Summary of Wave Life Sciences FY Conference Call Company Overview - **Company**: Wave Life Sciences (NasdaqGM: WVE) - **Focus**: RNA medicine and oligonucleotide optimization, with a strong emphasis on human clinical genetics and innovative chemistry [2][4] Key Points from 4Q Results - **Pipeline Development**: Significant advancements in the pipeline, particularly in obesity treatment with Inhibin E and RNA editing for alpha-1 antitrypsin deficiency [3][4] - **Clinical Data**: Upcoming clinical data expected for Inhibin E, including 240 mg six-month data and 400 mg three-month data [3][15] Obesity Treatment Insights - **Targeted Mechanism**: Inhibin E aims to address obesity by reducing visceral fat without significant loss of lean muscle mass, which is crucial for metabolic health [6][10][14] - **Clinical Feedback**: Key opinion leaders (KOLs) are interested in the mechanism that focuses on body composition rather than just weight loss [5][12] - **Initial Results**: At three months, the 240 mg dose showed nearly 10% reduction in visceral fat and a 0.9% increase in lean mass, indicating a favorable profile compared to existing therapies [13][19] Future Expectations - **Data Anticipation**: The 400 mg cohort data is expected to provide insights into dose dependency and time dependency of fat loss [17][19] - **Regulatory Considerations**: The company is confident in meeting FDA requirements for obesity drugs, focusing on both weight loss and body composition improvements [45][49] RNA Editing Program - **Alpha-1 Antitrypsin Deficiency**: The program aims to correct the deficiency by producing endogenous proteins, with potential for accelerated approval based on existing data [66][75] - **Regulatory Engagement**: Discussions with regulators are ongoing to establish a clear path for market entry and confirmatory studies [75][76] Strategic Insights - **Combination Therapies**: There is potential for Inhibin E to be used in combination with GLP-1 therapies, enhancing treatment options for obesity [52][54] - **Market Potential**: The company sees a significant opportunity in addressing the obesity epidemic, with a scalable and accessible treatment model [58][60] Innovations and Future Pipeline - **Bifunctional Modality**: The company is exploring bifunctional modalities that can target multiple pathways, enhancing therapeutic efficacy [78][80] - **Targeting Multiple Cell Types**: Future developments may include targeting both adipocytes and hepatocytes, broadening the therapeutic applications in cardiometabolic diseases [80] Conclusion - Wave Life Sciences is positioned at a critical juncture with innovative approaches to RNA medicine, particularly in obesity and genetic disorders. The upcoming clinical data and regulatory discussions will be pivotal in shaping the future of its therapies and market presence.

Wave Life Sciences (NasdaqGM:WVE) Q4 2025 Earnings call February 26, 2026 08:30 AM ET Company ParticipantsCatherine Novack - Director of Healthcare Equity ResearchCha Cha Yang - Senior Associate, Biotech Equity ResearchCheng Li - Director of Biotech Equity ResearchChristopher Wright - CMOChristopher Wright - Chief Medical OfficerErik Ingelsson - Chief Scientific OfficerJoon Lee - Managing DirectorJoseph Schwartz - Senior Managing DirectorKate Rausch - VP of Corporate Affairs and Investor RelationsKyle Moran ...

Wave Life Sciences (NasdaqGM:WVE) Q4 2025 Earnings call February 26, 2026 08:30 AM ET Company ParticipantsCatherine Novack - Director of Healthcare Equity ResearchCha Cha Yang - Senior Associate, Biotech Equity ResearchCheng Li - Director of Biotech Equity ResearchChristopher Wright - CMOErik Ingelsson - Chief Scientific OfficerJoon Lee - Managing DirectorKate Rausch - VP of Corporate Affairs and Investor RelationsKyle Moran - CFOLillian Yuen-Ho - Financial PlannerPaul B. Bolno - President and CEOPaul Bolno ...

Wave Life Sciences (NasdaqGM:WVE) Q4 2025 Earnings call February 26, 2026 08:30 AM ET Speaker14Good morning, welcome to the Wave Life Sciences fourth quarter and full year 2025 earnings conference call. At this time, all participants are in a listen-only mode. As a reminder, this call is being recorded and webcast. I'll now turn the call over to Kate Rausch, Vice President of Corporate Affairs and Investor Relations. Please go ahead.Speaker6Thank you, Sophie. Good morning to everyone on the call. Earlier th ...

CAMBRIDGE, Mass., Feb. 25, 2026 (GLOBE NEWSWIRE) -- Korro Bio, Inc. (Korro) (Nasdaq: KRRO), a biopharmaceutical company developing a new class of genetic medicines based on editing RNA for rare and highly prevalent diseases, today announced that Ram Aiyar, Ph.D., Chief Executive Officer and President, will present at the TD Cowen 46th Annual Health Care Conference being held in Boston, MA at the Boston Marriott Copley Place on March 2nd – 4th, 2026. Dr. Aiyar’s presentation will take place on Tuesday, March ...

While PAH is a rare condition, it represents a sizable commercial opportunity. According to Research and Markets, the global PAH market was valued at $8.29 billion in 2024 and is projected to reach nearly $12 billion by 2030, as demand for novel therapies continues to build.The first Bank of America pick we’re looking at is Inhibikase Therapeutics, a small-cap biotech focused on kinase biology, with its current lead effort aimed at pulmonary arterial hypertension, or PAH. It’s a brutal disease driven by pro ...

Core Insights - Korro Bio, Inc. is hosting a virtual Analyst Day on January 27, 2026, to discuss KRRO-121, a potential treatment for hyperammonemia, featuring insights from management and personal experiences from caregivers [1][2][3] Company Overview - Korro is focused on developing genetic medicines through RNA editing, targeting both rare and prevalent diseases, with a proprietary platform designed for precise and transient single base edits [7] - The company is based in Cambridge, Massachusetts, and aims to leverage established regulatory pathways for oligonucleotide drugs [7] Product Development - KRRO-121 is being developed for the treatment of hyperammonemia in patients with urea cycle disorders (UCDs) and hepatic encephalopathy (HE), utilizing a GalNAc-conjugated construct to activate a biological pathway [2][9] - The company plans to file for regulatory approval for the first-in-human trial of KRRO-121 in the second half of 2026, supported by preclinical data [2][3] Pipeline Updates - At the J.P. Morgan Healthcare Conference, Korro highlighted its momentum across multiple programs, including a GalNAc-conjugated antisense oligonucleotide for Alpha-1 Antitrypsin Deficiency (AATD), which has achieved over 90% in vivo RNA editing [4] - Korro is also expanding its RNA editing platform, OPERA, with promising preclinical data for additional targets, including AMPKγ1 for longevity and TDP-43 for amyotrophic lateral sclerosis (ALS) [4]

Summary of Korro Bio Conference Call Company Overview - **Company**: Korro Bio - **Industry**: Biotechnology, specifically focusing on RNA editing technologies for therapeutic applications Core Points and Arguments 1. **Vision and Technology**: Korro Bio aims to develop transformative medicines for both rare and prevalent conditions by activating biological pathways through RNA editing, which allows for precise modifications without altering DNA [2][4] 2. **RNA Editing Modality**: The company utilizes chemically modified oligonucleotides to edit RNA, enabling single base changes that can impact protein structure and function, offering a transient solution rather than permanent genetic modifications [3][10] 3. **Pipeline Focus**: The lead program, Korro 121, is set to enter clinical trials in the latter half of the year, targeting ammonia reduction in patients with liver conditions [4][11] 4. **Clinical Development**: Korro Bio plans to provide insights into their clinical approach and patient needs during an upcoming educational session on January 27 [5] 5. **Korro 110 Termination**: The company has decided to terminate the Korro 110 program due to unsatisfactory clinical data and structural integrity issues observed in patient trials [12][20] 6. **Market Potential**: Korro Bio identifies significant unmet medical needs in conditions associated with elevated ammonia levels, with a patient population of approximately 4,500 in the U.S. and a similar number in Europe [16][17] 7. **Regulatory Filing**: Anticipation of a regulatory filing for Korro 121 in the second half of the year, with expectations for rapid clinical data generation [11][18] 8. **Safety Profile**: RNA editing is posited to have a high specificity and low likelihood of off-target effects compared to DNA editing, enhancing the safety profile of Korro's therapies [30][31] Additional Important Content 1. **Biological Pathways**: The technology allows for modulation of proteins to activate biological pathways, which has not been achievable with traditional gene therapies [10][28] 2. **Next-Generation Programs**: Korro Bio is advancing towards a next-generation Alpha-1 product that shows promise for achieving over 90% editing in vivo, significantly improving upon previous attempts [22][23] 3. **Unique Therapeutic Approach**: The company emphasizes the transient nature of their therapies, which is particularly beneficial for chronic conditions where permanent changes could lead to complications [36] 4. **Dosing Frequency**: Korro Bio's therapies are designed for infrequent dosing, potentially once a month, which contrasts with the more frequent dosing required by traditional therapies [15][36] 5. **Research and Development**: The company is leveraging machine learning to enhance target identification and therapy design, indicating a forward-thinking approach to drug development [8][30] This summary encapsulates the key points discussed during the conference call, highlighting Korro Bio's innovative approach to RNA editing and its implications for future therapeutic developments.

