John Steven Mullaly is a Cohasset, Massachusetts based financial services professional with more than 25 years of experience focused on the biotechnology and life sciences sectors. He earned a Bachelor of Arts in economics from Northeastern University and later completed a Master of Business Administration at the Boston University Questrom School of Business. Over the course of his career, he has worked extensively with private and public life sciences companies across a wide range of development stages, advising on strategic growth, capital markets positioning, and investor communication. Previously serving as a Managing Director at LifeSci Advisors in Boston, he supported small and mid-cap biotechnology companies through institutional investor relations and market engagement. Today, as a private investor, he applies this background to evaluate emerging platforms, including advances in RNA-based therapeutics that are reshaping modern medicine.
Advances in RNA-Based Therapeutics
RNA-based therapeutics are an emerging class of medicines harnessing ribonucleic acid molecules to treat а disease. RNA molecule acts inside cells as a messenger/regulator carrying instructions from DNA to the protein-making system. RNA-based therapeutics modulate gene expression, correct genetic mistakes, or direct cells to make helpful proteins. The RNA-based therapeutics field has grown – with recent progress widening clinical use, improving delivery, and opening paths to treat complex diseases.
Knowledge of the main classes of RNA therapeutics helps rationalize recent progress. One class of such agents, antisense oligonucleotides (ASOs), is short, single-stranded synthetic nucleic acids that bind to specific RNA targets. Small interfering RNAs (siRNAs) contain two strands and guide а cell process that breaks down matching messenger RNA, which stops protein production. mRNAs or Messenger RNAs serve as templates that cells use to produce а chosen protein.
MicroRNAs (miRNAs) help regulate gene activity by binding to mRNA and slowing or stopping protein output. RNA aptamers are another class that assume shapes that adhere tightly to proteins or other targets. They prevent proteins from acting, much as antibodies do.
A key progress in RNA therapeutics is improved delivery systems. RNA molecules often break down quickly and struggle to reach many tissues. New delivery methods protect RNA, move it into cells, and reduce immune reactions. One method is through lipid nanoparticles (LNPs). LNPs are tiny fat-based particles that carry RNA medicines into cells. They shield RNA, help it enter cells, and support its release where it can act.
New designs show better stability and lower immune effects. Another delivery method uses small-molecule ligands linked to siRNA. These ligand conjugates direct treatment to specific cells, allow lower doses, and support longer effects. Researchers use them for liver, metabolic, and genetic disorders.
Virus-like particles (VLPs) and extracellular vesicles (EVs) are other new delivery systems that show good promise. VLPs copy а virus’s outer structure but lack infectious genetic material. Their protein shells protect RNA drugs from enzymes that would normally degrade them. These particles can enter cells with ease since cells recognize them in ways similar to viruses. This property helps RNA cross cell membranes. Scientists can engineer VLPs to target specific cells and reduce effects on healthy tissue.
Meanwhile, EVs are small sacs that cells release to send signals. They can carry RNA through the bloodstream without triggering strong immune reactions. These vesicles can cross walls such as the blood-brain barrier and deliver treatment to the brain. Compared to many synthetic carriers, they have fewer side effects.
Another major progress involves precision RNA editing tools. Many RNA therapies aim to fix or control gene activity, but older methods often lack accuracy and raise the risk of unwanted changes. New editing tools change single RNA letters without altering DNA. This approach allows correcting disease-causing errors at the RNA level only. Tech like CRISPR-Cas13 -and related RNA editing platforms – can recognize and modify specific RNA sequences with high precision, reducing risk of unintended off-target effects.
Scientists are also using AI to drive design and optimization in RNA therapeutics. AI models help guide RNA drug development with data. They can analyze large genomics datasets to identify disease targets and support selection among ASO, siRNA, or mRNA strategies. AI systems can also improve RNA sequence design so molecules remain stable and function as intended. By combining genetic markers with clinical data, AI also supports patient-specific treatment design. Moreover, safety assessment improves because AI models can help predict off-target effects and immune responses before advanced studies.
Circular RNA therapeutics represent another vital progress. This special type of RNA forms a closed loop instead of the usual linear strand. This shape increases stability since enzymes struggle to break the structure down inside cells. Circular RNA also tends to trigger fewer immune reactions than many linear RNA formats. Some companies have already launched human trials of circRNA-based cancer vaccines and are testing circRNAs in Alzheimer’s disease models.
About John Steven Mullaly
John Steven Mullaly is a life sciences focused investor based in Cohasset, Massachusetts, with a professional background spanning more than two decades in healthcare finance. He previously served as a Managing Director at LifeSci Advisors, where he advised small and mid-cap biotechnology companies on investor relations, capital markets strategy, and institutional engagement. Drawing on earlier experience as an institutional equity healthcare specialist at leading investment banks, he brings deep familiarity with public and private company financing environments. In addition to his professional work, he volunteers with a New England adaptive ski program.
