Andelyn Biosciences, Inc : Andelyn Biosciences and Armatus Bio Partner to Manufacture Suspension-Based AAV Gene Therapy for Rare Neurological Disease Charcot-Marie-Tooth Type 1A (CMT1A)
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HUIADGENE ANNOUNCES RARE PEDIATRIC DRUG DESIGNATION GRANTED TO HG302, A NOVEL CRISPR DNA-EDITING THERAPY, FOR THE TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY
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IPS HEART Receives U S FDA Rare Pediatric Drug Designation for ISX9-CPC Stem Cell Therapy for Treatment of Cardiomyopathy Associated with Danon disease
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Marks third Rare Pediatric Drug Designation granted by FDA for pipeline candidateHOUSTON (BUSINESS WIRE) IPS HEART, a privately held cell therapy company advancing its stem cell platform to develop new skeletal muscle and cardiac muscle generation treatments for Duchenne muscular dystrophy (a rare disease) and hea.