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Oligonucleotides Clinical Trial Pipeline Analysis Demonstrates 280+ Key Companies at the Horizon Expected to Transform the Treatment Paradigm, Assesses DelveInsight
Oligonucleotides are short chains of nucleic acid molecules that can be used to treat or manage a variety of diseases. Oligonucleotides are being increasingly explored for a variety of therapeutic areas, such as rare diseases, cancer, infectious diseases, and genetic disorders. Technologies like antisense oligonucleotides (ASOs), small interfering RNAs (siRNAs), and aptamers are at the forefront of innovation. New York, USA, June 17, 2025 (GLOBE NEWSWIRE) -- Oligonucleotides Clinical Trial Pipeline Analysis Demonstrates 280+ Key Companies at the Horizon Expected to Transform the Treatment Paradigm, Assesses DelveInsight Oligonucleotides are short chains of nucleic acid molecules that can be used to treat or manage a variety of diseases. Oligonucleotides are being increasingly explored for a variety of therapeutic areas, such as rare diseases, cancer, infectious diseases, and genetic disorders. Technologies like antisense oligonucleotides (ASOs), small interfering RNAs (siRNAs), and aptamers are at the forefront of innovation. DelveInsight's 'Oligonucleotides Competitive Landscape 2025' report provides comprehensive global coverage of pipeline oligonucleotides in various stages of clinical development, major pharmaceutical companies are working to advance the pipeline space and future growth potential of the oligonucleotides pipeline domain. Key Takeaways from the Oligonucleotides Pipeline Report DelveInsight's oligonucleotides competitive report presents a robust market with over 280 active players developing more than 320 pipeline oligonucleotides. Key oligonucleotide companies such as Novartis, Astellas, Alnylam Pharmaceuticals, Ionis Pharmaceuticals, 4D Molecular Therapeutics, Avidity Biosciences, Suzhou Ribo Life Science, Amgen, ProQR Therapeutics, Stoke Therapeutics, MiNA Therapeutics, Sylentis, GSK, Silexion Therapeutics, Novo Nordisk A/S, Bio-Path Holdings, Sunhawk Vision Biotech, Isarna Therapeutics, Sirnaomics, Laboratoire Thea, Dyne Therapeutics, Vertex Pharmaceuticals, Korro Bio, Praxis-Precision-Medicines, Vico Therapeutics, BioMarin Pharmaceutical, TransCode Therapeutics, TME Therapeutics, ARTHEx Biotech, aptaTargets, CSPC Zhongnuo Pharmaceutical, ExoRNA Bioscience, Visirna Therapeutics, AiCuris, Comanche Biopharma, Tallac Therapeutics, and others are evaluating new oligonucleotides to improve the treatment landscape. Promising pipeline oligonucleotides such as Pelacarsen, Izervay, Nucresiran, ALN-6400, Zilganersen, 4D-150, Delpacibart Etedesiran, RBD1007, Olpasiran, GSK3228836, Sepofarsen, STK-001, MTL-CEBPA, SYL-1801, Loder, CDR132L, BP1001, SHJ002, ISTH0036, STP705, Ultevursen, DYNE-101, VX-670, KRRO 110, PRAX-222, VO659, BMN 351, TTX-MC138, TME151, ATX-01, ApTOLL, SYH2062, ER2001, VSA012, AIC468, CBP-4888, ALTA-002, and others are under different phases of oligonucleotide clinical trials. Request a sample and discover the recent advances in oligonucleotide drugs @ Oligonucleotides Competitive Report Oligonucleotides Overview Oligonucleotides are short chains of nucleotides, composed of repeating units that include a ribose or deoxyribose sugar, nitrogenous bases, and a phosphate backbone. Their ability to selectively bind to complementary DNA or RNA strands allows them to form duplexes or, less commonly, more complex structures. This property makes oligonucleotides valuable tools for detecting specific nucleic acid sequences. Typically ranging from 13 to 25 nucleotides in length, oligonucleotides can hybridize with target DNA or RNA. They are classified into several types, including antisense oligonucleotides (ASOs), small interfering RNAs (siRNAs), microRNAs (miRNAs), and aptamers. These molecules are being actively explored for their therapeutic potential in treating conditions such as neurodegenerative diseases, cancer, and rare diseases. Clinical trials are also evaluating their use in dermatological, gastrointestinal, and endocrine disorders. Oligonucleotides influence gene expression through various mechanisms, such as RNA interference (RNAi), RNase