Passage Bio Earnings Call Transcripts
Fiscal Year 2026
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AAV gene therapy for FTD-GRN is advancing with a focus on earlier-stage patients and a safer, lower dose, showing promising biomarker and safety data. Regulatory discussions are ongoing, and a Huntington’s program is progressing toward clinical candidate selection.
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Lead gene therapy program for FTD-GRN shows robust, durable target engagement and favorable safety, with a focus on earlier-stage patients and improved trial design. Huntington's program targets MSH3, with clinical candidate selection expected in H2. Cash runway extends into Q1 2027.
Fiscal Year 2025
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The lead gene therapy program targets early-stage FTD-GRN patients, aiming to raise progranulin levels more effectively than competitors. Encouraged by FDA guidance, the team plans early regulatory engagement and expects key data and feedback in the first half of next year.
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Gene therapy programs for neurodegenerative diseases are advancing with local delivery, AI-driven data analysis, and strong clinical results. Regulatory agencies are increasingly flexible, and access is expected to improve as procedures align with existing hospital practices.
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PBFT02, a one-time AAV1 gene therapy for FTD, shows robust, durable increases in progranulin and early signs of slowing neurodegeneration, with a favorable safety profile and strong competitive positioning. Expansion to new patient groups and key regulatory milestones are planned through 2026.
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PBFT02, a one-time AAV1 gene therapy for FTD-GRN, shows durable CSF progranulin elevation and promising early biomarker effects on neurodegeneration. The program is advancing with robust manufacturing, regulatory alignment, and plans for further data readouts in 2025.
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PBFT02, a one-time AAV1 gene therapy for FTD with granulin mutations, shows durable, elevated CSF progranulin and promising early biomarker effects, with expanded indications and strong manufacturing capabilities. Additional clinical data and regulatory milestones are expected through 2026.
Fiscal Year 2024
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PBFT02, a one-time AAV1 gene therapy for FTD-GRN, shows high, durable, and consistent CSF progranulin levels with a favorable safety profile. Regulatory feedback is positive, with pivotal discussions planned and expansion to FTD-C9 underway. Differentiation is based on durability and convenience.
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A gene therapy program for FTD-GRN showed strong, durable increases in CSF progranulin and a favorable safety profile, prompting expansion into additional neurodegenerative diseases. Key milestones include upcoming data presentations and regulatory discussions, with cash runway through Q2 2026.
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A one-time gene therapy for FTD with granulin mutation shows strong, durable target engagement and favorable safety in early clinical data. Expansion to additional neurodegenerative indications and pivotal study planning are underway, with key data updates expected at ISFTD and throughout 2025.
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PBFT02, an AAV1 gene therapy, shows best-in-class, durable CSF progranulin elevation in FTD-GRN patients, with a strong safety profile and potential to address other neurodegenerative diseases. Strategic focus has shifted to adult indications, with robust cash runway and upcoming data milestones.