RARE Daily

PPMD Venture Pathways Invests $500,000 in Kinea Bio Next-Gen Gene Therapy Development

February 14, 2024

Rare Daily Staff

PPMD Venture Pathways, the venture philanthropy arm of Parent Project Muscular Dystrophy, said it has invested $500,000 in Kinea Bio to 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.

The funding will enable Kinea to complete studies needed to secure funding required to advance its experimental therapy to the clinic.

Duchenne muscular dystrophy 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 versions of the dystrophin producing gene into the muscle cells, where no dystrophin is produced.

One limitation to existing gene replacement strategies for Duchenne is the limited packaging capacity of AAV, which means a shortened micro-dystrophin transgene must be used. While micro-dystrophin proteins can provide benefit to patients, they are missing significant protein domains compared to the full-length dystrophin protein.

Additionally, the current gene therapy strategies require high doses of viral vector to effectively get the micro-dystrophin transgenes into muscle. These high vector doses can increase the safety risk associated with receiving the therapy.

Kinea uses a dual vector approach to enable delivery of a larger construct (called a midi-dystrophin) to individual muscle cells. This midi-dystrophin is a larger dystrophin protein, containing more key domains of the dystrophin protein than current micro-dystrophins, and preclinical data suggests it may provide more benefit to muscle.

It also uses next-generation AAVs that increase the efficiency of viral delivery to muscle cells, which lowers the total AAV doses and decreases the risk of serious adverse events. It also uses miniaturized and highly active muscle-specific expression cassettes to produce high levels of midi-dystrophin exclusively in muscle cells.

“Our goal is to use a midi-dystrophin transgene, which contains more functional protein domains than current micro-dystrophins, along with a muscle regulated and targeted AAV to increase transduction efficiency to muscle tissue and dramatically improve muscle function and quality of life for patients living with this genetic disorder,” said Jeff Chamberlain, chief scientific advisor of Kinea Bio. “Our gene therapy approach potentially overcomes many of the limitations of currently approved and investigational micro-dystrophin replacement therapies and builds on the learnings and research from my lab that has helped to shape where gene therapy for Duchenne is today.”

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