Gene editing, CRISPR specifically, has become much more well-known and heavily used in the recent past. Advances in this field have allowed medical professionals to treat conditions that had seen no successful therapies in the past. Now, Scribe Therapeutics wants to push it to do more. They have raised $100M to further develop their CRISPR technology for the treatment of amyotrophic lateral sclerosis (ALS).
Amyotrophic lateral sclerosis (ALS) is a progressive, neurological disease in which nerve cells in the brain stem, brain, and spinal cord deteriorate. Due to this deterioration, muscles weaken and people lose control of them and their voluntary movement. In the late stages of ALS, the muscles necessary for breathing weaken, resulting in death. There are two forms of this disease: sporadic and familial. The former is the most common, with 90-95% of cases falling into this category. In the familial form of the disease, it is known that a mutated gene is inherited from parents, but it is still not fully understood and only accounts for 5-10% of cases. Otherwise, the cause of ALS is unknown. Researchers believe that there is a connection between frontotemporal dementia and ALS. Another theory is that exposure to certain substances or toxins leads to the development of ALS.
Symptoms of ALS vary between individuals. They also worsen as the disease progresses. Symptoms begin with difficulty with small movements and everyday things like walking. At the onset of the disease, people may trip and feel weakness in their arms, hands, and legs. As it progresses, people experience difficulties with speaking and swallowing, slowed and slurred speech, twitches and cramps in the muscles, and difficulty holding good posture. In the later stages people will be unable to move their muscles gradually, which affects the entire body. This inability affects movements like blinking. While people with ALS experience loss of muscle function, they do not lose any of their cognitive abilities.
CRISPR For ALS
Scribe is focusing on improving its CRISPR technology so that it is better able to treat neurodegenerative diseases like ALS. This technology is meant to remove, add, or otherwise change a certain section of one’s DNA, making it perfect for treating rare, genetic disorders.
Using X-editing technology, a gene-editing platform created by Scribe, this company is able to curate treatments for diseases previously thought to be incurable. One example is amyotrophic lateral sclerosis (ALS).
In 2020, Biogen and Scribe came to an agreement to dedicate $400 million to find a CRISPR-based treatment for ALS. They’re focusing specifically on the underlying causes of the disease. If they make discoveries that could aid in the drug development or treatment of other neurodegenerative disorders, they will work to do so as well.
Now, an additional $100 million will go towards this research. Money came from Andreessen Horowitz, Avoro Capital Advisors, Avoro Ventures, Wellington Management, OrbiMed Advisors, T. Rowe Price Associates, Perceptive Advisors, Menlo Ventures, and RA Capital Management.
Hopefully this money and research will lead to a viable treatment of ALS. Read more here.