SAN DIEGO, May 15, 2023 – Locanabio, Inc., a genetic medicines company developing RNA-targeted therapeutics for patients with rare genetic neuromuscular and neurodegenerative diseases, today announced that CureDuchenne Ventures, the strategic investment arm of CureDuchenne, has made an equity investment in Locanabio. This investment will support the development of the company’s snRNA-mediated exon skipping programs intended to restore muscle health in people living with DMD.
“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, Ph.D., Locanabio’s chief executive officer. “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. Our exon 51 program, which enables the production of a near full-length dystrophin protein, is in IND-enabling studies and we look forward to advancing product candidates for additional exon mutations leveraging our snRNA-mediated exon skipping approach in DMD.”
“CureDuchenne Ventures is delighted to support Locanabio as part of our mission to fund ground-breaking research and facilitate its translation into better therapies for patients with DMD,” said Debra Miller, founder and chief executive officer of CureDuchenne. “Our support of Locanabio’s work in snRNA-mediated exon skipping is a logical next step after our funding of Dr. Kevin Flanigan’s work at Nationwide Children’s Hospital exploring snRNA-mediated exon skipping to treat Duchenne patients harboring a duplication of exon 2 of the dystrophin gene which provided early clinical proof of concept. We strongly believe that Locanabio’s approach has the potential to lead to meaningful therapeutic advances that could transform the lives of people with DMD whose disease is amenable to exon skipping.”
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% 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.
Locanabio’s Vectorized snRNA Platform
Locanabio is using engineered small nuclear RNAs, or snRNAs, delivered using AAV gene therapy to target disease-causing RNA with a one-time administration. snRNAs target RNA exclusively and precisely and are non-immunogenic. They can effect a wide variety of mechanisms including exon-skipping to either restore a reading frame or regulate mRNA and protein expression, target disease causing toxic RNA repeat sequences and recruit endogenous adenosine deaminases acting on RNA (ADARs) for therapeutic RNA editing. Their small size allows the delivery of multiple snRNAs in a single AAV vector which can be used to increase potency or allow targeting of multiple RNAs.
About Duchenne Muscular Dystrophy (DMD)
DMD is a rare fatal X-linked recessive degenerative neuromuscular disorder caused by mutations in the dystrophin gene. The disease affects approximately 1 in every 3,500 to 5,000 males born worldwide.
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.
About Locanabio, Inc.
Locanabio is a leader in developing a new class of genetic medicines that has the potential to significantly improve the lives of patients with devastating genetic diseases by correcting the message of disease-causing RNA. Our proprietary platform uses gene therapy to deliver RNA-binding systems, including snRNA, Cas13d and PUF, that can be engineered to selectively manipulate disease-causing RNA by multiple mechanisms. Our systems are designed to provide a durable therapy with a single administration without altering a cell’s DNA. Locanabio’s platform has applications across a range of tissues and diseases, and we are currently advancing programs in rare genetic neuromuscular and neurodegenerative diseases. For more information, visit www.locanabio.com.
About CureDuchenne Ventures
CureDuchenne Ventures, LLC is the investment arm of CureDuchenne, a non-profit patient advocacy organization and global leader in funding research, patient care, and innovations for improving and extending the lives of those with Duchenne muscular dystrophy (DMD). CureDuchenne Ventures has supported transformative treatments for DMD, including 17 projects that advanced to human clinical trials, and multiple equity investments in companies striving to overcome the limitations of first-generation exon-skipping and gene therapy. For more information, please visit cureduchenne.org.