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- Results presented from over a decade of treatment with velmanase alfa in alpha-mannosidosis – - Company also presents immunogenicity and tolerability data from pegunigalsidase alfa clinical trial program in Fabry disease – - Additional presentations provide new insights on prevalence, diagnosis, and progression of these rare lysosomal storage disorders – BOSTON, Feb. 8, 2024 /PRNewswire/ -- Chiesi Global Rare Diseases, a business unit of the Chiesi Group established to deliver innovative therapies and solutions for people living with rare diseases, today announced the presentation of long-term data from up to 12 years of treatment with velmanase alfa in patients with alpha-mannosidosis (AM). The company also announced the presentation of immunogenicity and tolerability data from its pegunigalsidase alfa clinical trial program in Fabry disease, as well as analyses of the prevalence, diagnosis, and progression of these rare lysosomal storage disorders. The data are being presented during the 20th Annual WORLDSymposium™ Research Meeting that is being held February 4-9, 2024, in San Diego, California. "This year's WORLDSymposium marks a significant moment for people living with rare lysosomal storage disorders including alpha-mannosidosis and Fabry disease, and their healthcare teams including clinicians and nurses, who now have access to recently approved treatment options and an important opportunity to understand the impact of these therapies and the barriers that must be addressed to improve diagnosis and disease management," said Giacomo Chiesi, head of Chiesi Global Rare Diseases. "Our company is driven by a commitment to rare disease communities around the world and we are taking full advantage of this conference to share our extensive clinical research in AM, which now spans more than a decade, and in Fabry disease, where we are continuing to evaluate data from our comprehensive development program. We are also emphasizing the importance of the caregiver and patient voice, reporting results from a range of surveys and analyses, hosting symposia, and presenting a special film screening that will help healthcare professionals identify and treat people in these communities in the years ahead." Results from long-term treatment with velmanase alfa in AM In an oral session, Prof. Nathalie Guffon, M.D., Reference Centre of inherited metabolic disease in Femme Mère Enfant Hospital, Hospices Civils of Lyon, France, is presenting results of an analysis that evaluated long‑term efficacy of up to 12 years of treatment with velmanase alfa, which is the first and only enzyme replacement therapy (ERT) available for people with AM. Pooled analyses demonstrate tolerability of pegunigalsidase alfa in Fabry disease and low incidence of de novo anti-drug antibodies Several poster presentations are highlighting immunogenicity and tolerability data from the pegunigalsidase alfa clinical trial program in Fabry disease. John Bernat, M.D., Ph.D., Medical Director of the Iowa Lysosomal Storage Disorders Center, is presenting a poster describing characteristics of anti-drug antibodies (ADAs) reacting with pegunigalsidase alfa, and the patients who develop ADAs, using data from an integrated clinical trial dataset. The analysis includes 108 patients, 27 (25%) of whom were ADA+ before receiving their first dose of pegunigalsidase alfa. There was a low incidence of de novo ADAs (n=17) occurring predominantly in the first year of treatment with pegunigalsidase alfa. Neutralizing antibodies were detected in <25% of patients with de novo ADAs versus in almost all patients who were ADA+ at baseline. In separate poster presentations, Derralynn Hughes, M.D., Professor of Experimental Haematology at the University College London, and Ankit Mehta, M.D., FASN, Baylor University Medical Center, are sharing results from analyses of the tolerability of pegunigalsidase alfa across the clinical program per dosing regimen and prior enzyme replacement therapy (ERT) experience, respectively. Dr. Hughes found that less than a third of patients receiving pegunigalsidase alfa experienced infusion related reactions (IRRs), most of which occurred from the start of the infusion until up to two hours after its end among patients with pre-existing or de novo ADAs. The frequency of IRRs decreased over time and in parallel with a decrease in premedication use among those switching from prior ERT. Dr. Mehta found that IRRs from the start of the infusion until up to two hours after its end occurred in about a quarter of either ERT-naive or ERT-switch patients following treatment with pegunigalsidase alfa. Most of these IRRs were mild to moderate in severity and occurred predominantly in the first year after initiating the therapy. Surveys and epidemiological analyses shed new light on the prevalence, diagnosis, and progression of AM and Fabry disease Chiesi Global Rare Diseases is announcing five additional presentations focused on improving