Vocal Cord Paralysis

Fireside Chat, moderated by Hartaj Singh of Oppenheimer & Co., will be held on Wednesday, January 24, 2024, at 12:00pm ET PLYMOUTH MEETING, Pa., Jan. 22, 2024 /PRNewswire/ -- INOVIO (NASDAQ: INO), a biotechnology company focused on developing and commercializing DNA medicines to help treat and protect people from HPV-associated diseases, cancer and infectious diseases, today announced that it will participate in a virtual fireside chat on the potential treatment of Recurrent Respiratory Papillomatosis (RRP). INOVIO's President and CEO, Dr. Jacqueline Shea, and Chief Medical Officer, Dr. Michael Sumner, will be joined by Dr. Simon Best, Otolaryngologist at Johns Hopkins University and a Principal Investigator in INOVIO's Phase 1/2 trial with INO-3107. The fireside chat, titled "RRP - Disease Treatment Dynamics & Urgent Need for Therapeutic Modalities," will take place on Wednesday, January 24, 2024, at 12:00pm ET. Hartaj Singh, MBA, Managing Director of Biotechnology Equity Research at Oppenheimer & Co. Inc., will moderate the discussion. A replay of the event will be made available shortly after its conclusion and will be archived for 90 days under the Events and Presentations page on the investor relations section of the INOVIO website following the event. Panel Participants Simon Best, MD, is an Associate Professor of Otolaryngology – Head & Neck Surgery at Johns Hopkins Hospital. He is a fellowship-trained laryngeal surgeon whose clinical interests are focused on voice and airway disorders, with a particular interest in RRP. He had led an NIH-funded laboratory focused on RRP, participated in clinical trials, spearheaded novel treatment paradigms, and sees patients from across the world for this disease. In addition, he treats the full range of voice disorders, including in professional voice users and singers, vocal cord paralysis, laryngeal cancer, precancerous vocal cord dysplasia, benign laryngeal lesions (polyps, cysts, nodules), and laryngeal and tracheal stenosis. Dr. Jacqueline Shea has served as INOVIO's President and Chief Executive Officer (CEO) since May 2022. Dr. Shea joined the company in March 2019 as Chief Operating Officer (COO), serving as a key member of the executive team. Dr. Shea has more than 25 years' experience in the life sciences and biotech industries. Prior to joining INOVIO, she was the CEO at Aeras, a not-for-profit organization dedicated to developing new vaccines against tuberculosis (TB). She also held executive roles at Emergent BioSolutions and was the General Manager and Vice President of The Oxford-Emergent Tuberculosis Consortium. Dr. Shea has dedicated much of her career to developing innovative products to prevent and treat infectious diseases. Dr. Shea received a BSc Hons in Applied Biology from the University of Bath and a Ph.D. from the National Institute for Medical Research in the UK. Dr. Michael Sumner leads INOVIO's clinical-stage pipeline of DNA medicines. Dr. Sumner received his medical degree from the University of London, is a member of the Royal College of Physicians, and holds a Master of Business Administration. He brings 30 plus years of extensive pharmaceutical, medical and clinical experience driving many late-stage product approvals and supporting successful commercial products globally across multiple therapeutic areas. He previously served as CMO of Orexo AB since 2013 and prior to that held numerous European- and US-based leadership roles at Novartis, CSL Behring, Novo Nordisk and Shire Pharmaceuticals. About INOVIO INOVIO is a biotechnology company focused on developing and commercializing DNA medicines to help treat and protect people from HPV-related diseases, cancer, and infectious diseases. INOVIO's technology optimizes the design and delivery of innovative DNA medicines that teach the body to manufacture its own disease-fighting tools. For more information, visit . Contacts Media: Jennie Willson, (267) 429-8567, [email protected] Investors: Thomas Hong, (267) 440-4298, thomas[email protected] SOURCE INOVIO Pharmaceuticals, Inc.

Deploying a suite of proprietary tools and deep genetics expertise to develop therapeutics against targets shared between rare and more prevalent diseases Lead small molecule program targeting TRPV4 mutations advancing toward clinic for treatment of Charcot-Marie-Tooth disease type 2C and other serious bone diseases Innovative partnership with The Jackson Laboratory to develop and implement new transgenic models of rare disease pathways Investor syndicate comprised of leading healthcare investors, including Canaan, Deerfield Management, DROIA Ventures, EcoR1 and Euclidean Capital SAN DIEGO--(BUSINESS WIRE)-- Actio Biosciences, a biotechnology company leveraging a novel platform approach to genetics and precision medicine to develop new therapeutics that target shared underlying biology in both rare and common diseases, today announced the completion of a $55 million Series A financing. The financing was led by Canaan and DROIA Ventures, with participation from existing investors Deerfield Management and EcoR1, as well as new investor Euclidean Capital. “Defining a high-impact drug target for a common disease with a heterogenous population has a very low probability of success and has long been a major challenge for drug discovery,” said David Goldstein, Ph.D., co-founder and CEO of Actio. “By starting with a focus on rare diseases, we gain invaluable insights that not only define new therapeutic benefits in high-need indications but also de-risk and guide therapeutic expansion to more common conditions, benefiting even more patients. We are thrilled to have the backing of this esteemed group of investors, along with our world-class team, whose collective support will enable the advancement of our programs to bring life-changing medicines to those that need them most.” Nina Kjellson, board member and general partner at Canaan, commented, “In today’s financing environment, investors can be quite selective where to place our bets. At Canaan, we had a thesis in precision genetics, and Actio stood out for scientific excellence, a seasoned team and very impressive pipeline progress in the short time since inception. We are passionate about investing in teams that have deep, relevant expertise, yet aren’t afraid to tackle the hard problems from new angles and with multidisciplinary approaches.” A Precision Approach to Medicine – From One to Many While it is well known that a single gene can lead to rare disease, the function of mutations in those single genes and how they might be modulated for therapeutic intervention is often not well understood. To overcome this and better utilize the breadth of genetic information now available for drug discovery and development, Actio is leveraging its human genetics platform to identify relevant disease targets, understand their function in disease and create medicines that modulate them. The company’s platform comprises a vast proprietary target database, called the Rare Disease Target Atlas, and unparalleled expertise in bioinformatics, biology and chemistry. Actio will initially focus on identifying targets and advancing programs for rare disease indications and plans to leverage insights throughout the development process to inform potential relevance of those targets in more common diseases. The company has established a partnership with the Rare Disease Translational Center at The Jackson Laboratory (JAX), a leading nonprofit biomedical research institution, dedicated to understanding the molecular underpinning of rare diseases and their treatments. Actio is partnering with JAX to evaluate targets and phenotypes of interest in high-quality genetically engineered mouse models at scale, enabling a high level of confidence in the outcomes observed. Actio is also collaborating with prominent academic centers to provide further validation of the role of certain genes in disease, as well as insights into disease pathology to better inform the design of preclinical studies, rare patient natural history studies and future clinical trials. Lead Program Targeting TRPV4 Advancing Toward the Clinic Actio’s pipeline is led by a program targeting TRPV4, an ion channel target identified as high-value through the company’s platform. Actio plans to initially evaluate it for the treatment of rare diseases caused by TRPV4 mutations, including Charcot-Marie-Tooth disease type 2C (CMT2C), characterized by severe muscle weakness, vocal cord paresis and respiratory complications, as well as serious bone diseases, such as metatropic dysplasia. Evaluation of this program in novel construct-valid preclinical TRPV4 rare disease models has demonstrated marked improvements in motor function, mobility and bone morphology, as well as increased lifespan compared to untreated controls. “Since our founding, we’ve built a proprietary platform designed to de-risk targets of interest and establish biological connections between rare and common disease states that is unlike anything else in the industry today,” said John McHutchison, AO, M.D., co-founder and board member of Actio. “That work has led to the prioritization of several high-value targets, including our lead TRPV4, program for the treatment of CMT2C and other bone diseases with high unmet need. Beyond this program, we are working expeditiously to expand our pipeline and have identified two additional programs that we plan to evaluate for genetic epilepsies and genetic skin