Curapath is participating in the NEUROAN project, a strategic collaborative initiative led by ARTHEx Biotech that brings together ARTHEx Biotech, ProtoQSAR, AMS Biopharma and Curapath, to develop next-generation targeted RNA therapies for neuromuscular diseases.
Through this collaboration, the entities will combine their scientific and technological capabilities to address one of the most significant challenges in RNA‑based drug development: achieving efficient, safe, and highly specific delivery of antisense oligonucleotides (AONs) to neuromuscular tissues.
Project goals and scientific challenges
The project focuses on the rational design of therapeutic antisense oligonucleotides (AONs) for neuromuscular diseases, aiming to optimize efficacy, safety, and target specificity. This includes tissue-specific oligonucleotide engineering combined with targeted delivery strategies and preclinical validation, providing a strong foundation for the development of treatments for currently untreatable diseases with no disease-modifying options.
The collaboration aims to overcome key biological and technological barriers that currently limit the clinical translation of RNA therapeutics:
- Biological barriers preventing AONs from reaching neuromuscular tissues efficiently.
- Avoiding liver and kidney accumulation, a common issue in systemic RNA delivery.
- Increasing in vivo stability to enhance therapeutic durability.
- Ensuring high specificity without off‑target effects.
- Integrating biotechnology, computational chemistry, AI, and next‑generation delivery platforms into a unified development strategy.
Curapath’s role in the collaboration
Curapath will play a central role by contributing expertise in lipid–AON conjugate design to enhance delivery, stability, biodistribution, and therapeutic efficacy in neuromuscular tissues. This will be supported by advanced synthesis, shielding strategies, analytical characterization, and scalable manufacturing to enable precise targeting and translational development of RNA therapeutics. Its contribution will focus on:
- Synthesis of a library of lipids and linkers with selective tissue affinity to optimize therapeutic efficacy while minimizing adverse effects.
- Physicochemical and supramolecular characterization of the conjugates using advanced analytical techniques, including HPLC-MS, dynamic light scattering (DLS), zeta potential measurements, and gel electrophoresis.
- Evaluation in reporter cell lines to assess conjugate transfection efficiency and stability under different experimental conditions.
- Scale-up studies for in vivo-grade conjugates, ensuring reproducibility as well as stability during storage and in biological media.
Expected impact
This collaboration is poised to drive meaningful advancements in RNA medicine by:
- Enhancing delivery efficiency to hard‑to‑reach neuromuscular tissues.
- Reducing systemic toxicity and off‑target exposure.
- Establishing new delivery platforms applicable to future RNA and gene‑editing therapies.
- Strengthening the technological foundation for next‑generation precision treatments.
About Curapath
Curapath is a science‑driven CDMO specializing in the design, development, and GMP manufacturing of advanced drug delivery systems. The company supports biopharma partners from early research through commercial production, with a strong focus on non‑viral gene delivery using polymer‑ and lipid‑based nanoparticles. Curapath’s capabilities span formulation development, analytical characterization, and scalable GMP manufacturing for next‑generation therapeutics.
To learn more about Curapath you can contact us here: https://curapath.com/
About ARTHEx Biotech
ARTHEx is a biotechnology company focused on the development of RNA-targeted therapies for rare neuromuscular and neurodegenerative diseases. Leveraging proprietary antisense oligonucleotide (ASO) technologies, the company develops innovative therapeutic approaches to address diseases with high unmet medical needs. ARTHEx combines expertise in RNA biology, drug development, and translational research to advance next-generation precision medicines from discovery to clinical development.
To learn more about ARTHEx visit: Home Arthex Biotech
About ProtoQSAR 2000 S.L.
ProtoQSAR is a computational biotechnology company specializing in artificial intelligence, molecular modeling, and in silico drug discovery. The company develops predictive models and computational platforms to support the design, optimization, and safety assessment of novel therapeutics, helping accelerate pharmaceutical R&D while reducing development costs and timelines.
To learn more about ProtoQSAR visit: Inicio | ProtoQSAR
About AMS Biopharma Laboratory S.L.
AMS Biopharma Laboratory S.L. is a contract research organization (CRO) specializing in preclinical pharmacology and translational research. The company provides integrated in vitro and in vivo services to support drug discovery and development, including efficacy, pharmacokinetic, toxicology, and biomarker studies. With extensive expertise in disease models and regulatory-compliant research, AMS Biopharma supports the advancement of innovative therapeutics from preclinical evaluation toward clinical development.
To learn more about AMS Biopharma visit: Home | AMSbiopharma