awareness and understanding of the patient experience with rare lysosomal storage disorders including AM and Fabry disease. In an oral presentation, Stuart Gaffney, M.D., Medical Manager, Chiesi Global Rare Diseases, is sharing results from interviews and surveys with Fabry disease patients (n=20), nurses, and clinicians to understand the diagnostic journey and identify unmet medical education needs of UK primary and specialty healthcare professionals. The most common first symptom of Fabry disease was neuropathic pain, and the first severe indication for many patients was chest pain and general heart complaints. Cardiologists were the main specialists identifying Fabry disease, and 55% (11/20) of patients saw a cardiologist who was the first to suspect Fabry disease was the cause of their symptoms. In a poster presentation, Patricia Dorling, Head of Global Health Economics and Outcome Research (GHEOR), Chiesi USA, is reporting results from a study that analyzed data from the Adelphi Fabry Disease Specific Programme™ (DSP), a national representative online survey of physicians treating patients with a confirmed diagnosis of Fabry disease in the United States. Results demonstrate that the burden of complications and use of medical services (HCRU) is substantial with 76% of patients undergoing routine clinical examinations over two years and 19% of patients being hospitalized due to Fabry disease over one year. In a poster presentation, Khashayar Azimpour, M.D., Ph.D., Director, Global Health Economics and Outcome Research (GHEOR), Chiesi USA, is reporting results from a study aimed to estimate diagnosed Fabry disease prevalence and treatment rates in the U.S. via a retrospective cohort analysis. The 2021 diagnosed prevalence was 5,462 people, and 3,747 of those people with Fabry disease had received treatment. The findings highlight potentially higher gender disparities in treatment than previous estimates, signifying greater need for treatment in females. In a poster presentation, Nicole Muschol, M.D., International Center for Lysosomal Disorders (ICLD) at the University Medical Center Hamburg-Eppendorf in Germany, is reporting results from a Delphi study that generated 60 internationally applicable best practice recommendations for the initial assessment of newly diagnosed AM patients and their routine follow-up and integrated care coordination. In a poster presentation, Prof. Guffon is highlighting results from an international caregiver and patient survey designed to assess the impact of AM on patients' functional ability and quality of life. Results confirm that an important treatment goal is stabilizing and slowing disease progression. Untreated patients reported deterioration at a functional, pain and quality of life level, while patients treated with ERT or allogeneic haematopoietic stem cell transplantation reported less decline in walking ability and improved ability to self-care. Chiesi Global Rare Diseases is sponsoring three satellite symposia during WORLDSymposium to review perspectives on the management of Fabry disease and AM including insights from clinicians, caregivers, and patients. The company is also presenting a special screening of "Rare Land," a film focused on AM, with an expert panel featuring Robert Hopkin, M.D., Clinical Geneticist at Cincinnati Children's Hospital Medical Center and Martin Magner, M.D., Ph.D., Department of Paediatrics and Hereditary Metabolic Disorders of the General University Hospital of Prague. In addition, the company is presenting during the Industry Expert Theater session and is participating in the Patient Voice and Preference meeting. Indication and Important Safety Information for Elfabrio® (pegunigalsidase alfa-iwxj) Indication Elfabrio® (pegunigalsidase alfa-iwxj) is indicated for the treatment of adults with confirmed Fabry disease. Important Safety Information Prior to Elfabrio administration, consider pretreating with antihistamines, antipyretics, and/or corticosteroids. Inform patients and caregivers of the signs and symptoms of hypersensitivity reactions and infusion-associated reactions (IARs), and instruct them to seek medical care immediately if such symptoms occur. If a severe hypersensitivity reaction (including anaphylaxis) or severe IAR occurs, immediately discontinue Elfabrio administration and initiate appropriate medical treatment. If a mild to moderate hypersensitivity reaction or IAR occurs, consider slowing the infusion rate or temporarily withholding the dose. In clinical trials, 20 (14%) Elfabrio-treated patients experienced hypersensitivity reactions. Four Elfabrio-treated patients (3%) experienced anaphylaxis reactions that occurred within 5 to 40 minutes of the start of the initial infusion. The signs and symptoms of hypersensitivity reactions and anaphylaxis included headache, nausea, vomiting, throat tightness, facial and oral edema, truncal rash, tachycardia, hypotension, rigors, urticaria, intense pruritus, moderate upper airway obstructions, macroglossia, and mild