Imagine your child never speaking a word, never taking their first step, completely dependent on lifelong care. This is the reality for children born with Angelman syndrome, a rare genetic disorder that profoundly affects the nervous system.

We are Asgent – a Czech nonprofit organization founded by the parents of Oliver, a boy diagnosed with Angelman syndrome. We've decided to take our destiny into our own hands. In collaboration with the Czech Centre for Phenogenomics, led by Associate Professor Radislav Sedláček, we're committed to developing a groundbreaking gene therapy that could change lives globally.

Thanks to your support, we have made incredible progress over the past 12 months:

✅ Nearly 23 million CZK raised from public donations for preclinical research. 
✅ Successfully developed a functional mouse model for treatment testing. 
✅ Established a clear therapeutic approach for Angelman syndrome with promising results.

Our next crucial steps include:
- Securing effective project management and coordination.

- Ensuring patent protection and maintaining independence from commercial interests.
- Initiating clinical trials within the next two to three years.

Research led by docent Sedláček has made significant progress, successfully developing a therapeutic approach. This advancement greatly increases the chance of creating a treatment that could significantly change the lives of thousands of children affected by this rare genetic disorder. Unlike large pharmaceutical companies often burdened with costly administration and profit demands, our nonprofit model ensures that the developed therapy will be more affordable and accessible to everyone in need. An additional advantage is that Czech patients could gain priority access to treatment during clinical trials.

We need your help to continue. Every contribution brings us closer to our dream—a therapy accessible to every family in need.

You can learn more about Association of Gene Therapy on our official website: www.asgent.org
FB       @Asociace genové terapie, z.s.
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