Our strategy has always been to derisk SYNGAP1 so that industry will invest and bring their skill and capital to bear. We leverage donor dollars to maximize impact. Yesterday was a great example:
We submitted our first regulatory filing for CMP-002 in Australia which positions us to initiate a global first-in-human Phase 1/2 clinical trial in the second half of 2026.
Lead optimization is underway to identify a clinical candidate for the treatment of SYNGAP1 in 2026. SYNGAP1 is a severe and rare genetic neurodevelopmental disease.
Praxis remains on track to nominate a development candidate for each of its three early stage ASO therapeutic initiatives in the first half of 2026: PRAX-090 is designed to address SYNGAP1 loss-of-function (LoF) mutations, a leading cause of severe intellectual disability and epilepsy in DEEs.
Watch and share the new SYNGAP1 Video, it is excellent and helps explain our cause to families and friends. We need to turn them into supporters and donors.
Podden och tillhörande omslagsbild på den här sidan tillhör
Syngap Research Fund, 501(c)(3). Innehållet i podden är skapat av Syngap Research Fund, 501(c)(3) och inte av,
eller tillsammans med, Poddtoppen.