Oregon Health & Science University

MADRID, SPAIN and CAMBRIDGE, MA, UNITED STATES, January 13th, 2025 - Oryzon Genomics, S.A. (ISIN Code: ES0167733015, ORY), a clinical-stage biopharmaceutical company leveraging epigenetics to develop therapies in diseases with strong unmet medical need, announced today that the first patient has been dosed in a Phase I dose-finding clinical trial of iadademstat, Oryzon’s potent and selective LSD1 inhibitor, in combination with venetoclax and azacitidine in newly diagnosed acute myeloid leukemia (AML), sponsored by the National Cancer Institute (NCI), part of the National Institutes of Health. The trial (NCT06514261), titled “Phase 1 Trial of Iadademstat in Combination With Venetoclax and Azacitidine in Patients With Treatment Naive AML”, will evaluate the safety, tolerability, and optimal dose of iadademstat when administered together with the standard-of-care venetoclax and azacitidine in treatment-naïve AML patients. Preliminary efficacy of the triple combination will also be evaluated. This Phase I study will be conducted and sponsored by the NCI, and will be led by Principal Investigator, Dr. Natalie Galanina, from the University of Pittsburgh Cancer Institute. The trial plans to enroll 45 patients and is carried out under a Cooperative Research and Development Agreement (CRADA) that Oryzon has in place with the NCI. Dr. Carlos Buesa, Oryzon’s CEO, added: “We are excited to have the first patient dosed in this study. The trial expands on the findings from our ALICE trial, where combining iadademstat with azacitidine demonstrated robust antileukemic effects in first-line AML, producing deep and durable responses along with a manageable safety profile, including in patients with high-risk prognostic factors that respond poorly to venetoclax plus azacitidine.” In AML, iadademstat is also being evaluated in combination with venetoclax and azacitidine in newly diagnosed AML patients in an investigator-initiated Phase I clinical trial at Oregon Health & Science University (OHSU) Knight Cancer Institute (NCT06357182), and in a company-sponsored Phase Ib clinical trial in combination with gilteritinib in patients with relapsed/refractory AML harboring a FMS-like tyrosine kinase mutation (FLT3mut+) (NCT05546580).

BARCELONA, Spain I December 12, 2024 I SpliceBio, a genetic medicines company pioneering Protein Splicing to address diseases caused by mutations in large genes, today announced that the U.S. Food & Drug Administration (FDA) has cleared its investigational new drug (IND) application for lead program SB-007. SB-007 is the only clinical-stage therapeutic addressing the root genetic cause of Stargardt disease with the potential to treat all patients across all ABCA4 mutations. Miquel Vila-Perelló, Ph.D., Chief Executive Officer, and Co-Founder of SpliceBio, said: “The FDA IND clearance of SB-007 is a significant achievement for SpliceBio and Stargardt disease patients. As the first-ever IND for a Protein Splicing gene therapy, it is a huge step forward to demonstrate the potential of this new therapeutic modality to address diseases caused by mutations in large genes such as ABCA4 . SB-007 is an adeno-associated viral (AAV) vector gene therapy aimed at restoring expression of the full-length ABCA4 protein, and the only clinical-stage therapy with the potential to help all Stargardt patients. We look forward to accelerating the clinical development of SB-007, building on the Orphan Drug Designation granted by the FDA in 2024, and advancing this potentially life-changing therapeutic for patients with Stargardt disease.” “Stargardt disease has been a challenge for the development of gene therapies due to the large size of the ABCA4 gene, and currently has no approved therapies available,” said Professor Paul Yang, M.D., Ph.D., Chief of the Paul H. Casey Ophthalmic Genetics Division at Casey Eye Institute at Oregon Health & Science University . “This new therapy utilizes a unique approach to replace the full-size, normal ABCA4 protein at high efficiency, which addresses the root cause of Stargardt disease across any pathogenic variant in the ABCA4 gene. This IND clearance represents a major milestone in the field and I am thrilled to be part of the clinical studies exploring this promising approach that could transform the lives of Stargardt disease patients.” SpliceBio plans to initiate enrolment in the Phase 1/2 ASTRA study in the first half of 2025. ASTRA will evaluate the safety and efficacy of a single dose of SB-007 administered subretinally in patients with Stargardt disease. In March 2024, SpliceBio launched the POLARIS trial, a pioneering company-sponsored natural history study of Stargardt disease designed to evaluate disease progression, refine endpoints, and streamline eligibility criteria for accelerated enrolment into the Phase 1/2 ASTRA study. This study will enable Stargardt disease patients to benefit from more precise diagnoses, more rigorous disease monitoring, and potentially faster access to innovative therapies. The SpliceBio Management team will be attending J.P. Morgan’s 43rd Annual Healthcare Conference 2025, being held in San Francisco from 13-16 January 2025. Please get in contact to schedule a meeting at bd@splice.bio . About Stargardt disease Stargardt disease is the most common form of inherited juvenile macular degeneration, affecting approximately 1 in 8,000 to 10,000 individuals. Caused by mutations in the ABCA4 gene, Stargardt disease leads to the progressive loss of central vision due to damage to the central region of the retina known as the macula. The disease is variable in the age of onset, including early onset in children and adolescents and late-onset forms in adulthood. There are no approved treatments, and patients face significant challenges in daily life as the disease progresses. Stargardt disease has remained elusive to genetic medicines due to the large size of the ABCA4 gene. About SB-007 SB-007 is a Protein Splicing dual AAV gene therapy that delivers the full-length ABCA4 gene and aims to restore expression of the native ABCA4 protein in the retina. It has demonstrated robust pharmacological activity in animal models of Stargardt disease and durable expression and safety in non-human primates. SB-007 has been granted Orphan Drug Designation by the FDA in the US and the European Commission in Europe. About SpliceBio SpliceBio is a genetic medicines company pioneering Protein Splicing to address diseases caused by mutations in large genes. The Company’s lead program, SB-007, targets the root cause of Stargardt disease, a genetic eye disease that causes blindness in children and adults. SpliceBio’s pipeline comprises additional gene therapy programs across therapeutic areas, including ophthalmology and neurology. SpliceBio’s platform is based on technology developed in the Muir Lab at Princeton University after more than 20 years of pioneering intein, Protein Splicing, and protein engineering research. For additional information, please visit www.splice.bio . SOURCE: SpliceBio

