Parent Project Muscular Dystrophy (PPMD), a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne), is excited to announce that PPMD has provided $500,000 in funding to Kinea Bio, Inc. (Kinea) through PPMD Venture Pathways, the organization’s venture philanthropy program which provides industry funding to accelerate therapeutic development.
This funding commitment aims to address current limitations in gene therapy delivery and will advance the development of the company’s next-generation adeno-associated virus (AAV) mediated midi-dystrophin gene replacement therapy for the treatment of individuals with Duchenne by enabling Kinea to complete critical studies needed to secure funding required to advance their investigational product to the clinic.
Duchenne is the most common genetic disorder diagnosed in childhood, affecting approximately one in 5,000 live male births. Duchenne is caused by a change in the DMD gene that codes for the dystrophin protein. Gene therapy holds the promise of providing benefit to patients with Duchenne by introducing replacement (shortened) versions of the dystrophin producing gene into the muscle cells, where no dystrophin is produced.