NEW YORK, NY — Myrtelle Inc., a clinical stage gene therapy company focused on developing transformative treatments for neurodegenerative diseases, today announced that the U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) Designation to the Company’s lead gene therapy product candidate, rAAV-Olig001-ASPA for the treatment of...
treatment News
Nagoya, Japan – Researchers from Nagoya University Graduate School of Medicine in Japan have successfully treated the skin diseases epidermolytic ichthyosis (EI) and ichthyosis with confetti (IWC) by transplanting genetically healthy skin to inflamed areas. Transplanting healthy skin to inflamed areas has been used as a treatment option for severe...
PARIS & CAMBRIDGE, Mass. – NANOBIOTIX (Euronext : NANO –– NASDAQ: NBTX – the ‘‘Company’’), a late-clinical stage biotechnology company pioneering physics-based approaches to expand treatment possibilities for patients with cancer, today announced the initiation of a new phase I study evaluating NBTXR3 activated by radiation therapy (RT) for patients with...
São Paulo, Brazil – A novel therapeutic strategy developed by researchers at São Paulo State University (UNESP) could revolutionize the treatment of visceral leishmaniasis, a neglected tropical disease (NTD) transmitted by sandflies. Some 12 million people suffer from the disease worldwide, and 700,000 to 1 million new cases of occur...
São Paulo, Brazil – A low-cost technology involving nanoparticles loaded with antibiotics and other antimicrobial compounds that can be used in multiple attacks on infections by the bacterium responsible for most cases of tuberculosis has been developed by researchers at São Paulo State University (UNESP) in Brazil and is reported...
DALLAS, TX — Nanoscope Therapeutics Inc., a late-stage clinical biotechnology company developing gene therapies for inherited retinal diseases and age-related macular degenerations (AMD), today announced positive top-line results after the completion of the 2-year Phase 2b RESTORE randomized, controlled clinical trial of its lead program, MCO-010, a mutation-agnostic gene therapy...
Nanoscope Therapeutics Inc., a clinical-stage biotechnology company developing gene therapies for retinal degenerative diseases, today announced that the International Nonproprietary Names (INN) Expert Committee of the World Health Organization (WHO) has selected "sonpiretigene isteparvovec" for the nonproprietary name of the Company's MCO-010, an ambient-light activatable Multi-Characteristic Opsin (MCO) optogenetic monotherapy to restore vision in patients with retinitis pigmentosa (RP) and Stargardt disease.
DALLAS, Texas — Nanoscope Therapeutics Inc., a late-stage clinical biotechnology company developing gene therapies for inherited retinal diseases and age-related macular degenerations (AMD), today provided an update following recent regulatory discussions with the U.S. Food and Drug Administration (FDA) and from the Scientific Advice meeting with the Icelandic Medicines Agency (IMA),...
Philadelphia, PA – Thursday afternoon marked a major regulatory milestone. In one of the year’s most highly anticipated FDA decisions, the regulator approved Madrigal Pharmaceuticals’ Rezdiffra (resmetirom) as the first-ever therapy for the treatment of adults with noncirrhotic nonalcoholic steatohepatitis (NASH). Or was the approval for metabolic dysfunction-associated steatohepatitis (MASH)? If...
DUBLIN – Jazz Pharmaceuticals plc (Nasdaq: JAZZ) today announced that the National Comprehensive Cancer Network® (NCCN) added Rylaze™ (asparaginase erwinia chrysanthemi (recombinant)-rywn) to the Clinical Practice Guidelines in Oncology (NCCN Guidelines®) for Acute Lymphoblastic Leukemia (ALL), for both pediatric and adult patients. The NCCN Guidelines for ALL and the NCCN Guidelines for Pediatric...