Bloomsbury Genetic Therapies, a clinical-stage biotechnology company developing potentially curative treatments for patients suffering from rare neurological and metabolic diseases based on clinically proven gene therapy technologies, has announced that both the United States Food and Drug Administration (FDA) and the European Commission (EC) have granted Orphan Drug Designation (ODD) for BGT-DTDS, the company’s neuron-targeted AAV2 investigational gene therapy for the treatment of Dopamine Transporter Deficiency Syndrome (DTDS). BGT-DTDS has completed preclinical efficacy studies and preparations for a first-in-human clinical trial are ongoing.
“These Orphan Drug Designations recognise the significant unmet need in patients in the United States and the European Union living with DTDS; a devastating disease with poor prognosis. We look forward to continuing to investigate BGT-DTDS and to leveraging the benefits that ODD brings, including significant developmental benefits and the provision of post-approval market exclusivity.” Adrien Lemoine, Co-Founder & Chief Executive Officer of Bloomsbury.
“These Orphan Drug Designations recognise the significant unmet need in patients in the United States and the European Union living with DTDS; a devastating disease with poor prognosis. We look forward to continuing to investigate BGT-DTDS and to leveraging the benefits that ODD brings, including significant developmental benefits and the provision of post-approval market exclusivity.”
Read the full story from Blooomsbury GTx.
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