KRRO — what changed in the latest 10-Q

Text added vs the prior filing · source: 10-Q · 2026-05-07

• We are a biopharmaceutical company leveraging a novel oligonucleotide promoted editing of RNA, or OPERA, platform to develop a new class of genetic medicines for rare and highly prevalent diseases. OPERA provides precise, tissue-directed delivery of oligonucleotides that modify the targeted native m…

• The versatility of RNA editing combined with our OPERA platform broadens the therapeutic target space significantly. The advent of large-scale genome sequencing has progressively revealed causal genetic variation underlying several human diseases, both rare and highly prevalent. Genetic mutations, i…

• We continue to make meaningful advancements across our programs, including KRRO-121 as a potential first-in-class treatment for hyperammonemia that has the potential to address substantial unmet need in patients with poor ammonia control, including those with urea cycle disorders, or UCD, and hepati…

• We are also advancing early-stage research and development programs highlighted by a GalNac-conjugated oligonucleotide for AATD. We are on track to announce our AATD development candidate in second quarter of 2026 and plan to nominate a development candidate for a third GalNAc-conjugated program in …

• Since inception, we have focused primarily on organizing and staffing our company, business planning, raising capital, securing related intellectual property, and conducting research and development activities for our potential programs and development candidates. Since inception, we have funded our…

Text removed vs the prior filing · source: 10-Q · 2025-11-12

• We are a clinical-stage biopharmaceutical company with a mission to discover, develop and commercialize a new class of genetic medicines based on editing RNA, enabling the treatment of both rare and highly prevalent diseases.

• 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. By editing RNA instead of DNA, we are expanding the reach of genetic medicines by delivering additional precision and tunab…

• The versatility of RNA editing combined with our RNA platform broadens the therapeutic target space significantly. While our approach can be used to repair pathogenic single nucleotide variants, or SNVs, we can also engineer de novo SNVs and change amino acids on proteins to endow them with desired …

• We have investigated KRRO-110 as a treatment for AATD where, using our proprietary RNA editing approach, we aimed to repair a pathogenic variant on RNA. KRRO-110 did not reach projected levels of functional protein following a single administration as of the data cutoff date, and we are now pivoting…

• KRRO-110 Safety Observations (as of the November 6, 2025 Data Cutoff Date):

Text added vs the prior filing · source: 10-Q · 2026-05-07

• We expect that it will be many years, if ever, before we have an RNA editing product ready for commercialization. To become and remain profitable, we must develop and, either directly or through collaborators, eventually commercialize a product or products with market potential. This will require us…

• In November 2025, we announced that we did not reach projected levels of protein in patients in our Phase 1/2a REWRITE clinical trial of KRRO-110 for AATD (which we subsequently terminated), and our other programs are in early stages of development. In addition, we undertook workforce reductions in …

• Our expenses could increase beyond expectations if we are required by the FDA, the EMA, HREC, or TGA, or other regulatory authorities to perform clinical and other studies in addition to those that we currently anticipate. Even if one or more of the candidates we may develop are approved for commerc…

• approved development candidate. Additionally, such products may become subject to unfavorable pricing regulations, third-party reimbursement practices or healthcare reform initiatives. Even if we are able to generate revenues from the sale of any approved development candidates, we may not become pr…

• We expect our expenses to continue to increase in connection with our ongoing activities, particularly as we identify, continue the research and development of, initiate preclinical studies and clinical trials of, and seek marketing approval for, our lead candidates and development candidates. Becau…

Text removed vs the prior filing · source: 10-Q · 2025-11-12

• We expect that it will be many years, if ever, before we have an RNA editing product ready for commercialization. To become and remain profitable, we must develop and, either directly or through collaborators, eventually commercialize a product or products

• with market potential. This will require us to be successful in a range of challenging activities, including identifying product candidates, completing preclinical studies and clinical trials of product candidates, obtaining marketing approval for these product candidates, manufacturing, marketing a…

• We did not reach projected levels of protein in patients in our Phase 1/2a REWRITE clinical trial of KRRO-110 for AATD and our other programs are in early stages of development. Because of the numerous risks and uncertainties associated with developing oligonucleotide product candidates and the risk…

• Our expenses could increase beyond expectations if we are required by the FDA, the EMA, HREC, TGA, HDEC or other regulatory authorities to perform clinical and other studies in addition to those that we currently anticipate. Even if one or more of the product candidates we may develop are approved f…

• We expect our expenses to continue to increase in connection with our ongoing activities, particularly as we identify, continue the research and development of, initiate preclinical studies and clinical trials of, and seek marketing approval for, product candidates.