Klotho Neurosciences Moves Forward with Manufacturing Gene Therapy for the Treatment of ALS
Klotho Neurosciences (NASDAQ: KLTO) announced advancement in manufacturing and process development for KLTO-202, its investigational gene therapy treatment for amyotrophic lateral sclerosis (ALS). The company has licensed a unique RNA splice variant of the human alpha-Klotho gene from the Autonomous University of Barcelona for developing advanced gene therapies.
The company's research has demonstrated that overexpression of secreted alpha-Klotho (s-KL) using gene therapy has shown positive therapeutic outcomes in multiple animal studies, including mouse and non-human primate models. The timeline for development indicates approximately 8 months for manufacturing and 4-6 months for regulatory processes, with Phase I/II clinical trials expected to begin by Q3 2026.
KLTO plans to collaborate with contract research organizations (CROs) to manage manufacturing and clinical trials, maintaining operational efficiency without significant staff expansion. The company aims to use an AAV vector to deliver the s-KL gene directly to motor neurons affected by ALS, a disease that typically leads to paralysis and death within 2-3 years of diagnosis.
Klotho Neurosciences (NASDAQ: KLTO) ha annunciato progressi nella produzione e nello sviluppo dei processi per KLTO-202, il suo trattamento sperimentale di terapia genica per la sclerosi laterale amiotrofica (SLA). L'azienda ha ottenuto la licenza di una variante unica di RNA splicing del gene umano alfa-Klotho dall'Università Autonoma di Barcellona per sviluppare terapie geniche avanzate.
Le ricerche dell'azienda hanno dimostrato che la sovraespressione dell'alfa-Klotho secreta (s-KL) tramite terapia genica ha mostrato risultati terapeutici positivi in diversi studi su animali, inclusi modelli murini e primati non umani. Il calendario di sviluppo prevede circa 8 mesi per la produzione e 4-6 mesi per le procedure regolatorie, con l'inizio delle sperimentazioni cliniche di Fase I/II previsto entro il terzo trimestre 2026.
KLTO intende collaborare con organizzazioni di ricerca a contratto (CRO) per gestire la produzione e le sperimentazioni cliniche, mantenendo l'efficienza operativa senza un'espansione significativa del personale. L'azienda mira a utilizzare un vettore AAV per veicolare direttamente il gene s-KL ai neuroni motori colpiti dalla SLA, una malattia che solitamente porta a paralisi e morte entro 2-3 anni dalla diagnosi.
Klotho Neurosciences (NASDAQ: KLTO) anunció avances en la fabricación y desarrollo de procesos para KLTO-202, su tratamiento experimental de terapia génica para la esclerosis lateral amiotrófica (ELA). La compañía ha licenciado una variante única de empalme de ARN del gen humano alfa-Klotho de la Universidad Autónoma de Barcelona para desarrollar terapias génicas avanzadas.
La investigación de la empresa ha demostrado que la sobreexpresión de alfa-Klotho secretado (s-KL) mediante terapia génica ha mostrado resultados terapéuticos positivos en múltiples estudios en animales, incluyendo modelos de ratón y primates no humanos. El cronograma de desarrollo indica aproximadamente 8 meses para la fabricación y 4-6 meses para los procesos regulatorios, con ensayos clínicos de Fase I/II previstos para comenzar en el tercer trimestre de 2026.
KLTO planea colaborar con organizaciones de investigación por contrato (CROs) para gestionar la fabricación y los ensayos clínicos, manteniendo la eficiencia operativa sin una expansión significativa del personal. La compañía busca usar un vector AAV para entregar el gen s-KL directamente a las neuronas motoras afectadas por la ELA, una enfermedad que típicamente conduce a la parálisis y muerte dentro de 2-3 años tras el diagnóstico.
Klotho Neurosciences (NASDAQ: KLTO)는 근위축성 측삭 경화증(ALS) 치료를 위한 실험용 유전자 치료제 KLTO-202의 제조 및 공정 개발에서 진전을 발표했습니다. 회사는 첨단 유전자 치료제 개발을 위해 바르셀로나 자치대학교로부터 인간 알파-클로토 유전자의 독특한 RNA 스플라이스 변이체를 라이선스 받았습니다.
회사의 연구에 따르면, 유전자 치료를 통해 분비형 알파-클로토(s-KL)의 과발현은 쥐 및 비인간 영장류 모델을 포함한 다수의 동물 연구에서 긍정적인 치료 효과를 보였습니다. 개발 일정은 제조에 약 8개월, 규제 절차에 4-6개월이 소요될 예정이며, 1/2상 임상시험은 2026년 3분기 시작이 예상됩니다.
KLTO는 제조 및 임상시험 관리를 위해 계약 연구 기관(CRO)과 협력할 계획이며, 인력 대규모 확장 없이 운영 효율성을 유지할 방침입니다. 회사는 AAV 벡터를 사용해 ALS에 영향을 받은 운동 신경세포에 s-KL 유전자를 직접 전달하는 것을 목표로 하고 있으며, ALS는 진단 후 보통 2-3년 내에 마비와 사망에 이르는 질환입니다.
Klotho Neurosciences (NASDAQ : KLTO) a annoncé des avancées dans la fabrication et le développement des processus pour KLTO-202, son traitement de thérapie génique expérimental contre la sclérose latérale amyotrophique (SLA). L'entreprise a obtenu une licence pour un variant unique d'épissage ARN du gène humain alpha-Klotho auprès de l'Université autonome de Barcelone afin de développer des thérapies géniques avancées.
