Penicillamine

Vivet Therapeutics Announces Presentations at Upcoming European Society of Gene and Cell Therapy (ESGCT) 2023 Annual Congress Paris, France, October 23, 2023 (GLOBE NEWSWIRE) – Vivet Therapeutics (“Vivet”), a clinical-stage biotechnology company developing novel gene therapies for rare, inherited metabolic conditions, announced its participation and presentations at the 30th European Society of Gene and Cell Therapy (ESGCT) 2023 Annual Congress. The congress will be held from October 24 – 27, 2023, in Brussels, Belgium. Dr. Nicholas Weber, Principal Scientist and Lead for New Programs and Innovations at Vivet, will present in an invited talk about adeno-associated virus (AAV) therapies targeting inherited cholestasis. Gloria Gonzalez-Aseguinolaza, Vivet Therapeutics Chief Scientific Officer, will also present new preclinical results related to its lead program VTX-801, which utilizes the proprietary recombinant AAV (rAAV) technology to target Wilson disease. The presentation and poster details are listed below; the full program is available on the ESGCT website.Session 3c: Gene therapy for metabolic diseases at preclinical stageDate/Time: Wednesday 25 Oct at 8:30.Title: INV21 – Advances and pitfalls in AAV therapy targeting inherited cholestasisPresenter: Nicholas Weber, Ph.D , Principal Scientist, Vivet TherapeuticsRoom: Maison de la Poste, PARALLEL Poster Number: P035Category: AAV & non integrative vectorsPoster Title: Improvement of progressive familial intrahepatic cholestasis type 2 via AAV-mediated hepatic BSEP expression in mice Presenter: Angie Molina, Ph.D. (Fundación para la Investigación Medica Aplicada in partnership with Vivet Therapeutics) Poster Number: P585Category: Metabolic DiseasesPoster Title: Therapeutic efficacy of VTX-801 Wilson’s disease mice at an advanced stage the disease Presenter: Gloria Gonzalez-Aseguinolaza, Ph.D ,Chief Scientific Officer, Vivet Therapeutics Number: P567Category: Metabolic DiseasesPoster Title: Therapeutic Efficacy of the Combination of Long-Term Penicillamine Treatment with a Single Administration of VTX-801 in Wilson’s Disease MicePresenter: Gloria Gonzalez-Aseguinolaza, Ph.D, Chief Scientific Officer, Vivet Therapeutics About Vivet TherapeuticsVivet Therapeutics is a biotechnology company developing transformative and novel gene therapy treatments for rare, inherited metabolic diseases.Vivet’s lead program is VTX-801, a novel investigational gene therapy being evaluated in the GATEWAY clinical trial, a Phase I/II clinical trial to determine the safety, viability, and pharmacological activity of a single intravenous infusion in adults with Wilson disease. Wilson disease is a rare genetic disorder caused by mutations in the gene encoding the ATP7B protein, which reduces the ability of the liver and other tissues to regulate copper levels, causing severe hepatic damage, neurologic symptoms and potentially death. VTX-801 has been granted Orphan Drug Designation (ODD) by the U.S. Food and Drug Administration (FDA) and the European Commission (EC). It has also received Fast Track Designation by the FDA.Vivet is building a diversified gene therapy pipeline based on novel rAAV (recombinant AAV) with five preclinical programs and new technologies addressing key challenges of AAV-based gene therapy related to the sustainability of expression in young patients and immunomodulation for potential retreatment. Vivet develops these technologies through its partnership with, and exclusive licenses from, the Fundación para la Investigación Médica Aplicada (FIMA), a not-for-profit foundation at the Centro de Investigación Medica Aplicada (CIMA), University of Navarra based in Pamplona, Spain. Visit www.vivet-therapeutics.com for more information. If you would like to meet with the Vivet team during the congress, or at any other time, please contact: info@vivet-therapeutics.com. For more information on the ongoing VTX-801 GATEWAY clinical trial, visit https://www.gatewaytrialwilsondisease.com/. https://www.linkedin.com/company/vivettherapeutics/https://twitter.com/vivet_tx

