Klotho Neurosciences Advances Gene Therapy for ALS Treatment
Klotho Neurosciences, Inc. (NASDAQ: KLTO) is taking a major step forward in its mission to develop innovative therapies for neurodegenerative diseases. The company has announced its plans to progress with the manufacturing and process development of KLTO-202, a novel investigational gene therapy aimed at treating amyotrophic lateral sclerosis (ALS).
The foundation of KLTO-202 is based on a unique RNA splice variant of the human gene known as alpha-Klotho. This gene has been licensed from the Autonomous University of Barcelona and includes patents, applications for patents, and a wealth of research and intellectual property. Alpha-Klotho itself is a five-exon gene encoded within cells throughout the human body, leading to two distinct protein isoforms: membrane-bound Klotho (m-KL), primarily functioning in kidneys to regulate phosphate balance, and secreted alpha-Klotho (s-KL), which plays a neuroprotective role primarily in the brain and spinal cord neurons.
Recent studies conducted on mice and non-human primates over the past two years have provided compelling evidence of s-KL's therapeutic potential. These studies have indicated significant therapeutic outcomes for ALS, rapid aging, and Alzheimer’s disease models, supporting the efficacy of increasing s-KL protein levels through gene therapy.
The timeline for Klotho Neurosciences is ambitious, projecting approximately eight months for the complete process development and manufacturing of KLTO-202. Following this period, the company anticipates an additional four to six months for essential FDA meetings, preclinical animal safety studies, and the submission of an investigational new drug application (IND). The goal is to initiate Phase I/II clinical trials with ALS patients by the third quarter of the upcoming year.
The company's strategy involves collaboration with contract research organizations (CROs), which will enable them to streamline the manufacturing and clinical trial processes without incurring significant operational overhead by avoiding extensive hiring.
Dr. Joseph Sinkule, the founder and CEO of Klotho Neurosciences, expressed optimism about the company’s recent fundraising success. He highlighted a more efficient method of producing the AAV vector required to deliver the s-KL gene to motor neurons—cells that are significantly affected in ALS cases. The primary objective remains to boost local s-KL protein levels, thereby shielding these neurons from damage, a process that can lead to muscle paralysis and even death in ALS patients.
Given ALS's aggressive progression, often resulting in loss of mobility and life within just a few years of diagnosis, the urgency of this development cannot be overstated.
About Klotho Neurosciences, Inc.
Klotho Neurosciences is dedicated to advancing the frontiers of biogenetics by developing innovative gene and cell therapies. Their unique focus on the human Klotho gene—a protein associated with aging—positions them at the forefront of treatments for various neurodegenerative and aging-related diseases. The clinical development of KLTO-202 is just one of many avenues the company is exploring through its proprietary research and therapeutic programs.
For more information on Klotho Neurosciences and their ongoing research, you can visit their official website at
www.klothoneuro.com. Investors can reach out to Jeffrey LeBlanc, CFO, for corporate communications and inquiries.
This announcement comes with forward-looking statements about their expected operational strategies and projected timelines; thus, stakeholders are encouraged to consider potential risks and uncertainties related to the company's future endeavors. Overall, the advancement of KLTO-202 reinforces the ongoing commitment of Klotho Neurosciences to transform the landscape of ALS treatment and improve the lives of those affected by this devastating disease.