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Foundation Supports Muscular Dystrophy RNA Therapy

Nerve cells illustration

(Colin Behrens, Pixabay)

12 June 2019. A university spin-off company is receiving a foundation grant to advance its Crispr technology targeting RNA to treat a form of muscular dystrophy. Locana Inc. in San Diego is the recipient of a $550,000 venture philanthropy award from Muscular Dystrophy Association.

Locana is a 3 year-old spin-off enterprise from University of California in San Diego, and the computational biology and neuroscience lab of Eugene Yeo, a scientific co-founder of the company. Yeo’s lab studies gene expression through RNA and its role in the development of disease, including inherited neuromuscular disorders. A key focus of the group is RNA expressed by mutations that turns toxic to nerve cells, and implicated in inherited diseases such as myotonic dystrophy, Huntington’s disease, and inherited forms of amyotrophic lateral sclerosis or ALS.

Among the tools developed by Yeo and colleagues is the gene-editing technique Crispr, short for clustered regularly interspaced short palindromic repeats. Most Crispr applications edit DNA with RNA-guided enzymes, mainly an enzyme called Cas9. The Yeo lab adapts Crispr for editing components of RNA expressed by mutations responsible for the disease-causing toxicity to nerve cells.

In a paper published in the journal Cell in August 2017, a team from Yeo’s lab used Crispr and Cas9 to edit RNA expressed by mutations responsible for myotonic dystrophy, an inherited neurological disorder and a form of muscular dystrophy. With edited RNA, the researchers were able to stop production in lab cultures of toxic proteins in muscle cells of patients with myotonic dystrophy and other neuromuscular disorders.

The Muscular Dystrophy Association award aims to advance the work published in Cell closer to the clinic. The project is led by Ron Batra, vice-president for R&D at Locana and first author of the Cell paper. Lynn O’Connor Vos, president and CEO of Muscular Dystrophy Association says in a joint statement, “This gives us hope that novel approaches, such as Locana’s for designing highly specific RNA-targeting therapeutic candidates, for treatment of myotonic dystrophy can address the significant unmet needs for patients who live with this genetic disease.”

The $550,000 funding is made under the foundation’s venture philanthropy program. The program supports higher-risk therapy development, where the degree of risk or small patient population may limit conventional investor interest. Muscular Dystrophy Association says it structures the financing much like venture capital, with specified milestones that grant recipients are expected to meet.

Locana licenses the Yeo lab technology from UC-San Diego, and its chief technology officer is co-founder David Nelles, a former graduate student in Yeo’s lab and co-author of the Cell paper. The company received a large financial boost last month from $55 million raised in its first venture funding round. The venture financing was led by life science investment company ARCH Venture Partners, and joined by several other investors including GV, formerly Google Ventures.

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