krabbe disease

Human iPSC-derived myelinating organoids and globoid cells to study Krabbe disease

Research / December 12, 2024

The article, “Human iPSC-derived myelinating organoids and globoid cells to study Krabbe disease”, explores innovative in vitro methods to study Krabbe disease (KD), a severe lysosomal storage disorder. Using induced pluripotent stem cells (iPSCs), the research develops human-based models that replicate key aspects of the disease, providing insights into its mechanisms and potential therapeutic approaches. …

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An Update from Passage Bio on Out-Licensing of Pediatric Programs to GEMMA Biotherapeutics

News / August 2, 2024

On August 1, 2024, Passage Bio shared they entered into a new agreement with GEMMA Biotherapeutics for the out-licensing of three pediatric gene therapy programs. Through this transaction, Passage Bio has granted GEMMA Biotherapeutics the rights for the development and commercialization of PBGM01 for GM1 gangliosidosis, PBKR03 for Krabbe disease, and PBML04 for metachromatic leukodystrophy.  …

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FDA Clearance of Investigational New Drug for Patients with Krabbe Disease

uncategorized / January 5, 2021

January 4, 2021 by Dan Salvo Forge Biologics has received IND, IBC, and IRB clearance for FBX-101, a gene therapy for patients with Krabbe disease, a rare and fatal genetic disorder FBX-101 is a first-in-human potential treatment utilizing an adeno-associated virus (AAV) to deliver a functioning copy of the GALC gene to cells in the CNS and …

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Patient Focused Drug Development Meeting on Krabbe Disease

News / June 8, 2020

NORD invites you to register for an externally-led Patient-Focused Drug Development (EL- PFDD) virtual meeting on Krabbe disease, taking place Thursday, October 29, 12:30 – 4:30 pm ET. Patients and caregivers impacted by Krabbe disease who are interested in potentially speaking at the meeting as panelists should be sure to fill out this form by August 7. If you …

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