KLTO-202 / Klotho Neurosciences - LARVOL DELTA

Klotho advances ALS gene therapy KLTO‑202 to manufacturing phase (ALS News Today) - "Klotho Neurosciences is advancing KLTO‑202, its gene therapy candidate for amyotrophic lateral sclerosis (ALS), to the manufacturing and process development phase — with an eye toward trials in humans beginning next year...The manufacturing phase is expected to last about eight months, and will be followed by 4-6 months of regulatory preparation, including meetings with the U.S. Food and Drug Administration (FDA), Klotho stated...After that will be the completion of animal safety studies required by the FDA, and the filing of an application to the regulatory agency requesting clearance to start clinical testing of the gene therapy...If all goes according to plan, the company expects to launch its first human study — a single-dose trial testing KLTO‑202 in people with ALS — by the third quarter of next year."

Commercial • New trial • Amyotrophic Lateral Sclerosis

Klotho Neurosciences, Inc. Granted FDA Orphan Drug Designation for KLTO-202 for Treatment of Amyotrophic Lateral Sclerosis ("ALS" or "Lou Gehrig's Disease") (PRNewswire) - "Klotho Neurosciences, Inc...announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to the company's novel secreted-Klotho (s-KL) promoter, gene and delivery system (KLTO-202, or s-KL-AAV.myo) for the treatment of ALS...Klotho Neurosciences will have completed 'proof of concept' studies in two animal models of human ALS and the Company is currently initiating manufacturing of the ALS-targeted product candidate, followed by meetings with the U.S. FDA and EMA in Europe to concur with the development path going forward."

Orphan drug • Amyotrophic Lateral Sclerosis

Klotho Neurosciences Moves Forward with Manufacturing Gene Therapy for the Treatment of ALS (PRNewswire) - "Animal studies supported by the Company over the past two years in mouse and non-human primate models of rapid aging, in models of human Alzheimer's disease, and in models of ALS have shown that over-expression and amplification of the tissue levels of s-KL using a gene therapy approach result in highly favorable therapeutic outcomes in every model tested...The Company expects that it will take approximately eight months to complete process development and manufacturing of KLTO-202, and about four to six months to conduct meetings with FDA, complete all FDA-mandated animal safety studies, file an investigational new drug application (IND), train and prepare clinical sites where the Phase I/II studies can be conducted, and then begin the single-dose gene therapy studies in ALS patients by the third quarter of next year."

IND • New P1/2 trial • Preclinical • Amyotrophic Lateral Sclerosis

ANEW MEDICAL, INC. Announces Patents Issued in Major Asian Markets for use of Genetic Therapy in the Treatment of Alzheimer’s Disease and ALS (GlobeNewswire) - "ANEW MEDICAL, INC...announces the grant and issuance of patent number HK1259628 protecting the development of novel genetic approaches for the treatment of neurodegenerative diseases to include Alzheimer’s disease, Parkinson’s diseases and amyotrophic lateral sclerosis ('ALS')...'We are particularly excited about the preclinical results and plan to begin human testing in late 2025.'"

New trial • Patent • Alzheimer's Disease • Amyotrophic Lateral Sclerosis • CNS Disorders • Parkinson's Disease

ANEW MEDICAL to Advance Patented Klotho Gene Therapy for Neurodegenerative Disorder Treatments (GlobeNewswire) - "ANEW MEDICAL, INC...announces plans to advance its recently patented Klotho gene therapy program for neurodegenerative disorders. ANEW has compelling initial data suggesting that maintaining elevated levels of Klotho in the body significantly contributes to longer, healthier lifespans...ANEW will also investigate other age-related disorders associated with depleted Klotho levels...ANEW has development plans to advance its program in three distinct areas: FDA Clinical Trials: The development of therapeutics to treat neurodegenerative diseases and age-related disorders involves using an adenovirus (AAV) vector to deliver the secreted Klotho gene DNA sequence to neurons in the central nervous system (CNS). This approach aims to correct deficiencies in the levels of s-KL"

Pipeline update • Alzheimer's Disease • Amyotrophic Lateral Sclerosis • CNS Disorders • Parkinson's Disease