Kinea Bio Inc., a biotechnology company developing genetic medicines for severe neuromuscular disease, today highlighted the publication in Science Translational Medicine of preclinical data supporting KNA-155, its dual-AAV gene therapy for dysferlinopathy (LGMDR2/LGMD2B). “Use of split intein and myotropic AAV vectors enables effective preclinical gene therapy for dysferlinopathy” was published on October 7, 2026, led by investigators at the University of Washington and Children’s National Hospital. Corresponding author Hichem Tasfaout, PhD, serves on Kinea Bio’s Scientific Advisory Board, and several of the study’s authors are co-founders of the company.

The dysferlin coding sequence is roughly 6.2 kilobases, beyond the approximately 4.7 kilobase capacity of a single AAV vector, and dysferlin requires its full length to function. The published work splits the sequence across two AAV vectors and uses split inteins to reconstitute the complete protein inside the muscle cell, delivered with a myotropic capsid. This is the principle underlying Kinea Bio’s SIMPLI-GT™ platform.

Therapeutic effect at substantially lower vector doses. The highest dose tested was 10-fold lower than doses commonly used with conventional AAV serotypes, and approximately 5-fold lower than the dose previously required for a dual-AAV strategy based on homologous recombination, against which the split-intein approach produced more full-length dysferlin and greater functional improvement. Dose governs manufacturing cost and capacity, and it sits at the center of the immune and liver toxicity risks that have stopped programs across the field. Working an order of magnitude below the conventional range relieves pressure on both at once.

Benefit in advanced disease. Treatment improved contractile force and reduced fibrosis and fatty degeneration both when given near disease onset and in models with established, advanced disease. Because people with dysferlinopathy are typically diagnosed years after symptoms begin, activity in already-damaged muscle addresses a central translational risk.

“Restoring full-length dysferlin has been the central obstacle in this field, and this work shows it can be done,” said Martin K. Childers, DO, PhD, Chief Executive Officer and co-founder of Kinea Bio, and a co-author of the study. “What matters most to us as a company is the context in which the benefit was achieved: in muscle that was already badly damaged, at doses well-below what conventional AAV programs require. Most people with this disease are diagnosed long after the damage has started. A therapy that only works before that point would not help them, and that is the bar we are building KNA-155 to clear.”

The publication builds on the Jain Foundation’s $1.1 million investment in Kinea Bio, announced in September 2025, which is supporting IND-enabling activities for KNA-155 including dose-finding and GLP toxicology studies.

“The Jain Foundation and the dysferlin community are excited to see this work published, showing the therapeutic potential of an intein-based dual AAV gene therapy for dysferlinopathy. The advances Kinea have made are remarkable and we are excited to support this program and its further development into human studies”, said Doug Albrecht, PhD, Co-President of the Jain Foundation.

Kinea Bio is advancing KNA-155 through IND-enabling studies. Further work is required before clinical testing, including immune responses to the AAV vectors and split inteins, long-term durability, and uniformity of dysferlin expression.

About Dysferlinopathy

Dysferlinopathy, also known as LGMD2B/R2 or Miyoshi Myopathy type 1, is a progressive and debilitating muscular dystrophy caused by mutations in the dysferlin (DYSF) gene. It affects approximately one in 100,000 individuals worldwide, with higher incidence in some populations. Patients experience progressive muscle weakness and loss of ambulation, and there are currently no approved therapies.

About Kinea Bio, Inc.

Kinea Bio, Inc. is a biotechnology company pioneering a novel dual AAV vector platform, SIMPLI-GT™, to deliver large therapeutic genes that exceed the natural packaging capacity of AAVs. The company was among the first to demonstrate the potential of this approach in systemic disorders such as Duchenne muscular dystrophy and is now expanding its pipeline to include dysferlinopathy and other severe genetic conditions. For more information, please visit www.kineabio.com.

About the Jain Foundation

The Jain Foundation is a private foundation based in Seattle dedicated to developing therapies for dysferlinopathy. Founded by Ajit Jain in 2005, the Foundation funds promising research, supports clinical trial readiness, and makes program-related investments in companies to expedite commercialization of innovative therapeutic approaches. For more information, please visit www.jain-foundation.org.

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