Initial AX-0810 data show no safety signals after 4 weeks of dosing and pharmacokinetics consistent with non-clinical data; Phase 1 enrollment and dosing in healthy volunteers ongoing with target engagement data expected in H1 2026, followed by inclusion of a patient cohortDevelopment Candidates selected for pipeline programs AX-2402 for Rett syndrome (MECP2, R270X) and AX-2911 for MASH (PNPLA3)Strategic collaboration with Eli Lilly achieved $4.5 million in milestones in 2025, contributing to strong financi ...

CAMBRIDGE, Mass., Jan. 08, 2026 (GLOBE NEWSWIRE) -- Korro Bio, Inc. (Korro) (Nasdaq: KRRO) today announced that Ram Aiyar, Ph.D., Chief Executive Officer and President, will present at the 44th Annual J.P. Morgan Healthcare Conference on Thursday, January 15, 2026, at 10:30 a.m. PT (1:30 p.m. ET). Todd Chappell, Chief Operating Officer, and Loic Vincent, Ph.D., Chief Scientific Officer, will also be participating at the conference. A live webcast of the presentation can be accessed on the “Events & Presenta ...

ProQR Therapeutics Conference Call Summary Company Overview - ProQR Therapeutics is a biopharmaceutical company based in the Netherlands, focused on developing RNA editing medicines to treat genetic and common diseases by modifying human messenger RNA [1][2]. Core Technology and Partnerships - The company has developed a proprietary RNA editing technology over the past 10 years and controls all foundational intellectual property [2]. - In 2021, ProQR entered a $4 billion collaboration with Eli Lilly, receiving $125 million upfront and the potential for $250 million in milestone payments for each of the 15 targets [2]. Pipeline and Clinical Programs - ProQR is pursuing a wholly owned pipeline focusing on liver and CNS indications, with the lead program AX-0810 targeting cholestatic diseases [3]. - The company has initiated a clinical study for AX-0810, expecting first human clinical data in the first half of next year [3][11]. Trial Design and Expectations - The AX-0810 trial will enroll 33 healthy volunteers across three cohorts, with a randomized, placebo-controlled, and double-blinded design [10]. - Initial safety and pharmacokinetic data are expected by the end of this year, while pharmacodynamic data will be available in the first half of next year [11]. Unmet Medical Needs - The target diseases, Primary Sclerosing Cholangitis (PSC) and Biliary Atresia (BA), have no approved therapies and represent a significant unmet medical need [7][14]. - The company aims to develop a medicine that addresses this need while generating a robust dataset to validate its technology [8]. Safety and Efficacy Considerations - ProQR believes that pruritus (itching) associated with high bile acid levels is driven by inflammation rather than bile acids themselves, supported by data from healthy individuals with high bile acid levels who do not experience pruritus [17][22]. - The company aims for a twofold increase in bile acid levels in serum to halt disease progression, with a linear correlation between editing percentage and bile acid concentration [23][25]. Future Development Plans - Following the healthy volunteer study, ProQR plans to conduct a Phase 1B study in PSC patients, with results expected before the end of next year [13]. - The company is exploring accelerated development plans for potential approval based on efficacy data [31]. Rett Syndrome Program - ProQR is also developing a program for Rett syndrome, a neurodevelopmental disease caused by the absence of the MECP2 protein, with the potential to restore normal function through RNA editing [34]. - The program is co-financed by the Rett Syndrome Research Trust, and a clinical candidate is expected to be selected soon [35]. Differentiation from Gene Therapy - The RNA editing approach allows for precise restoration of the MECP2 protein without the risk of overexpression, which is a concern in gene therapy [37]. - ProQR plans to target specific mutations, particularly stop codon mutations, which affect approximately 5,000 patients each [38]. Conclusion - ProQR Therapeutics is positioned to address significant unmet medical needs in liver diseases and Rett syndrome through innovative RNA editing technologies, with ongoing clinical trials and strategic partnerships enhancing its development pipeline [1][2][34].

