La Jolla Labs partner, 1STRAND, quotes CEO of La Jolla Labs, Jeff Milton in article discussing San Diego's RNA cluster.

Our new ML models provide a roadmap for designing optimal splicing screens for antisense therapeutics.

La Jolla Labs CEO, Jeff Milton, joined the Innovation in Rare Disease Panel Discussion during LaunchBio's Invest In Cures Event to discuss how computational biology and machine learning may be used to address the unmet needs of rare disease patients.

"This is not a science problem anymore’: Paths emerge for scaling up rare disease medicine"(ENDPOINT NEWS).

"ASOs are an established class of drug showing increasing success in treating neurological diseases. To determine if ASOs would make good therapeutics for Pitt Hopkins Syndrome (PHS), the PHRF has arranged a collaboration with Mahzi Therapeutics and La Jolla Labs. Currently, this project is in the very early phases of scientific development, working to establish proper biological systems to evaluate this strategy. Once experimental conditions are worked out, this collaboration will test the ability of ASOs to restore the protein levels of TCF4 and effectively reverse the cause of PTHS" (Pitt Hopkins Research Foundation).

"Looking for a refresher on KIF1A and KAND? This week La Jolla Labs featured KAND in its Revealing Rare Genes blog with input from KIF1A.ORG. This article provides an excellent overview of the history and biology of KAND with a focus on therapeutic development, so we’ll let it speak for itself. A huge thank you to Perla Sandoval and Allison Brown for creating this resource"(KIF1A.ORG).

Learn more about how La Jolla Labs is developing the world's most efficient RNA drug discovery pipeline.

Tune in to hear Jeff Milton, CEO of La Jolla Labs, join the inspirational Effie Parks on the Once Upon a Gene podcast.

In this episode, they discuss the development of a drug discovery process that opens the doors to a competitive rare disease therapeutics market and isn't trapped behind the walls of "big" pharmaceutical companies.

La Jolla Labs participates in the Global Chain of Lights to show support and raise awareness for rare diseases and patients on Rare Disease Day.

Tune in to hear Founder and CEO of La Jolla Labs, Jeff Milton, explain how new artificial intelligence will drive rare disease therapeutics at iXCells Biotechnologies' Rare Disease Day Webinar.

Millions of patients world-wide suffer from rare disease and while non-profit organizations provide a platform for certain indications, only a few have succeeded in delivering meaningful therapeutics to patients. To address rare disease in a comprehensive and sustainable manner requires a fundamental change to drug discovery; a change that opens a competitive landscape with risks and rewards that match those found in big pharma. I will present a model that leverages modern technology and the interconnected nature of disease to provide “economic runways” for many rare disease programs.

Today, we have the science to help dying children, but little to no access because our system wasn't designed for medicines for one or very few people. The drug development process may work for large diseases, but to help those with rare diseases, we must now pivot and build a parallel system for individualized medicines.

Revealing Rare Genes is a blog covering topics at the intersection of rare diseases and RNA therapeutics. We feature individual diseases, highlight patient stories, and delve into the challenges of bringing these therapies to market. Sometimes science can feel disconnected from the real world and everyday people. Through this blog we aim to forge connections that drive drug development for rare diseases forward: to connect all people with stories to inspire action; scientists with research challenges to overcome; and clinicians with healthcare gaps to address.

Partnership leverages La Jolla Labs’ automated, high-throughput screening technology to identify optimal ASO candidates.