ADDED
In this Annual Report, when we refer to our leads or lead candidates, we are referring to our oligonucleotides that we are researching for potential nomination as a development candidate.
When we refer to our development candidates, we generally mean an oligonucleotide lead candidate that we have nominated for clinical development.
If preclinical studies or clinical trials of any lead candidates or development candidates fail to demonstrate safety and efficacy to the satisfaction of regulatory authorities or do not otherwise produce positive results (such as we experienced in the Phase 1/2a REWRITE clinical trial of KRRO-110 for AATD), we may incur additional costs or experience delays in completing, or ultimately be unable to complete, the development and commercialization of such development candidates.
We may not realize the anticipated benefits of our organizational streamlining and workforce reduction..
We have yet to successfully complete clinical development of any development candidate, and any favorable results we have may not be predictive of results that may be observed in later preclinical studies or clinical trials, such as our experience in the Phase 1/2a REWRITE clinical trial of KRRO-110 for AATD.
We may not be successful in finding strategic collaborators for continuing development of certain of our development candidates or successfully commercializing or competing in the market for certain indications; and we may not see any benefit from our collaboration agreement with Novo Nordisk, which, as of November 2025, is paused for 12 months.
Our business may be impacted by macroeconomic conditions, including inflation, rising interest rates and volatile market conditions, and other uncertainties beyond our control, including geopolitical events, such as recent U.S.
We are generating a portfolio of differentiated programs that are designed to harness the body s natural RNA editing process to effect a precise yet transient change to a single nucleoside (adenosine to an inosine edit).
We use an oligonucleotide-based approach and expect to bring our medicines to patients by leveraging our proprietary platform with precedented delivery modalities, including N-acetylgalactosamine, or GalNAc, -conjugated delivery for subcutaneous administration, manufacturing know-how, and established regulatory pathways of approved oligonucleotide medicines.
By editing RNA to mimic a SNV, we believe we will be able to address unmet patient need by transiently modifying gene expression and the resultant protein function.
REMOVED
We have dosed very few participants in our Phase 1/2a REWRITE clinical trial of KRRO-110 for Alpha-1 Antitrypsin Deficiency, or AATD, and not received any clinical trial results for any of our proposed delivery methods or RNA editing approaches in clinical trials.
Any favorable results we may have may not be predictive of results that may be observed in later preclinical studies or clinical trials.
If preclinical studies or clinical trials of any product candidates we may identify and develop fail to demonstrate safety and efficacy to the satisfaction of regulatory authorities or do not otherwise produce positive results, we may incur additional costs or experience delays in completing, or ultimately be unable to complete, the development and commercialization of such product candidates.
If we experience delays or difficulties in the enrollment of patients in clinical trials, or other delays in our clinical trials, our receipt of necessary regulatory approvals could be delayed or prevented.
Our business may be impacted by macroeconomic conditions, including fears concerning the financial services industry, inflation, rising interest rates and volatile market conditions, and other uncertainties beyond our control.
We are generating a portfolio of differentiated programs that are designed to harness the body s natural RNA editing process to effect a precise yet transient single base edit.
Using an oligonucleotide-based approach, we expect to bring our medicines to patients by leveraging our proprietary platform with precedented delivery modalities, manufacturing know-how, and established regulatory pathways of approved oligonucleotide medicines.
By editing SNVs on RNA, we believe we will be able to address unmet patient need by transiently modifying gene function.
Our RNA editing approach involves co-opting this endogenous editing system via a proprietary engineered oligonucleotide to introduce precise edits to RNA.
The application of such an approach can provide the ability to alter an SNV and affect biology in meaningful ways.