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Over 1,600 families are caring for a loved one with the rare disease ”SynGAP” resulting from a variant of the SYNGAP1 gene. This weekly podcast is for them. -A quick summary of the latest news in the space. The host is Mike Graglia, co-founder & managing director of CURE SYNGAP1. CURE SYNGAP1 is a parent-led public charity in the US that strives to accelerate research into treatments for SYNGAP1 so that we can help our loved ones in a timeframe that matters. Learn more at https://cureSYNGAP1.org
Episodes
Monday Dec 01, 2025
Monday Dec 01, 2025
#CyberMonday, December 1, 2025. Week 49.
Go buy The Monster Inside My Brother by Nicole Ciccone illustrated by Lena Bardy.
Barnes and Noble: https://www.barnesandnoble.com/w/the-monster-inside-my-brother-nicole-m-ciccone/1148368158
Amazon: https://a.co/d/imfxmwG
See you Thursday at the CURE SYNGAP1 Conference 2025 Atlanta: https://curesyngap1.org/events/conferences/cure-syngap1-conference-2025-hosted-by-srf/
SOCIAL MATTERS
4,474 LinkedIn. https://www.linkedin.com/company/curesyngap1/
1,480 YouTube. https://www.youtube.com/@CureSYNGAP1
11.2k Twitter https://twitter.com/cureSYNGAP1
45k Insta https://www.instagram.com/curesyngap1/
$CAMP stock is at $3.80 on 26 Nov. ‘25 https://www.google.com/finance/beta/quote/CAMP:NASDAQ
Episode 191 of #Syngap10 #CureSYNGAP1
Thursday Nov 27, 2025
Thursday Nov 27, 2025
Happy Thanksgiving… Thursday, November 27, 2025. Week 48.
Continued from #S10e189…
And the AAV Paper (https://pubmed.ncbi.nlm.nih.gov/40988338/) from #S10e187… https://curesyngap1.org/podcasts/syngap10/clinical-research-ai-dx-nl47-survey-autism-press-6-days-to-register-for-syngap1conf-s10e187/
Issac’s story, Transmitter reprint, Scramble 4 write up and JK on #Autism, #MustRead
https://curesyngap1.org/resources/webinars/ 119 - 112
Register for livestream of the conference, AAV from Allen Inst., dos en espanol, Missense, Unlock and Rare-X for ProMMiS.
https://curesyngap1.org/podcasts/syngap1-stories/
A gold mine have you listened to #38, the Virginie Pod, really must listen, she is our leader. https://www.linkedin.com/posts/graglia_syngap1stories-syngap1-syngap1storiesty-activity-7387203351907708928-liNL
CLINICAL TRIAL & GENETIC MEDICINE CORNER
Example of Ultragenyx FAST Angelman follow on trial to look at other ages and genotypes, key message, never give up. https://www.linkedin.com/posts/cureangelman_the-global-aurora-study-will-enroll-approximately-activity-7389647402690957312-Bihi
Congrats to Novartis on approval of the first Gene Therapy to Cure SMA!
https://www.linkedin.com/posts/graglia_sma-fdaapproval-rarediseaseinnovation-activity-7398939783005347840-Ocd_ Remember Spinraza was approved in December 2019.
TODOS
- Sign up for Citizen Health: https://www.citizen.health/partners/srf
- USE YOUR ICD-10 F78.A1 #S10e185 https://www.youtube.com/watch?v=dale0NbxDpU
- Go to CURE SYNGAP1 Conference 2025 Atlanta: https://curesyngap1.org/events/conferences/cure-syngap1-conference-2025-hosted-by-srf/
SOCIAL MATTERS
4,468 LinkedIn. https://www.linkedin.com/company/curesyngap1/
1,480 YouTube. https://www.youtube.com/@CureSYNGAP1
11.2k Twitter https://twitter.com/cureSYNGAP1
45k Insta https://www.instagram.com/curesyngap1/
$CAMP stock is at $3.62 on 26 Nov. ‘25 https://www.google.com/finance/beta/quote/CAMP:NASDAQ
Episode 190 of #Syngap10 #CureSYNGAP1
Thursday Nov 27, 2025
Thursday Nov 27, 2025
Happy Thanksgiving… Thursday, November 27, 2025. Week 48.
https://curesyngap1.org/clinical-trials/ Emerald from Praxis for Relutrigine and DeeP Ocean from Longboard for Bexicaserin. Check them out, screen for both. See what you can do!