lip edema. In clinical trials, 41 (29%) Elfabrio-treated patients experienced one or more infusion-associated reactions, including hypersensitivity, nausea, chills, pruritus, rash, chest pain, dizziness, vomiting, asthenia, pain, sneezing, dyspnea, nasal congestion, throat irritation, abdominal pain, erythema, diarrhea, burning sensation, neuralgia, headache, paresthesia, tremor, agitation, increased body temperature, flushing, bradycardia, myalgia, hypertension, and hypotension. A case of membranoproliferative glomerulonephritis with immune depositions in the kidney was reported during clinical trials. Monitor serum creatinine and urinary protein-to-creatinine ratio. If glomerulonephritis is suspected, discontinue treatment until a diagnostic evaluation can be conducted. When switching to Elfabrio from a prior enzyme replacement therapy, the risk of hypersensitivity reactions and infusion-associated reactions may be increased in certain patients with pre-existing anti-drug antibodies (ADAs). Consider monitoring IgG and IgE ADAs and clinical or pharmacodynamic response (eg, plasma lyso-Gb3 levels). The most common adverse reactions (≥15%) were infusion-associated reactions, nasopharyngitis, headache, diarrhea, fatigue, nausea, back pain, pain in extremity, and sinusitis. Please see Full Prescribing Information for Elfabrio. Indication and Important Safety Information for Lamzede® (velmanase alfa-tycv) Indication Lamzede® (velmanase alfa-tycv) is indicated for the treatment of non-central nervous system manifestations of alpha-mannosidosis in adult and pediatric patients. Important Safety Information Considerations Due to Hypersensitivity Reactions and/or Infusion-Associated Reactions (IARs) Prior to Lamzede administration, consider pretreating with antihistamines, antipyretics, and/or corticosteroids. Inform patients and caregivers of the signs and symptoms of hypersensitivity reactions and IARs and instruct them to seek medical care immediately if such symptoms occur. If a severe hypersensitivity reaction (including anaphylaxis) or severe IAR occurs, immediately discontinue Lamzede administration and initiate appropriate medical treatment. In the event of a mild to moderate hypersensitivity reaction or a mild to moderate IAR, consider temporarily holding the infusion for 15 to 30 minutes, slowing the infusion rate to 25% to 50% of the recommended rate, and initiating appropriate medical treatment. Hypersensitivity Reactions Including Anaphylaxis Anaphylaxis and severe hypersensitivity signs and symptoms included cyanosis, hypotension, emesis, urticaria, erythema, facial swelling, pyrexia, and tremor. Infusion-Associated Reactions (IARs) The most frequent symptoms of IARs that occurred in >10% of the population were pyrexia, chills, erythema, vomiting, cough, urticaria, rash, and conjunctivitis. Females of Reproductive Potential Advise females of reproductive potential to use effective contraception during treatment and for 14 days after the last dose if Lamzede is discontinued. For females of reproductive potential, verify that the patient is not pregnant prior to initiating treatment with Lamzede. Embryo-Fetal Toxicity Based on findings from animal reproduction studies, Lamzede may cause embryo-fetal harm when administered to a pregnant female. Common Adverse Reactions The most common adverse reactions (incidence >20%) are hypersensitivity reactions including anaphylaxis, nasopharyngitis, pyrexia, headache, and arthralgia. Please see Full Prescribing Information for Lamzede. About Chiesi Global Rare Diseases Chiesi Global Rare Diseases is a business unit of the Chiesi Group established to deliver innovative therapies and solutions for people affected by rare diseases. As a family business, Chiesi Group strives to create a world where it is common to have a therapy for all diseases and acts as a force for good, for society and the planet. The goal of the Global Rare Diseases unit is to ensure equal access so as many people as possible can experience their most fulfilling life. The unit collaborates with the rare disease community around the globe to bring voice to underserved people in the health care system. For more information visit . About Chiesi Group Chiesi is an international, research-focused biopharmaceuticals group that develops and markets innovative therapeutic solutions in respiratory health, rare diseases, and specialty care. The company's mission is to improve people's quality of life and act responsibly towards both the community and the environment. By changing its legal status to a Benefit Corporation in Italy, the US, and France, Chiesi's commitment to create shared value for society as a whole is legally binding and central to company-wide decision-making. As a certified B Corp since 2019, we're part of a global community of businesses that meet high standards of social and environmental impact. The company aims to reach Net-Zero greenhouse gases (GHG) emissions by 2035. With over 85 years of experience, Chiesi is headquartered in Parma (Italy), operates in 31 countries, and counts more than 6,500 employees. The Group's research and development centre in Parma works alongside 6 other important R&D hubs in France, the US, Canada, China, the UK, and Sweden. For further information please visit . Chiesi Group Media Contacts Chiara Travagin Rare Communication Manager Tel: +39 348 8818985 Email: [email protected] Alessio Pappagallo Press Office Manager Tel: +39 339 5897483 Email: [email protected] Adam Daley Berry & Company Public Relations Tel: +1 212 253 8881 Email: [email protected] PP-MULTI-0288 V2.0 SOURCE Chiesi Global Rare Diseases

