Welcome to our dedicated page for Passage Bio news (Ticker: PASG), a resource for investors and traders seeking the latest updates and insights on Passage Bio stock.
Passage Bio, Inc. develops clinical-stage genetic medicines for neurodegenerative diseases, with news centered on PBFT02 and the upliFT-D study in frontotemporal dementia. Company updates commonly address safety and biomarker data, progranulin and neurofilament light-chain measures, patient groups involving FTD-GRN and FTD-C9orf72, and FDA interactions tied to potential registrational development.
Recurring announcements also cover periodic financial results, corporate presentations, investor conference participation, manufacturing comparability work, and strategic or operating updates that affect the company's development plans and expense profile.
Passage Bio (NASDAQ: PASG) announced participation in the Goldman Sachs 41st Annual Global Healthcare Conference on June 11, 2020, at 11:20am ET. The event will be a fireside chat, and a live audio webcast can be accessed through the company's website. A replay will be available for 30 days post-event. Passage Bio focuses on developing therapies for rare, monogenic central nervous system disorders and has a collaboration with the University of Pennsylvania. Their portfolio includes six product candidates targeting conditions like GM1 gangliosidosis and Krabbe disease.
Passage Bio (NASDAQ: PASG) has received FDA's Rare Pediatric Disease (RPD) designation for its gene therapy PBGM01, aimed at treating infantile GM1 gangliosidosis, a severe CNS disorder. This designation reflects the urgent need for therapies for this rare condition. PBGM01 has also gained Orphan Drug designation, reinforcing its significance. The company plans to start a Phase 1/2 trial of PBGM01 in Q4 2020, with initial safety data expected by mid-2021. GM1 gangliosidosis, caused by GLB1 gene mutations, severely impacts infants, leading to shortened life expectancy.
Preclinical data from the University of Pennsylvania's Gene Therapy Program highlights the efficacy of a single injection of AAVhu68 delivering the GALC gene in animal models of Krabbe disease. The study revealed normalization of GALC enzyme activity, improved nerve conduction, and enhanced brain health in treated mice and dogs. The data suggests PBKR03 could be a major breakthrough for infantile Krabbe disease, aiming for IND submission later this year. The findings indicate significant potential for this gene therapy to restore myelination and nerve functions, ultimately increasing survival rates.
Passage Bio (NASDAQ: PASG) has expanded its collaboration with the Gene Therapy Program (GTP) at the University of Pennsylvania. This includes licensing an additional five programs, extending the collaboration to 2025, and committing $5 million annually for research funding. The agreement grants Passage exclusive rights to technologies developed during this partnership for their gene therapy products. This move is expected to enhance Passage's pipeline and expertise in creating transformative therapies for rare CNS disorders.