Core Viewpoint - Klotho Neurosciences, Inc. has received Orphan Drug Designation from the FDA for its gene therapy product KLTO-202, aimed at treating ALS, highlighting the company's commitment to developing innovative treatments for rare neurodegenerative diseases [1][3][5]. Group 1: Company Overview - Klotho Neurosciences, Inc. focuses on gene and cell therapies for neurodegenerative and aging-related diseases, utilizing a patented form of the "anti-aging" human Klotho gene [7]. - The company is developing KLTO-202, which targets motor neuron diseases and muscular dystrophies through a muscle-specific promoter and aims to deliver therapeutic concentrations of the s-KL protein [6][7]. Group 2: Orphan Drug Designation - The FDA's Orphan Drug Designation is granted to treatments for rare diseases affecting fewer than 200,000 people in the U.S., providing incentives such as tax credits and seven years of market exclusivity [2]. - The designation for KLTO-202 validates the scientific approach of Klotho Neurosciences in addressing ALS, a disease with significant unmet medical needs [2][3]. Group 3: ALS Context - ALS, also known as Lou Gehrig's disease, affects fewer than 200,000 people in the U.S., with approximately 5,000 new cases diagnosed annually [4]. - The disease is characterized by motor neuron damage, leading to severe physical decline and is universally fatal [3][4]. Group 4: Development Progress - Klotho Neurosciences has completed proof of concept studies in animal models and is initiating manufacturing of KLTO-202, with plans for further discussions with the FDA and EMA regarding development [5].
Klotho Neurosciences, Inc. Granted FDA Orphan Drug Designation for KLTO-202 for Treatment of Amyotrophic Lateral Sclerosis ("ALS" or "Lou Gehrig's Disease")