duchenne-muscular-dystrophy
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The $85 Million Bet That a DNA Scaffold Can Make Gene Therapy Re-Dosable
Code Biotherapeutics is trying to turn DNA into the FedEx network for genetic medicine. Its modular 3DNA scaffold promises bigger payloads, precise addresses and repeat delivery - but the public evidence is still preclinical.
Allysta Pharmaceuticals, Inc.
Allysta Pharmaceuticals is a private, venture-backed, clinical-stage biopharmaceutical company developing first-in-class therapeutic peptides that target adiponectin signaling. Its lead compound, ALY688, is described as the first adiponectin receptor agonist to enter human testing, with anti-inflammatory, anti-fibrotic, cell-regenerative and metabolic properties. Allysta is advancing ALY688 as an ophthalmic solution for dry eye disease and as an extended/sustained-release formulation for systemic conditions such as NASH/MASH and Duchenne muscular dystrophy.
SonoThera
SonoThera is a South San Francisco biotech developing an ultrasound-guided, nonviral platform for delivering genetic medicines. Instead of the viral vectors that dominate gene therapy, it uses focused ultrasound and injectable microbubbles - a process called sonoporation - to open transient pores in target cells and shuttle DNA or RNA payloads into specific organs. The pitch is delivery that is targeted, potentially redosable, and decoupled from the immune and manufacturing headaches of viruses. The company raised a $125M Series B in June 2026 and is pushing lead programs in Duchenne muscular dystrophy and polycystic kidney disease toward the clinic.