Scribe, Biogen Partner to Develop Gene Therapy for ALS, Other Neurological Diseases

Patricia Inácio, PhD avatar

by Patricia Inácio, PhD |

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Scribe Therapeutics and Biogen, a biotechnology company focused on neurological diseases, have established a research partnership to develop new gene therapies for amyotrophic lateral sclerosis (ALS) using the gene-editing technology CRISPR-Cas9.

The collaboration will focus initially on the development and commercialization of CRISPR-based therapies that address some of the underlying genetic causes of ALS. However, the partnership between the two companies eventually may extend to other neurological diseases that currently lack effective treatments.

“We’re proud to collaborate with Biogen and apply our uniquely customized approaches with the goal of developing new, safe and effective genetic medicines for neurodegenerative disease,” Benjamin Oakes, CEO and co-founder of Scribe Therapeutics, said in a press release.

Mutations in several genes have been associated with both familial and sporadic forms of ALS. The C9orf72 gene is the most frequently mutated gene in ALS patients, along with the SOD1, TARDBP and the FUS genes. Additional genes relating to ALS include VCP, ATXN2, NEK1, ANG, TBK1VAPB, and SQSTM1, among others.

While it is not fully understood how these mutations contribute to ALS, therapeutic approaches that help lower the levels of mutated proteins encoded by these genes were found to be beneficial in animal models of the disease.

First discovered as a molecular defense system of microbes to fend off viruses, the gene editing tool CRISPR-Cas 9 has since been explored as a technology to rewrite human genes. Conventional CRISPR uses the enzyme Cas9 that recognizes and cuts a specific portion of DNA, like scissors, to remove or alter sequences that make genes go awry.

Founded by the scientists who originally invented CRISPR, Scribe has developed novel, custom-CRISPR tools, using a technology called X-Editing. This advanced gene-editing platform has greater editing activity and higher specificity, meaning that it can edit the target gene with greater precision than other CRISPR tools currently available.

“Scribe has designed, engineered and tested thousands of evolved CRISPR enzymes to build an advanced platform for creating breakthrough in vivo treatments,” Oakes said.

As part of the collaboration, Scribe will receive a $15 million upfront payment from Biogen. The company also will be eligible to receive more than $400 million from the potential clinical and commercial development of the neurological-target gene therapies created within the partnership, as well as other tiered royalty payments.