Gene therapy has successfully transitioned from basic research to medicine. Science to Engineering.
While gene therapy has been a subject of extensive research for over 50 years, with greater than 62,000 publications on PubMed and 1,000 registered trials on ClinicalTrials.gov, the approvals of the previous decade have been focused on rare disease indications. With the advent of DNA-based Adenoviral vaccines and RNA-based lipid nanoparticle vaccines (de facto in vivo gene therapies, delivering protein coding genetic instructions to cells), gene therapy has been elevated to the status of common-place medicine. At Remedium Bio, we engineer medicine by modularly applying proven scientific fundamentals in novel ways to treat well characterized disease pathologies.
A vector is the method of delivery for the genetic material, it can be viral, lipid, or polymeric (with or without targeting).
GENE OF INTEREST
The cargo typically contains a gene or genetic sequence of interest, this gene can express a protein that treats the disease.
The cargo is the genetic construct delivered by the vector, it can be DNA, RNA, or other nucleic acid derivative.
Expression of the therapeutic protein can be regulated by a tissue- or cell-specific promoter and other genetic elements.
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