Kinea Bio announced two major strategic achievements that position the biotechnology company to accelerate development of KNA-155, its dual-AAV gene therapy for dysferlinopathy. The Jain Foundation committed to invest up to $1.1 million based on milestone achievements, while Solid Biosciences granted a non-exclusive license for its proprietary AAV-SLB101 capsid technology.
Jain Foundation Investment Supports Critical Preclinical Development
The Jain Foundation's milestone-based funding will support key IND-enabling activities, including dose-finding and GLP toxicology studies in dysferlin-deficient animal models. These studies aim to define the therapeutic window of KNA-155, generate pivotal safety and biodistribution data, and de-risk the path to first-in-human trials.
"Supporting promising therapeutic strategies and guiding them through rigorous preclinical development is central to our mission," said Dr. Laura Rufibach, Co-President of the Jain Foundation. Dr. Doug Albrecht, the Foundation's other Co-President, added that "Kinea Bio's innovative dual-vector approach has already demonstrated compelling preclinical evidence, and we are proud to help accelerate its translation toward the clinic."
AAV-SLB101 Capsid Technology Enhances Delivery Platform
Kinea Bio licensed Solid Biosciences' AAV-SLB101 capsid, a clinically validated myotropic AAV engineered for efficient skeletal muscle delivery with reduced liver uptake. This next-generation capsid will serve as the delivery backbone for KNA-155, enhancing potency and safety in systemic administration.
AAV-SLB101 is a rationally designed capsid developed for enhanced muscle tropism and reduced biodistribution to the liver. Preclinical studies have demonstrated robust cardiac and skeletal muscle transduction, with clinical validation emerging from Solid's ongoing Phase 1/2 INSPIRE DUCHENNE trial evaluating SGT-003. As of August 12, 2025, AAV-SLB101 has been well tolerated in 15 participants who have been dosed in the trial.
"We are excited to partner with Kinea Bio to apply our muscle-tropic capsid, AAV-SLB101, to advance the field of gene therapy for dysferlinopathy," said Bo Cumbo, President and CEO at Solid Biosciences. "We believe that the compelling combination of Kinea's scientific expertise, our next-generation delivery technology and the Jain Foundation's commitment will help bring new hope to patients."
Addressing Significant Unmet Medical Need
Dysferlinopathy, also known as LGMD2B/R2 or Miyoshi Myopathy type 1, is a progressive and debilitating type of muscular dystrophy caused by mutations in the dysferlin (DYSF) gene. The condition affects approximately one in 100,000 individuals worldwide, with higher incidence in some populations. Patients experience progressive muscle weakness and loss of ambulation, and currently have no approved therapies.
The dysferlin gene exceeds the packaging capacity of a single AAV, necessitating a dual-vector approach for AAV restoration of the full-length dysferlin gene. Kinea Bio's SIMPLI-GT™ platform addresses this challenge by enabling delivery of large therapeutic genes that exceed the natural packaging capacity of AAVs.
Strategic Partnerships Drive Development Forward
Under the licensing agreement with Solid Biosciences, Kinea Bio receives a non-exclusive worldwide license to utilize AAV-SLB101 for KNA-155 development and commercialization. Solid receives an upfront fee and is eligible for additional payments upon achievement of certain development and sales milestones, plus tiered royalties on net sales.
"Combining the Jain Foundation's support with Solid's capsid technology creates a powerful foundation for success," said Casey Childers, CEO of Kinea Bio. "These achievements allow us to execute our development plan with confidence and move closer to bringing a much-needed therapy to patients living with dysferlinopathy."
Solid Biosciences has expanded collaborative efforts for AAV-SLB101 with more than 25 agreements and licenses executed to date, demonstrating the broad applicability of the capsid technology across multiple therapeutic applications.
