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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 the granting of inducement stock options to its new Chief Financial Officer, Simona King, and eight additional hires. Ms. King received 360,000 shares with an exercise price of $11.43, reflecting the closing stock price on August 23, 2021. The other employees were awarded options for 205,100 shares at an exercise price of $10.84, based on the closing price on August 16, 2021. These stock options will vest over four years, aligned with Nasdaq listing Rule 5635(c)(4).
Passage Bio (Nasdaq: PASG) expanded its collaboration with the University of Pennsylvania’s Gene Therapy Program to enhance genetic medicines for CNS disorders, focusing on Alzheimer's Disease and Temporal Lobe Epilepsy. The company expects to initiate Phase 1/2 trials for PBFT02 and PBKR03 by Q3 2021 and report initial safety data from PBGM01 in Q4 2021. Financially, Passage Bio reported a net loss of $48.4 million for Q2 2021, an increase from $27.2 million in Q2 2020, while cash reserves grew to $407.8 million, ensuring operational funding for at least 24 months.
Passage Bio (Nasdaq: PASG) expresses deep sorrow over the unexpected passing of founder and chairman, Dr. Tadataka Yamada, on August 3, 2021. Dr. Yamada contributed significantly to the company and the life sciences industry, serving as chairman since July 2017 and previously holding prestigious roles at Takeda and GlaxoSmithKline. His strategic insight shaped the company’s development of transformative genetic therapies for CNS disorders. Passage Bio aims to continue his legacy while maintaining a commitment to patients and innovation in gene therapy.
Passage Bio, Inc. (NASDAQ: PASG) announced that CEO Bruce Goldsmith will participate in a virtual panel at the 12th Annual Wedbush PacGrow Healthcare Virtual Conference, scheduled for August 10-12, 2021. Goldsmith's participation is set for August 11 at 8:35 a.m. ET. The event will be accessible via live webcast on the company's website, with a replay available for 30 days post-event. Passage Bio is dedicated to developing gene therapies for rare central nervous system disorders and has a collaboration with the University of Pennsylvania’s Gene Therapy Program.
Passage Bio (NASDAQ: PASG) will host a conference call on August 5, 2021, at 8:30 a.m. ET to discuss its second quarter 2021 financial results and recent business highlights. Investors can join the call by dialing 833-528-0605 (domestic) or 830-221-9711 (international), using conference ID 4037236. The call will also be available as a live audio webcast on the company’s website, with an archived version accessible for 30 days post-event. Passage Bio focuses on gene therapies for rare CNS disorders and collaborates with the University of Pennsylvania for its research.
Passage Bio, a clinical-stage genetic medicines company, granted an inducement award to its new Chief Commercial Officer, M. Maria Törnsén. This includes options to buy 200,000 shares at $13.04 each and 20,000 restricted stock units, vesting over four years. The award aligns with Nasdaq Rule 5635(c)(4) and aims to attract talent to advance therapies for rare CNS disorders. Passage Bio is focused on innovative gene therapies in partnership with the University of Pennsylvania's Gene Therapy Program to enhance its pipeline.
Passage Bio, Inc. (Nasdaq: PASG) has announced key leadership appointments, enhancing its capability in developing transformative therapies for rare CNS disorders. Effective immediately, Maria Törnsén takes on the role of Chief Commercial Officer, while Simona King will join as Chief Financial Officer on August 23, and Mark Forman, M.D., Ph.D. will assume the role of Chief Medical Officer on July 30. These appointments aim to bolster Passage Bio's pipeline and drive growth, positioning the company well for future clinical developments in gene therapy for patients in need.
Passage Bio (Nasdaq: PASG) announced the election of Dr. Derrell D. Porter to its Board of Directors and Audit Committee, effective May 27, 2021. Dr. Porter brings extensive experience in corporate strategy and product development from his leadership roles at Cellevolve Bio and Atara Biotherapeutics. His addition is expected to enhance the company's strategic direction in developing therapies for rare CNS disorders. Dr. Porter replaces Patrick Heron, who did not stand for re-election. The board aims to leverage Dr. Porter's expertise to strengthen commercial strategies for their gene therapies.
Passage Bio (NASDAQ: PASG) announced its participation in two virtual investor conferences in June 2021. The Jefferies Healthcare Conference will take place on June 2 at 4:30 p.m. ET, featuring a presentation format. On June 10, Passage Bio will engage in a fireside chat at the Goldman Sachs 42nd Annual Global Healthcare Conference at 11:20 a.m. ET. Both events will be available via live webcast on the company's website, with replays accessible for 30 days post-event. As a clinical-stage genetic medicines firm, Passage Bio focuses on innovative therapies for rare CNS disorders.
Passage Bio (Nasdaq: PASG) announced a significant presentation by the University of Pennsylvania’s Gene Therapy Program at the ASGCT annual meeting, showcasing a new mouse model to evaluate gene therapy efficacy for metachromatic leukodystrophy (MLD). Preliminary results indicate that Passage Bio’s candidate PBML04 showed promising efficacy, reducing neurological deficits in this model. This advancement addresses the challenge of limited mouse models for MLD, facilitating the preclinical evaluation of AAV-mediated gene therapy. The company aims to advance its IND-enabling studies for PBML04.