Vtesse, Inc.

CAMBRIDGE, Mass., April 12, 2023 (GLOBE NEWSWIRE) -- Arbor Biotechnologies®, a biotechnology company discovering and developing the next generation of genetic editing medicines, today announced the appointment of Daniel Ory, M.D., as Chief Medical Officer. Dr. Ory joins Arbor from Casma Therapeutics, a Third Rock Ventures start up, where he served most recently as Chief Medical Officer, responsible for overseeing its muscular dystrophy and rare oncology programs. “I am pleased to welcome Dr. Ory to our executive team, who has extensive experience in the neurology, metabolic, and rare disease space, spanning both industry and academia,” said Devyn Smith, Ph.D., CEO of Arbor. “We look forward to Dr. Ory’s immediate contributions in evolving our pipeline development strategy as we advance our next-generation genomic editors toward the clinic.” In addition to his senior leadership roles at Casma, throughout the span of his 20+ year career, Dr. Ory served as the Alan A. and Edith L. Wolff Professor of Cardiology at Washington University in St. Louis, authored over 160 peer-reviewed publications, and led multiple clinical trials in Niemann-Pick Type C (NPC) disease—a rare, neurodegenerative disorder. He was also scientific co-founder of Vtesse Therapeutics, a rare disease company, that was later acquired by Sucampo. Added Dr. Ory: “I am thrilled to join and lead a team of passionate scientists at this pivotal time in Arbor’s trajectory as a leader in next-generation gene editing biotechnology. I look forward to advancing our wholly owned portfolio of editing technologies into clinical studies for patients.” Dr. Ory has served on scientific advisory boards of various biotech companies, including Kisbee Therapeutics and Stride Bio, as well as disease foundations such as Ara Parseghian Medical Research Foundation and the Salla Treatment and Research Foundation. As a champion of rare disease research, Dr. Ory has been honored many times for his work, including the Global Genes RARE Champion of Hope Award. He has an A.B. from Harvard College and an M.D. from Harvard Medical School. He completed his residency in Internal Medicine at Brigham and Women’s Hospital, his fellowship in Cardiology at MGH, and his postdoctoral studies at the Whitehead Institute at MIT. About Arbor Biotechnologies® Arbor Biotechnologies is a next-generation gene editing company focused on discovering and developing potentially curative genomic medicines, with the most extensive toolbox of proprietary genomic editors in the industry to date. Founded by Feng Zhang, David Walt, David Scott, and Winston Yan, our proprietary discovery engine is focused on discovering and evolving technologies that enable gene knockout, RT editing, precise excisions and large insertions into endogenous loci. Leveraging our wholly owned nucleases as the chassis for genetic modification, we have the unique ability to work backward from disease pathology and choose the optimal editors or combination of editors for the disease in question, with a focus on areas of high unmet need. As Arbor continues to advance its pipeline toward the clinic with an initial focus in liver and CNS disease, the Company has also secured several partnerships around gene editing and ex vivo cell therapy programs to broaden the reach of its novel nuclease technology. For more information, please visit: arbor.bio. MediaAmy Bonanno, Solebury Strategic Communications abonanno@soleburystrat.com 914-450-0349

Company pursuing an advanced small molecule MOS118 (formerly known as AVE0118) discovered by Sanofi Experienced team with strong background in Metabolic Diseases and Obstructive Sleep Apnea Seed financing led by Forty51 Ventures BASEL, Switzerland, July 13, 2022 /PRNewswire/ -- Mosanna Therapeutics AG, a Swiss Biotech company today announced that it has secured seed financing led by Forty51 Ventures to pursue the development of AVE0118 which will be referred to as MOS118 going forward, a small molecule developed by Sanofi S.A. (Paris, France). Mosanna will utilize the funds for formulation and manufacturing optimization and regulatory activities to enable the company's first Phase 1b clinical trial by end-2023. The company confirmed the appointment of co-founder Dr. Jonathan Talbot as Chief Executive Officer, who said, "I'm excited to develop a treatment for one of the modern world's growing, global health crises. Mosanna aims to bring the first, targeted precision medicine to Obstructive Sleep Apnea patients with a focus on the devastating cardiometabolic impact of this disease." Dr. Talbot brings broad industry experience with a strong track record in Pharma. The company also announced the appointment of industry veteran Ben Machielse as Chairman of the Board. Mr. Machielse's impressive career includes a former role as CEO of Vtesse, a company developing treatments for Niemann Pick Disease that was acquired by Sucampo in 2017, and COO at Omthera Pharmaceuticals, a company in the cardiovascular space acquired by AstraZeneca in 2013. Dr. Patrick Lévy, Professor at Université de Grenoble (France) and leading Key Opinion Leader in the field of Obstructive Sleep Apnea celebrated Mosanna's emergence saying, "I am delighted that all the excellent basic research and clinical work performed by Sanofi with this compound will now be taken forward by Mosanna with a focus on the important metabolic impacts of OSA." Mosanna Therapeutics was co-founded by Forty51 Ventures, a new Biotech Venture Capital fund focused on company formation in Biotech. Forty51 Ventures led the Seed Financing and will join the Board of Directors. About Metabolic Obstructive Sleep Apnea MOSA is characterized by obstruction of the upper airway during sleep that occurs when the muscles that support the soft tissues in the throat temporarily relax. Breathing is impaired or completely prevented causing blood oxygen levels to drop below the normal range. Low oxygen levels increase cardiac load and stress the nervous system which if left untreated increases significantly the risk for cardiovascular mortality. MOSA can also result in excessive daytime sleepiness and memory or concentration problems. Hundreds of millions of people around the globe are affected by MOSA. For further info please visit or email [email protected]. About Forty51 Ventures Forty51 is a recently founded Venture Capital firm focused on early stage venture creation in Europe backed by a strong investor syndicate with Stable Asset Management, several Pension funds, family offices and accomplished Biotech entrepreneurs. For further information please visit or email [email protected]. Logo - SOURCE Mosanna Therapeutics AG