Summary of Wave Life Sciences Conference Call Company Overview - **Company**: Wave Life Sciences (NasdaqGM: WVE) - **Focus**: Building a fully integrated RNA medicines company capable of unlocking the potential of genetic medicines, particularly in RNA editing and siRNA technologies [3][4][39] Key Programs and Developments 1. Inhibin E (WVE-007) - **Target**: Obesity, focusing on fat loss without impacting lean mass - **Mechanism**: Utilizes siRNA to knock down inhibin E, leading to significant fat loss while preserving muscle mass - **Preclinical Results**: Demonstrated a 70% reduction in activin E levels in DIO mice, translating to fat loss equivalent to GLP-1s without affecting lean mass [10][12][13] - **Clinical Data**: - 75 mg cohort showed a 55% reduction in activin E sustained over six months - 240 mg cohort achieved a 75% reduction at day 29, with ongoing decline [13][14] - **Future Outlook**: Q4 will provide three-month data on the 240 mg cohort, with further insights expected in Q1 2026 [14][20] 2. Alpha-1 Antitrypsin Deficiency (AATD) - **Mechanism**: RNA editing to convert Z protein to M protein, enhancing protein levels in patients - **Clinical Results**: At the lowest dose, patients showed a return to near-normal levels of total protein, with significant increases in M protein [32][34] - **Upcoming Data**: Focus on the 400 mg dose cohort to assess durability and editing efficiency [32][37] 3. PNPLA3 Program (WVE-008) - **Target**: Liver disease associated with homozygous mutations - **Potential Impact**: Could address a large patient population (approximately 9 million) at risk of liver diseases, with a unique mechanism that siRNAs cannot treat [39][40] Industry Context and Market Opportunity - **Obesity Treatment Landscape**: The company aims to shift the paradigm in obesity treatment from frequent dosing (weekly/monthly) to potentially once or twice a year with their siRNA therapies, addressing a significant global health issue [27][28] - **Market Size**: The global obesity market presents a substantial opportunity, with the potential to treat a billion patients worldwide [27] Important Considerations - **Kinetics of Fat Loss**: The company emphasizes the importance of understanding the kinetics of fat loss versus total weight loss, particularly in the context of preserving muscle mass [18][19][21] - **Regulatory Perspective**: The FDA's framing of healthy weight loss has influenced the company's approach to defining success in their clinical trials [17][18] Conclusion - Wave Life Sciences is positioned to make significant advancements in genetic medicine, particularly in obesity and liver disease, with promising data expected in the near future. The focus on innovative RNA technologies and the potential for long-term treatment regimens could disrupt current treatment paradigms in these areas [39][40]

Core Insights - Korro Bio, Inc. has provided updates on its Phase 1/2a REWRITE clinical trial for KRRO-110, reported third-quarter financial results, and outlined strategic business decisions [1][5]. Clinical Trial Update - KRRO-110 successfully generated functional M-AAT protein in patients with Alpha-1 Antitrypsin Deficiency (AATD), although the protein levels were below preclinical projections [2][5]. - The REWRITE trial has completed all six planned single ascending dose cohorts in healthy volunteers, with no dose-limiting toxicities observed [4][7]. - The trial is currently evaluating KRRO-110 in two AATD patient cohorts at doses of 0.6 mg/kg and 0.8 mg/kg [4][7]. Development Candidates - Korro has nominated KRRO-121, a GalNAc-conjugated construct aimed at treating hyperammonemia, marking an expansion of its RNA editing platform [3][5]. - The company plans to advance KRRO-121 and a GalNAc version for AATD patients into clinical trials in the second half of 2026 and 2027, respectively [3][5]. Financial Results - As of September 30, 2025, Korro reported cash, cash equivalents, and marketable securities totaling $102.5 million, down from $163.1 million at the end of 2024 [12][26]. - Collaboration revenue for the third quarter of 2025 was $1.1 million, compared to no revenue in the same period of 2024 [13]. - Research and development expenses decreased to $13.8 million from $16.0 million year-over-year, while general and administrative expenses also saw a decline [14][15]. Strategic Restructuring - The company is implementing a strategic restructuring that will reduce its workforce by approximately one-third to focus on generating clinical data and advancing GalNAc-conjugated programs [3][18]. - This restructuring is expected to extend Korro's cash runway into the second half of 2027, with one-time restructuring charges estimated at $2.4 million [18]. Regulatory Milestones - KRRO-110 has received Investigational New Drug clearance from the FDA, along with Fast Track and Orphan Drug Designations [7][19]. - The company is prioritizing high-potential GalNAc-conjugated programs targeting the liver, including KRRO-121 [7][8].

