Possible herpes keratitis cure takes another step: BDGene of China applies for Orphan Drug Designation at the FDA

Chinese company BDGene from Shanghai continues to lead in the global race for a herpes keratitis cure. The highly anticipated treatment, BD111 has reached a big milestone as it applied for Orphan Drug designation from the US FDA. Previously, BD111 has received international attention for its progress with a gene editing cure for herpes keratitis. We reported on its progress – which it has published in the top international academic journals Nature Biotechnology and Nature Biomedical Engineering.

BD Gene has (in collaboration with the Eye and ENT Hospital of Fudan University) carried out 3 cases of IIT human clinical trials, and has reported pre-clinical results where study subjects were cleared of the virus. The potential to change the landscape for Herpes treatment has patient advocates very excited.

Orphan drugs, also known as rare disease drugs, refer to drugs used for the prevention, treatment and diagnosis of rare diseases. The orphan drug designation granted by the FDA applies to drugs and biologics for rare diseases that affect less than 200,000 people in the United States each year, and provides policy support for related products. Therefore, obtaining orphan drug designation is of great advantage for manufacturers bringing new medicines to market.

Possible herpes cure takes another important step

According to the U.S. FDA Orphan Drug Act, new drugs that have obtained orphan drug qualification allows for a series of advantages for BDGene including quicker, easier processes to get to market in the US. Supports include tax credit for clinical research expenses, Exemption of NDA/BLA application fees, access to special R&D funds, special approval channels, exemption from the declaration of some clinical data, and a seven-year market exclusivity period after the drug is approved. Orphan Drug Designation can also bring investment as the company continues to lead the market.

About Herpes Keratitis: Herpes virus keratitis is caused by herpes simplex virus (HSV-1) infection and is the most common infectious blindness disease. Current first-line antiviral drugs can only inhibit viral replication by interfering with viral DNA synthesis. These drugs can inhibit HSV-1 DNA replication, but cannot clear the latent viral genome in the cornea and trigeminal ganglion, which leads to the disease. Repeated attacks can lead to blindness in severe cases.

About BD111: CRISPR-based gene editing technology can directly degrade the viral genome, providing the possibility of fundamentally curing the disease. BD111 gene editing drugs get rid of the drawbacks of traditional related drugs that need to be repeatedly administered, and only need to be injected once. The drug uses the original delivery technology of VLP to transduce the CRISPR gene editing tool to directly target and cut the HSV-1 genome, so as to achieve the purpose of removing the HSV-1 virus genome, thereby realizing the treatment of herpes virus keratitis. The characteristics of the BD111 drug are: (1) Cas9 mRNA is delivered, and the gene enzyme stays in the body for a short time, which can reduce the risk of immune response and gene editing off-target; (2) It cuts the viral genome and does not need to change anyone’s genes, not detected to off-target effects on the human genome.

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