Hectic with #MIHealthSummit, Tony at home, an NHS visit, Ultrabootcamp, not to mention prepping for the most elaborate conference yet!
ProMMiS - Meet committed clinicians, get great care and build the future of SYNGAP1 clinical medicine.
Upcoming Stanford enrollment dates: December 10–11, 2025: 2 slots, February 25–26, 2026: 2 slots, May 27–28, 2026: 2 slots to enroll email prommis@curesyngap1.org
GIVING TUESDAY
1 week to go. Set up an Unlock fundraising page now and send it out BEFORE Tuesday December 2nd! cureSyngap1.org/unlock or for more info: https://curesyngap1.org/podcasts/syngap10/time-to-unlock-their-tomorrow-raise-funds-camp4-our-name-s10e188/
Video: https://www.linkedin.com/posts/curesyngap1_curesyngap1-syngap1-activity-7399492685474009088-DXWI
CONFERENCE
- Record Headcount over 20% increase: Scientific: 237 & Caregiver Connect: 180 (of which 26 patients)
- Agendas are up: https://curesyngap1.org/calendar/cure-syngap1-conference-2025-hosted-by-srf/
- Volunteer with us: stacey@cureSYNGAP1.org
PAPERS
PUBMED at 52, and low. Clinical research! 181 / 359 = more than half of the knowledge on S1 in the past 4 year!
https://pubmed.ncbi.nlm.nih.gov/?term=syngap1&filter=years.2025-2025&sort=date
Movement disorder comparison: https://www.medrxiv.org/content/10.1101/2025.11.04.25339413v1.full.pdf
SLEEP - SRD Patients are the worst https://pubmed.ncbi.nlm.nih.gov/41138043/ aligned with CSH paper https://pmc.ncbi.nlm.nih.gov/articles/PMC8472329/
TODOS
- Sign up for Citizen Health: https://www.citizen.health/partners/srf
- USE YOUR ICD-10 F78.A1 #S10e185 https://www.youtube.com/watch?v=dale0NbxDpU
- Go to CURE SYNGAP1 Conference 2025 Atlanta: https://curesyngap1.org/events/conferences/cure-syngap1-conference-2025-hosted-by-srf/
SOCIAL MATTERS
4,468 LinkedIn. https://www.linkedin.com/company/curesyngap1/
1,480 YouTube. https://www.youtube.com/@CureSYNGAP1
11.2k Twitter https://twitter.com/cureSYNGAP1
45k Insta https://www.instagram.com/curesyngap1/
$CAMP stock is at $3.62 on 26 Nov. ‘25 https://www.google.com/finance/beta/quote/CAMP:NASDAQ
Episode 189 of #Syngap10 #CureSYNGAP1
Sunday Nov 09, 2025
Sunday Nov 09, 2025
Sunday, November 9, 2025. Week 46.
End of year campaign: UNLOCK THEIR TOMORROW, curesyngap1.org/unlock
Why now? UNLOCK It’s giving season. Count our blessings and share what we have. CS1 has moved mountains this year: Staff, ProMMiS, etc. But we are just starting…
Every single family should give and should solicit their friends. Yes, it’s a tough time. Yes, people have other causes. No (most of) your friends don’t have a SynGAPian.
Miss 100% of the shots you don’t take. And remember, donors learn about what they give to, so even small donations cause huge changes in awareness.
Go to our Champions page: https://curesyngap1.org/champions-of-hope/
Tony, Myla, Hattie, Jansen, Kai, Sophia, Kaylie, Gracie are at $50k+. Eight families. It’s only five at $100k+. I think we can do better. I also think we should have more participation at every level, we can’t lean on a handful of families to carry this organization.
These families get something key: CURE SYNGAP1 is in it for the long run, unlike EVERYONE ELSE you are supporting. Great that you are doing schools, horses, girl scouts, etc, but the only people asking you for money right now that are going to be fighting for your kid in 5, 10, or 50 years, is CURE SYNGAP1. The only group who won’t kick your family/loved one out of our community when your SynGAPian bites one of our staff or elopes from a meeting across streets. Also us. The people who understand SYNGAP1 the best. Right here.
Technicals, we could not make this easier!
Take 5 minutes to build your own fundraiser or simply email friends/family/co-workers/church associates/other kids’ sports team parents/etc. It’s uncomfortable but essential.
