KrabbeConnect Secures Generous Funding from the Rosenau Family Research Foundation for Groundbreaking Krabbe Disease Study
Minneapolis, MN, November 30th, 2023 — KrabbeConnect, a pioneering organization dedicated to supporting individuals and families affected by Krabbe disease, is proud to announce the receipt of an important grant from the Rosenau Family Research Foundation (RFRF). This grant, titled…
Forge Biologics Reports Positive FBX-101 Clinical Updates for Patients with Krabbe Disease at WorldSymposium
COLUMBUS, OHIO – Forge Biologics, a genetic medicines development and manufacturing organization, announced today that Maria Escolar, M.D., Chief Medical Officer, will present updated data from the RESKUE Phase 1/2 clinical trialA clinical trial is research designed to understand the safety and efficacy of a drug, biologic or device. There are 4 phases to most clinical trials from Phase 1 that seeks to answer safety concern… for FBX-101, the Company’s novel AAV gene therapy…
Passage Bio Announces Closure of Krabbe Disease Program
Our Krabbe disease community received some disappointing news today. PassageBio will stop further clinical development of PBKR03 for Krabbe disease. “There’s no doubt this news is upsetting but we won’t allow it to disrupt our momentum in achieving advances in…
Continue Reading Passage Bio Announces Closure of Krabbe Disease Program
Forge Biologics Announces Updated Positive Clinical Data in RESKUE, a Novel Phase 1/2 Gene Therapy Trial for Patients with Krabbe Disease
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 trialA clinical trial is research designed to understand the safety and efficacy of a drug, biologic or device. There are 4 phases to most clinical trials from Phase 1 that seeks to answer safety concern… for FBX-101-the Company’s novel gene therapy…
Passage Bio Receives European Commission Orphan Designation for PBKR03 for Treatment of Krabbe Disease
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 systemThe central nervous system (CNS) is made up of the brain and the spinal cord. (CNS) disorders, today announced that the European Commission has granted Orphan designation…
U.S. Food and Drug Administration Grants Fast Track Designation to Three Passage Bio Gene Therapy Candidates Targeting Rare CNS Disorders
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 systemThe central nervous system (CNS) is made up of the brain and the spinal cord. (CNS) disorders, today announced the U.S. Food and Drug Administration (FDA) has granted…
FDA Fast Track, ODD, and RPDD Designations for FBX-101 Gene Therapy for Patients with Krabbe Disease
COLUMBUS, OHIO, February 16, 2021 – Forge Biologics Inc., a fully integrated clinical stage gene therapyA type of therapy that offers hope and promise for a cure for many genetic disorders. A working copy of the gene replaces the non-working copy of the gene. Gene therapy is at the forefront of many … 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…
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
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 systemThe central nervous system (CNS) is made up of the brain and the spinal cord. (CNS) disorders, today announced that the U.S. Food and Drug Administration (FDA) has…
Passage Bio’s PBKR03 Receives Orphan Drug and Rare Pediatric Disease Designations from FDA for Treatment of Krabbe Disease
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 systemThe central nervous system (CNS) is made up of the brain and the spinal cord. disorders, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug and Rare…
Passage Bio Announces Third Gene Therapy Development Program in Krabbe Disease and Supports Million Dreams Fundraising Gala
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 systemThe central nervous system (CNS) is made up of the brain and the spinal cord. diseases, today announced that its third clinical trialA clinical trial is research designed to understand the safety and efficacy of a drug, biologic or device. There are 4 phases to most clinical trials from Phase 1 that seeks to answer safety concern… program will be for infantile Krabbe…