Non-exclusive license for Solid Biosciences’ proprietary, next-generation myotropic capsid, AAV-SLB101, to accelerate development of KNA-155 for dysferlinopathy

SEATTLE–(BUSINESS WIRE)–Kinea Bio Inc., a biotechnology company dedicated to developing transformative genetic medicines, today announced two major achievements in its gene therapy program: a commitment by the Jain Foundation to invest up to $1.1 million based on the achievement of certain milestones to support critical preclinical studies, and a strategic license from Solid Biosciences for its proprietary, next-generation myotropic AAV capsid, AAV-SLB101, to enable safe and effective muscle-targeted delivery. Together, these advances provide the scientific and financial foundation to accelerate development of KNA-155, Kinea Bio’s dual-AAV gene therapy for dysferlinopathy.

“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.”Share

The Jain Foundation’s funding will support key IND-enabling activities, including dose-finding and GLP toxicology studies in dysferlin-deficient animal models. These studies will define the therapeutic window of KNA-155, generate pivotal safety and biodistribution data, and de-risk the path to first-in-human trials.

The Jain Foundation’s Co-Presidents, Doug Albrecht and Laura Rufibach, expressed enthusiasm for Kinea’s dysferlinopathy program. “Supporting promising therapeutic strategies and guiding them through rigorous preclinical development is central to our mission,” said Dr. Rufibach. Dr. Albrecht agreed, saying, “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.”

To complement this funding, Kinea Bio has in-licensed Solid Biosciences’ AAV-SLB101 capsid, a clinically validated myotropic AAV engineered for efficient skeletal muscle delivery with reduced liver uptake. This capsid will serve as the delivery backbone for KNA-155, enhancing potency and safety in systemic administration.

“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.”

“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.”

The collaboration with the Jain Foundation underscores the Foundation’s commitment to the development of treatments for dysferlinopathy through targeted investment and research oversight, while the partnership with Solid Biosciences adds cutting-edge delivery technology to maximize clinical impact. Together, these initiatives validate confidence in Kinea Bio’s innovative dual vector strategy and highlight the urgency and momentum driving its advancement toward the clinic.

About Dysferlinopathy

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. 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 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.

About Kinea Bio, Inc.

Kinea Bio, Inc. is a biotechnology company pioneering a novel dual AAV vector platform SIMPLI-GT^TM 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. Through innovative science and strategic collaborations, Kinea Bio is dedicated to translating breakthrough biology into transformative medicines for patients with high unmet needs. For more information, please visit www.kineabio.com.

About Jain Foundation

The Jain Foundation is a private foundation based in Seattle which is dedicated to developing therapies for dysferlinopathy. Founded by Ajit Jain in 2005, the Foundation engages in multiple complementary approaches which include funding promising research and supporting clinical trial readiness. The Foundation’s activities have advanced understanding of dysferlinopathy, defined the natural history of the disease, and developed outcome measures for clinical trials. As part of its ongoing funding activities, the Foundation makes program-related investments in companies to expedite commercialization of innovative therapeutic approaches, such as Kinea Bio’s KNA-155 program. For more information, please visit www.jain-foundation.org.

About Solid Biosciences

Solid Biosciences is a precision genetic medicine company focused on advancing a portfolio of gene therapy candidates targeting rare neuromuscular and cardiac diseases, including SGT-003 for Duchenne muscular dystrophy (Duchenne), SGT-212 for Friedreich’s ataxia (FA), SGT-501 for catecholaminergic polymorphic ventricular tachycardia (CPVT), SGT-601 for TNNT2-mediated dilated cardiomyopathy and additional fatal, genetic cardiac diseases. The Company is also focused on developing innovative libraries of genetic regulators and other enabling technologies with promising potential to significantly impact gene therapy delivery cross-industry. Solid is advancing its diverse pipeline and delivery platform in the pursuit of uniting experts in science, technology, disease management, and care. Patient-focused and founded by those directly impacted by Duchenne, Solid’s mission is to improve the daily lives of patients living with devastating rare diseases. For more information, please visit www.solidbio.com.

Contacts

Media Contact:
Nicholas Wageling
Director of Operations
contact@kineabio.com