LA JOLLA, CA — April 30, 2026 — La Jolla Labs, Inc. today announced that the first patient has been treated with GBFsen, a custom-designed antisense oligonucleotide (ASO) therapy developed to target a TDP-43 patient-specific mutation associated with an inherited, ultra-rare form of amyotrophic lateral sclerosis (ALS).
The patient was diagnosed one year ago with a rapidly progressive form of ALS driven by a mutation in the prion-like domain of TDP-43. With no approved therapies, no clinical protocols, and no established development pathway for his specific mutation, traditional drug development frameworks offered no viable options.
La Jolla Labs initiated a therapeutic program at its own expense, with the support from Illumina, ChemGenes Corporation, and IDT, to pursue this targeted genetic intervention. The program was initiated in September 2025, the team identified highly selective and potent compounds targeting mutant TDP-43. The program was reviewed in March 2026 by the 1M1M consortium and proceeded into clinical development at Heidelberg University Hospital. Safety, tolerability, and exploratory biomarkers are being monitored. “The first patient treated with GBFsen reflects what is possible when scientific urgency is matched with human urgency,” said Dr. Tamar Grossman, Co-founder and Chief Executive Officer of La Jolla Labs. “This program was built from the ground up around a single mutation, with the goal of intervening directly in the underlying biology of disease.”
“This milestone is not only about a single patient, but about challenging the assumptions that have historically limited drug development for ultra-rare conditions,” said Jeff Milton, Co-founder, Chief Technology Officer of La Jolla Labs. “We believe individualized therapies can be developed with both speed and scientific rigor, and that new models are needed to serve patients who fall outside conventional frameworks.” La Jolla Labs believes this effort may inform future approaches to enable more rapid, cost-effective therapies for patients with rare and genetically defined diseases. “This is about more than one case,” added Dr. Grossman. “It is about demonstrating that therapies to rare diseases does not have to mean unreachable and our vision at La Jolla Labs is to make genetic medicine accessible to all patients”
About GBFsen
GBFsen is an investigational antisense oligonucleotide therapy designed to selectively target a disease-causing RNA transcripts associated with a rare genetic form of ALS involving TDP-43 pathology. GBFsen has not been approved by the U.S. Food and Drug Administration, and studies are ongoing for safety and efficacy.
About
La Jolla Labs, Inc. La Jolla Labs is a
biotechnology company revolutionize genetic medicine discovery and development
for rare and complex diseases. La Jolla Labs proprietary
AI-powered software streamlines drug design and screening, resulting in
efficient and rapid drug discovery of safe and effective candidates. La Jolla Labs has a clinically validated
technology for novel therapeutics.
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