Friday, September 26, 2025. Week 39.

 
In #S10e182 I told you about CAMP4, don’t miss that, watch here: https://www.youtube.com/watch?v=PZ0Oj-Zz-B0 Sharing research comments from William Blair & Wedbush
 
William Blair Initiation of Coverage: “Among several quality investors, the private placement included the Syngap Research Fund, which is active in the Syngap1 patient community and will be an important resource in aiding patient identification and enrollment in the Phase 1/2 trial in our view.”
 
Wedbush
Ph1/2 Will Likely Begin From Ex-U.S., Aiming for Early Intervention. Citing precedents of other intrathecally delivered antisense oligonucleotide programs for CNS indications, where the FDA oftentimes required sponsors to begin at a dose level well below the efficacious dose, CAMP plans to begin patient dosing outside the U.S. for the potential to go directly to doses that are expected to show efficacy. The selection of patient age range will depend on regulatory discussions, and management highlighted the impact of the disease on neurodevelopment, so early intervention could allow patients to have a better opportunity to achieve as normal as possible development.
CAMP Will Have Access to Natural History Data being Collected by SRF and CHOP. According to management, a natural history study is being conducted by SynGAP Research Fund (SRF), which also participated in the private placement, in collaboration with Children's Hospital of Philadelphia (CHOP), and CAMP will have access to data as well as patients for future clinical study enrollment.
Donate now: https://curesyngap1.org/donate/
Beacon of Hope was a great success, raised over $100k.  We need to do this every year.  Thanks to Navarros for getting this launched, also to SJ, Emily Barnes, Peter Halliburton, & Kathryn Helde who helped make this event incredible.
Emmy’s video (top of) https://curesyngap1.org/resources/movies/
Blog: https://cureSYNGAP1.org/Beacon25 (will be live Friday night 9/26)
Pairs well with Gala Blog: https://curesyngap1.org/Gala25 
Research is non-stop:
 - CRID, get one. https://curesyngap1.org/blog/every-syngap1-related-disorders-patient-needs-a-crid/
 - ProMMiS, incredible coordination meeting today.  Sign up. https://curesyngap1.org/resources/studies/syngap1-ProMMiS/
 - Sign up for Citizen Health too!  AI Advocate is live for us an awesome. https://www.citizen.health/partners/srf
 
Episode 183 of #Syngap10 #CureSYNGAP1
#Advocate #PatientAdvocacy #UnmetNeed #SYNGAP1 #SynGAP #SynGAProMMiS

Ver todos los episodios