conditions.” Distinguished Founders and Leadership Actio was founded by human genetics expert David Goldstein, Ph.D., who serves as the company’s CEO and a board member, and pioneering drug developer John McHutchison, AO, M.D., who also serves as a board member. The full Actio leadership team and board include the following: Leadership Team David Goldstein, Ph.D., co-founder and CEO David Breckenridge, Ph.D., chief scientific officer John Link, Ph.D., senior advisor, drug discovery Sunil Sahdeo, Ph.D., vice president, biology Nick Stock, Ph.D., senior vice president, chemistry Ali Torkamani, Ph.D., vice president, bioinformatics and genetics Anne Sandan, CPA, vice president, human resources and finance operations Board of Directors George Golumbeski, Ph.D., board chairman and partner, DROIA Ventures Jason Fuller, Ph.D., board member and partner, Deerfield Management David Goldstein, Ph.D., board member and CEO, Actio Nina Kjellson, board member and general partner, Canaan John McHutchison, AO, M.D., board member and co-founder, Actio Alex Tkachenko, Ph.D., board member and executive in residence, EcoR1 “The Actio team is comprised of proven geneticists and drug hunters that have both the expertise and drive to advance a company rooted in the science of precision medicine, with a big-picture vision of translating that science into medicines that could ultimately address the most common diseases,” said Dr. Golumbeski, board chairman and partner at DROIA Ventures. “DROIA is honored to partner with this team to advance a platform and pipeline that could change the trajectory of how we leverage the complexities of biology and human genetics to create promising new therapies.” About Actio Biosciences Actio Biosciences is leveraging advances in precision medicine to develop new therapeutics that target shared genetics in rare and common diseases—bringing meaningful medicines from one to many. Applying its expertise in genetics, drug discovery and data sciences, Actio seeks to identify programs where both biological and technical risk can be minimized to streamline the drug development process and bring forward exceptionally potent and precisely targeted therapeutics. Founded in October 2021, the San Diego-based company is led by leaders in genetics and drug development and backed by top healthcare investors. For more information, please visit ActioBiosciences.com. View source version on businesswire.com: Contacts Katie Engleman, 1AB katie@1abmedia.com Source: Actio Biosciences View this news release online at:

Antagonist anti-CD89 will be developed for the treatment of Linear IgA Bullous Dermatosis. WARSAW, Poland--(BUSINESS WIRE)-- JJP Biologics, a company that specializes in the development of proprietary biologic agents for personalized medicine approaches, has announced that the European Commission has approved the company’s application for designation of its product candidate JJP-1212 as an orphan medicinal product for treatment of Linear IgA Bullous Dermatosis (LABD). Following the European Medicines Agency’s recommendation, the European Commission has confirmed that the intention to treat this condition with JJP-1212 justified orphan drug status based on in vitro and in vivo non-clinical data showing the reduction in the influx of granulocytes into the basement membrane zone of the skin and reduction of inflammatory biomarkers, addressing the key elements of LABD skin blistering pathogenesis. LABD is a chronically debilitating disease and may lead to blindness and blister formation. Moreover, currently no approved treatments are available for LABD in the European Union. The European Commission has confirmed that, as of the day of submitting the application, LABD could affect approximately 0.3 in 10,000 persons in the European Union.1 “The orphan drug designation of JJP-1212 by the EMA validates the scientific and business choices of JJP Biologics. It was confirmed that JJP-1212 targets the central mechanism in LABD by antagonizing the IgA-CD89 interaction” according to Louis Boon, the Chief Scientific Officer at JJP Biologics. “We are very excited to offer a potential novel therapy for patients suffering from this ultra-rare skin disease with currently unmet needs”. Having this drug classified into the group of orphan medicinal products provides scientific advice and protocol assistance of the agency. “This decision by the European Commission has also a substantial streamlining effect on the clinical development timeline. We feel the responsibility to translate this decision into prompt availability of the therapy for patients” states Paweł Szczepański, the Chief Operations Officer at JJP Biologics. About JJP Biologics: JJP Biologics sp. z o.o. is a biotechnology company that specializes in the development of proprietary biologic agents accompanied by companion diagnostics to allow therapy personalization. JJP Biologics is a privately funded biotech which pursues the development of its own product candidates as well as projects executed in cooperation with scientific partners. The company’s programs target general immune pathways that have application in autoimmune diseases and cancer. JJP Biologics currently has a range of programs including the most advanced JJP-1212 program and an anti-CD270 program for immune oncology. 1 JJP Biologics performed a systematic review of the published literature and the relevant EU databases (list items)