RARE Daily

Locanabio Gets Equity Investment from CureDuchenne Ventures

May 15, 2023

Rare Daily Staff

Locanabio said that the venture philanthropy fund CureDuchenne Ventures has made an equity investment in Locanabio to support the development of the company’s snRNA-mediated exon skipping programs intended to restore muscle health in people living with Duchenne muscular dystrophy.

The company did not disclose the amount of the investment.

Duchenne muscular dystrophy (DMD) is a rare and fatal neuromuscular disorder caused by mutations in the dystrophin gene. The dystrophin gene is the largest human gene. DMD causing mutations can occur at various places in the gene and most result in large exon deletions or duplications and dysfunctional dystrophin protein. Dystrophin plays a key structural role in muscle. It is one of a group of proteins whose function is to strengthen muscle fibers and protect them from injury as muscles contract and relax. Without it, muscle cells become damaged which leads to muscle wasting. Patients with DMD experience progressive muscle wasting, difficulty controlling movement, respiratory failure and heart failure leading to full time wheelchair use in teens and early 20’s and reduced life expectancy.

Locanabio’s exon skipping approach for DMD uses its vectorized snRNA platform in which an AAV vector is used to deliver engineered snRNAs that target multiple splicing regulatory sites in the dystrophin mRNA to promote efficient exon skipping and production of a minimally truncated and functional dystrophin protein. Exon skipping therapies have the potential to offer clinical benefit for the approximately 80 percent of DMD patients whose disease is caused by dystrophin mutations that are amenable to exon skipping. Locanabio expects to present additional data at an upcoming peer-reviewed scientific conference.

“CureDuchenne is a leader in funding pioneering research aimed at advancing the best possible therapies for DMD patients, and we value their vote of confidence and financial support of our snRNA-mediated exon skipping platform,” said Jim Burns, Locanabio’s CEO. “Locanabio has developed a proprietary snRNA-AAV packaging and manufacturing approach that addresses the challenges of delivering multiple snRNAs, enabling us to target multiple exons or multiple sites in a single exon for enhanced exon skipping efficiency and opening up the full potential of snRNAs in therapeutic applications.”

Photo: Jim Burns, CEO of Locanabio

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