Les recherches de la société ont démontré que la surexpression de l'alpha-Klotho sécrété (s-KL) via la thérapie génique a montré des résultats thérapeutiques positifs dans plusieurs études animales, incluant des modèles de souris et de primates non humains. Le calendrier de développement prévoit environ 8 mois pour la fabrication et 4 à 6 mois pour les procédures réglementaires, avec des essais cliniques de phase I/II attendus pour débuter au 3e trimestre 2026.
KLTO prévoit de collaborer avec des organisations de recherche sous contrat (CRO) pour gérer la fabrication et les essais cliniques, tout en maintenant une efficacité opérationnelle sans augmentation significative du personnel. L'entreprise vise à utiliser un vecteur AAV pour délivrer directement le gène s-KL aux motoneurones affectés par la SLA, une maladie qui conduit généralement à la paralysie et au décès dans les 2 à 3 ans suivant le diagnostic.
Klotho Neurosciences (NASDAQ: KLTO) gab Fortschritte in der Herstellung und Prozessentwicklung für KLTO-202 bekannt, seine experimentelle Gentherapie zur Behandlung der amyotrophen Lateralsklerose (ALS). Das Unternehmen hat eine einzigartige RNA-Spleißvariante des menschlichen Alpha-Klotho-Gens von der Autonomen Universität Barcelona lizenziert, um fortschrittliche Gentherapien zu entwickeln.
Die Forschung des Unternehmens hat gezeigt, dass die Überexpression von sekretierter Alpha-Klotho (s-KL) mittels Gentherapie in mehreren Tierversuchen, darunter Maus- und nicht-menschliche Primatenmodelle, positive therapeutische Ergebnisse erzielte. Der Entwicklungszeitplan sieht etwa 8 Monate für die Herstellung und 4-6 Monate für regulatorische Prozesse vor, wobei Phase I/II-Studien voraussichtlich im 3. Quartal 2026 beginnen.
KLTO plant die Zusammenarbeit mit Auftragsforschungsinstituten (CROs), um Herstellung und klinische Studien zu managen und dabei die operative Effizienz ohne signifikante Personalaufstockung zu erhalten. Das Unternehmen beabsichtigt, einen AAV-Vektor zu verwenden, um das s-KL-Gen direkt an die von ALS betroffenen Motoneuronen zu liefern, einer Krankheit, die typischerweise innerhalb von 2-3 Jahren nach Diagnose zu Lähmung und Tod führt.
- None.
- Extended timeline to clinical trials (approximately 12-14 months)
- Complex manufacturing process requiring significant development time
- Multiple regulatory steps needed before trial initiation
Insights
Klotho advances ALS gene therapy toward clinical trials with promising preclinical data, though significant development milestones remain before efficacy can be determined.
Klotho Neurosciences is progressing toward clinical development of KLTO-202, a gene therapy targeting ALS that's built on a unique scientific approach. The therapy centers on the secreted alpha-Klotho (s-KL) protein variant that occurs naturally in brain and spinal cord neurons, where it provides neuroprotection by reducing oxidative stress and neuroinflammation.
The company's timing projections are quite specific: approximately 8 months for manufacturing and process development, followed by 4-6 months for regulatory interactions and preparations, targeting first patient dosing by Q3 2026. This timeline is relatively aggressive but not unreasonable for gene therapy development.
What's particularly notable is their preclinical evidence spanning multiple disease models including rapid aging, Alzheimer's, and ALS models in both mice and non-human primates—with published results showing "highly favorable therapeutic outcomes" across all tested scenarios. The breadth of positive preclinical data across different neurological conditions strengthens their scientific rationale.
Their strategic decision to utilize CROs rather than expanding internal headcount demonstrates financial discipline, particularly important for smaller biotechs where cash runway is critical. The recent "fundraising success" mentioned (though without specific figures) appears to have enabled this development push.
For context, ALS represents a severe unmet medical need with rapid progression and high mortality, typically claiming patients within 2-3 years of diagnosis. While gene therapies offer transformative potential, they face significant technical and regulatory hurdles, with many promising candidates failing in later stages. KLTO-202 remains years away from potential commercialization, with the critical proof-of-concept data in humans still pending.
A unique RNA splice variant of the human gene called alpha-Klotho has been licensed by the Company from the Autonomous University of
The Company expects that it will take approximately eight months to complete process development and manufacturing of KLTO-202, and about four to six months to conduct meetings with FDA, complete all FDA-mandated animal safety studies, file an investigational new drug application (IND), train and prepare clinical sites where the Phase I/II studies can be conducted, and then begin the single-dose gene therapy studies in ALS patients by the third quarter of next year. The Company will work with contract research organizations (CROs) to facilitate all activities including manufacturing and clinical trials without the need to hire several dozen staff members, which would significantly increase our operating overhead.
Dr. Joseph Sinkule, the Company's CEO and founder commented: "With our recent fundraising success, we're moving forward with manufacturing the s-KL transgene DNA for KLTO-202. We've identified a more efficient method of producing the AAV vector to deliver the s-KL gene directly to motor neurons—the cells most affected by ALS. Our goal is to increase local s-KL protein levels to protect these neurons from the damage that leads to voluntary and involuntary muscle paralysis and ultimately death."
ALS typically progresses rapidly, with most patients losing mobility, respiratory function, and life within just 2–3 years of diagnosis.
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 its 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:
This press release contains forward-looking statements. These statements are made under the "safe harbor" provisions of the
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FAQ
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