La Jolla Labs Inc.

#lajollalabs #raredisease #rnatherapeutics #rna #rarediseases

Welcome new interns to La Jolla Labs

🌟Excited to share news about the Promising Pathways Act 2.0, a newly proposed bill designed to expedite and streamline approval pathways for rare disease therapies in the US! 🌟 This Act holds great promise but also faces challenges, especially for small and medium-sized biotech firms that often lack the financial power of Big Pharma and struggle with complex regulatory hurdles. As a frequent PharmaBoardroom contributor and rare disease patient advocate, I played a crucial role in crafting this bill and explains more about its potential impact. Let's support this initiative to bring hope to those affected by rare diseases! #RareDisease #HealthcareInnovation #PromisingPathwaysAct Patients Rising I AM ALS The Cure Starts Now Cancer Foundation CARES Foundation, Inc Beyond Batten Disease Foundation COMBINEDBrain APS TYPE 1 FOUNDATION Cure MLD Glut1 Deficiency Foundation SynGAP Research Fund (SRF) for SYNGAP1-Related Disorders La Jolla Labs Inc. Project Alive PharmaEssentia Soleno Therapeutics, Inc. Stealth BioTherapeutics Angelman Syndrome Foundation Lennox-Gastaut Syndrome (LGS) Foundation SCN2A- The FamilieSCN2A Foundation Prader-Willi Syndrome Association | USA SYNGAP1 Foundation Team Gleason Foundation Foundation for Government Accountability PROJECT SEBASTIAN Dravet Syndrome Foundation Please tag yourself and share if you support this bill!

Fantastic science and amazing families at the Pitt Hopkins meeting in Denver

Today, June 20th, is World FSHD Day!  FSHD or Facioscapulohumeral Muscular Dystrophy is a rare genetic muscle disorder that can affect and cause atrophy in the facial, shoulder, upper arm, and lower leg muscles of an individual.   Learn more about FSHD in our infographic below! #FSHD #WorldFSHDDay #Neuromuscular #RareDisease #CureFSHD #FSHDResearch #RareResearch #MuscularDystrophy #LJL #LaJollaLabs #drugdiscovery

The ASO off-target impact of transposons/retrotransposons is significant!

Introducing the LJL Genome Patent Database: Visualize patented sequence space. #genome #rna #aso #oligonucleotides

Using our splicing models we correctly predict TDP-43 (TARDBP) blocking cryptic-exon "inclusion" attributes in the frontotemporal dementia ALS gene UNC13A. Thus loss of TDP-43 (e.g. in ALS) favors cryptic exon inclusion.   https://lnkd.in/dcDFfp7C #ALS #ai #ASO #splicing

La Jolla Labs recently published the fastest algorithm for RNA-targeting off-target analysis. (up to 5 Levenshtein distances on a 20mer) #aso, #levenshtein #RNA #azure #ml #ai

I know I've posted before on this professional site about how my personal life drastically and unexpectedly changed my professional career. I'll say again that my time as a NICU parent was most definitely life-changing. I'm so happy to be in the professional spot I am now (La Jolla Labs Inc.! #raredisease), but I wasn't always sure I'd ever get back to science. If you have been touched by the NICU and/or want a way to give back, I'm running Sunday in Miracle Babies Non-Profit's 5k and I'm inviting you to join our "Reading Rhinos" team or donate to it. Thanks for your support! See our team page for an explanation of our name 💙 https://lnkd.in/gvG6mszD