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 cleared an investigational new drug (IND) application for PBKR03, an adeno-associated virus (AAV)-delivery 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 ... being studied for the treatment of early infantile Krabbe disease (Globoid Cell LeukodystrophyAnother name for Krabbe disease, a rare and usually fatal disorder of the nervous system.). Currently, there are no approved disease-modifying therapies available for Krabbe disease, a rare lysosomal storage diseaseLysosomal storage disorders comprise a group of ~50 metabolic disorders that result from a missing, deficient or lack of a specific enzyme. that most often presents early in a child’s life, resulting in rapid progressive damage to both the brain and peripheral nervous systemThe nervous system of the body is made up of the Central Nervous System (CNS) and the Peripheral Nervous System (PNS). 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.

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