Klotho Neurosciences, Inc. Partners with AAVnerGene Inc. to Make Klotho's Gene Therapy Assets

Rhea-AI Summary

Klotho Neurosciences (NASDAQ: KLTO) has announced a strategic partnership with AAVnerGene Inc. to manufacture its gene therapy candidates using AAVnerGene's advanced AAV manufacturing and tissue-targeted delivery technologies.

The collaboration aims to accelerate the development of KLTO's three key candidates: KLTO-101 for Alzheimer's and Parkinson's diseases, KLTO-202 for ALS and motor neuron diseases, and KLTO-303 for aging-related pathologies. AAVnerGene's proprietary platforms - AAVone system and ATHENA platform - promise to deliver more cost-effective, faster manufacturing with higher efficacy, purity, and lower toxicity through improved tissue targeting.

Positive

• None.

Negative

• No immediate revenue generation from early-stage partnership
• Success depends on unproven manufacturing technology integration

Insights

Biotechnology Research Scientist

KLTO partners with AAVnerGene to advance gene therapy candidates using superior manufacturing technology that promises faster development, lower costs, and improved efficacy.

This strategic partnership between Klotho Neurosciences and AAVnerGene represents a significant technological advancement for KLTO's gene therapy pipeline. AAVnerGene brings two proprietary platforms to the collaboration: the AAVone system for manufacturing and the ATHENA platform for tissue-targeted delivery. These technologies directly address the two major bottlenecks in adeno-associated virus (AAV) gene therapy development: high manufacturing costs and high dose-related toxicity.

The technical benefits described are substantial from a development perspective. AAVnerGene's platforms potentially offer KLTO more cost-effective production, faster manufacturing timelines, fewer impurities, higher efficacy, and lower toxicity through improved tissue targeting. These advantages could significantly accelerate the clinical development of KLTO's neurology-focused gene therapy candidates:

• KLTO-101 targeting Alzheimer's and Parkinson's diseases
• KLTO-202 for ALS and other motor neuron diseases
• KLTO-303 for aging-related pathologies

The fundamental science behind this partnership leverages tissue-specific promoters driving the Klotho gene to produce the Klotho protein at elevated concentrations in targeted tissues. The Klotho protein has shown neuroprotective properties and longevity effects in preclinical studies, which aligns with KLTO's focus on neurodegenerative conditions. If successfully implemented, this manufacturing approach could position KLTO to bring potentially transformative one-time treatments to market with improved economics and safety profiles compared to current AAV therapies.

Pharmaceutical Industry Analyst

This partnership addresses a critical industry challenge in the AAV gene therapy space. Despite the revolutionary potential of these one-time treatments, commercial viability has been constrained by two factors: prohibitively high manufacturing costs and dose-dependent toxicity concerns. AAVnerGene's technology appears positioned to tackle both simultaneously.

From a manufacturing economics perspective, the AAVone platform could significantly improve KLTO's production efficiency. Current AAV manufacturing processes are notoriously expensive, with production costs often running into millions per batch. Any substantial improvement in manufacturing efficiency directly enhances gross margins and commercial viability.

The ATHENA platform's improved tissue targeting could reduce required dosing, addressing both safety concerns and cost issues. Lower effective doses mean reduced manufacturing volume requirements and potentially fewer adverse events in clinical trials and commercial use. This dual benefit could substantially de-risk KLTO's clinical development pathway.

The partnership strategically positions KLTO to accelerate three key neurology programs while potentially sidestepping capital-intensive manufacturing infrastructure investments. Rather than building in-house capabilities, this collaboration leverages AAVnerGene's specialized expertise. For a company focused on neuroscience applications of the Klotho protein, this manufacturing partnership represents prudent resource allocation.

This development is particularly significant for KLTO's competitive positioning, as manufacturing expertise and cost structure increasingly differentiate winners from losers in the gene therapy space. Companies that solve the production efficiency equation gain substantial advantages in pricing flexibility, market access, and ultimately commercial success in bringing these complex biologics to patients with serious neurological conditions.

NEW YORK, July 22, 2025 /PRNewswire/ -- Klotho Neurosciences, Inc. (NASDAQ: KLTO), announced that it is partnering with AAVnerGene Inc. (AAVnerGene), a Rockville, MD-based innovation-driven biotech renowned for its transformative technologies in AAV manufacturing and tissue-targeted delivery. 

Dr. Joseph Sinkule, the CEO of KLTO stated "we announced previously that we have been looking for a more efficient, 'next-generation' method to manufacture several of our patented gene therapy candidates, and the two platform technologies offered by AAVnerGene will catapult our product candidates into the clinic at a faster pace, at a lower cost, a higher efficacy and purity compared to the current state-of-the-art AAV manufacturing processes. We are talking about KLTO-101 for Alzheimer's and Parkinson's diseases, KLTO-202 for ALS and other motor neuron diseases, and KLTO-303 for treatment of ageing-related pathologies resulting in a healthy, extended lifespan. I'm also looking at other complementary longevity-related assets that we may bring into KLTO."

"AAV gene therapy has been proven to be a revolutionary one-time, life-changing treatment, however it is experiencing some crisis caused by high drug price and high dose-related toxicity," said Dr. Daozhan Yu, Founder and CEO of AAVnerGene. "We envisioned these bottleneck problems six years ago and AAVnerGene was founded with the mission to solve them. With the two proprietary platforms, we can dramatically drive down the manufacture cost with the AAVone system and the dose required by the highly efficient and specific tissue-targeting AAVs developed with the ATHENA platform. It's a perfect synergy to partner with Klotho, a neuroscience-driven pathfinder.   Neurological disease drug development is always challenging and we need new approaches and mechanisms. Klotho's data on neuron protection and longevity are very impressive and it holds the potential to treat so many devastating diseases. We are very glad to work with the passionate and highly executive team, and strongly believe that the collaboration can bring more affordable and innovative drugs for patients with rare and common diseases."

Dr. Joseph Sinkule concluded "Klotho believes AAVnerGene has conceived a process method that is more cost effective, faster to make, has fewer impurities, shows higher efficacy and lower toxicity because of the higher targeted tissue delivery.  KLTO hopes to work with AAVnerGene for manufacturing several of our product candidates composed of a tissue-specific promoter driving our Klotho gene to produce the Klotho protein and elevated concentrations of the protein in specific, targeted tissues and organs affected by these neurologic and neuromuscular diseases."

About AAVnerGene, Inc. AAVnerGene is a creativity-powered biotechnology company pioneering next-generation AAV vector technologies. The company's proprietary platforms include AAVone, a high-yield, single-plasmid AAV production system that dramatically reduces manufacturing costs, and ATHENA, an advanced capsid engineering platform enabling precise tissue targeting. AAVnerGene is dedicated to delivering affordable, scalable, and precision-engineered gene therapy solutions that accelerate clinical development across diverse therapeutic areas.

Investor Contact and Corporate Communications – Daozhan Yu, CEO
daozhan.yu@aavnergene.com
Website: www.aavnergene.com

About Klotho Neurosciences, Inc. Klotho Neurosciences, Inc. (NASDAQ: KLTO), is a biogenetics company focused on the development of innovative, disease-modifying cell and gene therapies using a protein derived from a patented form of the "anti-aging" human Klotho gene (s-KL), and it's novel delivery systems to transform and improve the treatment of neurodegenerative and age-related disorders such as ALS, Alzheimer's, and Parkinson's disease. The company's current portfolio consists of its proprietary cell and gene therapy programs using DNA and RNA as therapeutics and genomics-based diagnostic assays. The company is managed by a team of individuals and advisors who are highly experienced in biopharmaceutical product development and commercialization.

Investor Contact and Corporate Communications - Jeffrey LeBlanc, CFO
ir@klothoneuro.com

Website: www.klothoneuro.com

Forward-Looking Statements:

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SOURCE Klotho Neurosciences, Inc.

FAQ

What is the partnership between Klotho Neurosciences (KLTO) and AAVnerGene?

KLTO has partnered with AAVnerGene to manufacture its gene therapy candidates using AAVnerGene's advanced AAV manufacturing and tissue-targeted delivery technologies, aiming for more efficient and cost-effective production.

What are the main drug candidates that KLTO is developing?

KLTO is developing KLTO-101 for Alzheimer's and Parkinson's, KLTO-202 for ALS and motor neuron diseases, and KLTO-303 for aging-related pathologies.

How will AAVnerGene's technology benefit KLTO's gene therapy production?

AAVnerGene's technology promises to provide faster manufacturing, lower costs, higher efficacy, better purity, and reduced toxicity compared to current AAV manufacturing processes.

What are the key platforms that AAVnerGene brings to the partnership?

AAVnerGene brings two key platforms: the AAVone system for cost-effective manufacturing and the ATHENA platform for highly efficient and specific tissue-targeting AAVs.

What therapeutic areas will benefit from the KLTO-AAVnerGene partnership?

The partnership will focus on developing treatments for neurological and neuromuscular diseases, including Alzheimer's, Parkinson's, ALS, and aging-related pathologies.