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Overview
Passage Bio, Inc. (PASG) is a clinical stage genetic medicines company focused on the development and advancement of transformative gene therapies for rare, monogenic central nervous system (CNS) disorders. The company is at the forefront of pioneering innovative treatments using sophisticated gene replacement strategies that leverage engineered adeno-associated virus (AAV) vectors. With an emphasis on addressing neurodegenerative diseases, Passage Bio targets conditions that have significant unmet needs in both pediatric and adult patient populations.
Innovative Gene Therapy Platform
At the heart of Passage Bio's approach is its robust gene therapy platform. The company utilizes proprietary AAV capsids, such as AAVHU68 and AAV1, to deliver functional copies of critical genes to the brain and peripheral tissues. This technology is designed to provide a one-time treatment capable of addressing the underlying genetic defects that lead to CNS disorders. By replacing dysfunctional genes, the therapies aim to restore lysosomal function and improve cellular health in affected tissues.
Key Clinical Programs
Passage Bio has focused its research on several critical therapeutic areas:
- Infantile GM1 Gangliosidosis: The company is advancing PBGM01, which employs its proprietary AAVHU68 capsid to deliver a functional GLB1 gene. This therapy aims to restore the production of the enzyme lysosomal acid beta-galactosidase to counteract the effects of GM1 gangliosidosis.
- Frontotemporal Dementia (FTD): PBFT02 is designed to address FTD due to progranulin deficiency. Using an AAV1 vector, this therapy delivers a functional GRN gene to elevate progranulin levels within the central nervous system, with the objective of improving lysosomal function and mitigating disease progression.
- Infantile Krabbe Disease: PBKR03 utilizes the same proprietary AAVHU68 platform to transport a functional GALC gene, aimed at restoring the activity of the galactosylceramidase enzyme, a key factor in the pathogenesis of Krabbe disease.
Strategic Collaborations and Licensing
To enhance its research and development capabilities, Passage Bio has engaged in several strategic collaborations. The company maintains a significant partnership with the University of Pennsylvania's Gene Therapy Program, which has bolstered its preclinical research initiatives. Additionally, Passage Bio has established agreements with key service providers, such as Catalent Maryland, to support clinical supply and development services. Notably, the company has executed out-license agreements with GEMMA Biotherapeutics for its pediatric lysosomal storage disease programs, thereby effectively expanding its operational focus towards adult neurodegenerative conditions and optimizing its resource allocation.
Operational Strategy and Research Excellence
Beyond its innovative therapeutic strategies, Passage Bio has implemented disciplined operational strategies, including workforce reorganization and the transition to an outsourced analytical testing model. These measures, which extend the company's operating cash runway, reflect a commitment to maintaining a lean operational structure while continuing to generate impactful clinical data. Such actions underscore the company’s dedication to delivering therapies that address the critical underlying pathology of neurodegenerative diseases.
Market Significance and Clinical Impact
Within the competitive landscape of genetic medicines and biopharmaceutical innovation, Passage Bio distinguishes itself through its rigorous scientific approach and strategic collaborations. The company’s focus on CNS disorders, supported by extensive preclinical evidence and evolving clinical data, positions it as a critical contributor to the transformation of therapies for rare neurodegenerative diseases. By targeting the root genetic causes with one-time treatments, Passage Bio is committed to not only advancing clinical research but also providing a clear framework for understanding the potential impact of gene therapies in an area of significant unmet medical need.
Commitment to Scientific Rigor and Patient Care
Passage Bio’s operations are driven by a rigorous scientific process combined with a deep understanding of the complexities inherent in CNS disorders. The company remains focused on constructing a coherent narrative that bridges advanced technology with clinical applicability, ensuring that every step—from research and development to strategic licensing—is meticulously planned and executed. This unwavering commitment to scientific excellence and patient care enhances its credibility among stakeholders while reinforcing its reputation as an expert in the field of genetic medicines.
Passage Bio (NASDAQ: PASG) reported its Q4 and full-year 2024 financial results, highlighting significant progress in its PBFT02 program for FTD-GRN patients. Interim data showed promising results, with Dose 1 PBFT02 increasing CSF progranulin levels from below 3 ng/mL to 13-27 ng/mL at six months and 22-34 ng/mL at 12 months.
The company has enrolled its first patient for Dose 2 PBFT02, which is 50% lower than Dose 1. Financial results show cash position of $76.8 million as of December 31, 2024, with runway extended into Q1 2027. Net loss for 2024 was $64.8 million ($1.07 per share), improved from $102.1 million in 2023. R&D expenses decreased to $40.2 million from $61.4 million, while G&A expenses reduced to $25.0 million from $41.6 million year-over-year.
Passage Bio (NASDAQ: PASG), a clinical stage genetic medicines company specializing in neurodegenerative diseases, has announced its participation in two major upcoming investor conferences.
The company will present at the TD Cowen 45th Annual Health Care Conference in Boston on March 5, 2025, at 9:10 a.m. ET, and the Leerink Global Biopharma Conference in Miami on March 12, 2025, at 8:00 a.m. ET. Both events will feature management presentations and investor meetings.
