La Jolla Labs

09/24/2026

La Jolla Labs is excited to announce a new partnership with the Charcot-Marie-Tooth Association to develop a novel ASO therapeutic for a subtype of Charcot-Marie-Tooth (CMT), CMT2A.

CMT2A is caused by a mutation in the MFN2 gene, which damages peripheral nerves. For patients, this leads to progressive muscle weakness, and increasing challenges with strength and mobility. This new research project will investigate if switching on a paralog, MFN1, could compensate for the faulty MFN2 gene.

We are thrilled to be collaborating with CMTA Strategy to Accelerate Research (CMTA-STAR) Advisory Board Chair John Svaren, PhD at the University of Wisconsin-Madison to accelerate a targeted therapeutic for CMT.

Read the full announcement here:

CMTA partners with La Jolla Labs on new project to develop an ASO medicine for CMT2A that increases MFN1 expression to compensate for MFN2 mutations.

09/22/2026

Our CEO Tamar Grossman will be presenting at the RNA Leaders conference in Boston, MA on September 23-25, 2026.

09/02/2026

La Jolla Labs is celebrating it's fifth anniversary! La Jolla Labs was founded with the goal of making RNA therapeutics more accessible to patients with genetic conditions that often lack targeted treatments. This year we are celebrating having delivered over 20 drugs to patients with rare diseases. We are grateful to our team and our collaborators, especially the patient families and rare disease advocates that help us make this work possible.

La Jolla Labs was founded to make modern RNA biology and advanced computational techniques directly useful to patients whose conditions may lack clear therapeutic paths.

08/31/2026

Thank you to Valerie Rogatsky and Tyler Anthony from Marsh McLennan Agency for hosting a lunch to celebrate our award as Top Startup Honoree at the 2026 San Diego Business Impact Awards!

04/29/2026

National DNA Day commemorates the successful completion of the Human Genome Project in 2003 and the discovery of DNA's double helix in 1953. La Jolla Labs' celebrates this day to honor past achievements in nucleic acid science and as a company we hope to build upon this knowledge through RNA therapeutics research.

04/28/2026

April 28th is KIF1A Day. KIF1A is a gene that is essential for the healthy function of the nervous system.

A mutation in this gene can cause KAND (KIF1A Associated Neurological Disorder), an ultrarare progressive neurodegenerative disorder.

Read more about the function of KIF1A and this ultrarare condition in our blog here:

KIF1A-related disorders are a group of genetic disorders caused by one or more mutations in the KIF1A gene [1]. These mutations cause changes in the microtubule (MT) motor protein, disrupting the motor domain’s ability to transport specific synaptic vesicle precursors in axons, leading to KIF1A-As...

04/23/2026

Today is World Laboratory Day! Perla J. Sandoval and Geramie Shaver celebrate today by wearing their PPE and exercising those thumbs with their pipettes.

04/15/2026

Today is April 15th, International Pompe Day, created to foster international awareness of Pompe Disease. Pompe disease is a rare lysosomal storage disorder caused by a dysfunctional acid alpha-glucosidase enzyme, responsible for the lysosomal degradation of glycogen. This leads to glycogen accumulation in the body which causes cellular damage and dysfunction, tissue damage and organ dysfunction.

04/07/2026

La Jolla Labs celebrates World Health Day, observed on April 7th every year. This year the theme is “Together for health. Stand with science.” La Jolla Labs shares this philosophy by leveraging modern biology and computing technology and working together with patient families and partners to enable targeted therapeutic development for underrepresented rare disease communities.

04/04/2026

April is Fabry Disease Awareness Month! Fabry Disease is a rare genetic lysosomal storage disorder estimated to affect 1 in 100,000 people. People with this condition do not produce enough healthy versions of an enzyme called alpha-galactosidase A that breaks down fats. Consequently, these fats collect in the body's blood vessels and tissues, raising the risk of heart attack, stroke, and kidney failure.