Klotho Neurosciences, Inc. (NASDAQ: KLTO) has announced a strategic manufacturing partnership with Rockville-based AAVnerGene Inc., aimed at accelerating the clinical development of its gene therapy candidates for neurodegenerative and aging-related diseases. The news was released by both companies on July 22, 2025, with confirmation of the collaboration coming from Klotho’s CEO Dr. Joseph Sinkule.
The partnership will utilize AAVnerGene’s proprietary platforms—AAVone and ATHENA—to manufacture Klotho’s targeted gene therapies, including KLTO-101 for Alzheimer’s and Parkinson’s disease, KLTO-202 for ALS and other motor neuron conditions, and KLTO-303 for age-related degeneration. These therapies are based on delivering the Klotho protein, known for its neuroprotective and longevity-enhancing properties.
Why did Klotho Neurosciences partner with AAVnerGene?
Klotho Neurosciences has been actively seeking next-generation manufacturing solutions to overcome the cost, scalability, and toxicity challenges typically associated with adeno-associated virus (AAV)-based gene therapies. According to Dr. Sinkule, the partnership with AAVnerGene will enable the production of higher-purity, more targeted therapies at reduced costs and faster development timelines compared to current AAV manufacturing techniques.
AAVnerGene’s platforms offer key advantages: the AAVone system reduces the cost of AAV vector production, while the ATHENA platform allows for highly efficient and specific tissue-targeted delivery, potentially reducing the overall therapeutic dose and associated toxicity.
Dr. Sinkule noted that these advantages will help “catapult our product candidates into the clinic at a faster pace, at a lower cost, a higher efficacy and purity” than what conventional methods currently allow.
What is Klotho’s focus in neuroscience and aging?
Klotho Neurosciences is named after the Klotho gene, which encodes a protein associated with cellular protection and lifespan extension. The company’s gene therapies aim to elevate Klotho protein levels in tissues affected by neurodegenerative diseases, with the goal of slowing or halting disease progression and supporting tissue regeneration.
KLTO-101, the company’s lead candidate, is under development for Alzheimer’s disease and Parkinson’s disease. KLTO-202 is being explored for amyotrophic lateral sclerosis (ALS) and other motor neuron disorders, while KLTO-303 targets aging-related decline. These therapies all incorporate tissue-specific promoters to drive targeted expression of the Klotho gene in affected organs.
According to the company’s disclosures, Klotho’s approach not only focuses on symptom alleviation but also aims to address the cellular mechanisms of aging and neuronal damage—paving a path toward disease-modifying therapies.
What makes AAVnerGene a critical player in this collaboration?
Founded to solve long-standing limitations in gene therapy, AAVnerGene has been working on strategies to address high vector production costs and dose-related toxicity—key bottlenecks in the commercial viability of AAV therapies.
In his statement, AAVnerGene CEO Dr. Daozhan Yu emphasized that “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.”
The AAVone platform, developed in-house, significantly improves manufacturing efficiency, while the ATHENA platform optimizes tissue targeting, ensuring that therapeutic doses are delivered precisely where they are needed—reducing systemic exposure and off-target effects.
Dr. Yu also expressed strong alignment with Klotho’s mission, describing the biotech as “a neuroscience-driven pathfinder” with promising data in neuron protection and healthy aging.
How does this partnership impact the future of neurological gene therapy?
This collaboration represents a potentially significant milestone in the gene therapy field, particularly for central nervous system (CNS) disorders, which have historically been challenging due to the complexity of brain-targeted delivery and the blood-brain barrier.
By leveraging AAVnerGene’s targeting platforms, Klotho may be able to overcome these barriers and develop safer, more effective therapies that require lower doses and demonstrate improved bioavailability in neural tissues. This could mark a new era in the treatment of conditions like Alzheimer’s, ALS, and Parkinson’s, which currently have limited treatment options.
Furthermore, the companies aim to bring affordability into focus. By tackling the high costs associated with vector production, they hope to make gene therapies more accessible to broader patient populations, including those in underfunded health systems or with rare disorders not typically targeted by large pharmaceutical players.
What’s next for Klotho and AAVnerGene?
Dr. Sinkule indicated that the companies will begin joint efforts to apply AAVnerGene’s platforms to multiple programs, starting with KLTO-101, KLTO-202, and KLTO-303. In addition, Klotho is evaluating further longevity-related assets that could complement its existing pipeline.
The collaboration is expected to accelerate Investigational New Drug (IND) submissions and preclinical development milestones, potentially bringing Klotho’s candidates into early-phase clinical trials sooner than originally anticipated.
Moreover, Klotho plans to work closely with AAVnerGene to design constructs that use a tissue-specific promoter system. This would enable the expression of the Klotho gene and production of its protein in selected tissues, such as the brain, spinal cord, and neuromuscular junctions—providing targeted intervention for diseases with localized pathology.
Broader industry implications and expert sentiment
While no third-party analysts were quoted in the official release, early indicators suggest the collaboration could be viewed positively by investors and researchers focused on aging biology and neurodegeneration. The partnership aligns with a broader trend in biotech toward platform-enabled innovation, especially in the gene and cell therapy space.
Many in the industry have raised concerns about the cost and safety of AAV gene therapies. Klotho and AAVnerGene’s shared strategy to address these concerns head-on through scalable, tissue-targeted delivery could set a precedent for next-generation gene therapy programs.
In addition, the collaboration highlights how specialized biotech firms are carving out niches in specific aspects of gene therapy—from payload design to manufacturing—and forming synergistic partnerships to bring high-potential candidates to market efficiently.
Prospects for potential clinical development and commercialisation
If successful, this partnership could position Klotho as a first mover in the delivery of Klotho-protein-based treatments for neurodegeneration and aging. The company’s clinical path will likely involve filing INDs with the FDA, initiating Phase 1 safety studies, and gathering early efficacy signals in small patient populations.
Regulatory scrutiny will remain high, particularly for CNS-targeted gene therapies, which require thorough demonstration of both safety and tissue selectivity. However, the combined technical expertise of Klotho and AAVnerGene could support robust preclinical packages, boosting regulatory confidence.
Commercially, therapies that can demonstrate reduced toxicity, cost-effective manufacturing, and improved outcomes may eventually gain competitive advantage, especially if targeting conditions with few effective options.
Klotho’s future plans could include licensing, strategic collaborations with larger biopharma companies, or further platform expansion to other age-related diseases. AAVnerGene, in turn, may attract more partners interested in its dual-platform delivery model, especially as pressure mounts to deliver affordable gene therapies at scale.
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