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Locanabio, a frontrunner in the genetic medicines domain, is set to receive an equity investment from CureDuchenne Ventures. This funding signifies a significant leap towards the development of snRNA-mediated exon skipping therapies aimed at restoring muscle health for people battling Duchenne Muscular Dystrophy (DMD).
CureDuchenne Ventures: The Strategic Investment Arm
CureDuchenne Ventures, a strategic investment entity of CureDuchenne, has been a key player in financially supporting innovative research efforts. Their mission is to accelerate the development of optimal therapies for patients suffering from DMD.
Locanabio's snRNA-Mediated Exon Skipping Approach
Locanabio's innovative approach utilizes its proprietary snRNA-AAV packaging and manufacturing methods to effectively deliver multiple snRNAs. This allows for enhanced exon skipping efficiency and reveals the full potential of snRNAs in therapeutic applications.
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Jim Burns, Locanabio's CEO, expressed his appreciation for CureDuchenne's confidence in their platform and their financial backing. He also highlighted their exon 51 program, which is currently undergoing IND-enabling studies.
"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' Support for Locanabio
Debra Miller, the founder and CEO of CureDuchenne, also expressed her enthusiasm about supporting Locanabio's research.
"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."
Locanabio's Exon Skipping Approach and Its Potential
Locanabio's exon skipping approach for DMD utilizes its vectorized snRNA platform. This platform employs an AAV vector to deliver engineered snRNAs that target multiple splicing regulatory sites in the dystrophin mRNA. This promotes efficient exon skipping and production of a minimally truncated and functional dystrophin protein.
The Potential of Exon Skipping Therapies
Exon skipping therapies have the potential to offer clinical benefits for approximately 80% of DMD patients whose disease is caused by dystrophin mutations that are amenable to exon skipping.
Upcoming Data Presentation
Locanabio plans to present additional data at an upcoming peer-reviewed scientific conference.
DMD: A Glimpse at the Disease
DMD is a rare fatal X-linked recessive degenerative neuromuscular disorder caused by mutations in the dystrophin gene. It affects approximately 1 in every 3,500 to 5,000 males born worldwide.
Locanabio's Proprietary Platform: A Game-Changer
Locanabio's proprietary platform utilizes gene therapy to deliver RNA-binding systems that can selectively manipulate disease-causing RNA by multiple mechanisms. This approach has the potential to significantly improve the lives of patients with devastating genetic diseases.
CureDuchenne Ventures: A Global Leader
CureDuchenne Ventures, a global leader in funding research and innovations for improving the lives of those with DMD, has supported transformative treatments for DMD. Their investments have led to 17 projects advancing to human clinical trials, and multiple equity investments in companies seeking to overcome the limitations of first-generation exon-skipping and gene therapy.
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