Year
DEALS // DEV.
Country
Therapeutic Area
Study Phase
Deal Type
Product Type
Dosage Form
Lead Product
Target
Lead Product(s) : PBFT02
Therapeutic Area : Neurology
Study Phase : Phase I/ Phase II
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Passage Bio Announces Interim Data from upliFT-D in FTD-GRN
Details : PBFT02 is an adeno-associated virus delivery gene therapy. It is being evaluated for the treatment of patients with frontotemporal dementia (FTD) with granulin (GRN) mutations.
Product Name : PBFT02
Product Type : Cell and Gene therapy
Upfront Cash : Inapplicable
January 10, 2025
Lead Product(s) : PBFT02
Therapeutic Area : Neurology
Highest Development Status : Phase I/ Phase II
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : PBGM01
Therapeutic Area : Genetic Disease
Study Phase : Phase I/ Phase II
Sponsor : GEMMA Biotherapeutics
Deal Size : $134.0 million
Deal Type : Licensing Agreement
Passage Bio Out-licenses Pediatric Gene Therapy Programs to GEMMA Biotherapeutics
Details : Through the license agreement, GEMMA will granted exclusive, worldwide rights for the development and commercialization of PBGM01 for GM1 gangliosidosis.
Product Name : PBGM01
Product Type : Cell and Gene therapy
Upfront Cash : Undisclosed
August 01, 2024
Lead Product(s) : PBGM01
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I/ Phase II
Sponsor : GEMMA Biotherapeutics
Deal Size : $134.0 million
Deal Type : Licensing Agreement
Lead Product(s) : PBFT02
Therapeutic Area : Neurology
Study Phase : Phase I/ Phase II
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Passage Bio Announces Positive Feedback from FDA on Expansion of upliFT-D Trial
Details : PBFT02 is an adeno-associated virus delivery gene therapy. It is being evaluated for the treatment of patients with frontotemporal dementia (FTD) with granulin (GRN) mutations.
Product Name : PBFT02
Product Type : Cell and Gene therapy
Upfront Cash : Not Applicable
July 16, 2024
Lead Product(s) : PBFT02
Therapeutic Area : Neurology
Highest Development Status : Phase I/ Phase II
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Lead Product(s) : PBFT02
Therapeutic Area : Neurology
Study Phase : Phase I/ Phase II
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Details : PBFT02 is an adeno-associated virus delivery gene therapy. It is being evaluated in phase 1/2 clinical trials for the treatment of patients with frontotemporal dementia (FTD) with granulin (GRN) mutations.
Product Name : PBFT02
Product Type : Cell and Gene therapy
Upfront Cash : Not Applicable
December 20, 2023
Lead Product(s) : PBFT02
Therapeutic Area : Neurology
Highest Development Status : Phase I/ Phase II
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Lead Product(s) : PBGM01
Therapeutic Area : Genetic Disease
Study Phase : Phase I/ Phase II
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Details : PBGM01 is an AAV-delivery gene therapy currently being developed for the treatment of infantile GM1, in which patients have mutations in the GLB1 gene causing little or no residual beta-galactosidase enzyme activity and subsequent neurodegeneration.
Product Name : PBGM01
Product Type : Cell and Gene therapy
Upfront Cash : Not Applicable
August 07, 2023
Lead Product(s) : PBGM01
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I/ Phase II
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Lead Product(s) : PBGM01
Therapeutic Area : Genetic Disease
Study Phase : Phase I/ Phase II
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Details : PBGM01 is an AAV-delivery gene therapy currently being developed for the treatment of infantile GM1, in which patients have mutations in the GLB1 gene causing little or no residual beta-galactosidase enzyme activity and subsequent neurodegeneration.
Product Name : PBGM01
Product Type : Cell and Gene therapy
Upfront Cash : Not Applicable
December 14, 2022
Lead Product(s) : PBGM01
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I/ Phase II
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Lead Product(s) : PBFT02
Therapeutic Area : Neurology
Study Phase : Phase I/ Phase II
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Details : PBFT02 utilizes an AAV1 viral vector to deliver, through ICM administration, a functional GRN gene to patients with mutations in the gene that encodes for progranulin (PGRN).
Product Name : PBFT02
Product Type : Cell and Gene therapy
Upfront Cash : Not Applicable
August 11, 2022
Lead Product(s) : PBFT02
Therapeutic Area : Neurology
Highest Development Status : Phase I/ Phase II
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Lead Product(s) : PBML04
Therapeutic Area : Genetic Disease
Study Phase : IND Enabling
Sponsor : University of Pennsylvania
Deal Size : Not Applicable
Deal Type : Not Applicable
Details : PBML04, an adeno-associated virus -delivery gene therapy to treat MLD, a recessive lysosomal storage disease caused by genetic mutations that result in demyelination of fibers of central and peripheral nervous systems, leading to progressive motor and co...
Product Name : PBML04
Product Type : Cell and Gene therapy
Upfront Cash : Not Applicable
June 08, 2022
Lead Product(s) : PBML04
Therapeutic Area : Genetic Disease
Highest Development Status : IND Enabling
Sponsor : University of Pennsylvania
Deal Size : Not Applicable
Deal Type : Not Applicable
Lead Product(s) : PBGM01
Therapeutic Area : Genetic Disease
Study Phase : Phase I/ Phase II
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Details : The data show that the low dose of PBGM01 is well-tolerated, has a favorable safety profile and exerts a biological effect that appears to translate to improvement in developmental milestones for these children.
Product Name : PBGM01
Product Type : Cell and Gene therapy
Upfront Cash : Not Applicable
May 18, 2022
Lead Product(s) : PBGM01
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I/ Phase II
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Lead Product(s) : PBKR03
Therapeutic Area : Genetic Disease
Study Phase : Phase I/ Phase II
Sponsor : University of Pennsylvania
Deal Size : Not Applicable
Deal Type : Not Applicable
Details : Preclinical results, report marked improvements in both disease progression and key biomarkers in large and small animal models of Krabbe disease following a single administration of PBKR03, with no observed dose-limiting toxicities.
Product Name : PBKR03
Product Type : Cell and Gene therapy
Upfront Cash : Not Applicable
March 29, 2022
Lead Product(s) : PBKR03
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I/ Phase II
Sponsor : University of Pennsylvania
Deal Size : Not Applicable
Deal Type : Not Applicable
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