H-mediated degradation, splicing modulation, inhibition of non-coding RNAs, gene activation, and programmable gene editing. As a result, oligonucleotide therapeutics, including ASOs, siRNAs, and splice-modulating oligonucleotides, are emerging as a powerful new class of drugs capable of targeting a broad range of genetic and non-genetic diseases. However, their clinical success depends heavily on overcoming significant delivery challenges. Due to their high molecular weight, hydrophilicity, and negative charge, oligonucleotides face difficulty crossing cell membranes. They are also prone to degradation by nucleases, have limited tissue penetration, are quickly cleared by the kidneys, and often require targeted delivery to specific tissues. Despite these hurdles, oligonucleotides have transformed both diagnostics and therapeutics thanks to their high specificity, programmable nature, and ease of synthesis. In diagnostics, they serve key roles as primers in PCR/qPCR, probes in assays like FISH, and functional elements in technologies such as microarrays and CRISPR-based platforms. Aptamers enhance imaging by targeting specific biomarkers, and chemical modifications can significantly improve their stability in biological environments. Therapeutically, oligonucleotides include drugs like pegaptanib (aptamer), nusinersen (ASO), and patisiran (siRNA), which work by silencing or modulating gene expression. They also play a foundational role in mRNA and DNA vaccines, miRNA inhibitors, and gene editing tools like CRISPR. While challenges such as off-target effects and rapid systemic clearance remain, innovations like GalNAc conjugation and AI-driven design are advancing the field, enhancing specificity, delivery efficiency, and clinical effectiveness, and propelling the era of precision medicine Market Dynamics The oligonucleotides market is experiencing significant growth, driven by advances in genomic research, increased prevalence of genetic disorders, and rising interest in personalized medicine. Key therapeutic modalities, including antisense oligonucleotides (ASOs), small interfering RNAs (siRNAs), and aptamers, are gaining traction due to their ability to target diseases at the genetic level. The success of RNA-based drugs like SPINRAZA (nusinersen) and ONPATTRO (patisiran) has validated the commercial potential of oligonucleotide therapeutics, spurring further investment and innovation in the space. The market dynamics are shaped by several influential trends. Technological advancements in oligonucleotide synthesis and chemical modifications have improved stability, bioavailability, and target specificity, thereby expanding clinical applications. Concurrently, the adoption of automated synthesis platforms and solid-phase synthesis technologies has increased manufacturing efficiency and scalability. However, production remains complex and cost-intensive, especially for therapeutic-grade oligonucleotides, creating opportunities for specialized CDMOs that can meet stringent regulatory and quality standards. Pharmaceutical and biotech companies are aggressively expanding their oligonucleotide-based therapeutic pipelines, targeting a broad spectrum of indications including rare genetic diseases, cancer, neurodegenerative disorders, and infectious diseases. The oncology segment, in particular, is attracting attention for oligonucleotide therapies due to their ability to silence oncogenes or modulate gene expression with high precision. Moreover, the regulatory environment is becoming more supportive, with agencies like the FDA and EMA providing clear guidance for oligonucleotide drug development, fast-track designations, and orphan drug incentives. Despite its promise, the oligonucleotides market faces several challenges. These include delivery barriers, especially for intracellular targets, off-target effects, and immunogenicity concerns. Lipid nanoparticles (LNPs), conjugation strategies (e.g., GalNAc), and novel carrier systems are under active investigation to address these issues. Furthermore, intellectual property landscapes are becoming increasingly complex as more players enter the field, potentially leading to patent disputes and licensing complexities. In conclusion, the