2023-10-10 23:00:00
Mark Heltz CEO Seungmin Lee
Customized AAV targeting carrier platform technology
Seongnam–(Newswire) October 11, 2023 — Mark Heltz (CEO Seung-min Lee) is developing an AAV gene delivery system using a promoter that specifically binds to cerebrovascular endothelial cells and expresses therapeutic genes specifically in endothelial cells. It was announced on the 11th that domestic patent registration for Korea was completed on September 8, and PCT applications are in progress for applications in the United States and Germany.
By successfully securing core technology related to the AAV gene delivery system that efficiently delivers drugs to brain tissue or cerebrovascular vessels, we are striving to pass rigorous clinical trials in the development of AAV gene therapy to treat brain diseases (dementia, stroke, brain cancer, Huntington’s disease, etc.). The possibility of securing a high level of stability and efficiency has increased.
Gene therapy products generally consist of a therapeutic gene and a vector. Recently, more than 3,500 therapeutic genes have been developed, but there is a global shortage of delivery technology to deliver them to the correct therapeutic cells. This is because the technology for mass production of delivery vehicles as well as technological development of therapeutic substances is still lacking. Accordingly, companies specializing in delivery systems will naturally possess mass production technology that allows scale-up.
Markheltz is one of the few domestic companies that possesses both ‘AAV delivery vehicle’ related technology development and mass production technology. The core technologies that Markheltz possesses are △target screening technology, △AAV target engineering technology, and △AAV automation mass production technology for mass production.
1. Targeted screening technology
Select the primary target motif using Phage-display target screening technology, analyze its 3D digital structure with artificial intelligence (AI) software to select the most likely candidates, and then select the best combination of 2-3 motifs using MS equipment. (Holds numerous papers and technologies).
2. AAV target engineering technology
Two to three selected targeting motifs are coated on the AAV surface in advance using nano-particles, and intracellular targeting and introduction efficiency are checked. Among these, one motif is selected as the most efficient candidate and inserted into the AAV capsid to develop an AAV agent that can be used as a drug (multiple papers and patents held).
3. AAV automated mass production technology
After producing R&D-grade AAV, we use AI software to △automate production monitoring of cells △monitor the productivity of AAV △each production system △automatically control each production equipment with digital devices, and process from 15ℓ level production to approximately 2000 level production. Apply digital analysis to automate mass production (design of Korea’s first single-use AAV production facility, automation equipment, and software development).
In particular, Markhelz demonstrated using experimental animal models that surface engineering of AAV Vector has a high impact on treatment efficacy. The achievements of AAV gene therapy, such as treating Duchenne muscular dystrophy, inducing neutrophils by expressing in blood vessels, or preventing atherosclerosis by expressing Anxa1 in endothelial cells to prevent the formation of plaques. By proving the possibility of treatment, the content has been introduced as cutting-edge technology in the international journals ‘Nature medicine (2020)’ and ‘Advanced science (2022)’.
The brain, heart, and the blood vessels that connect them can be said to be the three major organs that make up the human body, and if even one of them loses its function, death will result. Markhelz is developing myocardial tissue or cardiovascular AAV delivery vehicle technology and collaborating with a German preclinical institute, a world authority in the field of gene therapy, while also starting joint research with a famous cardiovascular center in Korea to ‘develop AAV gene therapy for the treatment of diabetes.’ In addition, we have promised to conduct research on degenerative brain diseases with the neurology department of a famous domestic hospital.
In addition, Markhelz announced that it has a branch in Frankfurt, Germany and is working with related companies in neighboring countries to create a large-scale ‘digital bio infrastructure’ in order to integrate Korea’s digital ICT technology with Germany’s cutting-edge bio technology.
About Markheltz
MarkHerz is a company that provides innovative treatments and development solutions for genetic diseases that address the root of diseases for humanity’s dream of a ‘disease-free and healthy life’. By establishing an AAV gene therapy development platform that drives the new global bio growth trend and presenting differentiated treatments with Mark Heltz’s unique core technology, we are laying the foundation for securing value and providing global standard services as a future value-oriented bio company.
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