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Over 1200 families are caring for a loved one with the rare disease ”SynGAP” resulting from a variant of the SYNGAP1 gene. This 10 minute 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 the SynGAP Research Fund. SRF is a parent-led, all volunteer 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://www.syngapresearchfund.org/
Episodes
Friday Jan 14, 2022
Friday Jan 14, 2022
Stoke Therapeutics & Acadia Pharmaceuticals are working on SYNGAP1
BIG NEWS! https://www.businesswire.com/news/home/20220110005334/en/
FIVE KEY LINKS
- Dr. Kaye’s presentation - Listen to it again and again https://investor.stoketherapeutics.com/events/event-details/40th-annual-jp-morgan-healthcare-conference
- AES 2018 Poster https://www.stoketherapeutics.com/wp-content/uploads/Stoke-Poster-Dec-1.pdf
- STOKE Patent https://patents.google.com/patent/WO2017106377A1/en
- #OneYearSooner - How we can make clinical trials happen faster https://www.syngapresearchfund.org/post/oneyearsooner
- Sign up for Ciitizen - www.Ciitizen.com/SYNGAP1
SPREAD THE WORD
Twitter.com/cureSYNGAP1/status/1480546972645793794?s=20
Linkedin.com/feed/update/urn:li:activity:6886314132866506753
Facebook.com/cureSYNGAP1/posts/946801809535615
WHO IS WHO AT STOKE
https://www.cshl.edu/research/faculty-staff/adrian-r-krainer/ PhD Harvard 1986
https://www.linkedin.com/in/huwnash/ PhD Harvard 1997, EIR ATP since 2014
https://www.oligotherapeutics.org/officers/isabel-aznarez-ph-d/ PhD Toronto 2006
https://www.linkedin.com/in/barryticho/ MD PhD Chicago
https://www.linkedin.com/in/edward-kaye-0a46a710/ MD Loyola Chicago
COMPANIES
Stoke https://www.stoketherapeutics.com/ $ACAD
Acadia https://www.acadia-pharm.com/ $STOK
OTHER GREAT LINKS
DSF on the Monarch https://www.dravetfoundation.org/wp-content/uploads/2020/04/Stoke-Community-FAQ-April-2020.pdf
It starts with Spinraza https://www.ninds.nih.gov/About-NINDS/Impact/NINDS-Contributions-Approved-Therapies/Nusinersen-Spinraza%C2%AE-%E2%80%93-Spinal-Muscular aka https://en.wikipedia.org/wiki/Nusinersen
Grant made in 2003, Phase 1 in 2011 (dec) FDA approval in 2016 (Dec) https://www.curesma.org/fda-approves-spinraza-for-sma/
From: https://www.bizjournals.com/boston/news/2018/01/04/ex-sarepta-ceo-takes-helm-of-genetic-disease.html
Kaye said he was recruited by Stoke co-founder Adrian Krainer, with whom he previously worked at Genzyme before joining Sarepta in 2010. Krainer is perhaps best known for being an inventor of another “antisense” drug targeting a genetic disease, Biogen’s spinal muscular atrophy treatment Spinraza.
“He was one of the real originators of RNA therapy,” Kaye said. “I thought (Stoke) was at a point where it needed to be shepherded from preclinical development into the clinic. It was a really exciting opportunity.”
Stoke Origins: https://endpts.com/gene-therapy-startup-stoke-therapeutics-secures-another-90m-in-series-b-funding/ $40M from ATP in 2018. https://www.appletreepartners.com/portfolio#stoke-therapeutics
IPO June 2019 $163M/ https://www.spglobal.com/marketintelligence/en/news-insights/trending/OTV6RnpzTCGYyRs_gx1m7A2
REMEMBER
Raise funds at https://syngap.fund/give
Sign up for this 10 minute #podcast #SYNGAP10 here https://syngap.fund/10 if you want a direct link for Apple: https://syngap.fund/10a
Episode 43 of #Syngap10 - January 14, 2022
#StokedAboutStoke #ASO #SYNGAP1 #AcadiaPharma #StokeTx
#F78A1 #Syngap #epilepsy #autism #intellectualdisability #id #raredisease #epilepsyawareness #autismawareness #rarediseaseresearch #SynGAPResearchFund #CareAboutRare #PatientAdvocacy #GCchat #Neurology #Genetics
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