oligonucleotides market is poised for sustained growth, driven by technological innovation, clinical success stories, and expanding therapeutic frontiers. As barriers to delivery and cost are gradually overcome, oligonucleotide therapeutics are expected to become a mainstay in precision medicine, especially in areas with unmet medical needs. Strategic partnerships, regulatory clarity, and continued investment in R&D will be crucial to fully unlocking the potential of this transformative modality. To know more about oligonucleotides, visit @ Oligonucleotides Market Insights Approved Oligonucleotides Drug Profile Analysis LEQVIO: Novartis LEQVIO (inclisiran) is a first-of-its-kind small interfering RNA (siRNA) therapy developed by Novartis that targets the mRNA of PCSK9 (proprotein convertase subtilisin/kexin type 9). Unlike traditional treatments, LEQVIO reduces the production of the PCSK9 protein in the liver, enhancing the liver's ability to absorb and eliminate LDL-C from the bloodstream. In 2023, Japan's Ministry of Health, Labour and Welfare (MHLW) approved LEQVIO for both familial and non-familial hypercholesterolemia, as well as for individuals at high risk of cardiovascular events. The treatment regimen involves an initial dose, a second injection after three months, and subsequent maintenance doses every six months. Clinical trials have shown that when used alongside statins, LEQVIO can lower LDL-C levels by around 50%. IZERVAY: Astellas Pharma Avacincaptad pegol, sold under the brand name IZERVAY, is an approved treatment specifically developed for geographic atrophy. It is an RNA aptamer chemically linked to a branched polyethylene glycol (PEG) molecule. The drug works by targeting and inhibiting complement factor C5, a critical element of the complement system involved in inflammation related to age-related macular degeneration (AMD). By preventing the conversion of C5 into its active forms (C5a and C5b), avacincaptad pegol helps reduce inflammation and slow GA progression. The FDA approved IZERVAY on August 4, 2023, for GA secondary to AMD. It is currently under evaluation by the European Medicines Agency and is also being studied for potential use in treating Stargardt disease. Find out more about oligonucleotide drugs @ Oligonucleotide Analysis A snapshot of the Pipeline Oligonucleotides mentioned in the report: Drugs Company Phase Indication Pelacarsen Novartis Pharmaceuticals III Hyperlipoproteinaemia Trabedersen Oncotelic III Glioblastoma 4D-150 4D Molecular Therapeutics III Wet age-related macular degeneration Delpacibart Etedesiran Avidity Biosciences III Myotonic dystrophy GSK3228836 GSK III Chronic hepatitis B virus infection WVE-N531 Wave Life Sciences II Duchenne muscular dystrophy ATX-01 ARTHEx Biotech II Myotonic dystrophy Type I & II TAC001 Tallac Therapeutics I/II Solid tumors ATB 301 Autotelic Bio I Pancreatic cancer Learn more about the emerging oligonucleotides @ Oligonucleotides Clinical Trials Key Developments in the Oligonucleotides Treatment Space In May 2025, Cure Rare Disease (CRD), announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to its investigational anti-sense Oligonucleotide therapeutic for the treatment of Spinocerebellar Ataxia (SCA), including Spinocerebellar Ataxia Type 3 (SCA3), a progressive and currently untreatable neurodegenerative disorder. In April 2025, Biogen Inc. announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to BIIB080, an investigational antisense oligonucleotide (ASO) therapy targeting tau, for the treatment of Alzheimer's disease. In March 2025, Korro Bio, Inc., a clinical-stage biopharmaceutical company focused on developing a new class of genetic medicines based on editing RNA for both rare and highly prevalent diseases, announced that the FDA had granted orphan drug designation to the investigational medicine KRRO-110 for the treatment of Alpha-1 Antitrypsin Deficiency (AATD). In February 2025, AusperBio Therapeutics, Inc. and Ausper Biopharma Co., Ltd. (together AusperBio), a clinical-stage biotechnology company, announced recent progress in the ongoing clinical development of its lead candidate AHB-137, an antisense oligonucleotide (ASO) therapeutic for functional cure of chronic Hepatitis B (CHB). In