Summary of Wave Life Sciences FY Conference Call Company Overview - **Company**: Wave Life Sciences (NasdaqGM: WVE) - **Industry**: RNA medicines, focusing on oligonucleotides, RNA editing, and genetic targets [3][4] Core Points and Arguments RNA Medicines and Pipeline - Wave Life Sciences is positioned at the forefront of RNA medicines, utilizing a proprietary chemistry engine to accelerate the development of oligonucleotides, including siRNA and RNA editing [3] - The company has successfully progressed from identifying genetic targets to generating meaningful human therapeutic data within 24 months, exemplified by their INHBE program for obesity [3] Alpha-1 Antitrypsin Program (WVE-006) - WVE-006 is the first RNA editing program to enter clinical trials, targeting alpha-1 antitrypsin deficiency [5] - The program achieved approximately 65% editing on protein levels, with a notable increase in protein response during acute exacerbations, demonstrating the potential for effective treatment [6][10] - The treatment paradigm is shifting from traditional IV protein replacement therapy to a more sustainable editing approach that can generate protein during acute events [9][10] - The FDA has not set a specific threshold for treatment efficacy, but the original approval for protein replacement therapy was based on an 11-micromolar threshold [19][20] Competitive Landscape - Wave Life Sciences is the only non-LNP derived therapy in the RNA editing space, differentiating itself through safety and durability by avoiding LNPs, which can cause liver injury [21][22] - The company emphasizes the specificity of its RNA editing approach, avoiding off-target effects and aberrant proteins, which are common in DNA editing [22][23] Obesity Program (INHBE - Program 007) - The INHBE program targets inhibin E, leveraging human clinical genetics to address obesity without inducing starvation or lean mass loss [28][29] - Wave has demonstrated significant reductions in activin E protein, correlating with weight loss similar to semaglutide, while maintaining lean muscle mass [30][31] - The potential for a once or twice yearly maintenance therapy is highlighted, with ongoing studies to assess fat loss and metabolic health [36][38] Future Studies and Market Strategy - Wave plans to conduct phase two studies on obese patients to evaluate fat reduction and sustainability of weight loss, with a focus on reducing reliance on GLP-1 therapies [42][43] - The company is exploring strategic collaborations while maintaining a wholly owned asset approach, aiming for clear registrational paths in the obesity landscape [42][43] Other Important Content - The company is optimistic about the potential for its therapies to transform patient outcomes in both alpha-1 antitrypsin deficiency and obesity [23][30] - Wave Life Sciences is focused on delivering data that demonstrates the efficacy and safety of its treatments, with upcoming data releases expected to provide further insights into their therapeutic potential [44][45]

Financial Data and Key Metrics Changes - Revenue for Q3 2025 was $7.6 million compared to a net loss of $7.7 million in the prior year quarter, attributed to the timing of revenue recognized under the collaboration agreement with GSK [22] - Research and development expenses increased to $45.9 million from $41.2 million year-over-year, driven by advancements in the inhibin E and RNA editing programs [22] - General and administrative expenses rose to $18.1 million from $15 million in the prior year quarter, primarily due to share-based compensation [22] - Net loss for Q3 2025 was $53.9 million, an improvement from a net loss of $61.8 million in the prior year quarter [23] - Cash and cash equivalents at the end of Q3 2025 were $196.2 million, down from $302.1 million as of December 31, 2024, but extended cash runway into Q2 2027 with additional proceeds [24] Business Line Data and Key Metrics Changes - The company highlighted significant advancements in the WVE-007 program, showing dose-dependent reductions in activin E levels, with reductions of 56%, 75%, and 85% for different dosing cohorts [8][9] - WVE-006 demonstrated the potential to restore physiological AAT production, achieving AAT levels of up to 13 micromolar and a 60% decrease in mutant ZAAT protein [13] - The DMD program, WVEN531, showed a statistically significant improvement in time to rise, with a 3.8 seconds improvement compared to natural history [18] Market Data and Key Metrics Changes - The company is addressing a significant market need with an estimated 9 million homozygous individuals living with PNPLA3I148M liver disease in the US and Europe, who are at a nine-fold higher risk of dying from their liver disease compared to non-carriers [16] - The obesity market is highlighted as having over 1 billion individuals living with obesity, many of whom lack access to current GLP-1 therapies, presenting a unique opportunity for the company's treatments [55] Company Strategy and Development Direction - The company is focused on advancing its pipeline, particularly in RNAi and RNA editing, with a strategy to leverage proprietary chemistry and platform innovations [5] - There is a strong emphasis on developing non-incretin treatment approaches for obesity, with WVE-007 positioned as a potential maintenance therapy to prevent rebound weight gain [6][11] - The company aims to build on its momentum in the obesity space, with plans for further studies and potential partnerships to enhance development [43] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the progress made in clinical trials, particularly with WVE-007 and WVE-006, and the potential for these therapies to transform treatment paradigms [25] - The management team acknowledged the competitive landscape in the obesity market and the need for innovative solutions that provide durable results without the side effects associated with current therapies [55] - There is confidence in the ability to meet regulatory requirements and achieve clinical milestones, with ongoing discussions with the FDA regarding imaging endpoints for Huntington's disease [29][30] Other Important Information - The company is preparing for a global potentially registrational phase 2-3 study of WVE-003 in adults with Huntington's disease, using caudate volume as a primary endpoint [19] - The company has received positive feedback from key opinion leaders regarding the potential of its RNA editing programs to address unmet medical needs [14] Q&A Session Summary Question: Inquiry about cholesterol fat mobilization post-inhibin knockdown - Management confirmed no observed changes in increased lipids and deposits in the liver from preclinical studies, indicating positive findings regarding fat mobilization [26][27] Question: Pre-IND meeting with FDA regarding Huntington's imaging endpoints - Management confirmed alignment with the FDA on using MRI as an imaging endpoint, emphasizing the importance of well-designed clinical trials [29] Question: Changes in gene expression timing in obesity studies - Management noted that while gene expression changes occur over time, early engagement of targets and sustained suppression of proteins are observed [34] Question: Insights on acute phase response in AATD patient - Management indicated that the patient responded as expected, with no new insights beyond the initial findings [48] Question: Future studies beyond NYTE for obesity program - Management expressed interest in exploring further studies and potential strategic partnerships, while noting that the GSK collaboration would not hinder these opportunities [62]

