Autologous bone marrow derived MSCs therapy(Corestem)

Recently, Biogen Inc. announced the outcome of the FDA Peripheral and Central Nervous System Drugs Advisory Committee meeting on tofersen, an investigational product for the treatment of superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS). The advisers voted 9-0 that the drug’s effect on an important protein linked to nerve damage was “reasonably likely” to predict a clinical benefit — a conclusion that aligns with the FDA's standards for granting a so-called accelerated approval. Yet when asked whether the available data provide “convincing evidence” of the medicine’s effectiveness, the group, which mostly consists of experts in neurology and clinical science, disagreed in a 5-3 vote. The ninth member abstained. While the FDA isn’t required to follow the recommendations of its advisory committees, it typically does. The agency is expected to issue a verdict on the medicine, which is being developed by Biogen under the name tofersen, by April 25. About Tofersen Tofersen is an antisense oligonucleotide (ASO) being evaluated as a treatment for SOD1-ALS. In people with this form of the disease, mutations in their SOD1 gene cause their bodies to create a toxic form of SOD1 protein. This toxic protein causes motor neurons to degenerate, resulting in progressive muscle weakness. Tofersen is designed to bind to SOD1 mRNA and reduce SOD1 protein production. Tofersen is currently in development for only one indication, ALS, and is in NDA in the US and Phase 3 in other countries. About Amyotrophic Lateral Sclerosis (ALS) Amyotrophic lateral sclerosis (ALS) is an ultra-rare, progressive and fatal neurodegenerative disease that results in the loss of motor neurons in the brain and the spinal cord that are responsible for controlling voluntary muscle movement. People with ALS experience muscle weakness and atrophy, causing them to lose independence as they steadily lose the ability to move, speak, eat, and eventually breathe. Average life expectancy for people with ALS is three to five years from time of symptom onset. SOD1-ALS is diagnosed in approximately 2 percent of all ALS cases, impacting about 330 people in the United States. Currently, there are no genetically targeted treatment options for ALS. Search the drug intelligence database: Synapse, a total of 346 drugs and 969 clinical trials were found for the treatment of Amyotrophic lateral sclerosis. For this indication, the top three research and development organizations are Sanofi, CORESTEMCHEMON, Inc. and Amylyx Pharmaceuticals, the major R&D targets are focused on BAX、HDAC and SCNA. The top three types of drug development for this indication are Small molecule drug, Mesenchymal stem cell therapy and Oligonucleotide antisense. There are four drugs approved for ALS: Riluzole、Edaravone、Lenzumestrocel (CORESTEM) and Sodium Phenylbutyrate/Tauroursodeoxycholic Acid. If you’re interested in learning more about this space or keeping track of drug development and clinical trials, sign up for Synapse（synapse.patsnap.com）, our freemium product offering.