MedPath

PBFT02

Generic Name
PBFT02
Brand Names
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Drug Type
Biotech
Chemical Formula
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CAS Number
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Unique Ingredient Identifier
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Background

PBFT02 is a non-replicating recombinant adeno-associated virus serotype 1 vector delivering human GRN gene encoding the protein progranulin.

Associated Conditions
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Associated Therapies
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morningstar.com
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Passage Bio Announces Interim Data from upliFT-D Study in FTD-GRN and Provides Update on PBFT02 Progress

PBFT02 showed increased CSF PGRN levels and reduced plasma NfL levels in FTD-GRN patients. Dose 2, 50% lower than Dose 1, is being evaluated. Passage Bio extended cash runway to 1Q 2027 by reducing expenses and transitioning to outsourced analytical testing. 12-month data from Dose 1 and interim data from Dose 2 expected in 2H 2025.
tipranks.com
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Passage Bio announces data from upliFT-D study in FTD

Passage Bio (PASG) updated Phase 1/2 upliFT-D trial data for PBFT02 in frontotemporal dementia treatment, showing durable CSF PGRN levels and early plasma NfL reduction. Evaluating lower Dose 2 for FTD-GRN and FTD-C9orf72 patients. Plans to report 12-month Dose 1 data and Dose 2 interim data by 2H 2025, seek regulatory feedback in 1H 2026. Completed PBFT02 manufacturing process development.

Passage Bio Announces Interim Data from upliFT-D Study in FTD-GRN and Provides Business Updates

Passage Bio reported PBFT02's Phase 1/2 upliFT-D trial data for FTD-GRN, showing increased CSF PGRN and reduced plasma NfL levels. Dose 2, 50% lower, is being evaluated. Cash runway extended to 1Q 2027 via operational adjustments. 12-month Dose 1 data and Dose 2 interim data expected in 2H 2025.
stocktitan.net
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Passage Bio's FTD Treatment Shows Promise in Clinical Trials

Passage Bio reports interim data from the upliFT-D study showing PBFT02 increased CSF PGRN levels and reduced plasma NfL levels in FTD-GRN patients. Plans to evaluate a lower Dose 2, report 12-month data in 2H 2025, and seek regulatory feedback in 1H 2026. Extended cash runway to 1Q 2027 by reducing expenses and transitioning to outsourced analytical testing.
globenewswire.com
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Passage Bio Announces Interim Data from upliFT-D Study

Passage Bio reports PBFT02's Phase 1/2 upliFT-D trial updates for FTD-GRN, showing increased CSF PGRN and reduced plasma NfL levels. Plans include Dose 2 evaluation, extending cash runway to 1Q 2027, and initiating FTD-C9orf72 dosing in 1H 2025. Manufacturing process scaled up for efficiency.
etfdailynews.com
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Passage Bio, Inc. (NASDAQ:PASG) Major Shareholder Lynx1 Capital Management Lp Acquires 373,645 Shares

Lynx1 Capital Management Lp increased its stake in Passage Bio by purchasing 373,645 shares at $0.65 each, totaling $242,869.25, now owning 9,256,953 shares valued at ~$6,017,019.45. Institutional investors hold 53.48% of Passage Bio, with several analysts setting 'buy' ratings and price targets up to $13.00. Passage Bio develops gene therapies for CNS diseases.
biospace.com
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Passage Bio to Present Positive Interim Data from Cohort 1 Patients with FTD-GRN in upliFT

Dose 1 of PBFT02 in upliFT-D trial for FTD-GRN achieved robust CSF progranulin levels, sustained up to 12 months, and was well-tolerated with revised immunosuppression. Updated data to be presented at ISFTD2024.
globenewswire.com
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Passage Bio to Present Positive Interim Data from Cohort 1

Dose 1 of PBFT02 in upliFT-D trial achieved robust CSF progranulin levels in FTD-GRN patients, sustained for up to 12 months, and was well-tolerated with no serious adverse events.
theaftd.org
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A Conversation with Juan Chavez, MD, of Passage Bio

Passage Bio utilizza terapie geniche con virus adeno-associati per trattare la FTD, mirando a correggere i livelli carenti della proteina progranulina. La FTD richiede un trattamento che rallenti la progressione della malattia, e la partecipazione alla ricerca clinica è cruciale per il progresso. Il panorama della ricerca mostra progressi significativi nella comprensione della biologia della malattia, aumentando le possibilità di sviluppare trattamenti efficaci.
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