The next generation
of RNA therapies is
just beginning

Our science

RNA-based therapies is an exciting new class of drugs with the potential to change the standard-of-care for many life-threatening diseases. These medicines can progress quickly from concept to patient, with some treatments already approved and many more in development.

The recent success of COVID-19 vaccines, for example, demonstrated that highly effective medicines could be produced faster, better, and at a lower cost than traditional approaches.

mRNA as
a vaccine or therapy

mRNA as
a therapeutic target

Targeting mRNA-RBP interactions

The power and promise of RNA therapies lies in the adaptability and versatility of RNA itself. As in the case of the COVID vaccines, a particular form of RNA called messenger RNA (mRNA) can serve directly as a vaccine or therapy.

mRNA can also serve as a therapeutic target; mRNA downregulation can be used to inhibit the expression of a disease-related gene, while mRNA upregulation may be used to enhance the expression of normal genes to compensate for a genetic deficiency.

Another therapeutic avenue involves so-called RNA binding proteins (RBPs). These proteins regulate the cellular activity of mRNA molecules, and understanding this regulation is important for the continued development of mRNAs as effective drugs and drug targets. In addition, drugs that alter the interaction between mRNAs and RBPs have the potential to become an entirely new class of therapeutics.

At Sanford Labs, our scientists are at the forefront of developing new therapeutic approaches and technologies based on these and other recent scientific advances. Advising these efforts is Gene Yeo, a world-renowned investigator in RNA biology and seasoned entrepreneur.

Meet Gene Yeo

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