A natural history study is a preplanned ... of the disease. Should the data demonstrate compelling clinical activity, Polaryx may pursue an expedited approval pathway. For more information about the SOTERIA trial, please visit https://clinicaltrials.gov/study/NCT07740512. ... Polaryx Therapeutics, Inc. is a clinical-stage biotechnology company focused on developing patient-friendly small molecule and gene therapy treatments for rare orphan lysosomal ... A natural history study is a preplanned observational study intended to track the course of the disease. Should the data demonstrate compelling clinical activity, Polaryx may pursue an expedited approval pathway. For more information about the SOTERIA trial, please visit https://clinicaltrials.gov/study/NCT07740512. ... Polaryx Therapeutics, Inc. is a clinical-stage biotechnology company focused on developing patient-friendly small molecule and gene therapy treatments for rare orphan lysosomal storage disorders (LSDs). Charlton is a board-certified pediatric endocrinologist with nearly two decades of leadership in rare disease and pediatric drug development across the full development lifecycle. According to Polaryx Therapeutics, his appointment is intended to strengthen clinical leadership and execution ahead of the planned SOTERIA Phase 2 trial of PLX-200, expected to initiate in Q4 2026. For investors, it matters because diagnosis, ongoing therapy, newborn screening and potential new drugs or gene therapies can drive medical spending, regulatory approvals and market opportunity in endocrinology and rare disease care. ... A pediatric endocrinologist is a medical specialist who diagnoses and treats hormone-related conditions in infants, children and adolescents, such as growth disorders, diabetes, thyroid problems and delayed puberty. Their expertise matters to investors when companies report clinical trial results, regulatory reviews, advisory-board appointments or guidance about pediatric use, because these doctors assess safety, dosing and effectiveness for younger patients—like a specialist mechanic for a particular class of engines. I look forward to bringing my experience to the SOTERIA trial and being of service to pediatric patients and families.” “Will has extensive experience serving as a Chief Medical Officer and leading rare disease clinical trials, coupled with his background as a pediatrician and passion for developing patient-friendly therapies, making him an excellent fit to lead our SOTERIA trial,” said Alex Yang, Polaryx Therapeutics’ Chief Executive Officer.

-- Chiesi’s abstract presentations highlight clinical insights and patient-reported outcomes inFabry disease and alpha-mannosidosis -- -- Chiesi’s abstract presentations highlight clinical insights and patient-reported outcomes inFabry disease and alpha-mannosidosis -- -- Rooted in patient voice and scientific rigor, this research reflects Chiesi’s long-standing role in advancing science for the lysosomal storage disorders community -- PARMA, Italy, Aug. 28, 2026 (GLOBE NEWSWIRE) -- Chiesi Global Rare Diseases, a business unit of the Chiesi Group established to deliver innovative therapies and solutions for people living with rare diseases, today announced presentations at the Society of Inborn Errors of Metabolism (SSIEM) 2026 Annual Symposium, held August 25-28, 2026, in Helsinki, Finland. The condition affects both males and females and can present from childhood through adulthood, often with delayed diagnosis or misdiagnosis. While Fabry disease is rare, early detection and access to appropriate treatment - such as enzyme replacement therapy or pharmacological chaperone therapy - are critical in managing symptoms and slowing disease progression. Chiesi-led presentations include long-term Phase 3 data from the F60/BRILLIANCE trial, patient-reported outcomes from a cross-sectional survey in untreated Fabry disease, and research exploring earlier diagnosis and genotype-phenotype relationships in alpha-mannosidosis. Independent studies supported by Chiesi examine real-world clinical experience, disease biology, biomarkers, and newborn screening. "From new tools that could help identify diagnosis earlier to long-term data on treatment outcomes, this research is united by a single goal: better understanding the experiences of people living with rare diseases and using those insights to help inform care. We're proud to have shared these findings with the broader scientific community and remain devoted to advancing solutions that make a meaningful difference.” ... By signing up with an email address, I acknowledge that I have read and agree to the Terms of Service and Privacy Policy. "This body of evidence spans long-term clinical trial data, real-world outcomes, and new biomarker research, contributing to a more comprehensive understanding of disease progression and unmet needs across the care journey,” said Alessio Amadasi, Vice President, Medical Affairs EU & International, Chiesi Global Rare Diseases.

