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GQ Bio’s high-capacity gene therapy vector platform (HCAd) enables gene therapy with large and multiple genes, which addresses one of the key challenges of conventional gene therapy vectors. Based on the HCAd platform, GQ Bio is developing transformative gene therapies for prevalent diseases such as osteoarthritis and intervertebral disc degeneration.
HCAd Vector Platform
GQ Bio’s high-capacity gene therapy vector platform (HCAd) represents the latest generation of adenoviral vectors, characterized by lower immunogenicity compared to earlier generations and long-term gene expression. One of the advantages of HCAds compared to other commonly used gene therapy vectors is the high efficiency to enter cells (transduction efficiency) in many tissues (in most cases more efficient than adeno-associated vectors (AAV)), in particular in joints and intervertebral discs. Another advantage is that HCAd can carry up to 30,000 base pairs of DNA (for comparison: AAV have a capacity of 2,500–5,000 base pairs), which enables gene therapy with large genes and multiple genes.
The HCAd technology was originally developed at Baylor College of Medicine (Houston, TX, USA). GQ Bio is working on expanding and optimizing the platform to make it even more efficient and more specific for certain tissue/cell types. For example, GQ Bio has developed a modular genetic kit to rapidly generate new HCAds with multiple genes. Furthermore, GQ Bio is working on developing a large-scale manufacturing process that will enable low manufacturing costs per dose making HCAd-based gene therapies available for large markets.
Gene Therapy for Prevalent Diseases
Gene therapy was originally developed to treat rare diseases that are based on genetic defects. GQ Bio takes gene therapy to the next level and leverages the technology to treat non-genetic, prevalent diseases in the musculoskeletal field. The aim is to establish long-term production of therapeutic proteins in affected tissues following a single injection. Based on this concept, GQ Bio develops drugs for diseases that can be treated with local injections to achieve high bioavailability of the therapeutic protein with low doses.
Gene Therapy for Osteoarthritis and Intervertebral Disc Degeneration
GQ Bio is focused on the development of drugs for the treatment of osteoarthritis and intervertebral disc degeneration – two very prevalent musculoskeletal disorders, which affect millions of patients and for which there are currently only symptomatic drugs available. Following a single injection into the affected joint or intervertebral disc, GQ Bio’s gene therapy drugs turn these tissues into factories for local production of therapeutic proteins, which protect or restore the joint and disc structure, inhibit inflammation and reduce symptoms. In contrast to conventional drugs, a single injection can lead to high bioavailability in the target tissue over a long period of time. Based on these properties, GQ Bio’s drug candidates have the potential to become the first medicines with sustained symptomatic and disease-modifying efficacy for osteoarthritis and intervertebral disc degeneration.