PITTSBURGH, April 19, 2023 /PRNewswire/ -- PANTHERx® Rare, a leader in rare disease product patient access and support services in the United States, is pleased to announce the launch of CUVRIOR to treat stable Wilson Disease in adults who are de-coppered and able to take penicillamine. "We are pleased to add CUVRIOR to the list of PANTHERx® medications. The PANTHERx team works hard to make a positive difference in the lives of our patients who are dealing with devastating and debilitating diseases and CUVRIOR is yet another way we can make an impact," said Rob Snyder, president of PANTHERx® Rare. Wilson Disease is a rare genetic disorder caused by a mutation in the ATP7B gene, resulting in the accumulation of copper throughout the body. Copper deposits in the body lead to a wide array of symptoms including, but not limited to tremors, anxiety, jaundice due to liver injury, and Kayser-Fleischer rings (brown rings in the corneas of the eyes). Untreated, Wilson Disease can be fatal. Serious complications include problems with the liver, kidneys, neurological (e.g., tremors, clumsy gait, speech difficulties), psychological (e.g., depression, psychosis) and blood problems (anemia, jaundice). CUVRIOR is an innovative oral formulation of the active ingredient trientine created by Orphalan to treat adult patients with stable Wilson Disease who have already been de-coppered. CUVRIOR works by chelating copper in the body to decrease copper absorption and increase urinary copper excretion. In clinical trials, CUVRIOR was found to be well tolerated and non-inferior to penicillamine in reducing non-ceruloplasmin copper (NCC) levels in Wilson Disease patients. About PANTHERx Rare PANTHERx Rare is one of the largest and fastest growing rare pharmacies in the United States. PANTHERx transforms lives by delivering medicine breakthroughs, clinical excellence, and access solutions to people living with rare and devastating conditions. Although the overall incidence of rare diseases is as common as diabetes, less than 7% of the 7,000 known rare and devastating disorders have an FDA-approved therapy. Changes in federal policy and advances in science have led to a surge in FDA orphan drug approvals, providing tremendous hope to the rare disease community. PANTHERx was recently awarded Specialty Pharmacy of the Year by the National Association of Specialty Pharmacy (NASP) and earned the inaugural Accredited Distinction in Rare Diseases and Orphan Drugs from the Accreditation Commission for Health Care (ACHC). PANTHERx is now a five-time winner of the prestigious MMIT Patient Choice Award, including the 2022 honor. PANTHERx is headquartered in Pittsburgh, licensed in all 50 states, and holds accreditations from URAC, NABP, and ACHC. IMPORTANT SAFETY INFORMATION Do not use CUVRIOR if you are allergic to trientine or to any of the ingredients in CUVRIOR. Your Wilson's Disease symptoms could get worse when you start treatment. This could happen because too much copper is removed from the body in a short period of time. Your doctor may need to reduce your dose or stop CUVRIOR treatment. Copper deficiency may develop following treatment with CUVRIOR. Your doctor will do tests to monitor your urine and blood for copper. Iron deficiency may develop while taking CUVRIOR. If this happens, your doctor may tell you to take iron supplements for a limited time. Allergic reactions, such as a rash can occur. If a rash or other allergic reaction occurs, stop taking CUVRIOR and get emergency medical help. The most common side effects are pain in the abdomen, change in bowel habits, rash, hair loss (alopecia), and mood swings. Take CUVRIOR at least 1 hour apart from any other oral medicine. Tell your doctor about all the medicines you're taking, including over-the-counter medicines, vitamins, and herbal supplements. Avoid taking supplements when taking CUVRIOR. Taking CUVRIOR with mineral supplements (e.g., iron, zinc, calcium, magnesium) can reduce the effectiveness of CUVRIOR. If iron supplementation is necessary, take CUVRIOR at least 2 hours before or 2 hours after taking iron; for other mineral supplements (e.g., zinc, calcium, magnesium), take CUVRIOR at least 1 hour before or 2 hours after. Do not start or stop any medicines while taking CUVRIOR without talking to your doctor. These are not all the possible side effects of CUVRIOR. You are encouraged to report negative side effects of prescription drugs. Contact Orphalan at 1-800-961-8320 or FDA at 1-800-FDA-1088 or medwatch. Please see full Prescribing Information. 03/2023 US-ORPH-CUVR-023* - Orphalan reviewed and approved this material exclusively regarding CUVRIOR, Wilson disease and Orphalan related information and claims. SOURCE PANTHERx Rare Pharmacy