University of L'Aquila

Data published in the Cell Press Journal Molecular Therapy Nucleic AcidsGUILDFORD, United Kingdom, Sept. 18, 2023 (GLOBE NEWSWIRE) -- SiSaf Ltd, an RNA therapeutics company, is pleased to announce the publication in the peer-reviewed journal Molecular Therapy Nucleic Acids(1) of positive in vivo data demonstrating the safety and efficacy of its siRNA therapy for Autosomal Dominant Osteopetrosis 2 (ADO2). The data reported in the paper demonstrate that siRNA complexed with SiSaf’s silicon stabilized hybrid lipid nanoparticles (sshLNP) was able to significantly downregulate expression of an ADO2-specific mutant gene. These results could have significant translational impact on bone disease therapies and open the path to human trials of SiSaf’s potentially curative treatment for Osteopetrosis ADO2. There are currently no approved treatments for this debilitating disease and no other treatments currently in clinical trials. SIS-101-ADO, SiSaf’s lead in house program, is an ADO2-specific siRNA specifically designed against the human CLCN7G215R mRNA formulated with the company’s sshLNP. When tested via single intraperitoneal injection in pre-puberal ADO2 mice, carrying a heterozygous mutation of the Clcn7 gene (Clcn7G213R), SIS-101-ADO significantly downregulated the Clcn7G213R related mRNA levels in femurs at 48 h. Confirmatory results were observed at 2 weeks and 4 weeks after treatments (3 intraperitoneal injections/week), with rescue of the bone phenotype and demonstrating safety. The publication builds on data presented at the American Society of Bone and Mineral Research (ASBMR) 2022 Annual Meeting and is jointly authored by SiSaf and its collaborators from the University of L'Aquila, Italy, led by Professor Anna Maria Teti. Co-author on the paper, Dr. Suzanne Saffie-Siebert, CEO of SiSaf Ltd, said, “Targeting the bone safely and effectively is a challenge for RNA-based therapies. The in vivo data for our ADO2 treatment show that SiSaf’s silicon stabilized LNPs are able to deliver siRNA to the bone without any side effects. This is an important milestone on the path to clinical trials of SiSaf’s treatment for autosomal dominant Osteopetrosis type 2. And it could have wider translational significance for RNA-based treatments of other skeletal disorders.” Professor Anna Maria Teti added, “We can’t wait to see SIS-101-ADO used to treat ADO2 patients and look forward to further collaboration with SiSaf on potential therapies for other skeletal disorders.” SiSaf has had ongoing productive collaboration with Professor Teti’s group for over three years and continues to progress studies in support of its IND data package for SIS-101-ADO. During 2023 it successfully submitted an application to the U.S. FDA for Orphan Drug Designation for SIS-101-ADO which was granted in May. In addition, due to the serious manifestations of this rare skeletal disorder in children, the FDA granted SIS-101-ADO Rare Pediatric Disease Designation for the treatment of Autosomal Dominant Osteopetrosis. 1. Novel hybrid silicon-lipid nanoparticles deliver a siRNA to cure autosomal dominant osteopetrosis in mice. Implications for gene therapy in humans: Molecular Therapy Nucleic Acids: VOLUME 33, P925-937, SEPTEMBER 12, 2023, Antonio Maurizi et. al. https://doi.org/10.1016/j.omtn.2023.08.020 – ABOUT SISAF SiSaf is an RNA therapeutics company with a proprietary delivery platform. Its lead programs are a siRNA treatment for Osteopetrosis ADO2 and a partnered siRNA treatment for Corneal Dystrophy. The company is in the process of expanding its pipeline to other fields, including oncology. SiSaf’s proprietary Bio-Courier™ delivery platform uses silicon stabilized hybrid lipid nanoparticles (sshLNP) to improve the stability, safety, and transfection efficiency of RNA. sshLNP remove the need for an ultra-cold chain and unlike conventional lipid nanoparticles, they can be manufactured empty and the RNA can be introduced in a separate step. Led by founder and leading biomaterials specialist Dr. Suzanne Saffie-Siebert, SiSaf is a venture capital-backed private company. Headquartered in Guildford, UK, it has fully integrated research labs and bio-analytical facilities, a scalable technology to fast-track development, and a growing patent estate with extensive freedom to operate. To learn more, please visit www.sisaf.com Follow us on LinkedIn and Twitter. For further information please contact: MEDIA ENQUIRIES Scius communications Sue Charles – sue@sciuscommunications.com +44 7968726585 Katja Stout – katja@sciuscommunications.com +44 7789435990

