Klotho Neurosciences Secures FDA Orphan Drug Designation for ALS Treatment with KLTO-202

Klotho Neurosciences Receives FDA Orphan Drug Designation for KLTO-202



Klotho Neurosciences, Inc., a company specializing in gene and cell therapy, has recently announced a significant achievement in its fight against Amyotrophic Lateral Sclerosis (ALS), also known as Lou Gehrig's Disease. The U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for the company’s innovative product, KLTO-202, which is particularly promising for the treatment of this neurodegenerative disorder that primarily affects motor neurons.

What is Orphan Drug Designation?


The FDA’s Orphan Drug Designation is a special status that promotes the development of medications intended for the treatment of rare diseases, affecting fewer than 200,000 individuals in the U.S. This designation provides Klotho Neurosciences with several incentives, including tax credits for clinical trial costs and a waiver for the associated user fee, ultimately benefiting the commercialization of their promising therapy.

Understanding KLTO-202


KLTO-202, also recognized as s-KL-AAV.myo, is a novel therapeutic approach derived from the secreted-Klotho (s-KL) promoter. This gene therapy aims to address the neurological damage that ALS inflicts on motor neurons and offers the potential for neurological protection through elevated levels of the s-KL protein in blood, brain, and muscle tissues. Dr. Joseph Sinkule, CEO of Klotho Neurosciences, expressed a personal connection to the disease, recalling his cousin's struggle with ALS, affirming the mission to develop effective treatments for individuals facing this devastating condition.

The Statistics Behind ALS


ALS, named after baseball great Lou Gehrig, impacts a significant number of people in the U.S., with approximately 5,000 new diagnoses each year. The condition is marked by progressive muscle weakness, leading to severe disability and ultimately death. This underscores the urgent need for effective therapies, especially considering that ALS can strike seemingly healthy individuals, as evidenced by Gehrig’s own history of athletic excellence before his diagnosis.

Scientific Validation and Future Plans


Klotho Neurosciences has completed

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