Interested parties can access live webcasts of the presentations through the Investors & Media section of Passage Bio's website at investors.passagebio.com. Replay recordings will remain available for 30 days after each event.
Passage Bio (NASDAQ: PASG) reported updated interim data from its Phase 1/2 upliFT-D clinical trial for PBFT02, targeting frontotemporal dementia (FTD) with granulin mutations. The data showed consistent increases in CSF PGRN expression and early signs of reduced disease progression compared to natural history data.
The company plans to evaluate a second dose that is 50% lower than Dose 1 for subsequent FTD-GRN and FTD-C9orf72 patients. Key upcoming milestones include reporting 12-month data from Dose 1 and interim data from Dose 2 in 2H 2025, with plans to seek regulatory feedback on FTD-GRN pivotal trial design in 1H 2026.
Passage Bio has successfully completed process development for a high-productivity, suspension-based manufacturing process for PBFT02. The company is transitioning to an outsourced analytical testing model and reducing its workforce by 55%, extending its cash runway into Q1 2027.
Passage Bio (NASDAQ: PASG) reports Q3 2024 financial results and business updates. The company has enrolled 4 patients in Cohort 2 of the upliFT-D trial for FTD-GRN, with dosing progressing as planned. Updated interim data from Cohort 1 showed PBFT02 was well-tolerated with elevated CSF progranulin levels for up to 12 months. Financial highlights include cash position of $84.8 million, R&D expenses of $8.7 million (down from $15.1 million YoY), and net loss of $19.3 million ($0.31 per share). The company expects current cash to fund operations through Q2 2026.
Passage Bio (NASDAQ: PASG), a clinical stage genetic medicines company specializing in neurodegenerative diseases, has announced its participation in the upcoming Guggenheim Securities Healthcare Innovation Conference. Will Chou, M.D., the company's president and CEO, will deliver a presentation on November 13, 2024, at 2:30 p.m. ET. The presentation will be accessible via live webcast on the company's investor relations website, with a replay available for 30 days after the event.
Passage Bio (NASDAQ: PASG) presented preclinical and interim clinical data for PBFT02, their gene therapy treatment for frontotemporal dementia (FTD-GRN), at the ESGCT 31st Annual Conference. Key findings showed that AAV1 vector achieved superior human progranulin levels compared to other vectors, and PBFT02 improved lysosomal histopathology and reduced neuroinflammation in test subjects.
The interim clinical data from the upliFT-D trial demonstrated that PBFT02 was well-tolerated and produced consistent, durable increases in CSF progranulin levels, maintained up to 12 months post-administration. The therapy showed promising results in achieving widespread vector distribution throughout the nervous system.
Passage Bio, a clinical stage genetic medicines company focused on neurodegenerative diseases, has announced its participation in Chardan's 8th Annual Genetic Medicines Conference. Will Chou, M.D., the company's president and CEO, will present on Tuesday, October 1, 2024, at 8:30 a.m. ET.
The presentation will be accessible via a live webcast on the Investors & Media section of Passage Bio's website at investors.passagebio.com. For those unable to attend the live event, a replay will be available for 30 days following the presentation.
Passage Bio is traded on the NASDAQ under the ticker symbol PASG.
Passage Bio (Nasdaq: PASG) will present updated data from its Phase 1/2 upliFT-D clinical trial of PBFT02, a gene therapy for frontotemporal dementia (FTD) with granulin mutations, at the 14th International Conference on Frontotemporal Dementias on September 20, 2024. The interim results from Cohort 1 (n=5) show that Dose 1 of PBFT02 was well-tolerated in patients receiving an enhanced immunosuppression regimen, with no serious adverse events observed.
Key findings include:
- Robust and durable increase in CSF progranulin (PGRN) expression, up to 6-fold at one month and 10-fold at six months
- CSF PGRN levels exceeded healthy adult control ranges
- Elevated CSF PGRN levels sustained up to 12 months post-treatment
- No evidence of dorsal root ganglion toxicity or clinically significant immune responses
These results suggest PBFT02's potential as a best-in-class progranulin-raising therapy for neurodegenerative diseases.
Passage Bio (Nasdaq: PASG) has appointed Tom Kassberg to its Board of Directors and Audit Committee. Kassberg, currently Chief Business Officer and Executive VP at Ultragenyx, brings extensive experience in strategic planning and corporate development. His background includes leadership roles at Proteolix, InterMune, Plexxikon, and SUGEN. Passage Bio CEO Will Chou highlighted Kassberg's expertise in advancing gene therapies for rare diseases, which aligns with the company's focus on neurodegenerative diseases. Kassberg expressed enthusiasm for joining Passage Bio, particularly noting the progress of the PBFT02 program and its potential expansion into additional indications.
Passage Bio (NASDAQ: PASG), a clinical stage genetic medicines company focusing on neurodegenerative diseases, has announced its participation in the H.C. Wainwright 26th Annual Global Investment Conference. The company's president and CEO, Will Chou, M.D., is scheduled to present on Monday, September 9, 2024, at 7:00 a.m. ET.
Investors and interested parties can access a live webcast of the presentation through the Investors & Media section of Passage Bio's website at investors.passagebio.com. For those unable to attend the live event, a replay will be available for 30 days following the presentation.
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