KRRO-110 has a potentially best-in-class profile with secretion of ~50µM functional AAT protein after a single dose in a preclinical mouse modelEvidence of greater than 40% editing of SERPINA1 RNA in non-human primates (NHPs) further supports the potential translation of KRRO-110 in humansDemonstrated greater than 50% editing using GalNAc conjugates in the liver in a preclinical mouse modelGenerated a de novo protein variant in NHPs, further highlighting the potential power of our RNA editing approach CAMBRIDGE, Mass., Jan. 18, 2024 (GLOBE NEWSWIRE) -- Korro Bio, Inc. (Korro) (Nasdaq: KRRO), a biopharmaceutical company focused on developing a new class of genetic medicines for both rare and highly prevalent diseases, presented new data for KRRO-110 and reported progress across its RNA editing portfolio at the J.P. Morgan Healthcare Conference on January 9, 2024. “KRRO-110 data showed the highest levels of corrected protein in a human transgenic PiZ mouse model, supporting a potentially best-in-class therapeutic for AATD patients,” said Chief Executive Officer and President, Ram Aiyar, Ph.D. “Beyond AATD, our ability to generate de novo mutations to activate a biological pathway is uncharted in the field of RNA editing. The progress we have made across our platform and pipeline underscores our commitment to bringing differentiated therapeutic options to patients with significant unmet medical needs.” Pipeline updates KRRO-110 As announced on December 7, 2023, Korro selected KRRO-110 as its first development candidate for the potential treatment of AATD, an inherited genetic disorder caused by single nucleotide variants (SNVs) in the SERPINA1 gene. AATD can lead to severe progressive lung disease, including emphysema and chronic obstructive pulmonary disease (COPD), and severe liver disease leading to inflammation, cirrhosis, and fibrosis. Preclinical data to date for KRRO-110 demonstrated: High specificity with no bystander effects in MZ human primary hepatocytesIntravenous administration at 2 mg/kg resulted in secretion of ~50µM functional AAT as early as 7 days post-single dose in a human transgenic PiZ mouse modelIncrease in AAT protein and the inhibition of elastase activity were sustained through week 9 when dosed every 2 weeks, demonstrating durability in miceGreater than 40% editing in NHPs utilizing an earlier generation oligonucleotide designed to edit a surrogate SERPINA1 RNA target site Preclinical development of KRRO-110 is ongoing in preparation for a regulatory filing expected in the second half of 2024, with a potential interim clinical readout in the second half of 2025. Platform Update Korro’s proprietary RNA editing platform, OPERA™, integrates a deep understanding of adenosine deaminase acting on RNA (ADAR) enzymology with expertise in oligonucleotide chemistry, machine learning optimization of oligonucleotides and fit-for-purpose delivery. CHORDs™, or Customized High-fidelity Oligonucleotides for RNA Deamination, are single-stranded, anti-sense oligonucleotides designed to have high target efficiency and specificity by leveraging the pillars of OPERA. Developed proprietary oligonucleotide chemistry using structural biology insights that enhances in vivo potency and durability of CHORDsComputational efficiency enables rapid iteration of CHORDs across targetsUsing CHORDs, Korro achieved greater than 50% editing in vivo utilizing both a ligand-based GalNAc conjugate and an LNP-based delivery approach Creating De Novo Mutation to Alter Protein Function In addition to repairing pathogenic SNVs, CHORDs can be used to engineer de novo SNVs and change amino acids on proteins to endow them with desired altered properties while still preserving their broader functional capabilities. In preclinical studies, Korro has demonstrated the ability to: Activate a transcription factor in an undisclosed target Demonstrated activation of a transcription factor by creating a de novo protein variant resulting in sustained downstream activity in NHPs lasting longer than 21 days, demonstrating potential to preserve transcription factor function Selectively modulate TDP-43 to reduce protein aggregation TDP-43 is a protein associated with ALS, FTD and other neurodegenerative diseases. A single RNA edit to TDP-43 is predicted to lead to the synthesis of a protein variant that does not aggregate, thereby preserving normal function and