Neurogenetic Diseases Treatment Platform: ASO-Based Treatments

ASO-Based Treatments

Antisense oligonucleotides (ASOs) are short, synthetic, single-stranded oligodeoxynucleotides that can modify RNA and reduce, restore, or alter protein production through several different mechanisms. In other words, they are short synthetic DNA fragments.

Treatment Efficacy

Since ASO-based treatments target the fundamental origin of the disease, they are more effective than targeting disease-causing pathways. Dramatic developments have occurred in this technology in recent years.

SMA Example

ASO-based treatment for spinal muscular atrofin, known as SMA disease among the public, has been approved by the FDA and the course of SMA disease has changed within a few years. Children who were lost before completing their first year of life before this treatment can now walk and survive for many more years.

Future Goals

This technology is expected to change the course of many neurological diseases. Neurological and Genetic Diseases Association, Istanbul University, and Cerrahpaşa Genetic Scientists are developing a treatment based on ASO technology for patients with Rett and Dravet Syndrome.

Targeted Treatments

With this treatment, we aim to:

• Increase BDNF protein in the brains of patients with Rett Syndrome
• Increase sodium channels in the neurons of patients with Dravet Syndrome

Study Process

First Stage

The design of the ASO molecule and testing it in healthy neuronal cell culture is the first phase of the study, which began in September 2022.

Timeline

• The design phase lasting 6-9 months is planned to be followed by animal experiments
• This trial is also expected to be completed in the first half of 2024
• Our goal is to start clinical trials with Turkish patients in 2024

Platform Goals

In this way, in addition to developing treatment for patients with Rett and Dravet Syndrome, we hope to establish a treatment platform for many genetic diseases that cause autism, epilepsy, and developmental delay in childhood.

Mission

We aim to provide personalized treatments for all our patients who have sufficient funding, no matter how rare they are.

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