Spark-101: Being developed for Leber congenital amaurosis (LCA) type 10.
Brief information about it:
Spark-101, also known as voretigene nepavehvec-rzyl, is a gene therapy being developed for the treatment of Leber congenital amaurosis (LCA) type 10, a rare genetic disorder that causes vision loss from birth.
Uses:
Leber congenital amaurosis (LCA) type 10: Spark-101 is designed to address the underlying genetic defect in LCA type 10, which leads to the loss of photoreceptor cells in the retina.
Mechanism of action:
Spark-101 is a viral vector that delivers a corrected version of the RPE65 gene to retinal cells. This corrected gene has the potential to restore vision in patients with LCA type 10.
Elimination half life:
The elimination half-life of Spark-101 can vary depending on individual factors. It is generally excreted from the body over a period of weeks or months.
Route of Administration:
Spark-101 is administered as a one-time subretinal injection. This involves surgically injecting the therapy directly into the retina of the eye.
Side effects:
Spark-101 can cause side effects, including:
- Eye inflammation
- Increased eye pressure
- Floaters
- Vision changes
- Infections
Dose:
The recommended dose of Spark-101 is determined based on the patient’s age and other factors.
Precautions:
· Consult a doctor: Before receiving Spark-101, consult with a healthcare professional to ensure it is safe for you and to discuss potential risks and benefits.
· Medical history: Inform your doctor about any personal or family history of conditions such as eye diseases or infections.
· Eye exams: Regular eye exams are important to monitor the effects of Spark-101 and to detect any potential side effects.