protecting downstream activity essential for neuronal health Modulate ion channels to within physiological levels in Nav1.7 Nav1.7 is a voltage-gated sodium channel that is essential for pain sensation and electrical signaling in the central nervous system. Korro has demonstrated that site-specific changes are sufficient to decrease the activity of Nav1.7. This approach has the potential to deliver potent analgesic activity without the dose-limiting toxicities that have been observed by sodium channel blockers J.P. Morgan Healthcare Conference Presentation A replay of Korro’s presentation at the J.P. Morgan Healthcare Conference can be accessed from the Investor section of Korro’s website at www.korrobio.com or directly with this link. The replay is available for 30 days following the January 9, 2024 conclusion of the presentation. About Korro Korro is a biopharmaceutical company focused on developing a new class of genetic medicines for both rare and highly prevalent diseases using its proprietary RNA editing platform. Korro is 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, Korro is expanding the reach of genetic medicines by delivering additional precision and tunability, which has the potential for increased specificity and improved long-term tolerability. Using an oligonucleotide-based approach, Korro expects to bring its medicines to patients by leveraging its proprietary platform with precedented delivery modalities, manufacturing know-how, and established regulatory pathways of approved oligonucleotide drugs. Korro is based in Cambridge, Mass. For more information, visit korrobio.com. Forward-Looking Statements Certain statements in this press release may constitute “forward-looking statements”. Forward-looking statements include, but are not limited to, express or implied statements regarding expectations, hopes, beliefs, intentions or strategies of Korro regarding the future including, without limitation, express or implied statements regarding: Korro’s RNA editing technology and the benefits of OPERA; the market opportunity for KRRO-110 and potential benefits over other AATD modalities; the potential of KRRO-110 to be a best-in-class drug candidate for AATD; the potential safety and efficacy of KRRO-110; Korro’s expected cash runway and plans for discovery and preclinical studies, as well as clinical trials, including timing of regulatory filings and data readouts and other developments or results in connection therewith. In addition, any statements that refer to projections, forecasts, or other characterizations of future events or circumstances, including any underlying assumptions, are forward-looking statements. The words “anticipate,” “believe,” continue,” “ could,” “estimate,” “ expect,” “ intend,” “may,” “might,” “plan,” “possible,” “potential,” “predict,” “project,” “ should,” “ strive,” “would,” “aim,” “target,” “commit,” and similar expressions may identify forward-looking statements, but the absence of these words does not mean that statement is not forward looking. Forward-looking statements are based on current expectations and assumptions that, while considered reasonable are inherently uncertain. New risks and uncertainties may emerge from time to time, and it is not possible to predict all risks and uncertainties. Factors that may cause actual results to differ materially from current expectations include, but are not limited to, various factors beyond management’s control including risks inherent in biopharmaceutical development; risks associated with pre-clinical studies and clinical trials; and other risks associated with obtaining regulatory approvals and protecting intellectual property; as well as risks associated with general economic conditions; the inability to recognize the anticipated benefits of the recently completed merger, which may be affected by, among other things, competition, Korro’s ability to grow and manage growth profitably, maintain relationships with customers and suppliers and retain key employees; costs related to merger; the possibility that Korro may be adversely affected by other economic, business, and/or competitive factors; other risks and uncertainties indicated from time to time in Korro’s filings with the SEC, including in Exhibit 99.2 to its Current Report on Form 8-K filed with the SEC on November 6, 2023, as such may be amended or supplemented by its other filings with the SEC. Nothing in this press release should be regarded as a representation by any person that the forward-looking statements set forth herein will be achieved or that any of the contemplated results of such forward-looking statements will be achieved. You should not place undue reliance on forward-looking statements in this press release, which speak only as of the date they are made and are qualified in their entirety by reference to the cautionary statements herein. Korro does not undertake or accept any duty to release publicly any updates or revisions to any forward-looking statements to reflect any change in their expectations or in the events, conditions or circumstances on which any such statement is based. This press release does not purport to summarize all of the conditions, risks and other attributes of an investment in Korro. Korro Contact Information Investors IR@korrobio.com Media Glenn Silver FINN Partners Glenn.silver@finnpartners.com

WVE-006 is uniquely designed to correct the disease-causing RNA mutation in AATD, thereby restoring circulation of wild-type M-AAT protein and reducing Z-AAT protein levels to address both lung and liver manifestations of the disease Wave earned $20 million USD milestone from GSK for initiation of dosing; eligible for additional development, launch, and sales-related milestones of up to $505 million for WVE-006 Proof-of-mechanism data in individuals with AATD, as measured by restoration of M-AAT protein, expected in 2024 As shared at recent R&D Day, Wave is also advancing a pipeline of wholly owned RNA editing therapeutics beyond WVE-006 and across a range of high-impact GalNAc-hepatic and extra hepatic targets CAMBRIDGE, Mass., Dec. 06, 2023 (GLOBE NEWSWIRE) -- WAVE Life Sciences Ltd. (Nasdaq: WVE), a clinical-stage RNA medicines company committed to delivering life-changing treatments for people battling devastating diseases, today announced the initiation of dosing in healthy volunteers in the RestorAATion clinical trial program, which is investigating WVE-006 as a potential treatment for alpha-1 antitrypsin deficiency (AATD). WVE-006 is a first-in-class, GalNAc-conjugated RNA editing oligonucleotide (AIMer). It is designed to restore circulation of healthy, wild-type alpha-1 antitrypsin (M-AAT) protein and reduce dysfunctional Z-AAT protein, thereby potentially addressing AATD-related lung disease, liver disease, or both. “Initiating dosing in RestorAATion represents an important milestone for the alpha-1 community, where treatment options are limited and there are no medicines that address the underlying genetic mutation that most commonly causes AATD. In preclinical studies, WVE-006 led to potent and durable RNA editing and restoration of AAT protein up to 30 micromolar, underscoring the impact of our novel chemistry. WVE-006 has the potential to transform the treatment paradigm for this disease, and we are well-positioned to achieve this vision as part of our collaboration with GSK,” said Paul Bolno, MD, MBA, President and Chief Executive Officer of Wave Life Sciences. “Moreover, with the first RNA editing therapeutic to ever be dosed in humans, we are making a significant contribution to the scientific field by bringing an entirely new class of medicines into clinical development, and we expect to continue unlocking the potential of RNA editing more broadly.” The RestorAATion clinical program includes healthy volunteers (RestorAATion-1) as well as individuals with AATD who have the homozygous PiZZ mutation (RestorAATion-2) and is designed to provide an efficient path to proof-of-mechanism as measured by restoration of M-AAT protein in serum. Wave expects to deliver proof-of-mechanism data in individuals with AATD in 2024. “This represents an important milestone for GSK’s growing oligonucleotide pipeline,” said Tony Wood, Chief Scientific Officer, GSK. “Throughout this year, we’ve demonstrated our commitment to realizing the potential of oligonucleotide therapeutics in areas of unmet need for patients. Beyond WVE-006, we’re also excited to continue our collaboration with Wave Life Sciences using their best-in-class PRISM™ platform.” With initiation of dosing in RestorAATion, Wave has achieved its first WVE-006 milestone in its collaboration with GSK, resulting in a $20 million payment to Wave. For WVE-006, Wave is eligible to receive up to $505 million in additional development, launch, and sales-related milestone payments, as well as tiered royalties on net sales, from GSK. Development and commercialization responsibilities will transfer to GSK after Wave completes the RestorAATion-2 study. Beyond WVE-006, Wave is advancing a pipeline of wholly owned RNA editing therapeutics designed to either correct or upregulate mRNA across a range of high impact targets. The company’s discovery and development efforts in RNA editing are powered by its proprietary “edit-verse”, which leverages genetic datasets and deep learning models to identify new RNA editing targets and edit sites. These targets leverage easily accessible biomarkers, offer efficient paths to proof-of-concept in humans, and represent meaningful commercial opportunities. At its R&D Day in September 2023, Wave shared in vivo and in vitro proof-of-concept data on several undisclosed targets, achieving at least 2-fold mRNA upregulation in liver and kidney targets and more than 60% mRNA correction in liver and lung targets. About Alpha-1 Antitrypsin Deficiency Alpha-1 antitrypsin deficiency (AATD) is an inherited genetic disorder that is commonly caused by a G-to-A point mutation (Z allele) in the SERPINA1 gene. This mutation leads to lung