Code Biotherapeutics is developing a multivalent synthetic DNA delivery platform, 3DNA®, to enhance the precision and bioavailability of non-viral gene therapies for life-threatening diseases. The company focuses on overcoming challenges such as dose-related toxicity and off-target effects, with a primary research emphasis on Duchenne Muscular Dystrophy.

ExeVir develops novel antibody therapies utilizing a modular platform based on camelid antibody fragments (VHH) for infectious disease control. This technology creates stable, cost-effective therapeutic compounds capable of binding multiple conserved epitopes on pathogens. The company is advancing a pipeline focused initially on COVID-19 and dengue to address public health threats, particularly for vulnerable patient populations.

Iris Medicine is developing small binding RNAs (sbRNAs) that selectively target expanded repeat sequences in mRNA to inhibit the expression of mutant genes associated with repeat expansion disorders. This technology aims to provide effective treatments for conditions like Huntington’s Disease and C9orf72 ALS, where current therapeutic options are limited.

Rinri Therapeutics is developing engineered stem cell therapies specifically for the regeneration of auditory tissues affected by sensorineural hearing loss. This approach aims to restore hearing functionality and improve patient quality of life by addressing the underlying cellular damage caused by various auditory conditions.

Seamless Therapeutics develops disease-modifying gene editing candidates using programmable recombinase technology. This approach enables precise, large DNA edits within the genome without introducing unintended mutations. The company aims to expand the therapeutic potential of gene editing to treat severe genetic diseases.

Switch Therapeutics develops precision neuroscience medicines using its proprietary Conditionally Activated siRNA (CASi) platform. This technology combines features of ASOs and siRNAs into a single molecule for efficient self-delivery and potent knockdown. The CASi platform enables cell-selective targeting to improve therapeutic windows for treating central nervous system diseases.

ViaNautis develops polyNaut®, a proprietary nanovesicle platform that utilizes advanced polymer materials and machine learning to deliver nucleic acid therapeutics with high precision to targeted tissues and cell types. This technology addresses the challenge of safely and effectively delivering genetic medicines, such as mRNA and siRNA, to treat previously untreatable diseases.

Walden Biosciences is developing targeted gene editing therapies to modify renal cell function in patients with chronic kidney disease. This approach aims to improve patient outcomes by reducing reliance on dialysis and kidney transplants.