Congenital muscular dystrophy

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.

Care is increasingly shifting from the hospital to the home, driven by factors such as a growing elderly population, the proliferation of virtual care options during the pandemic and reduced expenses for payers. Investors seem to have recognized this movement —at-home care was one of the three main categories within digital health that received the most venture investment during the first half of the year, along with value-based care and technology to improve healthcare’s workforce management. The home healthcare market was valued at $281.8 billion in 2020, but Grand View Research predicted last month that the market will reach $666.9 billion by 2030. Additionally, the White House’s budget includes $150 billion to improve and expand Medicaid home care services over the next decade. Yet, there are several misconceptions about this market, including the misbelief that at-home care is only for senior citizens and that this care model lacks scalability. The pandemic was the “initial forcing function” that necessitated the shift from hospital-based care to home-based care, said Yoni Shtein, CEO of Laguna Health, in a recent interview. His startup offers an AI-powered contextual care management platform that scales personalized care for patients as they transition from hospital care to at-home care. It raised $15 million in Series A financing in May. “During Covid, health systems expanded telehealth services out of necessity and, more importantly, expanded provider reimbursement to include parity for virtual care while also expanding coverage to include remote patient monitoring,” Shtein declared. While the healthcare industry is getting more and more comfortable with the idea that care needs to be expanded beyond the four walls of a hospital, he pointed out that some industry stakeholders still think that the physical provision of home care is too expensive and difficult to scale. In reality, some patients need in-home skilled nursing in which they are physically tended to, but many can manage their health via lower-cost options like telehealth appointments and wearable remote monitoring devices, Shtein explained. Another common misconception is the belief that home-based care is primarily for elderly individuals, said Vijay Kedar, CEO of Tomorrow Health. His company — which seeks to make it easier for patients, providers and payers to coordinate home-based — has raised $92.5 million since being founded in 2018. While older adults do represent a significant portion of the population receiving at-home care, this type of care can be provided to all age groups, Kedar declared. “For example, at Tomorrow Health, patients range from a 23-year-old female with congenital muscular dystrophy to a 15-year-old quadriplegic with chronic feeding difficulties as well as a 12-year-old male with a cerebellar tumor — with needs for everything from a hospital bed to nutrition supplies to continuous glucose monitors,” he said. Kedar also pushed back against the idea that care provided at home is lower in quality than care provided in a hospital or nursing home, arguing that well-managed home healthcare programs can provide comparable or even better outcomes in terms of patient satisfaction, health improvement and cost-effectiveness. He also pointed out that at-home care allows patients to receive personalized care in the comfort of their own homes, which can contribute to better overall wellbeing. Researchers will likely produce more data backing up the arguments made by Kedar and Shtein as the healthcare industry continues to embrace more home healthcare models. The first half of 2023 has already seen some megadeals in the at-home care space, including Monogram Health‘s $375 million fundraise and Author Health‘s $115 million financing round.

MDL-101 preclinical data support efficacy and safety of a differentiated precision medicine approach for LAMA2 Congenital Muscular Dystrophy (LAMA2-CMD) Preclinical data showing that our CRISPR-based epigenetic editing technology regulates the expression of target genes in mammals, suggesting the possibility of clinical efficacy as a therapeutic approach for serious genetic disorders TOKYO & WALTHAM, Mass.--(BUSINESS WIRE)-- Modalis Therapeutics Corporation (Tokyo Stock Exchange: 4883), a pioneering company developing innovative products for the treatment of rare genetic diseases utilizing its proprietary CRISPR-GNDM® epigenetic editing technology, today announced that the abstract has been accepted for a presentation in the late-breaking session at the 26th Annual Meeting of The American Society of Gene & Cell Therapy (ASGCT), being held in Los Angeles CA on May 16-20, 2023. The abstracts present preclinical data from the Company’s LAMA2 Congenital Muscular Dystrophy (LAMA2-CMD). Modalis presentations at ASGCT will include preclinical data demonstrating that: In LAMA-2 knockout mice (dyW disease model mice) and non-human primates (NHPs), a CRISPR-GNDM® based molecule (MDL-101) targeting the LAMA-1 gene introduced into a muscle-specific AAV vector raised LAMA-1 expression to levels that complement LAMA-2 function in the disease animals. This suggests that MDL-101 may have therapeutic potential in the clinic. At ASGCT, we will present preclinical data from MDL-101 validating the efficacy and safety of our differentiated therapeutic strategy utilizing our CRISPR-GNDM® technology for the previously difficult-to-treat LAMA2-CMD. "Our proprietary and world-first CRISPR-based epigenetic editing technology, CRISPR-GNDM®, controls the expression levels of disease-causing genes and provides a disease-modifying treatment for genetic disorders,” said Haru Morita, CEO of Modalis. “MDL-101 has raised its firepower as a potentially life-changing gene therapy for the treatment of LAMA2-CMD by employing a muscle-specific capsid that further increases the selectivity for muscle tissue. Development is currently on track for IND filing by the end of 2024. At this meeting, we will share data from our mice and NHPs studies that have shown efficacy and safety and discuss the potential implications for therapeutic efficacy in the clinic." stated. The Modalis Therapeutics presentation is as below. The abstract will become available on the ASGCT website from May 12th and the presentations will be posted on the Modalis website during the conference. Oral Presentation: Title: Advancing Epigenetic Editing with CRISPR-GNDM: Novel Muscle-Tropic AAV Vectors Deliver Promising Single-Dose Treatment for LAMA2-CMD Date and Time: 5/19/2023 10:45 AM Session Name: Late-breaking Abstracts 2 About MDL-101 MDL-101 is an experimental, epigenetic modulation therapy under investigation for the treatment of LAMA2-Congenital Muscular Dystrophy (LAMA2-CMD). MDL-101 is comprised of guide nucleotide targeting LAMA1 gene, a highly homologous sister gene of the disease-causing gene LAMA2, enzyme-null Cas9 (dCas9) fused with trans-activating domain driven by a muscle-specific promoter and coded in a muscle-specific AAV vector. MDL-101 upregulates LAMA-1 gene products in patients’ muscle tissue to compensate for loss-of-function caused by mutation of LAMA2, and therefore has the potential to provide a one-time, durable treatment benefit for people living with LAMA2-CMD. About Modalis: Modalis Therapeutics develops precision genetic medicines using epigenetic gene editing technology. Modalis is pursuing therapies for orphan genetic diseases using its proprietary CRISPR-GNDM® technology which enables the gene/locus-specific modulation of gene expression or epigenetic editing without the need for DNA cleavage or altering DNA sequence. Headquartered in Tokyo with laboratories and facilities in Waltham, Massachusetts, the company is listed on Tokyo Stock Exchange’s Growth market. For additional information, visit . View source version on businesswire.com: Contacts Modalis Therapeutics Sawako Nakamura media@modalistx.com Source: Modalis Therapeutics Corporation View this news release online at: