#United States #New York #Klotho Neurosciences #KLTO-202 #AAVnerGene

Klotho Neurosciences, Inc. Advances Gene Therapy Production

In a significant move impacting gene therapy technology, Klotho Neurosciences, Inc. (Nasdaq: KLTO) has announced the initiation of production for its KLTO-202 gene therapy candidate. The company has entered into a binding agreement to utilize the innovative platform technology from AAVnerGene Inc., marking a critical milestone in the development of disease-modifying therapies.

About Klotho Neurosciences and KLTO-202

Klotho Neurosciences is focused on pioneering new treatments for neurodegenerative diseases. The KLTO-202 therapy is anchored in the application of a human gene and protein derived from their patented Klotho gene, often referred to as an 'anti-aging' gene. Known for its significant role in age-related diseases, engineers have aimed to harness its potential for therapeutic use.

The CEO of Klotho, Dr. Joseph Sinkule, hailed this collaboration with AAVnerGene as a major breakthrough. He stated, "The AAVnerGene technology is a game-changer in manufacturing AAV vectors for intracellular delivery of genes." By employing AAVnerGene's cutting-edge 'AAVone' platform technology, Klotho aims to elevate production standards by generating higher concentrations of AAV particles with less impurity, ultimately leading to faster optimization than previous methodologies.

Tackling Gene Therapy Challenges

Developing AAV-vectored gene therapies, however, poses unique challenges, notably the tendency of AAVs to accumulate in the liver, potentially resulting in severe side effects. AAVnerGene has created a solution through its 'ATHENA' platform technology, which develops specific tissue-targeting AAVs, thus improving safety profiles significantly. With KLTO’s emphasis on safety and efficacy, utilizing tissue-specific promoters will be beneficial in mitigating risks traditionally associated with AAV vectors.

Historically, the process of AAV production has involved a cumbersome triple transfection method requiring three different plasmids to be introduced into a HEK293 cell line. This approach was labor-intensive and time-consuming, often prolonging development timelines. AAVnerGene’s introduction of the single-plasmid AAV packaging system, dubbed “AAVone,” promises to revolutionize these practices. This streamlined system enhances production capacity, minimizes impurities, and drastically reduces manufacturing costs, enabling a faster route to clinical applications for gene therapies.

The Path Ahead for Klotho and AAVnerGene

Klotho’s ambitious foray into developing disease-modifying therapies does not end with KLTO-202. Both Klotho and AAVnerGene are committed to advancing a portfolio of gene therapeutics. They aim to address pressing neurological concerns such as ALS, Alzheimer's disease, and Parkinson's disease, broadening the horizon for patients stricken with these debilitating conditions.

The partnership exemplifies how cutting-edge technology can culminate in effective therapy solutions, setting the stage for the future of biotech innovation. As they embark on this journey, the focused development and manufacturing capabilities laid out through this collaboration will be instrumental in reducing time to market for promising therapeutic solutions.

In conclusion, Klotho Neurosciences’ strategic move marks a pivotal moment in the biotechnology landscape, unlocking new opportunities for the application of gene therapy. The collaboration with AAVnerGene not only offers a more effective avenue for production but also enhances the safety and efficacy parameters crucial for the success of therapeutic interventions. Investors and stakeholders are keenly observing the advancements in the coming months as Klotho aims to bring their innovative therapies from the lab to the clinic efficiently.