Oct 6 (Reuters) - U.S. drugmaker Biogen Inc and gene-editing startup Scribe Therapeutics Inc will jointly develop therapies for treating underlying genetic causes of amyotrophic lateral sclerosis (ALS), a fatal neurological disorder better known as Lou Gehrig’s disease.
Scribe, co-founded by Jennifer Doudna, an early pioneer of the CRISPR gene-editing technology, said on Tuesday it would receive $15 million upfront and potentially over $400 million in milestone payments from Biogen.
Gene-editing tools such as CRISPR/Cas9 have been hailed as technologies that can rearrange the genetic code to potentially treat diseases driven by genetic mutations or abnormalities.
Scribe, backed by investors including top Silicon Valley venture capital firm Andreessen Horowitz, aims to custom engineer novel CRISPR molecules for gene editing.
ALS, often called Lou Gehrig’s disease after the New York Yankees player who was diagnosed with the disease, is a rare progressive condition that attacks cells located in the brain and spinal cord.
There are no treatments for the underlying cause of the disease, and current therapy focuses on slowing down the progression of symptoms.
Biogen suffered a setback reut.rs/3nxGFGy in 2013 after a promising experimental drug for ALS failed to work in a clinical trial. (Reporting by Manas Mishra in Bengaluru; Editing by Ramakrishnan M.)