Shanghai BDgene Technology Co., Ltd, a specialist in gene therapy, has announced that its drug candidate BD112, which utilizes virus-like particle (VLP) delivery technology for in vivo gene editing, has received orphan drug designation (ODD) from the US Food and Drug Administration (FDA). This follows a previous ODD granted by the European Commission for the treatment of Huntington’s disease (HD).
Understanding Huntington’s Disease (HD)
HD is an autosomal dominant genetic disorder that can result in the death of brain cells. The disease typically presents with mild emotional or intellectual issues initially, progressing to uncoordinated and unstable gait, and eventually leading to pronounced physical movement disharmony. As HD advances, patients experience a gradual deterioration in their ability to move and speak, with mental abilities often declining into dementia.
Current Landscape and Impact of HD
Huntington’s disease is currently incurable, and patients at the end stage require full-time care. The disease affects various ethnic groups, with the highest incidence found in Caucasian populations at a prevalence rate of 10.6-13.7 per 100,000. In China, the incidence rate of HD is reported to be 4.1 per million people. Statistics indicate that patients with HD face a significant risk of death 15-20 years after diagnosis.
Significance of Orphan Drug Designation
The orphan drug designation for BD112 highlights the potential impact of this gene therapy in addressing the unmet medical needs of HD patients. The designation can provide incentives for the development of drugs targeting rare diseases, such as HD, and may accelerate the path to market approval.
Conclusion
Shanghai BDgene’s achievement of obtaining FDA orphan drug designation for BD112 is a significant step forward in the development of gene therapy for Huntington’s disease. It underscores the company’s commitment to bringing innovative treatments to patients suffering from this debilitating genetic disorder.-Fineline Info & Tech