January 2025, Arrowhead Pharmaceuticals, Inc. announced that the US Food and Drug Administration (FDA) had accepted the New Drug Application (NDA) for investigational plozasiran for the treatment of familial chylomicronemia syndrome (FCS), a severe and rare genetic disease. In December 2024, Vir Biotechnology, Inc. announced that tobevibart and elebsiran have received U.S. Food and Drug Administration (FDA) Breakthrough Therapy designation and European Medicines Agency (EMA) Priority Medicines (PRIME) designation for the treatment of chronic hepatitis delta (CHD). In December 2024, the FDA granted breakthrough therapy designation to Stoke Therapeutics' investigational antisense agent STK-001 for the treatment of genetically confirmed Dravet syndrome (DS), a rare epilepsy disorder. In November 2024, Ionis Pharmaceuticals announced that the US Food and Drug Administration (FDA) had accepted for review the New Drug Application (NDA) for donidalorsen, an investigational RNA-targeted medicine for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adult and pediatric patients 12 years of age and older. The FDA has set an action date of August 21, 2025, under the Prescription Drug User Fee Act (PDUFA). In October 2024, Ribocure Pharmaceuticals AB and Suzhou Ribo Life Science Ltd received authorization from the Swedish Medicinal Product Agency (MPA) to initiate a Phase II clinical trial in Sweden with the lipid-lowering siRNA drug RBD5044 that targets APOC3. The trial will evaluate efficacy and safety in patients with mixed dyslipidemia In September 2024, WVE-N531, an exon-skipping oligonucleotide developed by Wave Life Sciences for the treatment of Duchenne muscular dystrophy (DMD) in patients amenable to exon 53 skipping, received Orphan Drug Designation from the US Food and Drug Administration (FDA). In September 2024, NS Pharma, Inc., a subsidiary of Nippon Shinyaku Co., Ltd. (Nippon Shinyaku), announced that the Food and Drug Administration (FDA) had granted rare pediatric disease designation to NS050/NCNP-03, which is being developed for the treatment of Duchenne muscular dystrophy (Duchenne). In May 2024, Sunhawk Vision Biotech announced that it had received authorization from the US FDA to commence a Phase II clinical trial for myopia control in children. In May 2024, Imvax, Inc., announced the completion of enrollment in its randomized, multicenter, double-blind, placebo-controlled Phase IIb clinical trial of IGV-001 in patients with newly diagnosed glioblastoma (ndGBM). Scope of the Oligonucleotides Pipeline Report Coverage: Global Key Oligonucleotides Companies: Novartis, Astellas, Alnylam Pharmaceuticals, Ionis Pharmaceuticals, 4D Molecular Therapeutics, Avidity Biosciences, Suzhou Ribo Life Science, Amgen, ProQR Therapeutics, Stoke Therapeutics, MiNA Therapeutics, Sylentis, GSK, Silexion Therapeutics, Novo Nordisk A/S, Bio-Path Holdings, Sunhawk Vision Biotech, Isarna Therapeutics, Sirnaomics, Laboratoire Thea, Dyne Therapeutics, Vertex Pharmaceuticals, Korro Bio, Praxis-Precision-Medicines, Vico Therapeutics, BioMarin Pharmaceutical, TransCode Therapeutics, TME Therapeutics, ARTHEx Biotech, aptaTargets, CSPC Zhongnuo Pharmaceutical, ExoRNA Bioscience, Visirna Therapeutics, AiCuris, Comanche Biopharma, Tallac Therapeutics and others. Key Oligonucleotides in Pipeline: Pelacarsen, Izervay, Nucresiran, ALN-6400, Zilganersen, 4D-150, Delpacibart Etedesiran, RBD1007, Olpasiran, GSK3228836, Sepofarsen, STK-001, MTL-CEBPA, SYL-1801, Loder, CDR132L, BP1001, SHJ002, ISTH0036, STP705, Ultevursen, DYNE-101, VX-670, KRRO 110, PRAX-222, VO659, BMN 351, TTX-MC138, TME151, ATX-01, ApTOLL, SYH2062, ER2001, VSA012, AIC468, CBP-4888, ALTA-002 and others are under different phases of oligonucleotide clinical trials. Dive deep into rich insights for new oligonucleotide treatments, visit @ Oligonucleotides Drugs Table of Contents 1. Oligonucleotides Pipeline Report Introduction 2. Oligonucleotides Pipeline Report Executive Summary 3. Oligonucleotides Pipeline: Overview 4. Oligonucleotides Marketed Drugs 4.1. LEQVIO: Novartis Pharmaceuticals 5. Oligonucleotides Clinical Trial Therapeutics 6. Oligonucleotides Pipeline: Late-Stage Products (Pre-registration) 7. Oligonucleotides Pipeline: Late-Stage