Passage Bio Inc
NASDAQ:PASG
If you don’t study any companies, you have the same success buying stocks as you do in a poker game if you bet without looking at your cards.
Utilize notes to systematically review your investment decisions. By reflecting on past outcomes, you can discern effective strategies and identify those that underperformed. This continuous feedback loop enables you to adapt and refine your approach, optimizing for future success.
Each note serves as a learning point, offering insights into your decision-making processes. Over time, you'll accumulate a personalized database of knowledge, enhancing your ability to make informed decisions quickly and effectively.
With a comprehensive record of your investment history at your fingertips, you can compare current opportunities against past experiences. This not only bolsters your confidence but also ensures that each decision is grounded in a well-documented rationale.
Do you really want to delete this note?
This action cannot be undone.
This alert will be permanently deleted.
Passage Bio Inc
Passage Bio, Inc. operates as a genetic medicines company. The company is headquartered in Philadelphia, Pennsylvania and currently employs 133 full-time employees. The company went IPO on 2020-02-28. The company is focused on developing transformative therapies for central nervous system (CNS) disorders. Its first product candidate, PBGM01, utilizes AAVhu68 capsid to deliver to the brain and peripheral tissues a functional GLB1 gene encoding lysosomal beta-galactosidase (β-gal) for gangliosidosis (GM1). Its second product candidate, PBFT02, utilizes AAV1 capsid to deliver to the brain a functional GRN gene encoding progranulin (PGRN) for frontotemporal dementia (FTD) caused by progranulin deficiency (FTD-GRN). Its third product candidate, PBKR03, utilizes AAVhu68 capsid to deliver to the brain and peripheral tissues a functional gene encoding the hydrolytic enzyme galactosylceramidase (GALC) for Krabbe disease. The company also has four programs in the research stage: PBML04 for metachromatic leukodystrophy (MLD); PBAL05 for amyotrophic lateral sclerosis (ALS); PBCM06 for Charcot-Marie-Tooth Type 2A (CMT2A), and other.
Passage Bio, Inc. operates as a genetic medicines company. The company is headquartered in Philadelphia, Pennsylvania and currently employs 133 full-time employees. The company went IPO on 2020-02-28. The company is focused on developing transformative therapies for central nervous system (CNS) disorders. Its first product candidate, PBGM01, utilizes AAVhu68 capsid to deliver to the brain and peripheral tissues a functional GLB1 gene encoding lysosomal beta-galactosidase (β-gal) for gangliosidosis (GM1). Its second product candidate, PBFT02, utilizes AAV1 capsid to deliver to the brain a functional GRN gene encoding progranulin (PGRN) for frontotemporal dementia (FTD) caused by progranulin deficiency (FTD-GRN). Its third product candidate, PBKR03, utilizes AAVhu68 capsid to deliver to the brain and peripheral tissues a functional gene encoding the hydrolytic enzyme galactosylceramidase (GALC) for Krabbe disease. The company also has four programs in the research stage: PBML04 for metachromatic leukodystrophy (MLD); PBAL05 for amyotrophic lateral sclerosis (ALS); PBCM06 for Charcot-Marie-Tooth Type 2A (CMT2A), and other.
Strong Cash Runway: Passage Bio ended 2022 with $189.6 million in cash, expected to fund operations into the first half of 2025.
Clinical Progress: The company advanced its two lead gene therapy programs, with new positive interim data in GM1 gangliosidosis and plans to dose additional patients at higher levels.
Key 2023 Milestones: Initial data readouts for high-dose infantile GM1 and the first cohort of the FTD trial are targeted for mid and late 2023, respectively.
Study Design Adjustments: Passage Bio is revising inclusion criteria to focus on earlier-stage GM1 patients and increasing dosing to optimize outcomes based on biomarker responses.
Cost Controls: Streamlined operations led to a significant reduction in both R&D and G&A expenses year-over-year.
Regulatory Strategy: The company will discuss registrational study design with regulators after generating additional high-dose patient data, delaying such conversations to 2024.
