Scientific Landscape: Gene editing has entered a new era beyond traditional CRISPR-Cas9.
Next-generation tools like base editors (e.g., cytosine and adenine base editors) and prime editors provide
unmatched precision by enabling single-nucleotide edits without introducing double-strand breaks. This mitigates the
risk of off-target effects and large-scale genomic rearrangements, making these tools more suitable for clinical
applications. The advent of programmable RNA-guided editors also facilitates more predictable and safer therapeutic
interventions.
Delivery Challenges: Despite their potential, the full therapeutic value of gene editing
technologies remains contingent upon delivery. In vivo editing requires efficient, cell-specific transport of gene
editing machinery. Lipid nanoparticles (LNPs), adeno-associated viruses (AAVs), and emerging non-viral vectors
(e.g., engineered exosomes) are being developed to overcome biological barriers. Companies like Verve Therapeutics
are pioneering LNP-based cardiovascular therapies, while Beam Therapeutics is advancing multiplex editing for sickle
cell and thalassemia.
Market & Pipeline: According to BIS Research, the global gene editing market is projected to
surpass $20 billion by 2030, growing at a CAGR of 30%+. Over 100 active CRISPR-based therapies are in various stages
of development globally. Concurrently, RNA therapeutics have expanded post-COVID, with more than 250 active clinical
programs in siRNA, mRNA, and self-amplifying RNA. Platforms such as Moderna, Arcturus, and Alnylam are now pushing
RNA beyond vaccines into rare diseases, oncology, and immune modulation.
Investment Relevance: Investors are targeting dual-platform companies that combine proprietary
editing enzymes with novel delivery mechanisms. The potential for milestone payments and licensing deals with large
pharma is substantial. Delivery-focused startups are particularly attractive due to their horizontal applicability
across multiple disease states.