Research & Development
Klotho Neurosciences receives FDA Orphan Drug Designation for ALS gene therapy candidate
10 July 2025 -

US biogenetics company Klotho Neurosciences, Inc. (NASDAQ:KLTO) announced on Thursday that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation to KLTO-202, its lead gene therapy candidate for the treatment of amyotrophic lateral sclerosis (ALS).

The designation applies to KLTO-202, also known as s-KL-AAV.myo, a gene therapy using a muscle-specific promoter to deliver the secreted-Klotho (s-KL) protein to neuromuscular junctions affected by motor neuron degeneration.

Orphan Drug Designation is reserved for treatments targeting rare diseases affecting fewer than 200,000 people in the United States and includes development incentives such as tax credits, market exclusivity for seven years, and fee waivers.

ALS, also referred to as Lou Gehrig's disease, is a fatal neurodegenerative condition with approximately 5,000 new US cases annually and currently has no cure.

Klotho Neurosciences has completed proof-of-concept studies in two animal models of ALS and is beginning manufacturing of the clinical candidate. KLTO-202 is not yet approved for human use by any regulatory authority.

The company plans to engage with the US FDA and European Medicines Agency to align on the next stages of development.

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