Products (Phase III) 7.1. Pelacarsen: Novartis Pharmaceuticals 8. Oligonucleotides Pipeline: Mid-Stage Products (Phase II) 8.1. Trabedersen: Oncotelic 9. Oligonucleotides Pipeline: Early-Stage Products (Phase I) 9.1. ATB 301: Autotelic Bio 10. Oligonucleotides Pipeline: Preclinical and Discovery Stage Products 11. Oligonucleotides Pipeline Therapeutics Assessment 12. Inactive Products in the Oligonucleotides Pipeline 13. Company-University Collaborations (Licensing/Partnering) Analysis 14. Unmet Needs 15. Oligonucleotides Market Drivers and Barriers 16. Appendix For further information on the oligonucleotides pipeline therapeutics, reach out @ Oligonucleotides Therapeutics Related Reports Antisense Oligonucleotide Therapeutics Pipeline Antisense Oligonucleotide Therapeutics Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key antisense oligonucleotide therapeutics companies, including Ionis Pharmaceuticals, Secarna Pharmaceuticals, Aro Biotherapeutics, NeuBase Therapeutics, Bio-Path Holdings, Inc., Scopus Biopharma, Dyne Therapeutics, CAMP4 Therapeutics, Pulmotect, GeneTx Biotherapeutics, Aligos Therapeutics, WaVe Life Sciences, among others. Oligonucleotide Synthesis Market Oligonucleotide Synthesis Market Insight, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of market trends, market drivers, market barriers, and key oligonucleotide synthesis companies, including Thermo Fisher Scientific Inc., Agilent Technologies, Merck KGaA, Bio-Synthesis Inc., Ajinomoto Bio-Pharma Services, CordenPharma, Creative Biolabs, Ella Biotech, Eurofins Genomics, Future Synthesis, Integrated DNA Technologies, Kaneka Eurogentec, LGC Biosearch Technologies, Microsynth, Nitto Avecia, Ribo Biotechnology, STA Pharmaceutical, Sumitomo Chemical, TriLink Biotechnologies, Sarepta Therapeutics, among others. RNA Interference Pipeline RNA Interference Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key RNA interference companies, including Silence Therapeutics, Janssen Research & Development, Eli Lilly and Company, Arrowhead Pharmaceuticals, Sylentis, Sirnaomics, Dicerna Pharmaceuticals, Suzhou Ribo Life Science, Alnylam Pharmaceuticals, Suzhou Ribo Life Science, Vir Biotechnology, Arbutus Biopharma, Silenseed, OliX Pharmaceuticals, Bio-Path Holdings, among others. Global Messenger RNA (mRNA)-based Vaccines and Therapeutics Market Global Messenger RNA (mRNA)-based Vaccines and Therapeutics Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key global messenger RNA -based vaccines and therapeutics companies, including Moderna, Inc., BioNTech SE, CureVac N.V., Arcturus Therapeutics, Translate Bio, Inc., GSK, among others. mRNA Vaccines and Therapeutics Market mRNA Vaccines and Therapeutics Market Insights, Competitive Landscape, and Market Forecast – 2032 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key mRNA vaccines and therapeutics companies, including Pfizer Inc., BioNTech SE, Moderna, Inc., Gennova Biopharmaceuticals Limited, GSK plc., Daiichi Sankyo, Arcturus, Boehringer Ingelheim International GmbH, Ethris GmbH, CureVac SE, AIM Vaccine Corporation, Charoen Pokphand Group, Argos Therapeutics Inc., Sanofi, Kernal Biologics Inc, among others. DelveInsight's Pharma Competitive Intelligence Service: Through its CI solutions, DelveInsight provides its clients with real-time and actionable intelligence on their competitors and markets of interest to keep them stay ahead of the competition by providing insights into the latest therapeutic area-specific/indication-specific market trends, in emerging drugs, and competitive strategies. These services are tailored to the specific needs of each client and are delivered through a combination of reports, dashboards, and interactive presentations, enabling clients to make informed decisions, mitigate risks, and identify opportunities for growth and expansion. Other Business Pharmaceutical Consulting Services Healthcare Conference Coverage Pipeline Assessment Healthcare Licensing Services Discover how a mid-pharma client gained a level of confidence in their soon-to-be partner for manufacturing their therapeutics by downloading our Due Diligence Case Study About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. Connect with us at LinkedIn CONTACT: Contact Us Shruti Thakur info@ +14699457679 in to access your portfolio
