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Pictured: RNA/iStock, Artur Plawgo Based on the significant progress made over the last few decades with RNA therapeutics, RNA editing is widely considered the next generation of promising medicines in this field. RNA therapies have made significant progress over the last few years, with an increasing number of FDA approvals beginning in 1998 with Vitravene for CMV retinitis, followed by Macugen for macular degeneration in 2004 and Spinraza for spinal muscular atrophy in 2016. There have also been multiple siRNA-based drugs, including Onpattro for polyneuropathy of hereditary transthyretin-mediated amyloidosis in 2018. And finally, in 2020, perhaps the most well-known products in the RNA space were introduced: the mRNA-based COVID-19 vaccines. All of these demonstrate the strength of RNA therapies and their potential impact on diseases with high unmet need. RNA therapeutics are indeed elegant approaches to altering RNA and thus protein expression, opening the potential to target a broad array of diseases. The field has seen a renewed and increased interest as reversible changes offer flexibility and RNA approaches introduce therapeutic opportunities that were not accessible before. RNA editing technology was first known and recognized as an interesting approach to treating genetic conditions and reversing disease-causing mutations at the RNA level. RNA editing is a naturally occurring and highly active process that uses the body’s existing capabilities to perform nucleotide changes. Since 2014, when ProQR Therapeutics invented the technique of using oligonucleotides recruiting endogenous adenosine deaminase action on RNA, known as ADAR-mediated editing, the field has rapidly progressed. This growth can be attributed to the rapid progression of knowledge about the technology. Alpha-1 antitrypsin deficiency (AATD) is the first indication that many RNA editing companies have decided to pursue, as it provides the opportunity to address liver and lung symptoms of the disease. Clinical trials for AATD run by both Wave Life Sciences and Korro Bio are planned to begin this year and next. Genetic and Common Disorders There is great excitement in the field about using learnings from decades of oligonucleotide-based drug development, natural RNA editing, and knowledge of biological pathways to make RNA editing technology a compelling approach to target various pathophysiological processes. This offers, for example, the possibility not only of reducing or restoring protein expression but also of modulating protein activity involved in diseases. This application of RNA editing offers the potential to impact both genetic disorders and common conditions, such as metabolic and cardiovascular diseases. ProQR’s approach differentiates RNA editing, as it provides the opportunity to target conditions that have thus far not been treatable with other technologies. Indeed, RNA editing offers the possibility of introducing protective variants, informed by human genetics, that could address or prevent diseases including certain cholestatic or cardiovascular conditions. For example, it has been reported in the literature that an Old Order Amish-enriched variant in a functional B4GALT1 was associated with lower serum LDL-C and lower plasma fibrinogen. This protective variant can be introduced via ADAR RNA editing technology, which has the potential to simultaneously address the two cardiovascular risk factors. Delivery is an important aspect of oligonucleotide base therapeutics. RNA therapies, including RNA editing, have again made great progress and generally use conjugation or lipid nanoparticle approaches. As an example, Alnylam made tremendous progress in its siRNA-based treatments for amyloidosis, with Onpattro in 2018 offering an intravenous treatment once every three weeks. Only four years later, Amvuttra arrived on the market for the same condition but with a subcutaneous 3-month dosing approach. For now, the majority of RNA editing programs are focused on targeting the liver where delivery is relatively de-risked, although progress is also being made in exploring new frontiers such as the central nervous system, as evidenced by the partnerships between Roche and Shape Therapeutics and ProQR and Eli Lilly. In summary, the RNA editing space is making impressive progress. The recent approvals and clinical results demonstrating the potential of RNA therapy to target a broad array of organs are extremely encouraging for RNA editing. Near term, we expect to see further development of the technology, more programs advancing to clinical development, expansion of therapeutic areas addressed, and ultimately, we are hopeful that the next few years will bring considerable progress for patients in need. Gerard Platenburg is a cofounder of ProQR and has served as the company’s chief scientific officer since 2022. Gerard has an extensive background in RNA modulation and orphan drug discovery and development and currently leads ProQR's Innovation unit.

