Forgot to add a link if you want to look it up for more details: rnahorizons.com/rna-horizons...

More posts on #RNA_Horizons25 from @grnfluoresceblg.bsky.social, Thank you Michal Lotem, Rotem Karni, Amalia Herszkowicz and all the other organizesrs for the invite and putting such a great event together! 😀🙏 - See you next time!

The above are just from the first ~1.5 day, just headlines, and I still missed a bunch! If I have energy I'll add more later...😅 Bottom line: I learned a *lot* about what's going on in the RNA TX space both academica and industry. High quality talks, in a fabolous venue 😀

12. Riccardo Panella descrived lab and company's approach to target miR-22 affects multiple metabolic pathways. Results compared to GLP-1 show faster, lasting, not affecting lean fat.

11. Oxana Beskrovnaya discussed her company's Dyne TX results on dushen and DM1 with targeted delivery to muscle and CNS claims 20fold increase, in mice results last at leats 2mo, in phase 1 much better than existing which is only 1%.

10. Adrian Krainer talked about SRSF1 regulating Aurka 5' UTR splicing. Normal tissues do not express this isoform, so potentially good target, ASO targeting collapses the SRSF1-AURKA-MYC circuit and induces apoptosis. Maybe combine with KRAS inhibitors, maybe other cancers too.

9. Gal Haimovich described Intercellular mRNA transfer via tunneling nanotubes (TNTs) as a potential new way for delivery, about 1% is being transferred want to dev tech to push that further.

8. Christopher Ricupero from Columbia described rare hnRNPH2 related neurdodevelopment, developed non-allele specific gapmer for n=3 with initial results, they think it's working even though non allele specific because H1 compenstates H2.

7. Christine Duthoit described her company's RNAlead alternative to LNP which is based on Lentivrial vectors (iLV) and bacterophage, says the mechanism is different so more efficient and not that defense response in cells.

6. Peter Weinstein discussed his company's Emervax technology for delivering with circRNA. Says that compared to linear RNA or existing designs they need much lower dosage, less frequent (it stays) and less immunogenicity. Showed results for yellow fever, Hantaan Virus.

5. Brage Storstein Andresen discussed his lab and company work on detecting and targeting Pseudo exons (PE) for theraputics (activate them via ASO to degrade the gene, but NMD is variable..) or in 5' UTR can affect translation.

4. Claudio Sette focused on PDAC cancer. Found RBM47 differentially expressed in low grade differentiated classical PDAC and via MAPK37 splicing and promotes TGF-beta signaling and pro mesenchmal features. Also QKI is associated with poor outcome, promotes cell migration.

2. Ruben Postel from Sapreme Technologies discussed their company's tech (proprietary functionalized saponin) to allow ASOs to escape the endosome, and related
3. François-Thomas Michaud from Feldan Tx discussed their tech for endosomal escape (a big challenge multi companies are working on)

1. Ernesto Guccione discussed his lab's efforts to improve ASO design - Both a reporter system and algorithms that take into account RBP binding, he shows an application for Urea Cycle Disorders (UCD) where people are missing diagnosis due to deep intronic mutations they detected and validated.