RNA

• “The Rett Syndrome Research Trust is the leading patient advocacy group championing a cure for Rett syndrome and we look forward to collaborating with them to further expand the broad applicability of our Axiomer RNA editing technology to Rett syndrome, a rare neurodevelopment disorder with significant unmet medical need,” said Daniel A. de Boer, Chief Executive Officer of ProQR.
• “Axiomer has the potential to restore precise levels of the MECP2 protein, which is lacking in Rett syndrome.
• It is the intent of the partnership to be continued by an expanded co-funding arrangement following the initial discovery work.
• The co-funding of the next phase of the collaboration would enable clinical development of an Axiomer-based therapeutic for Rett syndrome MECP2.

• Until the COVID-19 pandemic, however, vaccine development was still a long and idiosyncratic process.
• But the COVID-19 mRNA vaccines brought a new approach to vaccine development that has far-reaching implications for how researchers make drugs to treat many other diseases.

Some basics of mRNA drugs

• An mRNA drug comprises two essential components: mRNA molecules, which code for desired proteins, and the lipid molecules – such as phospholipids and cholesterol – that encapsulate them.
• From a drug development perspective, mRNA drugs offer significant advantages over traditional drugs because they are easily programmable.
• More importantly, different mRNA drugs produced by the same set of methods will have similar properties.
• This predictability significantly reduces the development risks and financial costs of developing mRNA drugs.

Self vs. nonself

• This may sound paradoxical – after all, your cells already contain large amounts of mRNAs.
• How does your immune system distinguish between self and nonself mRNAs?
• Therapeutic mRNAs enter cells using endosomes – sacs made of the cell’s membrane that take in materials from the cell’s environment.

• The 2023 Nobel Prize in physiology or medicine was awarded to the scientists who made this breakthrough discovery.
• RNA viruses also form double-stranded RNA when they replicate, and exposing cells to double-stranded RNA can lead to a strong immune response.
• Fortuitously, for mRNA vaccines, the residual amount of double-stranded RNA can stimulate the immune system to enhance antibody responses.

Moving beyond vaccines

• One promising example in development is using mRNA that encodes CRISPR-Cas9 gene-editing proteins to knock out genes that cause specific diseases.
• This disease is an ideal target for mRNA-based CRISPR gene therapy because the target protein is produced by the liver.

• Notable new developments in these areas include using computational algorithms to optimize mRNA sequences in ways that enhance their stability and engineering RNA polymerases that introduce fewer side products that may cause an immune response.
• Further advancements have the potential to enable a new generation of safe, durable and effective mRNA therapeutics for applications beyond vaccines.

Li Li receives funding from NIH.

ReNAgade Therapeutics, a company unlocking the limitless potential for RNA medicines, today announced that Amit D. Munshi, Chief Executive Officer, will present at the 42nd Annual J.P. Morgan Healthcare Conference on Tuesday, January 9, 2024, at 8:30 a.m. PT in San Francisco, CA.

• This release marks significant progress in the development of ChromaCode's software, ensuring accessibility, affordability, and security in laboratory-based diagnostic solutions.
• ChromaCode Cloud is an advanced analysis platform supporting real-time and digital PCR assays.
• The software empowers users to optimize analysis configurations seamlessly, with the cloud-based framework eliminating the need for local hardware or software installation.
• Stringent Regulatory Compliance: ChromaCode Cloud has achieved HITRUST Implemented, i1 certification again this year, reaffirming its commitment to data security.