X
Upload your Marketing & Sales content on your company Virtual Booth, click HERE.
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.
Lead Product(s): PBFT02
Therapeutic Area: Neurology Product Name: PBFT02
Highest Development Status: Phase I/ Phase IIProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable December 20, 2023
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.
Lead Product(s): PBGM01
Therapeutic Area: Genetic Disease Product Name: PBGM01
Highest Development Status: Phase I/ Phase IIProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable August 07, 2023
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.
Lead Product(s): PBGM01
Therapeutic Area: Genetic Disease Product Name: PBGM01
Highest Development Status: Phase I/ Phase IIProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable December 14, 2022
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).
Lead Product(s): PBFT02
Therapeutic Area: Neurology Product Name: PBFT02
Highest Development Status: Phase I/ Phase IIProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable August 11, 2022
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 cognitive impairments.
Lead Product(s): PBML04
Therapeutic Area: Genetic Disease Product Name: PBML04
Highest Development Status: IND EnablingProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: University of Pennsylvania
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable June 08, 2022
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.
Lead Product(s): PBGM01
Therapeutic Area: Genetic Disease Product Name: PBGM01
Highest Development Status: Phase I/ Phase IIProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable May 18, 2022
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.
Lead Product(s): PBKR03
Therapeutic Area: Genetic Disease Product Name: PBKR03
Highest Development Status: Phase I/ Phase IIProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: University of Pennsylvania
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable March 29, 2022
Details:
Interim safety data at three and nine months in Imagine-1 showed low dose PBGM01, a gene therapy for GM1 gangliosidosis well tolerated with no serious adverse events reported and no evidence of dorsal root ganglion toxicity.
Lead Product(s): PBGM01
Therapeutic Area: Genetic Disease Product Name: PBGM01
Highest Development Status: Phase I/ Phase IIProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable February 11, 2022
Details:
PBKR03 utilizes a next-generation proprietary AAV capsid to deliver, through intra-cisterna magna (ICM) administration, a functional GALC gene to Krabbe patients with mutations in the gene that codes for galactosylceramidase (GAL-C).
Lead Product(s): PBKR03
Therapeutic Area: Genetic Disease Product Name: PBKR03
Highest Development Status: Phase I/ Phase IIProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable April 05, 2021
Details:
PBKR03 utilizes a next-generation proprietary AAV capsid to deliver, through intra-cisterna magna (ICM) administration, a functional GALC gene to Krabbe patients with mutations in the gene that codes for galactosylceramidase (GAL-C).
Lead Product(s): PBKR03
Therapeutic Area: Genetic Disease Product Name: PBKR03
Highest Development Status: Phase I/ Phase IIProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable February 08, 2021
FULL SCREEN VIEW Click here to open all results in a new tab [this preview display 10 results] 
Market Place
