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Press Releases

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.

Link to full Release: https://investors.passagebio.com/news-releases/news-release-details/passage-bio-receives-european-commission-orphan-designation?fbclid=IwAR3HTidvHuvC3lPG2N1mzdYM0iRp9K5drcEYPehmRJsP0BPOJ-a-IX2fL_4

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.

Link to full Release: https://investors.passagebio.com/news-releases/news-release-details/us-food-and-drug-administration-grants-fast-track-designation

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.

Link to full Release: https://www.forgebiologics.com/2021/02/16/press-fda-fast-track-odd-and-rpdd-designations-for-fbx-101-gene-therapy-for-patients-with-krabbe-disease/?fbclid=IwAR1pOwLTsjDU4AnbUrJ8W-oPVg5KRDYnEogOjYbrnXV0a0FzMB0aT70dYs0

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.

Link to full Release: https://investors.passagebio.com/news-releases/news-release-details/fda-clears-ind-application-passage-bios-gene-therapy-candidate?fbclid=IwAR0C1qP1dEFrw0wGwPbPat74V5dAHRlW0HSex5GC_6NWGTYt6JjxlmmBiII

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.

Link to full Release: https://www.biospace.com/article/releases/passage-bio-s-pbkr03-receives-orphan-drug-and-rare-pediatric-disease-designations-from-fda-for-treatment-of-krabbe-disease/

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. The company also announced its support of the Million Dreams Fundraising Gala being held tomorrow, May 31st in Minneapolis, MN.

Link to full Release: https://www.passagebio.com/investors-and-media/news-and-events/press-releases/press-release-details/2019/Passage-Bio-Announces-Third-Gene-Therapy-Development-Program-in-Krabbe-Disease-and-Supports-Million-Dreams-Fundraising-Gala/default.aspx

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. The company also announced its support of the Million Dreams Fundraising Gala being held tomorrow, May 31st in Minneapolis, MN.

Link to full Release: https://www.passagebio.com/investors-and-media/news-and-events/press-releases/press-release-details/2019/Passage-Bio-Announces-Third-Gene-Therapy-Development-Program-in-Krabbe-Disease-and-Supports-Million-Dreams-Fundraising-Gala/default.aspx

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Currently Krabbe disease is not automatically screened for in the state of Pennsylvania. Parents must request or “Opt-In” to have Krabbe added to the newborn screening tests done at birth.

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