Benitogene's BD112 In Vivo Gene Editing Therapy Receives Orphan Drug Designation in Both the U.S. and EU for Huntington’s Disease

Shanghai Bendao Gene Technology Co., Ltd. (hereinafter referred to as "Bendao Gene"),BD112, an in vivo gene editing therapy, received Orphan Drug Designation from the U.S. FDA on October 6, 2023, for the proposed treatment of Huntington's disease.

Prior to receiving this approval, BD112 had already obtained Orphan Drug Designation (ODD) approval from the European Commission (EC) on August 17, 2023. This latest approval signifies that Bendao Gene’s BD112 has now secured ODD recognition in both the European and U.S. markets, marking another significant milestone for Bendao Gene.

Huntington's Disease (HD) is an autosomal dominant genetic disorder that leads to the death of brain cells. Early symptoms often include mild emotional or cognitive issues, followed by incoordination and an unsteady gait. As the disease progresses, motor incoordination becomes more pronounced, with capabilities gradually deteriorating until movement becomes difficult and speech is lost. Cognitive abilities typically decline into dementia. Specific symptoms vary among patients. Symptoms usually appear between the ages of 30 and 50, but can onset at any age. In families carrying the chromosomal abnormality associated with HD, members may develop the disease at a younger age. Approximately 8% of cases onset before the age of 20, with typical symptoms resembling those of Parkinson's disease. Patients with HD often underestimate the severity of their condition.

Huntington's disease is currently incurable. As the disease progresses to its terminal stage, patients require round-the-clock care. Huntington's disease has been reported across all ethnic groups, with the highest prevalence among Caucasians, at a rate of 10.6–13.7 per 100,000 individuals. In China, the incidence of HD is 4.1 per million people. Although the incidence of HD in China is lower than that in Western countries, the large population base means that a significant number of people are affected by this disease. The occurrence rate is similar between males and females. This condition can lead to complications such as pneumonia and heart disease, and physical injuries from falls, all of which reduce patients' life expectancy. Statistics show that mortality is highest among HD patients within 15 to 20 years after diagnosis.

Orphan Drug Designation (ODD) is a status granted by the FDA’s Office of Orphan Products Development (OOPD) to eligible drugs (including biologics) intended for the prevention, treatment, and diagnosis of rare diseases. The FDA has clearly defined criteria for rare diseases, namely those affecting fewer than 200,000 people in the United States.

An investigational drug (including biologics) can demonstrate its potential in treating, preventing, or diagnosing a rare disease in an Orphan Drug Designation (ODD) application. Upon review of the application materials, the Office of Orphan Products Development (OOPD) will grant ODD to the drug. Generally, clinical study data, animal model (in vivo) data, and in vitro study results may all serve as scientific evidence to demonstrate the potential efficacy of an investigational new drug. Therefore, obtaining orphan drug designation is of significant importance for new drug development.

Orphan Medicinal Products are used for the diagnosis, prevention, or treatment of life-threatening or very serious conditions affecting no more than 5 in 10,000 people in the European Union.The EU Orphan Medicinal Products Regulation establishes a centralized procedure for orphan drug designation and sets forth incentives for the research, development, and marketing authorization of orphan drugs, including but not limited to: (1) access to assistance with clinical trial protocols (the EMA provides dedicated scientific advice for designated orphan drugs); (2) the centralized marketing authorization procedure (companies may submit applications for marketing authorization or conditional approval directly to the EMA, with the resulting European Commission recommendations or decisions valid across all EU member states); (3) a 10-year market exclusivity period post-approval; and (4) fee reductions for regulatory activities (covering protocol assistance, marketing authorization applications and inspections, post-authorization variation applications, and annual fees).

Bendao Gene is an innovative gene therapy drug development company specializing in in vivo gene editing therapies. It is dedicated to developing globally significant innovative drugs for refractory diseases across multiple fields, including ophthalmology, the nervous system, the hematopoietic system, viral infections, and oncology. Bendao Gene possesses the internationally leading VLP mRNA delivery platform (BDmRNA) and the next-generation lentiviral vector platform (BDlenti). Centered on its core delivery technology platforms, the company has established multiple first-in-class product pipelines, conducted numerous first-in-human clinical studies, and engaged in collaborative R&D with renowned international pharmaceutical companies to develop novel therapies for major neurological disorders. Bendao Gene pioneered clinical research on in vivo gene editing for antiviral therapy globally and has achieved significant clinical breakthroughs in gene therapy for macular degeneration and thalassemia. The company’s core technologies have been published in prestigious journals such as Nature Biotechnology and Nature Biomedical Engineering. Its CRISPR-based antiviral therapy, HELP, was successfully selected as one of the “Top Ten Advances in Chinese Ophthalmology” in 2021. The company operates a nearly 1,000-square-meter pilot-scale R&D center and a 4,000-square-meter GMP pilot production base. Additionally, it has jointly established the “Joint R&D Center for Gene Therapy” with the Institute of Systems Biomedicine at Shanghai Jiao Tong University.