Camp4 Therapeutics (CAMP) Chardan's 10th Annual Healthcare Conference summary

RNA editing approach

• Korro’s oligonucleotide acts as a landing pad for endogenous ADAR, enabling a single-base RNA change; the drug is distinct from gene therapy and CRISPR and is designed for scalable manufacture.

• Endogenous A-to-I editing occurs in ~2%-3% of cells; Korro sees potential to repair variants and, especially, create amino-acid changes that activate biological pathways.

KRRO-121 in urea cycle disorders

• KRRO-121 edits glutamine synthetase to stabilize the liver protein, which converts ammonia into glutamine; the approach is intended to address urea cycle disorders across mutations.

• Preclinical studies showed blunted ammonia responses across multiple models, including OTC models, and demonstrated editing in monkeys; the program is designed as a pan-UCD treatment.

• A SAD/MAD study is underway, with the first cohort dosed; Korro guided to patient dosing in H1 2027 and data in H2 2027.

• Early clinical goals include lowering or blunting ammonia and showing potential for increased protein intake or reduced nitrogen-scavenger use; UCD patients are often limited to ~20% of normal protein intake.

• Korro estimated a $1.5 billion market opportunity for a pan-UCD therapy, citing ~6,500 U.S. patients and existing nitrogen-scavenger sales of $400+ million among 800 treated patients.

AATD program

• First-generation KRRO-110 used an IV lipid nanoparticle; safety and product stability in Alpha-1 patients were concerns, despite favorable findings in healthy volunteers.

• Korro halted investment in the first-generation product after finding stability differences in patients; the study did demonstrate repaired protein in each treated participant.

• The redesigned candidate uses a conjugate for subcutaneous dosing and achieved >90%-95% editing in animal models; Korro expects to provide 2027 clinical-entry and data timing guidance.

• Korro cited potential differentiation in liver rejuvenation versus DNA-editing approaches and said it aims to offer repeat dosing as an alternative for patients who prefer not to receive a one-time edit.