CHARLOTTESVILLE, Va., Dec. 12, 2024 /PRNewswire/ -- RIVANNA®, developers of world-first imaging-based medical solutions, has released a report exploring trends in anesthesiology employment across the United States. The findings detail significant regional demand for anesthesiologists while also examining the growth of the diagnostic ultrasound market, a development influenced by technological advancements and evolving healthcare needs. Anesthesiology workforce shows regional variations Employment trends for anesthesiologists demonstrate significant variability across states, highlighting challenges in balancing workforce supply and patient care demands. High-demand areas: New York and Florida reported the most active job postings for anesthesiologists, with 139 and 104 openings, respectively. Largest workforce: California led the nation with 5,380 anesthesiologists employed in 2023. Growth leaders: Pennsylvania showed the highest growth in total employment (202.08%) and jobs per 1,000 (197.56%). Declines: Missouri experienced a 75% decrease in anesthesiology positions during the same period, followed by Alabama (-66.7%) and North Carolina (-58.2%). "These data provide important insights into the evolving distribution of anesthesiology professionals, which is vital for addressing care gaps," said Dr. Brandon Togioka, Vice Chair of Clinical Research for the Department of Anesthesiology and Perioperative Medicine at Oregon Health & Science University. Ultrasound market continues rapid growth While workforce trends highlight significant demand for anesthesiologists in certain regions, the diagnostic ultrasound market has also experienced substantial growth, increasing from $8.2 billion in 2019 to an anticipated $20.4 billion by 2034. Ongoing technological advancements, the rising prevalence of chronic diseases, and a growing geriatric population fuel this trajectory. Additionally, healthcare providers and patients increasingly recognize the value of ultrasound in providing accurate and safe diagnostic solutions. "The growth reflects the increasing recognition of ultrasound-guided procedures as a valuable tool for enhancing accuracy and patient safety," noted anesthesiologist Dr. Steve Garber. "Ultrasound has become essential for procedures, including nerve blocks and vascular access." Ultrasound has become a cornerstone of modern anesthesiology; however, barriers to widespread adoption, including access to training and devices, remain. "Ultrasound's integration into clinical practice is accelerating as technology becomes more accessible and user-friendly," said Dr. Antonio Gonzalez, Associate Professor of Anesthesiology at Yale School of Medicine. "The development of smaller, AI-assisted devices will likely address many of the current challenges, further advancing procedural accuracy and patient care." Report methodology The data used in this analysis comes from publicly available sources, including the Bureau of Labor Statistics, employment platforms such as Indeed, and market data from Future Market Insights. While every effort has been made to ensure the accuracy of this report, RIVANNA does not guarantee the completeness or reliability of the external data and acknowledges the limitations inherent in the datasets used. About RIVANNA RIVANNA® is a commercial-stage medical device innovator and manufacturer in Charlottesville, VA. RIVANNA's mission is to develop and commercialize world-first imaging-based medical technologies that elevate global standards of care. RIVANNA is privately held and operates an FDA-registered and ISO 13485:2016-certified manufacturing facility. Learn more: RIVANNA. SOURCE RIVANNA WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED