Glycogen Storage Disease Type III

Genethon is a leader in developing gene therapies for rare and ultra-rare diseases PARIS, FRANCE (May 10, 2023) – Genethon, a unique non-profit gene therapy R&D, announced today its research will be featured in 4 oral presentations and 10 posters at the 26th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT), May 16-20, 2023, at the Los Angeles Convention Center in Los Angeles, CA. The international convention attracts thousands of scientists, physicians, patient advocates and government officials. “We are proud to report some of our most recent progress, covering the advancement of novel gene therapy candidates for multiple rare genetic diseases, and the design of novel vectors and tools,” said Frederic Revah, Genethon CEO. “The objective of Genethon’ scientists is to pursue research of excellence allowing more and more patients with rare genetic diseases to benefit from the treatments they need.” Genethon’s Ana Buj Bello, M.D., Ph.D., MBA, leader of the Neuromuscular Diseases and Gene Therapy Team, is co-chair of the oral abstract session on AAV Vectors – Clinical/Nonhuman primate studies, Wednesday, May 17, 3:45 pm – 5:30 pm US PDT (Rm 411). Dr. Buj Bello also is presenting a poster titled, AAV-mediated gene therapy corrects the severe phenotype of acid ceramidase deficient mice (Board No. 15918), Friday, May 19, 12 pm – 2 pm US PDT. Genethon has four scientists participating in the following oral poster presentation sessions (all times are US PDT): Wednesday, May 17 AAV Vectors – Preclinical and Proof-of-Concept In Vivo Studies I (3:45 pm – 5:30 pm) Rm 408 AB More Than Nine Year Survival of a GRMD Dog after Injection of AAV-Microdystrophin Gene Therapy (4:15 pm – 4:30 pm) – Caroline Le Guiner Thursday, May 18 Metabolic, Storage, Endocrine, Liver and Gastrointestinal Diseases I (1:30 pm to 3:15 pm) Rm 403 AB Single AAV vector gene therapy with a mini-GDE transgene corrects muscle impairment in mouse, rat and human cellular models of glycogen storage disease Type III (3 pm – 3:15 pm) – Antoine Gardin Novel Genetic Approaches for Muscle Diseases (3:35 pm – 5:30 pm) Rm 408 AB Development of MYOrganoids for therapeutic evaluation of gene therapy product in Duchenne Muscular Dystrophy (4:30 pm – 4:45 pm) – Laura Palmieri Saturday, May 20 AAV Development for Eye, Muscle, Kidney and CNS (8 am – 9:45 am) Concourse Hall 150 & 151) An integrin-targeting AAV developed by a novel computational rational design methodology presents an improved targeting to the skeletal muscle and reduced tropism to the liver (9:25 am to 9:42 am) – Ai Vu Hong In addition, Dr. Hong, a member of Genethon’s Progressive Muscular Dystrophies Team, received two ASGCT awards: one for Excellence in Research, which recognizes the top 10 – 15 posters submitted by students and fellows; and the other a Meritorious Abstract Travel Award given to associate members based on their high abstract scores. The following are poster presentations by Genethon scientists (all times are US PDT): Wednesday, May 17, 12 pm – 2 pm Extreme Inter-Individual Variability of Subretinal AAV-Induced Immune Response Even in a Highly Standardized Context [Board No. 721] – Duohao Ren and Sylvain Fisson Assessment of Therapeutical Potential and Advantage of a Dual AAV Approach for Duchenne Muscular Dystrophy [Board No. 663] – Sonia Albini In Vivo Gene Therapy for Lysosomal Acid Lipase Deficiency [Board No. 643] - Marine Laurent Characterization and Quantification of CRISPR/Cas9-Induced Genomic Alterations and AAV Targeted Integration in Human HSPC [Board No. 527] Alexandra Tachtsidi Thursday, May 18, 12 pm – 2 pm Evaluation of Gene Transfer Efficiency in a Mild Model of Dystrophic Muscle Disorder Performed by Machine Learning and Linear Discriminant Analysis [Board No. 1214] – Anthony Brureau Cardiomyocyte Death after AAV