Minerva Neurosciences Highlights Klotho's Advances in ALS Gene Therapy Development
- Klotho Neurosciences is advancing KLTO-202, a gene therapy targeting ALS through the alpha-Klotho gene.
- The company has conducted successful animal studies showing s-KL's efficacy in ALS and other neurodegenerative diseases.
- Klotho plans to initiate Phase I/II clinical trials for ALS patients within the next year, pending regulatory approvals.
Klotho Neurosciences Advances Gene Therapy Development for ALS
Klotho Neurosciences, Inc. is making significant strides in the fight against amyotrophic lateral sclerosis (ALS) with the advancement of KLTO-202, an investigational gene therapy. The company has recently transitioned into the manufacturing and process development stages for this promising treatment, which aims to harness the therapeutic potential of a unique RNA splice variant of the human alpha-Klotho gene. Licensed from the Autonomous University of Barcelona, this gene has been shown to produce two protein isoforms—membrane-bound Klotho (m-KL) and secreted alpha-Klotho (s-KL). Notably, s-KL is primarily present in the brain and spinal cord, where it provides neuroprotective effects by mitigating oxidative stress and neuroinflammation, both of which are critical factors in the progression of ALS.
Klotho Neurosciences has spent the past two years conducting animal studies that demonstrate the efficacy of amplifying s-KL levels through gene therapy. Their research, published in peer-reviewed journals, reveals promising therapeutic outcomes not only in ALS but also in models of rapid aging and Alzheimer's disease. As the company moves forward, it anticipates an eight-month timeline for process development and manufacturing, followed by four to six months dedicated to FDA consultations, safety studies, and the filing of an investigational new drug application (IND). These steps are crucial as Klotho aims to initiate Phase I/II clinical trials in ALS patients by the third quarter of next year.
In a statement, CEO Dr. Joseph Sinkule emphasizes the importance of efficiently producing the adeno-associated virus (AAV) vector for delivering the s-KL gene directly to the motor neurons that are critically affected by ALS. By increasing s-KL protein levels, the therapy aims to protect these neurons from the damage that leads to muscle paralysis and eventual death, addressing the urgent need for effective treatments in this rapidly progressing disease. The company’s focus on innovative gene therapy positions it at the forefront of ALS research, underscoring the potential impact of its work on patients suffering from this devastating condition.
In related news, Klotho’s progress comes at a time when the biotech industry is increasingly turning to gene therapies as a means of addressing complex neurodegenerative diseases. The licensing of the alpha-Klotho gene reflects a broader trend where collaborations between academia and industry are fostering innovation in therapeutic development. As Klotho Neurosciences prepares for its next steps, the anticipation surrounding KLTO-202 signals a potential breakthrough in the treatment landscape for ALS, offering hope to patients and their families.