September 23, 2025

The Jain Foundation is excited to announce that it has provided $1.1million in funding to Kinea Bio, Inc. to accelerate therapeutic development of KNA-155, Kinea Bio’s dual-AAV gene therapy for dysferlinopathy.

The investment from the Jain Foundation provides milestone driven support of essential preclinical studies. This funding will advance key IND-enabling activities, including dose-finding and GLP toxicology studies in dysferlin deficient animal models. With these studies, Kinea will be able to advance their investigational gene therapy product into human studies.

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 first demonstrated the potential of this approach in Duchenne muscular dystrophy and is now expanding its pipeline to include dysferlinopathy and other severe genetic conditions.

Dysferlinopathy, also known as LGMD2B/R2 or Miyoshi Myopathy type 1, is a progressive muscle wasting disease caused by mutations in the dysferlin (DYSF) gene. Dysferlinopathy has no approved treatments. While gene replacement therapy has shown promising results for other muscular dystrophies and neuromuscular diseases, gene replacement therapy using the dysferlin gene has been challenging because it is too large to fit in a single AAV virus. One possible solution  to the size limitation of AAV viruses is to engineer the viral cargo so that it is effective using a dual-vector approach.

Kinea plans to overcome these challenges by using:

1. A proprietary Split Intein Mediated Protein Ligation Gene Therapy (SIMPLI-GT^TM) that joins protein segments together to form large functional proteins
2. Next-generation myotropic AAV capsids to enable safe and effective muscle targeted delivery.

The Jain Foundation’s Co-Presidents, Drs. Doug Albrecht and Laura Rufibach, expressed enthusiasm for Kinea’s dysferlinopathy program.  Dr. Rufibach noted:

“Supporting promising therapeutic strategies and guiding them through rigorous preclinical development is central to our mission.”

Dr. Albrecht agreed, saying:

“Kinea Bio’s innovative dual-vector approach has already demonstrated compelling preclinical results, and we are excited to help accelerate its translation toward the clinic.”

In their latest press-release to simultaneously announce funding from the Jain foundation and a license for a myotropic capsid, AAV-SLB101, from Solid Biosciences, Casey Childers, DO, PhD, Chief Executive Officer of Kinea Bio, explains:

“Combining the Jain Foundation’s support with Solid’s capsid technology creates a powerful foundation for success. 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.”

For the past 20 years, the Jain Foundation has led the advancement of knowledge and drug discovery in dysferlinopathy by funding over 150 projects. We are excited by the advancements made by Kinea Bio and the potential it holds for our patient community. Learn more about Kinea Bio at  https://kineabio.com/