A natural history study is a preplanned ... of the disease. Should the data demonstrate compelling clinical activity, Polaryx may pursue an expedited approval pathway. For more information about the SOTERIA trial, please visit https://clinicaltrials.gov/study/NCT07740512. ... Polaryx Therapeutics, Inc. is a clinical-stage biotechnology company focused on developing patient-friendly small molecule and gene therapy treatments for rare orphan lysosomal ... A natural history study is a preplanned observational study intended to track the course of the disease. Should the data demonstrate compelling clinical activity, Polaryx may pursue an expedited approval pathway. For more information about the SOTERIA trial, please visit https://clinicaltrials.gov/study/NCT07740512. ... Polaryx Therapeutics, Inc. is a clinical-stage biotechnology company focused on developing patient-friendly small molecule and gene therapy treatments for rare orphan lysosomal storage disorders (LSDs). Company on track to initiate SOTERIA trial in the second half of 2026 · Clinical development activities advancing following FDA authorization, CRO engagement and Fast Track Designation across all four planned indication · PARAMUS, NJ, Aug. 25, 2026 (GLOBE NEWSWIRE) -- Polaryx Therapeutics, Inc. (Nasdaq: PLYX), a clinical-stage biotechnology company developing novel, disease-modifying therapies for rare pediatric lysosomal storage disorders (“LSDs”), today provided an operational readiness update for SOTERIA, its Phase 2 basket trial evaluating lead candidate PLX-200 across four rare pediatric LSDs. Company on track to initiate SOTERIA trial in the second half of 2026 Clinical development activities advancing following FDA authorization, CRO... Polaryx Therapeutics Announces Closing of $10 Million Private Placement Equity Financing to Support Launch of SOTERIA Phase 2 Basket Trial · PARAMUS, NJ, May 28, 2026 (GLOBE NEWSWIRE) -- Polaryx Therapeutics, Inc. (the “Company” or “Polaryx”) (Nasdaq: PLYX), a clinical-stage biotechnology company developing novel, disease-modifying...

Andelyn Biosciences is set to develop and manufacture a gene therapy known as AAV9-GM2, for Queen’s University to treat GM2 gangliosidoses, including Tay-Sachs and Sandhoff diseases. Andelyn Biosciences is set to develop and manufacture a gene therapy known as AAV9-GM2, for Queen’s University to treat GM2 gangliosidoses, including Tay-Sachs and Sandhoff diseases. Using its platform, Andelyn Biosciences plans to implement a multi-phase programme. Credit: Gorodenkoff / Shutterstock.com. ... The partnership aims to progress this gene therapy using Andelyn’s AAV Curator Platform. GM2 gangliosidosis is a rare genetic disorder that leads to the progressive loss of nerve cells in the brain and spinal cord. The condition belongs to the group of lysosomal storage disorders and results from genetic mutations that impair lysosomal function, leading to harmful accumulation within cells and severe neurological symptoms. Andelyn Biosciences will implement a multi-phase programme using its Curator Platform, which features a cell line and a modular approach intended to adapt manufacturing processes to specific programme needs. Queen’s University medical geneticist and department of paediatrics professor Dr Jagdeep Walia said: “This partnership with Andelyn is very strategic as they have the established expertise of producing vectors for many gene therapy programmes. In August 2025, Andelyn Biosciences entered a collaboration with Amplo Biotechnology to manufacture adeno-associated virus (AAV) gene therapies to address conditions affecting the neuromuscular junction.

The collaboration will test Apertura’s proprietary TfR1 CapX AAV capsid, a next-generation delivery technology designed to bind transferrin receptor 1 (TfR1) Apertura Gene Therapy is joining forces with two leading US research institutes to advance a potential new treatment approach for Niemann-Pick Disease Type C1 (NPC1), a rare and fatal genetic disorder that causes progressive neurological decline. NPC1 is a devastating autosomal recessive lysosomal storage disorder caused by mutations in the NPC1 gene. The disease leads to the buildup of cholesterol and lipids in cells, triggering severe neurological damage that can begin in early childhood. Patients often experience progressive cerebellar ataxia, dementia, and, in many cases, death during adolescence. "Systemic delivery of AAV gene therapies by intravenous administration has significant advantages over other methods of administration due to lower complexity and risks," said Forbes D. Andrew Steinsapir, Acting Chief Technology Officer of Apertura Gene Therapy and Gene Therapy Program Lead at Deerfield Management, said: "Apertura is excited to collaborate with NICHD and NHGRI and explore how an investigational gene therapy that uses TfR1 CapX can be delivered intravenously with the goal of treating Niemann-Pick Disease Type C1." "We are thrilled about this partnership. With multiple therapies now approved for NPC disease, our focus is on advancing the next generation of treatments, and we believe gene therapy holds tremendous promise," said Sean Kassen, Director of the Ara Parseghian Medical Research Fund.