while retrieving data Error while retrieving data Error while retrieving data
Yahoo
23-07-2025
- Yahoo
Topshop returning to high street in Donegal department store
Topshop is returning to the high street in a partnership with one of the Republic of Ireland's best known department stores. McElhinney's in Ballybofey, County Donegal, is to begin selling Topshop's clothing range next month. It will be the first time that Topshop clothes will be sold in a physical store since it moved solely into the online retail space. In 2021, Topshop was one of four brands acquired by online fashion group Asos from failed retail group Arcadia. McElhinney's general manager Sandra Devenney told BBC News NI shoppers can expect "the Topshop that we all knew and loved and have missed". "We are really optimistic and very excited about this." McElhinney's will be the first store to sell Topshop clothes as part of its high street relaunch. Its return to bricks and mortar retail will also see the brand partnering with stockists in Denmark and France later in 2025. Topshop, Ms Devenney said, is an iconic brand, and a perfect fit for the Donegal store. "We have an exceptionally dynamic team and are always looking to the future while, as one of the now very few independent family run stores in Europe, maintaining our heritage," she said. Is Topshop returning to the UK high street? Topshop has been teasing a return to high streets in the UK over recent weeks. Last month its managing director Michelle Wilson told industry magazine Drapers that Topshop is planning to be back on the UK high street by the autumn of 2025. In a post on social media on Tuesday, the company revealed actress and model Cara Delevingne as "the new face of Topshop", adding a "bold new era begins." Fashion blogger Denise Curran is excited for its return. "You were bang on trend if you were shopping in Topshop," Denise from County Armagh told BBC News NI. "I can't wait to see what their styles will be like." She said it was to the Donegal store's huge credit that they have partnered with Topshop and will definitely be making the trip down. "Well done to McElhinneys, it's amazing to see. "A lot of people obviously travel to Donegal on their holidays so it's a great location for people to actually plop on a map and say I want to go there." Topshop first opened in Sheffield in 1964 and at its peak had about 300 stores in the UK and Ireland, including 14 in Northern Ireland and a further six in the Republic. Asos bought the Topshop, Topman, Miss Selfridge and HIIT brands, but not the shops, after Arcadia went into administration in 2020. In 2024 Danish company Bestseller bought 75% of Asos' stake in Topshop. BBC News NI has approached Topshop for comment. Thousands of jobs at risk after Asos Arcadia deal A brief history of Topshop
Yahoo
11-07-2025
- Yahoo
ASOS launches loyalty program as part of turnaround effort
This story was originally published on CX Dive. To receive daily news and insights, subscribe to our free daily CX Dive newsletter. Fashion brand ASOS has launched a loyalty program for customers in the United Kingdom, the company announced last week. The fashion retailer began testing the program in March ahead of the full rollout. The program is divided into four tiers. The first tier, which is free, offers early access to product drops, a 20% birthday discount and access to ASOS' digital stylist, an AI tool that can curate looks for customers with a conversational interface. Members can reach higher tiers based on their annual spend and gain benefits including members-only sales, priority back-in-stock alerts and early access to curated product collections based on certain styles or trends. was designed to attract new shoppers while offering benefits that reward the company's most loyal customers. The company aimed to create a frictionless sign-up process and immediate access to perks to help the retailer stand out. Higher-tier rewards are focused on the experiential side, with top-tier members gaining access to in-person events. 'Our customers want to engage with ASOS in a way that goes beyond just shopping,' Macy Hong, head of loyalty at ASOS, said in a prepared statement. ' creates opportunities for them to connect with the brand, discover new fashion and gain access to exclusive experiences.' Exclusivity may be essential to the program's potential for success. A June 2024 Snappy survey found that access to exclusive merchandise was one of the top features of loyalty programs. Customers also expressed interest in special treatment, including discounts only available to members. The loyalty program is part of a broader emphasis on CX that ASOS CEO José Antonio Ramos Calamonte discussed on a Q2 2025 earnings call in April. Experience is one of the three pillars of the fashion retailer's turnaround strategy, and will play a key role, he said. The company is putting an emphasis on personalization as well, according to Ramos Calamonte. In addition to its AI stylist, the company is working on improving its search and recommendations engine. Still, ASOS has work ahead. Revenue was down 13% year over year in the first half of 2025, according to its most recent earnings report. The drop follows a 16% year-over-year revenue decline in fiscal 2024, according to an annual report. Recommended Reading How retailers can foster loyalty for Black Friday and beyond Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