Gene Transfer Can Be Related to Proteotoxicity or to Transgene Abnormal Function or Localization [Board No. 884] – Isabelle Richard Predictive Potential of Single Deleterious Variations to Infer on AAV2 and AAV2-13 Capsids Viability [Board No. 874] – Tiziana La Bella Friday, May 19, 12 pm – 2 pm Autophagy Induction Improves Dual AAV Vector Efficacy in a Mouse Model of Glycogen Storage Disease Type III [Board No. 1396] – Louisa Jauze Preliminary Results from a Prospective, Multicentric, Follow Up Standardized Cohort to Assess Natural History of Duchenne Muscular Dystrophy [Board No. 1603] – Fei Cao About Genethon A pioneer in the discovery and development of gene therapies for rare diseases, Genethon is a unique non-profit organization created by a patient association, the AFM-Telethon. A first gene therapy drug, to which Genethon contributed, has obtained marketing for spinal muscular atrophy. With 200+ scientists and professionals, Genethon is pursuing its mission to bring life-changing therapies to patients suffering from rare genetic diseases. 13 products resulting from Genethon’s research are in clinical trials for eye, liver, blood, immune system and muscle diseases. A further6 products are in the preparation phase for clinical trials over the next five years. Find out more: genethon.com Contact: Charles Craig Opus Biotech Communications 404-245-0591 charles.s.craig@gmail.com

Arcturus to present at the Guggenheim Healthcare Talks: Genomic Medicines & Rare Disease Day, April 3, 2023 SAN DIEGO--(BUSINESS WIRE)-- Arcturus Therapeutics, Inc. (the “Company,” “Arcturus,” Nasdaq: ARCT), a global late-stage clinical messenger RNA medicines company focused on the development of infectious disease vaccines and significant opportunities within liver and respiratory rare diseases, today announced that it will release its financial results for the quarter and year ended December 31, 2022 after the market close on Tuesday, March 28 and will also host a conference call and webcast at 4:30 p.m. Eastern Time on March 28, 2023. The Company also announced it will present at the Guggenheim Healthcare Talks: Genomic Medicines & Rare Disease Day on April 3, 2023, at 2:10 p.m. ET. Arcturus Therapeutics Fourth Quarter and Full Year 2022 Earnings Conference Call Tuesday, March 28, 2023 @ 4:30 p.m. ET Domestic: 1-877-407-0784 International: 1-201-689-8560 Conference ID: 13735876 Webcast: Link Guggenheim Healthcare Talks: Genomic Medicines & Rare Disease Day (Fireside Chat) Monday, April 3, 2023 (2:10 p.m. ET) A webcast and replay of the presentation will be available on the “Investor Relations/Events” section of the Company’s website at: About Arcturus Therapeutics Founded in 2013 and based in San Diego, California, Arcturus Therapeutics Holdings Inc. (Nasdaq: ARCT) is a global late-stage clinical mRNA medicines and vaccines company with enabling technologies: (i) LUNAR® lipid-mediated delivery, (ii) STARR™ mRNA Technology (samRNA) and (iii) mRNA drug substance along with drug product manufacturing expertise. Arcturus’ diverse pipeline of RNA therapeutic and vaccine candidates includes mRNA vaccine programs for SARS-CoV-2 (COVID-19) and Influenza, and other programs to potentially treat ornithine transcarbamylase (OTC) deficiency, and cystic fibrosis, along with partnered programs including glycogen storage disease type III. Arcturus’ versatile RNA therapeutics platforms can be applied toward multiple types of nucleic acid medicines including messenger RNA, small interfering RNA, circular RNA, antisense RNA, self-amplifying RNA, DNA, and gene editing therapeutics. Arcturus’ technologies are covered by its extensive patent portfolio (patents and patent applications issued in the U.S., Europe, Japan, China, and other countries). For more information, visit . In addition, please connect with us on Twitter and LinkedIn.

