Drug Profile

KLTO 202

Alternative Names: AAVmyo.desmin.s-KL; KLTO-202

Latest Information Update: 24 Feb 2026

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At a glance

Originator Klotho Neurosciences
Class Gene therapies
Mechanism of Action Gene transference

Orphan Drug Status

Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

Yes - Amyotrophic lateral sclerosis
New Molecular Entity Yes

Highest Development Phases

Preclinical Amyotrophic lateral sclerosis; Isaacs syndrome; Multiple sclerosis; Neuromuscular disorders

Most Recent Events

18 Feb 2026 Klotho Neurosciences receives patent claims for composition and use of human secreted Klotho protein ("s-KL") isoform of the human Klotho gene sequence in Australia
22 Jul 2025 Klotho Neurosciences and AAVnerGene agree to develop KLTO 202
10 Jul 2025 KLTO 202 receives Orphan Drug status for Amyotrophic lateral sclerosis in USA

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