Locanabio Raises $100M to Advance CRISPR-Mediated RNA-Targeting Therapies

On December 14th, Locanabio announced it had raised $100 million in a Series B financing. The funding will be used to advance the pre-clinical and clinical development of their CRISPR-mediated RNA-targeting gene therapies. The company is currently developing therapies for neuromuscular, neurodegenerative, and ophthalmology diseases.

Founded in 2016 in San Diego, California, Locanabio is a biotechnology company that aims to treat disease by modulating disease-causing RNA. Their approaches used an RNA-targeting CRISPR-Cas9, which is delivered via adeno-associated viral vectors. Their technology is versatile and allows for different approaches depending on the disease. For example, in diseases caused by an increase in nucleotide repeats such as myotonic dystrophy type 1 (DM1), Huntington’s disease, Amyotrophic Lateral Sclerosis, and others, Locanabio aims to destroy these nucleotide repeats. However, they claim this technology can be adapted not only to destroy disease-causing RNA but also to replace it with normal RNA. They are utilizing this approach with an undisclosed gene to treat retinal disease.

Their technology has been validated in pre-clinical models of disease. In a recent publication in the journal of Nature Biomedical Engineering, they demonstrated that their technology can selectively eliminate dysfunctional RNA in a mouse model of DM1. They show that intramuscular or systemic delivery of the therapeutic selectively eliminated the nucleotide repeats and reversed behavioral alteration in the mice with DM1.

By targeting the disease-causing RNA, Locanabio aims to avoid the drawbacks of normal CRISPR therapeutics. Targeting DNA could fix the mutated gene; however, recent studies have discovered that it can also lead to unintended mutations, or activate cancer-driving genes. Alternative approaches to target RNA such as antisense oligonucleotides, RNA-targeting small molecules, and others require frequent dosing and may have off-target effects.

Raising Capital

Last year, during the Series A financing round, Locanabio raised $55 million and they would be pleased to have doubled that amount this time. The Series B financing was led by Vida Ventures, and new investors RA Capital Management, Invus, Acuta Capital Partners, and others. This was in addition to prior investors ARCH Venture Partners, Temasek, Lightstone Ventures, UCB Ventures, and GV (formerly Google Ventures).

“This financing positions us to accelerate our efforts to advance multiple promising programs into IND enabling studies in 2021 and to further develop our novel 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,” said Jim Burns, Ph.D., chief executive officer at Locanabio. “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.”

In addition, Locanabio announced that Dr. Rajul Jain, director of Vida Ventures, will join the board of directors. “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,” said Dr. Jain. “They have built a strong management team to execute this bold vision and we are proud to support them.”

By Daniel Ojeda, Ph.D.

Related Article: Japan’s AnGes Acquires EmendoBio, Eying High Precision Gene-Editing Technology

References

1. https://locanabio.com/press-releases/locanabio-announces-100-million-series-b-financing-to-advance-portfolio-of-novel-rnatargeted-gene-therapies-for-neurodegenerative-neuromuscular-and-retinal-diseases/
2. https://www.prnewswire.com/news-releases/locana-raises-55-million-series-a-financing-to-advance-portfolio-of-rna-targeting-gene-therapies-300854809.html
3. https://www.nature.com/articles/s41551-020-00607-7
4. https://locanabio.com/pipeline/

Author

Daniel Ojeda