It’s so darn easy to give -
💻 ONLINE @ CURESYNGAP1.ORG/UNLOCK Use Credit Card, PayPal, Venmo, Google & Apple
📱 PHONE - Text UNLOCK to 71777, then follow the instructions.
📬 MAIL A CHECK CURE SYNGAP1, PO Box 515734 Los Angeles, CA 90051-5150
Two more questions:
?1: Aren’t we done now with CAMP4? No. Three reasons: Clock, Count, unknowns.
Clinical research. Behavioral interventions. Optimal medications. Missense research. Patient discovery. Drug repurposing. Family support. Global coordination.
?2: Our name is a demand: CURE SYNGAP1, and we are not there yet. SRF suggested that funding science was enough, boy were we wrong.
We need patients to be Support. Educate. Activate. Coordinate. I discussed the many ways a PAG matters in #S10e181. (I missed data & should have clinical separate, will redo.) https://curesyngap1.org/podcasts/syngap10/patient-advocacy-groups-matter-party-friday-syngap1conf-soon-elopement-s10e181/
Every single family needs to help us raise funds. Every single one.
SOCIALS
4,426 LinkedIn. https://www.linkedin.com/company/curesyngap1/
1,480 YouTube. https://www.youtube.com/@CureSYNGAP1
11k Twitter https://twitter.com/cureSYNGAP1
45k Insta https://www.instagram.com/curesyngap1/
$CAMP stock is at $4.25 on 7 Nov. ‘‘25 https://www.google.com/finance/beta/quote/CAMP:NASDAQ
Episode 188 of #Syngap10 #CureSYNGAP1
Saturday Oct 25, 2025
Saturday Oct 25, 2025
Saturday, October 25, 2025. Week 43.
Time to advocate, ELF on the Hill, support available, apply now: https://www.linkedin.com/posts/everylifeorg_were-excited-to-join-everyone-on-capitol-activity-7384625926333943808-mO1U/
PUBMED at 47, and low. Clinical research!
https://pubmed.ncbi.nlm.nih.gov/?term=syngap1&filter=years.2025-2025&sort=date
- CHOP EEG ProMMiS https://www.neurology.org/doi/10.1212/WNL.0000000000214148?url_ver=Z39.88-2003&rfr_id=ori:rid:crossref.org&rfr_dat=cr_pub%20%200pubmed
- COMMUNICATION #ORCA https://acamh.onlinelibrary.wiley.com/doi/full/10.1111/jcpp.70063
Doing surveys gets us into papers like ORCA, helps us raise awareness of SRD. This one on AI is really interesting: https://redcap.tch.harvard.edu/redcap_edc/surveys/?s=YFHYH7T7LTPAL44X
Newsletter #47
https://mailchi.mp/curesyngap1.org/unlock-their-tomorrow-issue47
IPM on SRD AAV https://www.insideprecisionmedicine.com/topics/precision-medicine/gene-therapy-reverses-syngap1-brain-disorder-symptoms-in-mice/
NYT Take on #Autism is very good, thank you Azeen Ghorayshi
Split the Autism Spectrum: https://www.nytimes.com/2025/10/01/health/autism-spectrum-neurodiversity-kennedy.html?unlocked_article_code=1.q08.NXEA.fg5ulHeTHUeJ&smid=url-share quotes Jackie K, explores argument for Profound & Severe Autism as a category.
Our own Jackie Kancir has a great substack, listen to it in her voice here: https://jkancir.substack.com/p/autism-is-not-my-daughter-nor-her
Sign up for Citizen Health:
https://www.citizen.health/partners/srf
CURE SYNGAP1 Conference 2025 Atlanta: https://curesyngap1.org/events/conferences/cure-syngap1-conference-2025-hosted-by-srf/
USE YOUR ICD-10 F78.A1 #S10e185 https://www.youtube.com/watch?v=dale0NbxDpU
SOCIALS
4,417 LinkedIn. https://www.linkedin.com/company/curesyngap1/
1,470 YouTube. https://www.youtube.com/@CureSYNGAP1
11k Twitter https://twitter.com/cureSYNGAP1
45k Insta https://www.instagram.com/curesyngap1/
Episode 187 of #Syngap10 #CureSYNGAP1
Friday Oct 03, 2025
Friday Oct 03, 2025
Friday, October 3, 2025. Week 40.