Time of India
07-06-2025
- Politics
- Time of India
HC directs govt to revise ASO seniority list based on appointment date
1 2 Cuttack: Orissa high court has directed the state govt to revise the seniority list of assistant section officers (ASOs)in the Odisha Secretariat Service, impacting hundreds of employees. The vacation bench's verdict settles a long-standing dispute concerning seniority among two batches of ASOs — direct recruits appointed through two separate advertisements and between promotees and direct recruits. Further, the verdict brings clarity to service-related disputes that lingered for years while providing relief to employees who were appointed earlier but placed lower in the final gradation list of ASOs published on June 11, 2020. A total of 10 writ petitions were clubbed together for hearing, categorised into two groups. The first group, comprising seven petitions, involved a dispute between two sets of direct recruits: one recruited under Odisha Public Service Commission (OPSC)'s advertisement no. 06 dated May 14, 2015, for ST category candidates (140), and another (811) under advertisement no. 08 of Oct 6, 2012. The second group of three petitions involved disputes between promotees and direct recruits. The HC noted that although advertisement no. 6 was issued earlier, appointments from the list were delayed until Oct 2016 due to legal hurdles and administrative delays. In contrast, candidates under the 2015 advertisement were appointed earlier — on Jan 27 and May 18, 2016. In the judgment on June 3, the vacation bench of Justice B P Routray upheld the principle that seniority in govt service should be based on the actual date of appointment, not the date of the recruitment advertisement or recommendation — unless a rule explicitly states otherwise. Justice Routray ruled that ASOs appointed earlier in 2016 under advertisement no. 6 must be placed above those appointed later in the same year under advertisement no. 8 in the final gradation list published on June 11, 2020. Justice Routray further ruled that promotees must be treated as en-bloc seniors to the direct recruits of the same calendar year. Accordingly, Justice Routray ordered the home department to revise the ASO gradation list in line with the HC's directives, ensuring that seniority reflects the actual date of appointment. The judgment reinforced a key service jurisprudence, "He who is appointed earlier, ranks earlier. " Get the latest lifestyle updates on Times of India, along with Eid wishes , messages , and quotes !
New Indian Express
07-06-2025
- Politics
- New Indian Express
Orissa HC directs govt to revise gradation list of secretariat ASOs
The bench of Justice AK Mohapatra noted that although Advertisement No. 08 was issued earlier in 2012, appointments from this list were delayed until October 2016 due to legal hurdles and administrative delays. In contrast, candidates under the 2015 advertisement were appointed on January 27, 2016 and May 18, 2016. Justice Mohapatra endorsed the principle that seniority in government service should be based on the actual date of appointment, not the date of the recruitment advertisement or recommendation, unless a rule explicitly states otherwise. While providing relief to candidates who were appointed earlier but placed lower in the seniority list, Justice Mohapatra ruled that the ASOs appointed earlier in 2016 under Advertisement No. 06 must be placed above those appointed later the same year under Advertisement No. 08 in the final gradation list published on June 11, 2020. The judge further ruled that promotees must be treated as en bloc senior to the direct recruits of the same calendar year and directed the Home department to revise the ASO gradation list in line with the court's directives, ensuring that seniority reflects the actual date of appointment. He also ruled that promotees must be treated as en bloc senior to the direct recruits of the same calendar year.
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
Business Wire
15-05-2025
- Business
- Business Wire
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)-- 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.
