La Jolla Labs has announced that it is approaching five years of work dedicated to advancing equitable access to precision ribonucleic acid (RNA) therapeutics. The company contributes to RNA discovery and provides translational support for families affected by rare conditions. Its early commitment to assisting clinicians and researchers in rare pediatric disorders continues to inform the design and evaluation of RNA therapies, with an emphasis on care and accessibility. This year, the company has delivered over 20 drugs to treat patients with rare diseases, including neurological paediatric diseases and rare Amyotrophic Lateral Sclerosis (ALS).
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. Its work spans screening automation, machine learning tools, high-throughput analysis, and the custom design of RNA-based molecules. By helping to reduce barriers that slow therapeutic development, the company aims to expand options for rare disease communities.
This service-driven model supports programs from early target identification through lead therapeutic design, with a focus on approaches that can advance toward clinical evaluation. “Our mission took shape after what we saw as a pivotal moment, an RNA-based therapeutic showing encouraging potential in an early-onset neurological disease model,” says Dr. Tamar Grossman, La Jolla Labs co-founder and CEO. “For us, it was a clear sign of both the possibilities within RNA science and the hurdles that still need thoughtful solutions. That experience inspired us to build an organization focused on turning promise into progress for families facing rare conditions.”
Drawing on years of shared expertise, the founders recognized the need for an organization dedicated to closing these gaps. Dr. Grossman has over two decades of experience developing RNA therapeutics from concept to the clinic at several biotech and Pharmaceutical companies. Early experimental work in severe pediatric models further guided the scientific direction and reinforced a commitment to responsive, compassionate development practices.
Building on this foundation, Dr. Grossman brought extensive experience across therapeutic modalities and advisory roles with emerging companies. Her active participation in scientific forums, regional initiatives, and community-focused biotech efforts has contributed to La Jolla Labs’ visibility and helped maintain patient-centered priorities within regional innovation.
Alongside Dr. Grossman, co-founder Jeff Milton has played a central role in shaping the company’s trajectory. With more than two decades of experience across life sciences and technology-driven drug discovery, he has helped integrate computational methods with laboratory innovation to accelerate therapeutic design. The leadership team’s combined expertise has supported the company’s strategic growth.
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This expertise translated into early innovations, most notably Precision Therapeutic Software Tools (PTX), a commercially available software platform intended to aid researchers in modeling RNA structure, splicing predictions, and exploring targeted RNA therapeutic interventions. Designed as a comprehensive tool that integrates predictive models of RNA binding, structural behavior, and splicing patterns, the platform has been positioned to support a network of research groups, pharmaceutical companies, and biotech companies.
“We believe Precision Therapeutic Software Tools (PTX) helped open new channels for scientific engagement,” Dr. Grossman states. “It supported collaborations through software licensing and offered tailored therapeutic design services, all of which aimed to strengthen sustainability while also expanding the reach of our work.”
According to La Jolla Labs, interest in its platforms and expertise has come from a diverse range of hospitals, research foundations, and patient-led organizations. The company suggests that this demand reflects both the reach of its software and the value of its scientific support. In practice, this has involved helping research teams incorporate RNA design principles into their work while also offering therapeutic proposals, which the company notes are occasionally offered at reduced or no cost to families and foundations addressing rare pediatric conditions. “Through these efforts, we have observed how computational innovation combined with laboratory expertise can encourage progress for communities that face significant challenges,” Dr. Grossman explains.
As La Jolla Labs approaches its fifth year, it’s preparing to expand its therapeutic programs into broader neurological indications, including conditions such as Alzheimer’s disease, Parkinson’s disease, and amyotrophic lateral sclerosis. La Jolla Labs plans to accelerate the timeline to get the first in human using the expertise gained from the delivery of the rare disease therapeutics.
The current rare ALS drug is planned to be administered to the patient in early 2026, less than 6 months after the mutation discovery. The company is also advancing new RNA therapeutic mechanisms to extend its scientific capabilities and plans to pursue partnerships that can help scale development. This next phase focuses on combining rigorous laboratory design and AI software tools with pathways for translation to broaden the range of patients who may eventually benefit from RNA-based interventions.
