{"id":11836,"date":"2024-11-08T15:03:38","date_gmt":"2024-11-08T07:03:38","guid":{"rendered":"https:\/\/flcube.com\/?p=11836"},"modified":"2024-11-08T15:03:38","modified_gmt":"2024-11-08T07:03:38","slug":"askbios-ab-1003-receives-rare-pediatric-disease-and-orphan-drug-designations-from-fda","status":"publish","type":"post","link":"https:\/\/flcube.com\/?p=11836","title":{"rendered":"AskBio’s AB-1003 Receives Rare Pediatric Disease and Orphan Drug Designations from FDA"},"content":{"rendered":"\n

Asklepios BioPharmaceutical, Inc. (AskBio), a wholly owned subsidiary of Bayer AG (ETR: BAYN<\/a>), has announced that it has received both rare pediatric disease designation (RPDD) and orphan-drug designation (ODD) from the US Food and Drug Administration (FDA) for its investigational gene therapy, AB-1003 (LION-101). The therapy targets limb-girdle muscular dystrophy type 2I\/R9 (LGMD2I\/R9), a rare and severe genetic disorder.<\/p>\n\n\n\n

AB-1003’s Mechanism and FDA Recognition<\/strong>
AB-1003 is an investigational AAV gene therapy that delivers the non-mutated FKRP gene into the body through a single intravenous infusion, aiming to treat the LGMD2I\/R9 disease. This approach has been recognized by the FDA with both RPDD and ODD, highlighting the therapy’s potential to address an unmet medical need for a pediatric population with a rare disease. The drug has also been previously awarded fast-track status in the US, underscoring its significance in expediting the development and review process.<\/p>\n\n\n\n

Phase I\/II Study and Assessment<\/strong>
AB-1003 is currently being assessed in a Phase I\/II study in the US, focusing on safety, tolerability, and efficacy. The study’s results will be crucial in determining the therapy’s potential role in improving outcomes for patients with LGMD2I\/R9, a condition that currently lacks effective treatment options.-Fineline Info & Tech<\/a><\/p>\n\n\n\n

<\/p>\n","protected":false},"excerpt":{"rendered":"

Asklepios BioPharmaceutical, Inc. 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