The company prepares for bringing the wearable prescribed device to Europe next year NETANYA, Israel, Aug. 9, 2023 /PRNewswire/ -- Theranica, a prescribed digital therapeutics company developing advanced neuromodulation devices for migraine and other pain conditions, today announced it received expanded CE (Conformité Européenne) mark approval under MDR European regulation for Nerivio® as dual-use therapy for both acute and preventive treatment of migraine for adults and adolescents. The CE mark expansion for Nerivio is a critical milestone to accelerate the availability of the novel, drug-free migraine device into the European market. Migraine is a major global neurological disorder that affects over 1 billion people worldwide, with a prevalence higher than one in ten people in Europe. Research shows it's one of the most prevalent disorders, with a high morbidity, especially among young adults and females. Nerivio has been available with prescription in the USA since 2020, initially for acute treatment and since April this year also as a preventive treatment. "Migraine is the leading cause of disability worldwide, including for for adolescents and young adults, and early intervention with preventive treatment is necessary to restrain the disease from escalating", said Dr. Simona Sacco, Professor of Neurology at the University of L'Aquila, Italy, and second vice president of the European Headache Federation. "It is important to expand the treatment options with a non-pharmacological treatment, especially one that can both treat and prevent migraine." "With more than 50,000 prescribed Nerivio patients now in the US, we receive numerous requests, on a weekly basis, to bring the device to Europe," says Alon Ironi, CEO and co-founder of Theranica. "We have been waiting for the prevention indication approval, and with that are now preparing for making Nerivio available for people with Migraine in Europe starting 2024. Our focus is to empower underserved populations, particularly adolescents, to safely and effectively prevent and treat migraines, and be fully present in everyday life." Controlled by a smartphone app and self-administered, Nerivio wraps around the upper arm and uses sub-painful Remote Electrical Neuromodulation (REN) to activate nociceptive nerves fibers in arm to send signals which trigger a descending pain management mechanism in the brain called conditioned pain modulation (CPM), which turns off migraine pain and associated symptoms without medication. In simpler terms, the upper arm is stimulated to unleash a natural process in the brain to abort or relieve migraine headache and other associated symptoms. Each treatment lasts 45 minutes and is applied every other day for prevention or at the start of a migraine attack for acute treatment. About Theranica Theranica is a prescribed digital therapeutics company dedicated to creating effective, safe, affordable, low-side-effect therapies for idiopathic pain conditions. The company's award-winning flagship wearable, Nerivio®, is the first FDA-cleared and CE marked prescription migraine bioband for acute and/or preventive treatment of migraine with or without aura in people 12 years or older. Nerivio already serves more than 50,000 people with migraine in the USA, including adolescents and veterans. Theranica is expanding its proprietary technology to develop solutions for additional idiopathic pain conditions. Learn more by visiting our websites, theranica.com and nerivio.com, and following us on LinkedIn, Twitter, Instagram and Facebook. Theranica Contact Ronen Jashek [email protected] +972-72-390-9750 Media Contact V.A. Lopes Grey Matter Marketing [email protected] SOURCE Theranica

– Highly coveted Innovation Grant by the Italian Government will measure impact of whole genome sequencing and pharmacogenomics starting from a specific population of cancer patients -- NEW YORK, April 27, 2023 /PRNewswire/ -- Dante Genomics, a global leader in genomics and precision medicine, today announced it has been awarded a highly coveted Innovation Grant from the Italian Government to measure the impact of whole genome sequencing and pharmacogenomics for patients with High Grade Serous Ovarian Cancer (HSOC). The immediate purpose is to measure the clinical utility of whole genome sequencing for this well-defined population of cancer patients in a clinical setting and the benefits for the entire healthcare system. "Our shared long-term goal is to create a pathway for the use of whole genome sequencing in real, clinical settings in a national healthcare system," said Andrea Riposati, Co-founder and CEO of Dante Genomics. "We are starting in Italy, a European Union founding member and G7 country with a population of 60 million people, and in a healthcare system keen to innovate." The grant will fund a patient pilot study in which Dante will provide sequencing, interpretation and genomic reports to demonstrate the validity of whole genome sequencing to support clinicians and their patients throughout all of the steps of the treatment journey from diagnosis to therapy selection and specialty consultation, eventually leading to better health outcomes. "Whole genome sequencing for cancer patients can mean all the difference when determining the best treatment plan in a timely manner. By starting with whole genome sequencing, we can offer pharmacogenomics reports available in minutes, determining the best drugs on the market and any resistance to existing therapies. This project with the Italian government will increase access to personalized medicine options for people with High Grade Serous Ovarian Cancer," said Mattia Capulli, PhD, Co-founder and CSO of Dante Genomics and Associate Professor of Human Embryology and Histology at the University of L'Aquila in Italy. The 36-month project has the ambition to set a proof of concept that demonstrates how a deep integration of genomics and bioinformatics in the treatment of oncology patients brings a dramatic improvement of cancer treatment outcomes. About Dante Genomics Dante Genomics is a global genomic information company building and commercializing a new class of transformative health and longevity applications based on whole genome sequencing and software. The Company uses its platform to deliver better patient outcomes, prevention, enhanced diagnostics and personalized medicine. The Company's assets include one of the largest private genome databases with research consent, proprietary software designed to unleash the power of genomic data at scale and proprietary processes which enable an industrial approach to genomic sequencing. Contact: Laura D'Angelo VP of Investor Relations ir@dantelabs.com +39 0862 191 0671