GAITHERSBURG, Md., Sept. 28, 2015 /PRNewswire/ -- Vtesse, Inc. today announced that the first three patients have been screened for inclusion in its pivotal Phase 2b/3 clinical trial with VTS-270 for treatment of Niemann-Pick Type C1 Disease (NPC). This clinical trial follows a Phase 1 study conducted by researchers at the National Institutes of Health (NIH) Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD). NPC is a progressive, irreversible, chronically debilitating and ultimately lethal genetic disease. It is caused by a defect in lipid transportation within the cell, which leads to excessive accumulation of lipids in the brain, liver and spleen. Vtesse has worked extensively with regulators in the United States and Europe with the goal of conducting its pivotal study under one global protocol that will evaluate safety and efficacy of VTS-270 to support approval of the drug by regulatory agencies in both regions. Ben Machielse, Drs., President and Chief Executive Officer of Vtesse, Inc., said, "Starting the pivotal clinical trial for VTS-270 within nine months of the company's launch underscores our dedication to serve the NPC community. A key ingredient that enabled us to start the trial so quickly is the complete package of high-quality pre-clinical data that clearly outlines the potential benefits of VTS-270, such as prolonged survival and preservation of neurons, as well as encouraging clinical data from the Phase 1 trial. The pivotal trial will allow us to further evaluate safety and efficacy of VTS-270 to build a foundational dataset for the NPC community." He added, "While initiation of the first site at Rush University Medical Center and the screening of three patients is a key milestone, we intend to open several sites in quick succession in the United States and the European Union to ensure availability of sites in close proximity to the patients who are eligible to enroll in the trial." Vtesse's Phase 2b/3 prospective, randomized, double-blind, sham-controlled trial of VTS-270 will be conducted in patients affected by NPC disease. The trial will take place in up to 20 centers in the United States and Europe. It is a three-part, efficacy and safety trial of VTS-270, administered by the lumbar intrathecal route (IT) every two weeks, with a planned enrollment of approximately 51 patients (in Parts A and B). In Part A of the study, researchers will evaluate three different dose levels of VTS-270 versus sham-control to determine the dose level for Parts B and C. All participants in the pivotal trial will be eligible to receive treatment with VTS-270 in Part C, the open-label extension, until the time of regulatory decisions. Preliminary analyses of the Phase 1 trial, conducted post-hoc, suggest that the rate of disease progression had slowed down (based on a standardized measure) in children treated with VTS-270 as compared to the rate in an age- and disease severity-matched cohort obtained from a separate natural history study of NPC patients. The VTS-270 pivotal study addresses a pressing need to discover new treatments for the disease, according to Forbes D. Porter, M.D., Ph.D., Co-Principal Investigator of the Phase 2b/3 study, Senior Investigator and Program Head in the intramural research program of the Developmental Endocrinology and Genetics Program (PDEGEN), NICHD. He added that the trial has the potential to provide important information about the disease as well as how patients may tolerate VTS-270 and its potential effects on the symptoms of NPC. "The recent analysis of the preliminary Phase 1 clinical data for VTS-270 has provided encouraging support suggesting this treatment for NPC may be able to alter the course of this fatal disease," said Elizabeth Berry-Kravis, M.D., Ph.D., Co-Principal Investigator of the Phase 2b/3 study, and Professor of Pediatrics, Neurological Sciences, Biochemistry, Rush University Medical Center. "The beginning of patient screening for the pivotal Phase 2b/3 study for VTS-270 marks another important step in the journey toward targeted treatment of NPC, as clinical trials are the only means to determine the effectiveness of VTS-270. This pivotal trial will allow us to gather the data required to advance the regulatory process and move toward U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) approval for VTS-270." More information on this Phase 2b/3 study can be found at Vtesse's clinical trial website: . Vtesse launched the website this summer as a resource to patients, parents, caregivers and physicians. For more information on enrollment criteria, please see: . About VTS-270 Vtesse's lead compound, VTS-270, has shown promise in pre-clinical and clinical studies as a potential treatment for NPC. It is a well-characterized mixture of (2-hydroxypropyl)-beta-cyclodextrin that has been extensively evaluated in pre-clinical and clinical studies at the National Institutes of Health, as well as under individual compassionate-use Investigational New Drug applications (iINDs) and in other academic labs. Most recently, Vtesse announced positive clinical results from the on-going Phase 1 study that demonstrated VTS-270 stabilizes the overall measures of NPC while improving certain disease measures. NPC is a genetic disease affecting an estimated one in 100,000 to 150,000 children and is often misdiagnosed and/or under-diagnosed. Affected patients are usually identified in early childhood with ataxia, exhibit progressive impairment of motor and intellectual function, and often die before adulthood. About Vtesse Vtesse, Inc. is a rare disease company dedicated to developing drugs for patients suffering from diseases that are underserved. Vtesse is working collaboratively with the NIH and other leading academic centers to advance clinical study of VTS-270 for NPC, and to conduct pre-clinical discovery and development of other novel drugs for NPC and other lysosomal storage diseases (LSDs). Vtesse is led by a highly experienced management team that has been involved in the development of more than 20 approved drugs and vaccines. Its experienced consortium of investors, led by New Enterprise Associates, has committed initial funding that is expected to bring this compound through pivotal clinical trial. Vtesse is based in Gaithersburg, Maryland and is the first spin-out company from Cydan Development, Inc. For more information, visit  . Logo - To view the original version on PR Newswire, visit: SOURCE Vtesse, Inc. Help employers find you! Check out all the jobs and post your resume.