disease due to insufficient levels of circulating M-AAT protein, which protects the lungs from proteolytic enzymes, and it leads to liver disease due to aggregation of misfolded Z-AAT protein in hepatocytes. There are approximately 200,000 patients in the United States and Europe who have Z mutations on both alleles, known as the PiZZ genotype. Augmentation therapy via delivery of AAT protein is currently the only treatment option for AATD lung disease and requires weekly intravenous infusions. There are no treatments for AATD liver disease, other than liver transplantation. About WVE-006 WVE-006 is a first-in-class, GalNAc-conjugated and subcutaneously administered RNA editing oligonucleotide designed to correct the single base mutation in messenger RNA (mRNA) coded by the SERPINA1 Z allele, thereby enabling restoration and circulation of functional M-AAT protein. In preclinical studies, WVE-006 demonstrated potent and durable editing of SERPINA1 Z transcript in mice, restoration of AAT protein up to 30 micromolar, and improvement in several markers of liver disease. WVE-006 is also highly specific with no evidence of bystander editing. Together, these data demonstrate the potential of WVE-006 to address AATD-related liver disease, lung disease, or both. About AIMers Wave’s A-to-I RNA editing oligonucleotides (AIMers) are designed to target single bases on an RNA transcript and recruit proteins that exist in the body, called ADAR (adenosine deaminases acting on RNA) enzymes, which naturally possess the ability to change an adenine (A) to an inosine (I), which cells read as guanine (G). This approach enables both the correction of G-to-A point mutations, as well as the modulation of RNA to upregulate protein expression, modify protein-protein interactions, or alter RNA folding and processing. AIMers enable simplified delivery and avoid the risk of permanent changes to the genome and irreversible off-target effects with DNA-targeting approaches. AIMers are short in length, fully chemically modified, and use novel chemistry, including proprietary PN backbone modifications and chiral control, which make them distinct from other ADAR-mediated editing approaches. About Wave Life Sciences Wave Life Sciences (Nasdaq: WVE) is a clinical-stage RNA medicines company committed to delivering life-changing treatments for people battling devastating diseases. Wave aspires to develop best-in-class medicines across multiple therapeutic modalities using PRISM, the company’s proprietary discovery and drug development platform that enables the precise design, optimization, and production of stereopure oligonucleotides. Driven by a resolute sense of urgency, the Wave team is targeting a broad range of genetically defined diseases so that patients and families may realize a brighter future. To find out more, please visit and follow Wave on X (formerly Twitter) @WaveLifeSci. Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, our expectations for our GalNAc-conjugated RNA editing oligonucleotides (AIMers), including WVE-006, and the anticipated therapeutic benefits thereof, including the potential of WVE-006 to treat AATD; the future performance and results of our clinical programs; our expectations and anticipated timing for delivering proof-of-mechanism clinical data in AATD patients treated with WVE-006; our understanding that WVE-006 is the most advanced candidate for AATD designed to restore functional wild-type AAT protein and reduce Z-AAT protein aggregation; our expectations regarding the ability of our AIMers, and RNA editing broadly, to address diseases of many different tissues and cell types; the potential benefits of our AIMers compared with other RNA base editing approaches; the potential benefits that our “edit-verse” map may offer to identify new RNA editing targets; and the potential achievement of development, launch and sales-related milestones for WVE-006 under our GSK collaboration and receipt of cash payments therefor. The words “may,” “will,” “could,” “would,” “should,” “expect,” “plan,” “anticipate,” “intend,” “believe,” “estimate,” “predict,” “project,” “potential,” “continue,” “target” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release and actual results may differ materially from those indicated by these forward-looking statements as a result of these risks, uncertainties and important factors, including, without limitation, the risks and uncertainties described in the section entitled “Risk Factors” in Wave’s most recent Annual Report on Form 10-K filed with the Securities and Exchange Commission (SEC), as amended, and in other filings Wave makes with the SEC from time to time. Wave undertakes no obligation to update the information contained in this press release to reflect subsequently occurring events or circumstance. Investor Contact: Kate Rausch +1 617-949-4827 krausch@wavelifesci.com Media Contact: Alicia Suter +1 617-949-4817 asuter@wavelifesci.com AATD Community Contact: Chelley Casey +1 617-949-2900 ccasey@wavelifesci.com