#SyngapCenus 1,675 https://curesyngap1.org/blog/syngap1-census-2025-update-39-q3-2025-total-1675/
Rachel J. made an educator handout https://curesyngap1.org/blog/supporting-students-with-syngap1-related-disorders/
Sign up for Citizen Health
https://www.citizen.health/partners/srf
CC/VNS Research w/ Citizen https://www.linkedin.com/posts/citizen-health-inc_syngap1-dee-raredisease-activity-7378823288950575105-YjP3
Rhymes with recent publication from Dr. Perry on Dravet
https://www.tandfonline.com/doi/full/10.1080/14737175.2025.2562118
Citizen AI Advocate avail. for SYNGAP1 families https://www.linkedin.com/posts/curesyngap1_syngap1-curesyngap1-raredisease-activity-7378443770201047040-ORGj
$CAMP starts GLP Tox https://www.linkedin.com/posts/camp4-therapeutics_syngap1-activity-7379142427149881344-cBFE
CF initiates coverage at Overweight https://www.investing.com/news/analyst-ratings/cantor-fitzgerald-initiates-camp4-therapeutics-stock-with-overweight-rating-93CH-4268395
$CAMP $2.98 at close on 10/2 https://www.google.com/finance/beta/quote/CAMP:NASDAQ
CIRM funds SYNGAP DISC0-17998 grant proposal from Iris Medicine team, in collaboration with Dr. Gene Yeo’s team (UCSD) https://www.cirm.ca.gov/about-cirm/newsroom/press-releases/cirm-approves-73-million-in-awards-for-discovery-research/
Scramble in SC on October 4th https://www.linkedin.com/posts/julie-miles-4294322ba_scramble-for-syngap-activity-7370558331611971585-iw0A
CURE SYNGAP1 Conference 2025 Atlanta https://curesyngap1.org/events/conferences/cure-syngap1-conference-2025-hosted-by-srf/
USE YOUR ICD-10 F78.A1
e185 https://www.youtube.com/watch?v=dale0NbxDpU
SOCIALS
4,381 LinkedIn. https://www.linkedin.com/company/curesyngap1/
1,450 YouTube. https://www.youtube.com/@CureSYNGAP1
11k Twitter https://twitter.com/cureSYNGAP1
45k Insta https://www.instagram.com/curesyngap1/
Episode 186 of #Syngap10 #CureSYNGAP1
Tuesday Sep 30, 2025
Tuesday Sep 30, 2025
Wednesday, October 1st, 2025. Week 40.
SYNGAP1 Related Disorders secured an ICD-10 code exactly four years ago today, through the advocacy of SRF and the hard work of volunteers like Hans Schlecht. Our code is F78.A1
Blog: https://curesyngap1.org/blog/syngap1-assigned-its-own-icd-10-code-f78-a1-srf/
Check out #S10e8 to learn more: https://www.youtube.com/watch?v=tZ5s5rQawXg
Read the case study: https://everylifefoundation.org/icd-code-roadmap/#toggle-id-13
Hear from other leaders: https://effieparks.com/podcast/episode-224-the-complicated-world-of-icd10-codes-with-ceo-and-co-founder-of-slc6a1-connect-amber-freed
Why does it matter and where are we now? It helps us find patients and it helps doctors and companies find YOU. We aren’t where we should be.
Dr. Lal’s sobering post: https://www.linkedin.com/posts/dennis-lal-71a8988a_raredisease-epilepsy-precisionmedicine-activity-7373307411383857152-dQS0
Preprint: https://www.medrxiv.org/content/10.1101/2025.09.12.25335652v1.full.pdf
TABLE 1. List of monogenic epilepsies with a syndrome-specific ICD-10 code, associated
genes, and code implementation dates.
