For the first time, researchers identify changes in brains of Krabbe disease infants that are similar to changes seen in dementia with Lewy bodies and Parkinson’s Disease, raising the possibility of future treatments.
Passage Bio (PASG) Receives European Commission Orphan Designation for PBKR03 for Treatment of Krabbe Disease streetinsider.com - get the latest breaking news, showbiz & celebrity photos, sport news & rumours, viral videos and top stories from streetinsider.com Daily Mail and Mail on Sunday newspapers.
- Urgency for effective treatment underscored by European Commission designation for investigational gene therapy PBKR03
- Global Phase 1/2 trial – GALax-C – PBKR03 planned to initiate in first half of 2021
PHILADELPHIA, April 05, 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)
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PARAMUS, N.J., Feb. 10, 2021 /PRNewswire/ Polaryx Therapeutics, Inc., a biotech company developing small molecule therapeutics for lysosomal storage disorders, announced today that it has received both Rare Pediatric Disease and Orphan Drug Designations for the treatment of Krabbe Disease (a.k.a. Globoid Cell Leukodystrophy) with PLX-300 from the U.S. Food and Drug Administration (FDA).
Krabbe disease (KD) is a rare, heritable metabolic genetic disorder that affects approximately one in 100,000 individuals in the United States. KD is caused by deficiency of the lysosomal enzyme, galactocerebrosidase (GALC). The function of GALC is to catabolize the cytotoxic lipid, galactosylsphingosine (also known as psychosine). When either the level of GALC or its activity is compromised, galactosylsphingosine accumulates in the nervous system, ultimately leading to neurodegeneration and demyelination in patients. KD is a devastating neurological di
Press release content from Globe Newswire. The AP news staff was not involved in its creation.
FDA Clears IND Application for Passage Bio’s Gene Therapy Candidate PBKR03 for Treatment of .
Passage BioFebruary 8, 2021 GMT
- Phase 1/2 trial expected to commence in first half of 2021
- Company has three INDs cleared for rare monogenic CNS disorders
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