Forge Biologics Announces Updated Positive Clinical Data in RESKUE, a Novel Phase 1/2 Gene Therapy Trial for Patients with Krabbe Disease
"There's no doubt this news is upsetting but we won't allow it to disrupt our momentum in achieving advances in research and patient care for those impacted by Krabbe disease. However, I do yearn for the right company to utilize the research and work conducted under Passage Bio for PBKR03 to bring the treatment to the finish line. We will have to wait and see if that's in the cards. To all of the families and patients, tough news and disappointing results are part of the path in all rare diseases. We must stay the course and stay focused because that's how we will #curekrabbe." - Stacy Pike-Langenfeld, President, KrabbeConnect
Published: November 10, 2022
Forge Biologics Announces Updated Positive Clinical Data in RESKUE, a Novel Phase 1/2 Gene Therapy Trial for Patients with Krabbe Disease
Published: October 11, 2022
COLUMBUS, OHIO – Forge Biologics, a gene therapy-focused contract development and manufacturing organization, announced today that Chief Medical Officer Maria Escolar, M.D, MS., presented updated clinical data from the RESKUE Phase 1/2 clinical trial for FBX-101—the Company’s novel gene therapy for the treatment of patients with Krabbe disease.
Passage Bio Receives European Commission Orphan Designation for PBKR03 for Treatment of Krabbe Disease
Published: April 5, 2021
PHILADELPHIA, April 5, 2021 (GLOBE NEWSWIRE) -- Passage Bio, Inc., (Nasdaq: PASG), a clinical-stage genetic medicines company focused on developing transformative therapies for rare monogenic central nervous system (CNS) disorders, today announced that the European Commission has granted Orphan designation for PBKR03, an adeno-associated virus (AAV)-delivery gene therapy for the treatment of Krabbe disease (Globoid Cell Leukodystrophy). The designation was based on a positive opinion from the European Medicines Agency Committee for Orphan Medicinal Products. Currently, there are no approved disease-modifying therapies available for Krabbe disease, a rare lysosomal storage disease that most often presents early in a child’s life. The U.S. Food and Drug Administration (FDA) previously granted Fast Track, Orphan Drug and Rare Pediatric Disease designations to PBKR03 for the treatment of Krabbe disease.
U.S. Food and Drug Administration Grants Fast Track Designation to Three Passage Bio Gene Therapy Candidates Targeting Rare CNS Disorders
Published: March 8, 2021
PHILADELPHIA, March 8, 2021 (GLOBE NEWSWIRE) -- Passage Bio, Inc., (Nasdaq: PASG), a genetic medicines company focused on developing transformative therapies for rare, monogenic central nervous system (CNS) disorders, today announced the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to the company’s three lead investigational gene therapies: PBGM01 for the treatment of GM1 gangliosidosis (GM1), PBFT02 for frontotemporal dementia with granulin mutations (FTD-GRN), and PBKR03 for Krabbe disease. Passage Bio plans to initiate clinical trials in GM1 in the first quarter of 2021 and in FTD-GRN and Krabbe disease in the first half of 2021.
FDA Fast Track, ODD, and RPDD Designations for FBX-101 Gene Therapy for Patients with Krabbe Disease
Published: February 16, 2021
COLUMBUS, OHIO, February 16, 2021 – Forge Biologics Inc., a fully integrated clinical stage gene therapy manufacturing and development company, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track, Orphan Drug, and Rare Pediatric Disease (RPD) designations to FBX-101 for the treatment of patients with Krabbe disease. Forge is now actively recruiting patients for enrollment in the RESKUE phase 1/2 clinical trial of FBX-101, a novel, first-in-human AAV gene therapy for the disease. FBX-101 is the first intraveniousgene therapy program for patients with Krabbe disease and marks a major step forward in building out the company’s hybrid model as a gene therapy manufacturing and development engine.
FDA Clears IND Application for Passage Bio’s Gene Therapy Candidate PBKR03 for Treatment of Patients with Early Infantile Krabbe Disease, A Rare Pediatric Disorder with No Approved Disease-Modifying Treatment Options
Published: February 8, 2021
PHILADELPHIA, Feb. 08, 2021 (GLOBE NEWSWIRE) -- Passage Bio, Inc. (Nasdaq: PASG), a genetic medicines company focused on developing transformative therapies for rare monogenic central nervous system (CNS) disorders, today announced that the U.S. Food and Drug Administration (FDA) has cleared an investigational new drug (IND) application for PBKR03, an adeno-associated virus (AAV)-delivery gene therapy being studied for the treatment of early infantile Krabbe disease (Globoid Cell Leukodystrophy). Currently, there are no approved disease-modifying therapies available for Krabbe disease, a rare lysosomal storage disease that most often presents early in a child’s life, resulting in rapid progressive damage to both the brain and peripheral nervous system and mortality by two years of age. Underscoring the urgent medical need in the patient population, the FDA has previously granted Passage Bio both Orphan Drug and Rare Pediatric Disease designations for PBKR03 for treatment in Krabbe disease.
Passage Bio’s PBKR03 Receives Orphan Drug and Rare Pediatric Disease Designations from FDA for Treatment of Krabbe Disease
Published: Oct 28, 2020
PHILADELPHIA, Oct. 28, 2020 (GLOBE NEWSWIRE) -- Passage Bio, Inc. (NASDAQ: PASG), a genetic medicines company focused on developing transformative therapies for rare, monogenic central nervous system disorders, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug and Rare Pediatric Disease (RPD) designations to PBKR03 for the treatment of Krabbe disease (Globoid Cell Leukodystrophy). Passage Bio expects to initiate a Phase 1/2 trial for PBKR03 in the first half of 2021. Krabbe disease is a rare and often life-threatening lysosomal storage disease that presents early in the patient’s life, resulting in progressive damage to both the brain and peripheral nervous system.
Passage Bio Announces Third Gene Therapy Development Program in Krabbe Disease and Supports Million Dreams Fundraising Gala
Published: MAY 30, 2019
PHILADELPHIA, May 30, 2019 (GLOBE NEWSWIRE) -- Passage Bio, a genetic medicines company developing AAV-delivered gene therapies for the treatment of rare monogenic central nervous system diseases, today announced that its third clinical trial program will be for infantile Krabbe disease, an inherited disease that causes progressive damage to the nervous system.