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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) announced participation in two virtual investor conferences in March 2021. The first is the Barclay’s Global Healthcare Conference on March 10, at 4:10 p.m. ET, featuring a presentation format. The second event is the Stifel 3rd Annual CNS Day on March 31, at 10:00 a.m. ET, in a fireside chat format. Both events will be webcast live, with replays available for 30 days. Passage Bio focuses on gene therapies for rare central nervous system disorders, collaborating with the University of Pennsylvania's Gene Therapy Program.
Passage Bio (NASDAQ: PASG) announced a conference call on March 3, 2021, at 8:30 a.m. ET to discuss its fourth quarter and full-year 2020 financial results. This call will provide key insights into the company's recent business highlights. Investors can join via phone or through a live audio webcast. The call will be archived for 30 days. Based in Philadelphia, Passage Bio is focused on developing gene therapies for rare central nervous system disorders, collaborating with the University of Pennsylvania to further its preclinical work and enhance its pipeline.
Passage Bio (Nasdaq: PASG) has appointed Dr. Maxine Gowen to its board of directors, effective February 18, 2021. Dr. Gowen, CEO of Tamuro Bio, brings extensive leadership experience from her tenure at Trevena and GSK. Her expertise is expected to support Passage Bio's transition into a clinical development organization. Simultaneously, Dr. Stephen Squinto has resigned from his board position after significant contributions, including advancing the company’s pipeline and supporting its IPO. Passage Bio aims to develop transformative gene therapies for rare CNS disorders.
Passage Bio (Nasdaq: PASG) announced FDA clearance for its IND application for PBKR03, a gene therapy targeting early infantile Krabbe disease, with clinical trials expected to start in 1H21. Krabbe disease, a rare CNS disorder, lacks approved therapies, highlighting the urgency of this development. PBKR03 utilizes a proprietary AAV capsid for gene delivery, aiming to restore GALC activity in affected patients. Preclinical studies show promising results, indicating potential for improved myelination and survival rates.
Passage Bio, Inc. (Nasdaq: PASG) announced FDA clearance for the IND application of PBFT02, a gene therapy for Frontotemporal Dementia (FTD) with GRN mutations, a condition with no approved disease-modifying treatments. Preclinical data from the University of Pennsylvania demonstrated PBFT02’s potential to elevate PGRN levels in the brain and cerebrospinal fluid while reducing lysosomal storage lesions. A Phase 1/2 clinical trial is expected to commence in H1 2021, focusing on safety, tolerability, and biomarker assessment, with initial data anticipated by late 2021 or early 2022.
Passage Bio, Inc. (Nasdaq: PASG) has priced its public offering of 7 million shares of common stock at $22.00 per share, aiming to raise $154 million in gross proceeds. The offering, which includes a 30-day option for underwriters to purchase an additional 1.05 million shares, is expected to close around January 26, 2021. The proceeds will support the company's efforts in gene therapies for rare central nervous system disorders. J.P. Morgan, Goldman Sachs, and Cowen are the joint bookrunning managers for the offering.
Passage Bio (Nasdaq: PASG) has announced a public offering of 7,000,000 shares of its common stock, with an additional 1,050,000 shares available through underwriters' options. This offering is intended to raise capital for the company's operations focusing on gene therapies for rare neurological disorders. J.P. Morgan, Goldman Sachs, and Cowen are managing the offering. The registration is pending approval from the SEC, and the offering's completion is uncertain.
Passage Bio (Nasdaq: PASG) announced a series of virtual Research & Development events for 2021, starting on January 25. The first event will focus on the investigational therapy PBGM01 targeting infantile GM1 gangliosidosis. Presenters include CEO Bruce Goldsmith, Chief Scientific Advisor James M. Wilson, and Chief Medical Officer Gary Romano, who will discuss the therapy's development and trial design. GM1 is a rare genetic disorder with a life expectancy of 2-4 years for infants. PBGM01 aims to deliver a functional GLB1 gene to mitigate neurodegeneration.
Passage Bio (Nasdaq: PASG) has submitted IND applications for PBFT02 aimed at treating Frontotemporal Dementia with Granulin Mutation and PBKR03 for Krabbe Disease. The company plans to initiate three clinical studies in the first half of 2021. It is also advancing PBML04 for Metachromatic Leukodystrophy into IND-enabling studies. Passage Bio continues to collaborate with the University of Pennsylvania’s Gene Therapy Program, having licensed a total of 17 programs. A new manufacturing lab will open in Q2 2021 to enhance testing capabilities.
On January 4, 2021, Passage Bio (Nasdaq: PASG) announced that its CEO, Bruce Goldsmith, will present a company overview at the J.P. Morgan 39th Annual Healthcare Conference on January 11, 2021, at 7:30 AM ET. A live webcast of the presentation will be available on the company's website for 30 days. Passage Bio focuses on developing gene therapies for rare central nervous system disorders, leveraging collaboration with the University of Pennsylvania’s Gene Therapy Program to advance its pipeline.
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