On October 6, 2020, molecular engineering company Scribe Therapeutics announced an exciting partnership. Originally founded by molecular engineers and CRISPR inventors, Scribe Therapeutics seeks to develop in vivo genetic medicine to treat patients with genetic disorders. Now, alongside Biogen, Scribe Therapeutics is seeking to develop CRISPR-related therapies to address the underlying cause of amyotrophic lateral sclerosis (ALS).
For years, researchers admitted there is no clear cause for ALS. However, many hypothesized that it could be cause by genetic mutations. Since approximately 5-10% of ALS cases are familial, there are some genetic variances associated with the condition. Through this collaboration, Biogen will provide Scribe Therapeutics with at least $15 million. However, as Scribe Therapeutics continues to develop customized CRISPR molecules, and editing with their proprietary X-Editing platform, the company could receive up to $400 million to assist with development.
Amyotrophic Lateral Sclerosis (ALS)
Also known as locked-in syndrome and Lou Gehrig’s disease, amyotrophic lateral sclerosis (ALS) is a progressive neurological disease characterized by brain stem, spinal cord, and brain nerve cell death. As these nerve cells die, patients experience continued muscle weakness. Eventually, ALS causes patients to lose voluntary muscle control. In many cases, patients lose the ability to breathe after their chest muscles are weakened. Generally, this condition affects males more than females. It is most common in white men between ages 60 and 69. Caucasians are also more likely to develop ALS than other ethnic or racial groups.
Symptoms and severity vary. Additionally, they are usually considered bulbar onset (beginning in the mouth or tongue, usually with speaking or swallowing difficulties) or limb onset (weakness or twitching in the arms or legs). However, symptoms usually include:
- Changes in speech (slurring, slowed speech)
- Difficulty swallowing or speaking
- Frequent tripping or falling
- Muscle twitching, particularly in the tongue, arms, shoulders, and legs
- Muscle weakness across the body
- Difficulty holding small objects or performing other small movements
- Muscle pain or cramping
- Changes in posture
- Depression and anxiety
Learn more about ALS.
