MARCH 2023LIFE SCIENCES REVIEW 19explore utilizing 3DNA for treating liver-mediated diseases and central nervous system (CNS) diseases. To treat CNS diseases, Code Bio has already shown data that demonstrates its capability to cross the blood-brain barrier and target specific brain cells."Our chemistry, translational research, and technology development teams comprise industry leaders whose experience and expertise are enabling us to make progress towards our mission of treating diseases that have to this point been untreatable," adds McVeigh.Code Bio's research and achievements are fuelled by various investors, including institutional investors like NEA, Northpond, 4BIO Capital and Hatteras; strategic investors like Amgen Ventures, Takeda Ventures, UCB Ventures, and UPMC Enterprises; and investors from disease venture philanthropy groups. It has collaborations with many pharmaceutical companies that enable it to advance multiple avenues with the 3DNA platform through financial and strategic assistance. Code Biotherapeutics' top-notch platform, expertise, and dedication to going above and beyond to develop treatments for difficult to treat conditions, while ensuring higher efficacy and accuracy, is a ray of hope for a world tormented by complex and deadly diseases. DMD. To improve the efficacy, researchers are using a micro-dystrophin or mini-dystrophin approach where they are trying to treat a truncated section of that dystrophin gene with AAV. However, the result is unsatisfactory because of AAV's inability to be redosed. Code Bio is working on the first stage of its discovery program, looking at delivering its version of the micro-dystrophin or mini-dystrophin to treat DMD. If successful, it could be used as a redosable therapy for new patients and those who have already been administered AAV. Code Bio is generating data to derive efficacious results from the therapy. Its next step is designing a formulation incorporating the Beckers' Gene sequence which is two-thirds the length of a full-length dystrophin gene. It is simultaneously researching to develop an entire 14-kilobase pair sequence to treat DMD. Code Bio's discovery research program starts by analyzing the diseases that have affected the tissue or cell to find the possible payloads to treat the ailment. Depending on the payload, it can send any medication--antibody, peptide, gene construct, or siRNA--to treat the disease. This shows potential for treating multiple conditions, even rare ones, in the future. A testament to the potential of Code Biotherapeutics' 3DNA genetic medicine delivery platform is its strategic collaboration with Takeda Pharmaceuticals'. They are working together to We are revolutionizing the prevailing ways of genetic medicine payload delivery through our transformative technology to treat debilitating genetic disorders. Our one-of-a-kind platform uses non-viral DNA-based vectors to deliver safer, redosable, and titratable treatments for genetic diseases
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