Scribe, Biogen expand ALS CRISPR agreement

Scribe Therapeutics and Biogen are taking their collaboration up a notch, adding another disease target in the study of a treatment for amyotrophic lateral sclerosis disease (ALS).

The therapy is an expansion of the duo's original agreement signed in October of 2020, to develop CRISPR-based genetic medicines that will address the underlying cause of ALS. According to the original agreement, Scribe received $15 million upfront from Biogen with an additional $400 million in potential development and commercial milestone payments.

Neither company said what the new target will be in the updated agreement, nor mentioned if Scribe will see any more milestone payments.

“At Scribe, we continue to push the boundaries of molecular engineering to fulfill the profound promise of CRISPR-based therapeutics and are pleased to have our collaborators at Biogen continue to support and expand our collaboration towards this goal,” said Benjamin Oakes, CEO and co-founder of Scribe Therapeutics.

Scribe, headquartered outside of San Francisco, focuses on creating gene therapies using CRISPR technology and custom engineered enzymes. The company’s custom platforms help it to overcome the technological hurdles that other companies in the field face, said Oakes.

The collaboration between Scribe and Biogen is not Biogen’s only attempt at finding a cure for ALS. In March of this year, a partnership with Ionis Pharmaceuticals fizzled out after disappointing results from a phase 1 trial. The two were exploring the use of BIIB078, a drug designed based on the hypothesis that disease mechanisms of ALS are associated with toxicity and repeats within RNA and corresponding dipeptides, short proteins that maintain the pH of cells or act as antioxidants. Results from that phase 1 study showed the drug did not produce results any different from the placebo group. In fact, participants who received a 90 mg dose of the drug saw a greater decline than those in the placebo group, Ionis said.