Veteran manufacturing and supply chain expert to oversee global manufacturing and QA CULVER CITY, Calif.--(BUSINESS WIRE)-ImmunityBio, Inc. (NASDAQ: IBRX), a clinical-stage immunotherapy company, today announced that it has named Enrique Diloné, Ph.D., to the newly created role of Chief Technology Officer. With more than three decades of pharmaceutical manufacturing expertise, Dr. Diloné will assume responsibility for all global manufacturing functions as the company prepares for potential FDA approval of a key bladder cancer drug. He will report directly to President and CEO Richard Adcock. Diloné’s career spans more than 30 years of chemistry, manufacturing and controls (CMC) management, pharmaceutical supply chain management, and regulatory responsibilities. During his professional tenure, he has contributed to the licensure of several commercial products including Galafold®, ZolpiMist® and MACUGEN®, and to the development and manufacture of several more clinical products. When he joins ImmunityBio on August 14, he will apply that experience to developing and managing ImmunityBio’s worldwide manufacturing capacity, both company owned and contract, along with responsibility for quality assurance, quality control, manufacturing operations, supply chain and project management. “Enrique joins us at an exciting and important time in ImmunityBio’s journey, at a time when we have multiple Phase 2 and 3 trials underway for therapeutics and vaccines, ready-to-scale manufacturing, and we continue to work diligently to get our drug approved for non-muscle invasive bladder cancer,” said Richard Adcock, President and CEO of ImmunityBio. “Enrique will play a critical role in helping us manage future growth through best-in-class manufacturing processes and supply chain and quality assurance processes that are second to none.” Prior to joining ImmunityBio, Diloné served as Chief Technology Officer of Prothelia, a privately held biotech company developing treatments for patients with congenital muscular dystrophy. He was previously Senior Vice President of Technical Operations at Amicus Therapeutics, a $3.9 billion company focused on rare diseases. He has also held leadership roles with NovaDel Pharma, OSI Pharmaceuticals (Eyetech), and Wyeth. Diloné received his Bachelor of Arts degree in chemistry from New York University and his Ph.D. in chemistry from Seton Hall University. He is also a graduate of the General Management Program at Harvard Business School. He will begin with ImmunityBio on August 14. Diloné will initially work closely with Dr. Lennie Sender, who had been overseeing manufacturing as part of his role as Chief Operating Officer (COO). Dr. Sender remains COO and will continue to focus on day-to-day oversight of the company’s other operations. About ImmunityBio ImmunityBio is a vertically-integrated, clinical-stage biotechnology company developing next-generation therapies and vaccines that bolster the natural immune system to defeat cancers and infectious diseases. The company’s range of immunotherapy and cell therapy platforms, alone and together, act to drive and sustain an immune response with the goal of creating durable and safe protection against disease. We are applying our science and platforms to treating cancers, including the development of potential cancer vaccines, as well as developing immunotherapies and cell therapies that we believe sharply reduce or eliminate the need for standard high-dose chemotherapy. These platforms and their associated product candidates are designed to be more effective, accessible, and easily administered than current standards of care in oncology and infectious diseases. For more information, please visit: Forward Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, such as statements regarding the anticipated hiring and commencement of employment of a Chief Technology Officer, clinical trials, manufacturing capabilities, the regulatory review and approval process and timing thereof, and the development of therapeutics for cancers and infectious diseases, among others. Statements in this press release that are not statements of historical fact are considered forward-looking statements, which are usually identified by the use of words such as “anticipates,” “believes,” “continues,” “goal,” “could,” “estimates,” “scheduled,” “expects,” “intends,” “may,” “plans,” “potential,” “predicts,” “indicate,” “projects,” “seeks,” “should,” “will,” “strategy,” and variations of such words or similar expressions. Statements of past performance, efforts, or results of our preclinical and clinical trials, about which inferences or assumptions may be made, can also be forward-looking statements and are not indicative of future performance or results. Forward-looking statements are neither forecasts, promises nor guarantees, and are based on the current beliefs of ImmunityBio’s management as well as assumptions made by and information currently available to ImmunityBio. Such information may be limited or incomplete, and ImmunityBio’s statements should not be read to indicate that it has conducted a thorough inquiry into, or review of, all potentially available relevant information. Such statements reflect the current views of ImmunityBio with respect to future events and are subject to known and unknown risks, including business, regulatory, economic and competitive risks, uncertainties, contingencies and assumptions about ImmunityBio, including, without limitation, (i) the risks and uncertainties associated with the regulatory review process, (ii) the ability of ImmunityBio and its third party contract manufacturing organizations to adequately address the issues raised in the FDA’s complete response letter, (iii) the ability of ImmunityBio to execute a partnering relationship with a large biopharmaceutical company for commercialization of N-803 plus BCG forintravesical administration on acceptable terms, if at all, (iv) the ability of ImmunityBio to continue its planned preclinical and clinical development of its development programs, and the timing and success of any such continued preclinical and clinical development and planned regulatory submissions, (v) ImmunityBio’s ability to retain and hire key personnel, including the role described herein, (vi) ImmunityBio’s ability to obtain additional financing to fund its operations and complete the development and commercialization of its various product candidates, (vii) ImmunityBio’s ability to successfully commercialize its product candidates and uncertainties around regulatory reviews and approvals, (viii) ImmunityBio’s ability to scale its manufacturing and commercial supply operations for its product candidates and future approved products, and (ix) ImmunityBio’s ability to obtain, maintain, protect and enforce patent protection and other proprietary rights for its product candidates and technologies. More details about these and other risks that may impact ImmunityBio’s business are described under the heading “Risk Factors” in the Company’s Form 10-K filed with the U.S. Securities and Exchange Commission (“SEC”) on March 1, 2023 and the Company’s Form 10-Q filed with the SEC on May 11, 2023, and in subsequent filings made by ImmunityBio with the SEC, which are available on the SEC’s website at . ImmunityBio cautions you not to place undue reliance on any forward-looking statements, which speak only as of the date hereof. ImmunityBio does not undertake any duty to update any forward-looking statement or other information in this press release, except to the extent required by law.