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What is mRNA Therapy?
mRNA or messenger RNA is a family of RNA molecules that contain genetic information which is carried from DNA to the ribosome such that the ribosome can manufacture amino acids.
mRNA Therapy falls under the designation of “Advanced Therapies” given its non-traditional focus on using the body’s translation mechanism to affect change.
Moderna, a leader in the research and advances in mRNA Therapy, describes it as a “set of instructions that direct cells in the body to make proteins to prevent or fight disease”. This is different from current modes of therapeutics, where proteins are manufactured in large production vessels at biopharmaceutical manufacturing plants. Therefore it comes as no surprise that the work being done on mRNA therapies is considered “Advanced Therapies”.
To better understand mRNA Therapy, one must first understand its use in the translation process.
What is mRNA?
To define mRNA is to first define the transcription and translation process that occurs within a cell. A DNA strand is transcribed into RNA inside the nucleus in a process called “Transcription”. This piece of RNA travels from the nucleus to the cell’s cytoplasm and is now called a “Messenger RNA” or “mRNA”. Once outside the nucleus and within the cytoplasm, the mRNA is translated into a protein through a process called “Translation”.
Needless to say, mRNA plays a critical role in molecular biology. And the advancements in mRNA Therapy do not focus on the cell or the gene, but rather on the communication pathways that affect how proteins are constructed.
How can mRNA be used in Patient Therapy?
Moderna is leading the charge on what’s possible with mRNA therapy. They have created a platform that uses messenger RNAs to affect change in the body – prompting proteins to be made in the body. However, the reason why mRNA remains an emerging technology within therapeutics is because when genetic material is found within the body (without an encapsulating vector or cell), it is targeted for deletion and removal by the body’s normal processes.
Therefore, if Moderna and others can identify a viable mRNA solution while overcoming the limitations of this technology, the world may well have a new bio technology that can be used to target disease and damaged cells.
Who are the Companies focusing on mRNA Therapy?
- CureVac: Founded by Ingmar Hoerr, the company claims to have identified a method to stabilize mRNA for therapeutic use leveraging an in-house nucleotide sequence library to assemble various mRNA pieces together without the need for further chemical modification of mRNA.
- BioNtech: Founded by Ugur Sahin in 2008, the company has various technology platforms, which includes engineered cell therapies, antibodies, small molecules and mRNA therapies. The current mRNA technologies under development at BioNtech use mRNA to deliver genetic information to cells, which is then translated into proteins that have a therapeutic effect. The portfolio includes four mRNA formats and three formulations to derive five platforms for treatment of cancer.
- Ethris: Founded by Carsten Rudolph and Christian Plank in 2009, the company has a technology platform (SNIM®RNA) to treat patients with respiratory disorders and has targeted rare and genetic respiratory diseases as a core strategic focus.
Lessons learned from Biopharma partnerships
Financial commitments from large pharms has been substantial in this field. Arcturus partnered with J&J and Takeda on various therapies including Hep B and GI-related disorders and CureVac received funding from Bill & Melinda Gates Foundation for development of vaccines to fight Zika and HIV.
Lessons learned in biopharma partnerships:
- IP can be gained through the right partnership
- The value of data to generate funding
- Do not dilute the technology by getting caught up in forging deals with one partner that has multiple targets
By: Kiran Chin
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