SAN DIEGO--(BUSINESS WIRE)-- Arcturus Therapeutics Holdings Inc. (the “Company”, “Arcturus”, Nasdaq: ARCT), a global, late-stage clinical messenger RNA medicines company focused on the development of infectious disease vaccines and significant opportunities within liver and respiratory rare diseases, today announced that the Compensation Committee of the Company’s Board of Directors and the Company’s Board of Directors approved inducement grants of stock options to purchase an aggregate of 48,200 shares of Arcturus common stock to seven newly-hired employees. The equity awards were granted pursuant to the Nasdaq Rule 5635(c)(4) inducement grant exception as a component of each individual’s employment compensation and were granted as an inducement material to his or her acceptance of employment with the Company. The options have an exercise price equal to $12.26 per share, the closing price of the Company’s common stock as reported by Nasdaq on June 16, 2022. The options have a ten-year term and vest over four years, with 25% of the number of shares underlying the stock option vesting on one-year anniversary of the applicable vesting commencement date (based on the quarter in which the applicable employee’s employment commenced) and the remaining shares vesting monthly over 36 months thereafter. The equity awards are subject to each individual’s continued service with the Company through the applicable vesting dates. About Arcturus Therapeutics Founded in 2013 and based in San Diego, California, Arcturus Therapeutics Holdings Inc. (Nasdaq: ARCT) is a clinical-stage mRNA medicines and vaccines company with enabling technologies: (i) LUNAR® lipid-mediated delivery, (ii) STARR™ mRNA Technology and (iii) mRNA drug substance along with drug product manufacturing expertise. Arcturus’ diverse pipeline of RNA therapeutic and vaccine candidates includes mRNA vaccine programs for SARS-CoV-2 (COVID-19) and Influenza, and other programs to potentially treat ornithine transcarbamylase (OTC) deficiency, and cystic fibrosis, along with partnered programs including glycogen storage disease type III, hepatitis B virus, and non-alcoholic steatohepatitis (NASH). Arcturus’ versatile RNA therapeutics platforms can be applied toward multiple types of nucleic acid medicines including messenger RNA, small interfering RNA, replicon RNA, antisense RNA, microRNA, DNA, and gene editing therapeutics. Arcturus’ technologies are covered by its extensive patent portfolio (patents and patent applications issued in the U.S., Europe, Japan, China and other countries). Arcturus’ commitment to the development of novel RNA therapeutics has led to collaborations with Janssen Pharmaceuticals, Inc., part of the Janssen Pharmaceutical Companies of Johnson & Johnson, Ultragenyx Pharmaceutical, Inc., Takeda Pharmaceutical Company Limited, CureVac AG, Duke-NUS Medical School, and the Cystic Fibrosis Foundation. For more information visit . In addition, please connect with us on Twitter and LinkedIn. Forward Looking Statements This press release contains forward-looking statements that involve substantial risks and uncertainties for purposes of the safe harbor provided by the Private Securities Litigation Reform Act of 1995. Any statements, other than statements of historical fact included in this press release, are forward-looking statements, including those regarding strategy, future operations, collaborations, the likelihood of success (including safety and efficacy) of the Company’s pipeline, the likelihood that a patent will issue from any patent application and the impact of general business and economic conditions. Arcturus may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in any forward-looking statements such as the foregoing and you should not place undue reliance on such forward-looking statements. Such statements are based on management’s current expectations and involve risks and uncertainties, including those discussed under the heading "Risk Factors" in Arcturus’ most recent Annual Report on Form 10-K, and in subsequent filings with, or submissions to, the SEC, which are available on the SEC’s website at . Except as otherwise required by law, Arcturus disclaims any intention or obligation to update or revise any forward-looking statements, which speak only as of the date they were made, whether as a result of new information, future events or circumstances or otherwise. View source version on businesswire.com: Contacts Kyle Gutstadt Senior Analyst, Investor and Public Relations (858) 732-0394 Arcturus Therapeutics IR@ArcturusRx.com Source: Arcturus Therapeutics Holdings Inc. View this news release online at: