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Common Rare Orthopedic Diseases
- Osteogenesis Imperfecta,OI
- Achondroplasia
- Fibrodysplasia Ossificans Progressiva, FOP
- Marfan Syndrome
- Melorheostosis
- X-linked Hypophosphatemia, XLH
- Paget's Disease of Bone
- amyotrophic lateral sclerosis, ALS
- spinal muscular atrophy, SMA
- Rare Bone Tumors
- Klippel-Trenaunay Syndrome
- Ehlers-Danlos Syndrome, EDS
- Ultimate Guide to Rare Orthopedic Diseases Research Services
- Special Technology Service Platform
Rare Orthopedic Diseases AAVLink™ Platforms
Protheragen possesses a deep understanding of the intricate processes involved in creating gene therapies for rare diseases. The AAVLink™ platform from our team supports biopharmaceutical innovation within these challenging fields. We provide complete research services that cover AAV vector design and delivery optimization, as well as therapeutic candidate development for significant scientific advancements.
Introduction to AAVLink™ Platforms
Protheragen developed the AAVLink™ platform which stands at the forefront of adeno-associated viral vector development as it targets gene therapy challenges for rare diseases. The platform provides advanced methods for AAV vector design optimization and production while resolving problems related to tissue targeting and packaging constraints. The AAVLink™ platform achieves precise therapeutic gene delivery through its unique AAV-Stable, AAV-Find, and AAV-Expand technologies which leads to better results in gene-based therapies.
Fig. 1 In vivo gene therapy with AAVs. (Mendell JR, et al., 2021)Our Services
At Protheragen, we understand the complexities of developing gene therapies for rare diseases. Our AAVLink™ platform is a state-of-the-art solution designed to advance biopharmaceutical innovation by enhancing AAV vector design, improving tissue-specific targeting, and overcoming packaging limitations. From designing these vectors to optimizing therapeutic delivery systems, we provide end-to-end research services, the services include:
AAV Vector Design and Production
- Specializes in the design of complementation systems, AAV capsid selection and optimization, and target gene integration.
- Innovatively uses inducible cell lines for high-yield, cost-effective AAV vector production.
AAV Vector Purification
Implements advanced purification methods, combining affinity chromatography and ion exchange chromatography, along with density gradient centrifugation.
Proven Delivery Capabilities
Develops AAV vectors for effective intravitreal injection and vectors targeting organs like the liver, heart, and muscles, validated in animal models.
Advanced Vector Platform
We have three major AAV vector platforms that simplify the development process of AAV vectors and enhance their functions. Our proprietary AAV platform is a testament to our commitment to innovation.
AAV-Find
Identifies novel AAV variants with enhanced tissue-specific targeting capabilities, overcoming translational barriers from animal models to human applications.
AAV-Stable
Utilizes inducible cell lines for the stable and scalable production of AAV vectors, ensuring consistency and efficiency.
AAV-Expand
Addresses packaging limitations by employing dual or multiple AAV systems, enabling the delivery of larger therapeutic genes.
Our Advantages
Time-saving services with high efficiency
Professional and experienced scientists
Complete project designing and reporting
Numerous service cases and customer praise
Protheragen's AAVLink™ platform uses our extensive knowledge in AAV vector development for rare diseases to enhance tissue-specific targeting and scalable production which results in next-generation gene therapies with improved efficacy and safety. You can learn more about our services by reaching out to us today.
Reference
- Mendell JR, et al. Current Clinical Applications of In Vivo Gene Therapy with AAVs. Mol Ther. 2021 Feb 3;29(2):464-488.
All of our services and products are intended for preclinical research use only and cannot be used to diagnose, treat or manage patients.