Core Insights - ProQR Therapeutics reported its financial and operational results for Q3 2025, highlighting significant advancements in its RNA editing technology platform, Axiomer, and the initiation of a first-in-human study for its lead program AX-0810 [1][2]. Financial Highlights - As of September 30, 2025, ProQR held cash and cash equivalents of approximately €106.9 million, a decrease from €149.4 million at the end of 2024 [4]. - The net cash used in operating activities for the nine-month period ended September 30, 2025, was €39.4 million, compared to €27.0 million for the same period in 2024 [4][6]. - The net loss for the nine-month period was €33.3 million, or €0.32 per diluted share, compared to a net loss of €18.5 million, or €0.23 per diluted share, for the same period last year [7][16]. Recent Progress and Upcoming Events - The company received CTA authorization for the Phase 1 trial of AX-0810, targeting NTCP for cholestatic diseases, and is initiating the study in healthy volunteers [4][5]. - Initial safety, tolerability, and pharmacokinetic (PK) data from Cohort 1 are expected by year-end 2025, with target engagement data anticipated in the first half of 2026 [5]. - ProQR's Axiomer pipeline includes programs targeting various diseases, such as AX-2402 for Rett syndrome and AX-2911 for fatty liver disease, with updates expected in the coming months [5]. Collaboration and Milestones - The company achieved certain milestones in its collaboration with Eli Lilly, amounting to $2.0 million (~€1.8 million) [6]. - ProQR continues to execute its partnership with Eli Lilly, with potential data updates and milestone income from the ongoing collaboration [5]. Research and Development Costs - Research and development costs for the nine-month period ended September 30, 2025, were €34.8 million, compared to €25.7 million for the same period last year [6]. - General and administrative costs were €11.2 million for the same period, up from €9.7 million in 2024 [6].

Company Overview - ProQR is pioneering ADAR-mediated RNA editing with its Axiomer platform, moving from invention to clinical trials [11, 17] - The company has a strong intellectual property estate and strategic partnerships with Eli Lilly and Rett Syndrome Research Trust [17, 135] - ProQR's cash and cash equivalents were €119.8 million as of the end of Q2 2025, providing a runway into mid-2027 [18, 136] Pipeline and Clinical Development - AX-0810, targeting NTCP for cholestatic diseases, has received CTA authorization and is entering a Phase 1 clinical trial [17, 21] - The Phase 1 trial aims to establish safety, PK, and target engagement in healthy volunteers [21] - AX-2402, for Rett syndrome, is rapidly advancing, positioning ProQR at the forefront of neurological RNA editing medicines [21] AX-0810 and Cholestatic Diseases - Cholestatic diseases, including Primary Sclerosing Cholangitis (PSC) affecting approximately 80,000 adults and Congenital Biliary Atresia affecting approximately 20,000 pediatrics, have high unmet medical needs [24, 38] - AX-0810 is a first-in-class RNA editing therapy designed to modulate the NTCP channel, limiting bile acid uptake while preserving other channel functions [31] - The therapeutic goal of AX-0810 is to reduce inflammation and fibrosis, alleviate symptoms in PSC and BA, and prevent or delay cirrhosis, organ failure, and transplant [36] AX-0810 Clinical Trial Design - The Phase 1 trial is a multiple ascending dose (MAD) study with N=33 participants (24 on treatment, 9 on placebo) across three cohorts: 3mg/kg, 6mg/kg, and 9mg/kg (TBC) [103, 104] - The trial will assess safety, tolerability, and PK of AX-0810, and confirm target engagement as measured by changes in bile acid levels, bile acid profile, and TUDCA challenge [107] - Initial Cohort 1 safety, tolerability, and PK data are expected toward the end of 2025, with target engagement data on all cohorts anticipated in H1 2026 [107, 131]

Core Insights - Wave Life Sciences is hosting its 2025 Research Day, focusing on RNA editing and RNA interference (RNAi) technologies [2]. Group 1: Event Overview - The event is being recorded and is accessible for future reference [1]. - The presentation materials will be available on the company's website in the Investors section after the call [2]. Group 2: Management Statements - Management may provide forward-looking statements during the presentation, which are subject to various risks and uncertainties [3].