Pipeline and Milestones - Korro Bio anticipates a regulatory filing for KRRO-121 in the second half of 2026 [26, 55] - The company expects to nominate a development candidate (DC) for its GalNAc-conjugated AATD program in the first half of 2026 [26, 55] - A development candidate is also expected for a third GalNAc-conjugated liver asset in the second half of 2026 [26, 55] KRRO-121 for Hyperammonemia - KRRO-121 targets hyperammonemia by stabilizing an intracellular protein in the liver to enhance ammonia clearance capacity [36, 39] - The addressable patient population for KRRO-121 includes 4,200 U S patients with Urea Cycle Disorders (UCD) and 80,000 U S patients with Hepatic Encephalopathy (HE) [37] - The market opportunity for KRRO-121 is estimated at $1 5 billion for UCD and $2 billion+ for HE [37] AATD Program - Korro Bio terminated the REWRITE clinical trial for KRRO-110 in AATD [27, 42] - The company achieved >90% editing of the SERPINA1 transcript using GalNAc delivery in vivo [43] AMPK Activation for Liver Function - In obese mice, approximately 20% editing was sufficient to normalize liver function and reduce body weight [47] Financial Runway - Korro Bio's cash runway extends into the second half of 2027 [14, 55]

Wave Life Sciences FY Conference Summary Company Overview - **Company**: Wave Life Sciences (NasdaqGM: WVE) - **Industry**: RNA Medicines and Biotechnology Key Points and Arguments RNA Medicines Potential - Wave Life Sciences aims to unlock the potential of RNA medicines to transform human health, emphasizing the foundational role of their proprietary RNA chemistry [2][3] Clinical Portfolio and Innovations - The company has developed a proprietary chemistry engine that allows rapid translation of genetic insights into medicines, exemplified by their obesity therapy, which progressed from mouse data to human clinical data in 18 months [3] - The dominant program discussed is the GalNAc-conjugated INHBE siRNA program, which shows promise in treating obesity by improving body composition through fat reduction while preserving muscle mass [4][11] Manufacturing and Financial Position - Wave Life Sciences has in-house GMP manufacturing capabilities, enabling rapid advancement of their clinical programs [4] - The company entered 2026 with $602 million in cash, sufficient to fund operations into Q3 2028 [5] Clinical Data and Efficacy - The INHBE program has shown a reduction in fat mass and preservation of lean mass, with a focus on visceral fat reduction, which is critical for cardiovascular health [12][27] - Preclinical data indicated a potent and durable reduction in Activin E levels, leading to significant fat loss and muscle preservation [18][27] Treatment Paradigm Shift - Current obesity treatments, particularly GLP-1s, are limited by muscle mass loss. Wave's approach aims to provide a treatment paradigm shift by preserving muscle while reducing fat, potentially allowing for less frequent dosing (once or twice a year) [11][12][20] Future Clinical Studies - The company plans to accelerate the development of WVE-007 in obesity and initiate new clinical studies, including combination and maintenance studies [10][32] - The Phase 2A portion of the INLIGHT study will explore the effects of INHBE in patients with higher BMI and comorbidities [10][32] RNA Editing Programs - Wave Life Sciences is advancing its RNA editing programs, particularly for Alpha-1 Antitrypsin Deficiency (AATD) and PNPLA3-related liver disease, with a focus on correcting genetic mutations to restore protein function [35][41] - The AATD program targets a rare disease affecting approximately 200,000 patients in the US and Europe, with no approved therapies for liver manifestations [35] Bifunctional Constructs - The company is exploring bifunctional conjugates that combine the benefits of RNA editing and siRNA, potentially allowing for a single therapeutic construct that can both silence and upregulate targets [44] Regulatory Pathway and Milestones - Wave Life Sciences is focused on a regulatory pathway that emphasizes body composition changes in obesity studies, with a goal of demonstrating significant fat loss while preserving muscle mass [29][60] - Anticipated milestones include multiple data sets on obesity and updates on RNA editing programs throughout 2026 [44][60] Additional Important Insights - The company emphasizes the importance of body composition in obesity treatment, highlighting the need for therapies that not only reduce weight but also improve metabolic health by targeting visceral fat [12][30] - Wave Life Sciences is positioning itself to address the challenges of long-term adherence to current obesity therapies by offering a more tolerable and effective treatment option [55][58]