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Potential FSHD Therapy GBC0905 Given Orphan Drug Status by FDA

Genea Biocells announced that the FDA has granted orphan drug status to GBC0905, a small molecule that may become the first targeted therapy for both type 1 and type facioscapulohumeral muscular dystrophy (FSHD).

Genea Biocells Announces FDA Orphan Drug Designation for GBC0905 for ...

SAN DIEGO-- (BUSINESS WIRE)-- Genea Biocells, a preclinical-stage company focused on drug development to treat neuromuscular diseases, today announced that it has been granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) for its therapeutic candidate, GBC0905, for the treatment of facioscapulohumeral muscular dystrophy (FSHD). This small molecule agent potently ...

FDA Grants Orphan Drug Designation to GBC0905 for FSHD

The US FDA has granted Genea Biocells Orphan Drug Designation to its product, GBC0905, for the treatment of facioscapulohumeral muscular dystrophy (FSHD).

FDA Grants Orphan Drug Designation to Treatment for Facioscapulohumeral ...

Genea Biocells has announced that the FDA has granted GBC0905, for the treatment of facioscapulohumeral muscular dystrophy (FSHD), an Orphan Drug Designation. FSHD, which affects between 4 and 10 ...

MYOCEA - Skeletal Muscle Experts

Myocea's GBC0905: Efficacy Recaptured in FSHD Primary Patient Biopsy Myotubes in a Dose-Dependent Fashion Normal Myogenesis is Not Affected GBC0905 reduces clinical DUX4 and H3.X/Y stress markers, does not exhibit a toxic or myogenic inhibitory effect. DUX4 and H3.X/Y are highly expressed in primary patient biopsy myotubes from FSHD1 patients.

GBC 0905 - AdisInsight

GBC 0905, a first-in-class small molecule, is being developed by Genea Biocells for the treatment of facioscapulohumeral muscular dystrophy (FSHD) (type 1 and

GBC-0905 - Drug Targets, Indications, Patents - Synapse

GBC-0905: a DUX4 inhibitors Drug, Initially developed by Genea Biocells Pty Ltd., Now, its global highest R&D status is Pending, Mechanism: DUX4 inhibitors (Double homeobox protein 4 inhibitors), Therapeutic Areas: Nervous System Diseases,Congenital Disorders,Skin and Musculoskeletal Diseases,Other Diseases.

New biotech is developing Genea Biocells' FSHD drug

By Alex Kiselyov, CEO, Myocea, Inc. Myocea is a biotechnology startup with a focus on neuromuscular disorders. Our product pipeline is championed by the small molecule GBC0905 (Rebastinib) aimed at the treatment of FSHD Type 1 and Type 2. Myocea was spun out of San Diego-based Genea Biocells in 2019 after its parent company Genea

Genea Biocells Announces FDA Orphan Drug Designation for GBC0905 for ...

SAN DIEGO-- (BUSINESS WIRE)--Genea Biocells, a preclinical-stage company focused on drug development to treat neuromuscular diseases, today announced that it has been granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) for its therapeutic candidate, GBC0905, for the treatment of facioscapulohumeral muscular dystrophy (FSHD). This small molecule agent potently ...

Treatment for Facioscapulohumeral Muscular Dystrophy Granted Orphan ...

Officials with the FDA have granted GBC0905, for the treatment of facioscapulohumeral muscular dystrophy (FSHD), an Orphan Drug Designation, Genea Biocells announced in a press release.

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