San Diego-based Locanabio secured $100 million in a Series B financing round that will be used to advance the companys portfolio of novel RNA-targeted gene therapies for neurodegenerative, neuromuscular and retinal diseases.

The funding will support pre-clinical and clinical development of its gene therapy treatments for diseases such as Huntington's disease, myotonic dystrophy type 1, genetic forms of amyotrophic lateral sclerosis and retinal disease, the company said.

Locanabio has a unique approach to gene therapy. The company has combined two validated gene therapy and RNA modifications to treat diseases. Locanabio uses a gene therapy vector to deliver an RNA-targeting protein tipped with an RNA-modifying enzyme. Through targeting RNA, the company said its approach avoids the risk of off-target effects in DNA and is suited to address many diseases linked to dysfunctional processing of RNA.

The $100 Million Series B builds on $55 million the company secured in a Series A last year. Chief Executive Officer Jim Burns, who joined Locanabio one year ago, said the financing round will allow the company to advance several of the companys most promising programs into IND-enabling studies in 2021. The financing will also allow the company to continue to advance its RNA-targeting platform, which has the potential to be a major new advance in medicine that can bring hope to patients with many devastating genetic diseases, Burns said.

While all of Locanabios assets are still in the research phase, its most advanced is a therapy for myotonic dystrophy type 1 (DM1), a genetic neuromuscular disorder caused by a mutation in the DMPK gene that results in trinucleotide (CUG) repeat expansion in the expressed RNA. Locanabios DM1 program targets and destroys the toxic CUG repeats, according to company information. Earlier this year, as BioSpace previously reported, Locanabio published a paper demonstrating the benefits of its technology as a potential one-time treatment of DM1.

The financing round was led by Vida Ventures LLC with participation from RA Capital Management, Invus, Acuta Capital Partners, an investment fund associated with SVB Leerink Prior Locanabio investors ARCH Venture Partners, Temasek, Lightstone Ventures, UCB Ventures and GV, also participated in the financing round.

"We are pleased that a team of highly sophisticated investors led by Vida Ventures has joined in this financing round, further validating our progress in research and the significant potential of our unique RNA-targeting platform, Burns said in a statement.

With the Series B, Rajul Jain, director of Vida Ventures, joined Locanabio's board of directors. Before Vida Ventures, Jain served on the executive team and headed development for Kite Pharma and was previously global development lead for Amgen.

The unique approach in RNA targeting using gene therapy to deliver RNA binding proteins developed by Locanabio represents the next frontier of genetic medicine with the ability to target the root cause of a range of genetic diseases, Jain said in a statement. They have built a strong management team to execute this bold vision and we are proud to support them.

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Locanabio Raises $100 Million to Advance RNA-Targeted Gene Therapies - BioSpace

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