Syndrome ICD-10 Code Gene Effective Date21
Rett syndrome F84.2 MECP2 10/01/2015
Glucose transporter protein type 1 deficiency syndrome (GLUT1-DS) E74.810 SLC2A1 10/01/2020
Cyclin-dependent kinase-like 5 deficiency disorder (CDD) G40.42 CDKL5 10/01/2020
Dravet syndrome G40.83 SCN1A 10/01/2020
SYNGAP1-related intellectual disability (SYNGAP1-ID) SYNGAP1 F78.A1 10/01/2021
MED13L syndrome Q87.85 MED13L 10/01/2023
Phelan-McDermid syndrome Q93.52 SHANK3 10/01/2023
SLC13A5 citrate transporter disorder E74.820 SLC13A5 10/01/2024
KCNQ2-related epilepsy G40.84 KCNQ2 10/01/2024
Kleefstra syndrome Q87.86 EHMT1 10/01/2024
5 Conclusion
Syndrome-specific ICD-10 codes for monogenic epilepsies are markedly underutilized, even for patients with confirmed molecular diagnoses and established clinical syndromes. In our cohort, fewer than two-thirds of eligible patients were ever documented with their syndrome-specific ICD-10 code, and when used, these codes were applied inconsistently across encounters, specialties, and time. Such gaps hinder the reliable identification of patients for precision therapies, clinical trials, and research studies, limiting the intended value of these codes. Although uptake of syndrome-specific ICD-10 codes showed gradual improvement over time, additional efforts, including automated and patient-driven coding support and integration of structured genetic data, are needed to ensure accurate and consistent use. Broader, multi-institutional studies will be essential to validate these findings and to guide strategies that maximize the clinical and research utility of syndrome-specific ICD codes as precision medicine advances.
Who else got them? New DEE Codes effective 10/1/2025! https://www.cdc.gov/nchs/icd/icd-10-cm/files.html
#FOXG1 Q04.8 https://www.foxg1research.org/news/foxg1-syndrome-icd-10-code
#Kabuki Q87.0
#CTNNB1 Q87.88 https://www.linkedin.com/posts/ctnnb1_ctnnb1-connectandcure-ctnnb1syndrome-activity-7376633308836683777-fRYC
#SCN2A QA0.0101 https://www.scn2a.org/from-advocacy-to-action-scn2a-now-has-its-own-icd-10-code/
#CACNA1A QA0.0102 https://www.linkedin.com/posts/cacna1a-foundation_huge-milestone-for-our-cacna1a-community-activity-7358883822282653696-xWr5
#SLC6A1 QA0.0131 https://www.linkedin.com/posts/slc6a1connect_raredisease-icd10-genetics-activity-7374801222056411136-wmAZ
#STXBP1 QA0.0141 https://www.stxbp1disorders.org/news/stxbp1-has-an-icd-10-code
#DLG4 QA0.0149
#Usher H35.5
CombinedBRAIN Rent a Neuro: https://combinedbrain.org/rent-a-neuroscientist/
CB Slide on ICD-10: https://docs.google.com/presentation/d/1wys1RLbJWBtK9eh7xSd_Lm-xwqbeZMSnM7xcCQznE8M/edit?usp=sharing
Everylife Roadmap: https://everylifefoundation.org/icd-code-roadmap/
REN ICD-10 page: https://www.rareepilepsynetwork.org/about-icd-codes
EVENTS!
Scramble this weekend in Greer, SC! https://donate.curesyngap1.org/event/scramble-for-syngap-2025/e667451
Conference on Dec 4 & 5 in Atlanta, don’t miss. https://donate.curesyngap1.org/event/cure-syngap1-conference-2025-hosted-by-srf/e661355
CURE SYNGAP1 CONNECT
https://curesyngap1.org/curesyngap1connect/
SOCIAL MATTERS
- 4,376 LinkedIn. https://www.linkedin.com/company/curesyngap1/
- 1,450 YouTube. https://www.youtube.com/@CureSYNGAP1
- 11,285 Twitter https://twitter.com/cureSYNGAP1
- 46k Insta https://www.instagram.com/curesyngap1/
NEWLY DIAGNOSED?
New families have resources here! https://syngap.fund/Resources
Podcasts, give all of these a five star review!
https://cureSYNGAP1.org/SRFApple
https://podcasts.apple.com/us/channel/syngap1-podcasts-by-srf/id6464522917
Episode 185 of #Syngap10 #CureSynGAP1
#Advocate #PatientAdvocacy #UnmetNeed #SYNGAP1 #SynGAP #SynGAProMMiS
Friday Sep 26, 2025
Friday Sep 26, 2025
Friday, September 26, 2025. Week 39.
In this episode of Syngap10, we continue the conversation from Episode 183, sharing the latest milestones and moments with our SYNGAP1 community.
DSC has announced!
