Personalized Genetic Therapy for Rare Form of ALS Reaches First Patient in Less Than One Year

For Immediate Release: Thursday, October 22, 2026, 4:20 p.m. U.S. Eastern Time

Media Contact: Evelyn Mantegani, +1 240-833-2014, [email protected]

Presented By: Tamar Grossman, PhD, La Jolla Labs

Rockville, Md. – Developing a therapy for a single patient is difficult under traditional drug-development models, particularly when the disease-causing genetic variant is exceptionally rare. A new program demonstrates the feasibility of designing and administering an individualized genetic therapy for a patient with rapidly progressing inherited amyotrophic lateral sclerosis, or ALS, less than one year after diagnosis.

The patient had a disease-causing variant in TARDBP, the gene that encodes the TDP-43 protein. Variants in this gene can cause genetically and clinically diverse forms of ALS, a progressive neurodegenerative disease with limited treatment options.

Researchers at La Jolla Labs developed GBFsen, an antisense oligonucleotide, or ASO, designed specifically for the patient’s mutation. ASOs are short strands of genetic material that can alter the production or activity of selected RNA transcripts. The researchers used an artificial intelligence-enabled discovery platform to identify highly selective candidates, which underwent preclinical evaluation and translational review before GBFsen was administered to the patient.

The program moved from identification of the mutation to individualized treatment in less than a year. It offers a potential model for developing precision medicines for patients whose variants are too rare to attract conventional commercial drug-development programs. The research team has since dosed a second patient – a family member of the initial patient – who is diagnosed with ALS and carries the same mutation. They plan to extend access to other family members and additional ALS patients carrying the same mutation.

The report establishes that rapid, patient-specific ASO development and administration are possible, but it does not demonstrate that GBFsen is safe or effective. Safety, tolerability, and exploratory biomarkers are still being assessed, and no clinical outcomes have been reported. Further evidence will be required to determine whether this approach can provide clinical benefit and be reproduced for other patients with rare genetic neurological conditions.

About the American Society of Human Genetics (ASHG)

The American Society of Human Genetics (ASHG) is the leading global organization advancing the science of human genetics and genomics. Representing nearly 8,000 researchers, clinicians, genetic counselors, educators, and other professionals, ASHG drives progress in understanding the human genome and its role in health and disease. The Society fosters innovation and collaboration through its flagship events and publications—including the ASHG Annual Meeting and its peer-reviewed journals The American Journal of Human Genetics and Human Genetics and Genomics Advances. ASHG also champions science advocacy, supports the next generation of genetics professionals, and promotes responsible policies and public engagement in genetics. Learn more at www.ashg.org.

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