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.