DSC (part of RDCRN, part of NCATS, part of NIH) also announced and continues to raise profile of SYNGAP1 Related Disorders (SRD)
Key post https://www.linkedin.com/posts/curesyngap1_86-million-nih-grant-renews-support-for-activity-7373870761230589952-aV1M
#RDCRN List with #DSC https://ncats.nih.gov/research/research-activities/rdcrn/consortia
In addition to that, the DSC was formally announced, and will result in five years of SYNGAP1 securing a spot on the map. This was because of an SRF grant years ago! Grant https://curesyngap1.org/blog/syngap-research-fund-announces-308-000-multidisciplinary-biomarker-grant-to-boston-childrens-hospital/
Pubmed is at 44! (+2 v ‘23, -10 v ‘24, 2nd place) https://pubmed.ncbi.nlm.nih.gov/?term=syngap1&filter=years.2025-2025&timeline=expanded&sort=date&sort_order=asc
Cell Paper on AAV in Mice: https://www.linkedin.com/posts/boaz-levi-07387741_aav-delivery-of-full-length-syngap1-rescues-activity-7376306391537532928-iT9u
Last week was a CB Conf in Nashville, attended by KAH and VA, thank you to both. KAH in Staff yesterday, the hardest thing is not seeing Joey. ☹️ Thanks to MS for going too.
Two big upcoming events:
Scramble in SC on October 4th https://www.linkedin.com/posts/julie-miles-4294322ba_scramble-for-syngap-activity-7370558331611971585-iw0A
CURE SYNGAP1 Conference 2025 in Atlanta https://curesyngap1.org/events/conferences/cure-syngap1-conference-2025-hosted-by-srf/
SOCIAL MATTERS
- 4,371 LinkedIn. https://www.linkedin.com/company/curesyngap1/
- 1,440 YouTube. https://www.youtube.com/@CureSYNGAP1
- 11,292 Twitter https://twitter.com/cureSYNGAP1
- 45k Insta https://www.instagram.com/curesyngap1/
COMPANIES WITH NAMED ASSETS FOR SYNGAP1
$CAMP $3.00 at close on 9/23
Episode 184 of #Syngap10 #CureSYNGAP1
#Advocate #PatientAdvocacy #UnmetNeed #SYNGAP1 #SynGAP #SynGAProMMiS
Friday Sep 26, 2025
Friday Sep 26, 2025
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
Wednesday Sep 10, 2025
Wednesday Sep 10, 2025
Wednesday, September 10, 2025. Week 37.
CAMP4 Press Release:
https://www.linkedin.com/posts/caleb-moore-4382704_syngap1-activity-7371545171047628800-zVqR
Let me tell you a story:
- EW Story, concern over viability of C4.
- Easy to follow financials, Mrkt Cap and Net assets of ~$40M. Net income/EBITDA of -$12.6M in Q2.
- Running Phase I / II trials and ramping up for Phase III, not cheap. They need more than they had and capital is hard to get in this market.
- But here is the good part, the data is solid, the team is strong, and the SYNGAP1 Ecosystem is excited to have a first mover.
- SRF was thrilled to be invited, not just because we believe in C4, but because we wanted to send a meaningful signal to other investors that we are working closely with C4 and are eager to support their success. I believe that our investment, while modest, sent that signal and helped this raise become oversubscribed.
- The board worked hard on this one.
Now for hard questions:
- Are we conflicted? No. We will transparently share info about all trials for products with good data. ( See #S10e172 for ASGCT Data https://youtu.be/9xO1TcO1Eus )
- Will other companies be upset? Unlikely. Stoke and Praxis are the only companies publicly working on SYNGAP1 that are close to this point and they are not worried about financial viability, but if they do want to do a raise for their SYNGAP1 program, they should certainly call us.
- What will other companies think? Indeed we are de-risking the disease by showing that our kids are modifiable with ASOs which are the majority of the therapies in scope. This is a huge favor to others looking at this space.
- Isn’t this taking a risk with our funds? Depends. But if it is, it’s a risk worth taking. Remember we are the smallest investor, we only committed up to $1M, so other professional biotech investors put in $99M.
- What was the process? C4 came to us, we decided it was worth talking to the board who had multiple discussions but we said yes in less than a week and that was last week.
When is the trial? 2H26 Less than a year from now. With this financing, I am sure of it.
As I write this, the $CAMP stock closed up $0.80 or +40%. Which is solid. The market is starting to agree with the wise investors and SRF!
Yes we need a cure.
https://www.linkedin.com/posts/curesyngap1_savekramerdavis-activity-7371607032807763968-PVfG
See you Friday: Beacon of Hope September 12, 2025 - Boston, MA cureSYNGAP1.org/Beacon25
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Episode 182 of #Syngap10 #CureSYNGAP1
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