Tune in on Tuesday, Oct 20th at 8 am PDT to hear the latest updates on our work with #exomers
Microsoft Teams meeting
Join: https://t.co/HT13f3Csvn
Meeting ID: 212 993 917 099 964
Passcode: i8iF9Rb7
Tune in on Tuesday, Oct 20th at 8 am PDT to hear the latest updates on our work with #exomers
Microsoft Teams meeting
Join: https://t.co/HT13f3Csvn
Meeting ID: 212 993 917 099 964
Passcode: i8iF9Rb7
This week’s #journalclub featured @T_Ikenouchi on modulators of #cardiomyocyte proliferation (yes, there are even more lurking out there), @AleCiulloPhD on cell-cell #mitochondrial transfer in #FreidreichsAtaxia (who’d have thought it happens frequently enough to make a therapeutic difference?), and me discussing a phase 1 trial of an anti-RAGE #siRNA for #asthma (nice template for our own upcoming FIH #exomer trials).
Terrific lab meeting this week by @ke_liaoPhD on the mechanism of #depression, which afflicts one third of patients after a #HeartAttack. Amazing discoveries involving #EVs, circular #RNA and depletion of miRs. We even have a prototype therapeutic candidate! Exciting times…
Late Breaking clinical trial #HOPE-3 OLE shows striking benefits of #deramiocel sustained over two years of therapy, and 76% slowing of #Duchenne myopathy progression in those who crossed over from placebo to deramiocel after one year. Work of ace #DMD trialist Craig McDonald highlighted at the @WorldMuscleSoc meeting in Hiroshima. Trial sponsored by @Capricor based on intellectual property from my team at Cedars-Sinai.
Delighted to have @MarbanLab for an inspiring talk on translating cardiosphere-derived cell biology into RNA therapeutics. Fascinating insights into extracellular vesicles, Y RNA, macrophage reprogramming, tissue repair, and TY1; from discovery to clinical translation. #UCLA
Suggesting a novel therapy for #SystemicSclerosis: https://t.co/EdUt8cSVrO
Eduardo Marbán & team @Marbanlab@CedarsSinai report that an orally delivered noncoding RNA (TY1) reduces inflammation and organ fibrosis in models of systemic sclerosis through inhibition of the cGAS/STING axis. The authors employed in vitro and in vivo models and evaluated skin, lung, and heart.
The video shows TY1 treatment in preclinical models of systemic sclerosis.
@AleCiulloPhD@TheIbrahimlab@the_asci
Excited to share our latest paper in @JCI_insight, with @XMichaelJonesMD & @TheIbrahimLab : #TY1, an #RNA #exomer drug, reverses the key disease manifestations of #scleroderma in 2 mouse models & in human #SSc cells. The drug works well orally & has unprecedented disease-modifying bioactivity in this horrible disease. Next step: clinical trials!
https://t.co/0EXDWd7CqI
We're pleased to share that @TheLancet has published results from Capricor's Phase 3 HOPE-3 trial evaluating Deramiocel in #Duchenne muscular dystrophy.
The independent peer-reviewed publication validates the trial's design, statistical methodology and findings. HOPE-3 met its primary endpoint, with Deramiocel slowing upper limb function decline by 54 percent versus placebo (PUL 2.0, p=0.03) and demonstrating a clinically meaningful cardiac benefit.
Read more here: https://t.co/WPBkXv0Ws9
We are grateful to the patients, families, investigators and study teams who made this research possible.