Vita Therapeutics, Inc.

DUBLIN--( BUSINESS WIRE )--The "Induced Pluripotent Stem Cell (iPSC) Industry Report - Market Size, Trends, and Forecasts, 2024" report has been added to ResearchAndMarkets.com's offering. This global strategic report reveals all major market competitors worldwide, including their core technologies, strategic partnerships, and products under development. It covers the current status of iPSC research, biomedical applications, manufacturing technologies, patents, and funding events, as well as all known trials for the development of iPSC-derived cell therapeutics worldwide. Importantly, it profiles leading market competitors worldwide and presents a comprehensive market size breakdown for iPSCs by Application, Technology, Cell Type, and Geography (North America, Europe, Asia/Pacific, and Rest of World). It also presents total market size figures with projected growth rates through 2030. Since the discovery of induced pluripotent stem cell (iPSC) technology in 2006, significant progress has been made in stem cell biology and regenerative medicine. New pathological mechanisms have been identified and explained, new drugs identified by iPSC screens are in the pipeline, and the first clinical trials employing human iPSC-derived cell types have been initiated. iPSCs can be used to explore the causes of disease onset and progression, create and test new drugs and therapies, and treat previously incurable diseases. Today, methods of commercializing induced pluripotent stem cells (iPSCs) include: Cellular Therapy: iPSCs are being explored in a diverse range of cell therapy applications for the purpose of reversing injury or disease. Disease Modelling: By generating iPSCs from patients with disorders of interest and differentiating them into disease-specific cells, iPSCs can effectively create disease models "in a dish". Drug Development and Discovery: iPSCs have the potential to transform drug discovery by providing physiologically relevant cells for compound identification, target validation, compound screening, and tool discovery. Personalized Medicine: The use of techniques such as CRISPR enable precise, directed creation of knock-outs and knock-ins (including single base changes) in many cell types. Pairing iPSCs with genome editing technologies is adding a new dimension to personalized medicine. Toxicology Testing: iPSCs can be used for toxicology screening, which is the use of iPSCs or their derivatives (tissue-specific cells) to assess the safety of compounds or drugs within living cells. Tissue Engineering: iPSCs can be seeded onto scaffolds made from biocompatible materials. These scaffolds mimic the structure and properties of the target tissue and can provide a supportive environment for cell growth and differentiation. Organoid Production: iPS cells can be coaxed to self-organize into 3D structures called organoids, which mimic the structure and function of organs. Organoids can be used for studying organ development, modeling diseases, and testing drugs. Gene Editing: iPS cells can be genetically modified using techniques like CRISPR-Cas9 to correct disease-causing mutations or introduce specific genetic changes. These edited iPS cells can then be differentiated into the desired cell type for transplantation or disease modeling. Research Tools: iPSCs and iPSC-derived cell types are being widely used within a diverse range of basic and applied research applications. Stem Cell Banking: iPSC repositories provide researchers with the opportunity to investigate a diverse range of conditions using iPSC-derived cell types produced from both healthy and diseased donors. Cultured Meat Production: iPSCs are being utilized in clean meat production by serving as the cellular foundation for the creation of lab-grown meat. 3D Bioprinting: iPSCs can be directed to differentiate into cell types of interest, such as skin, heart, or liver cells, which are then incorporated into bioinks. Wildlife Conservation and De-extinction Projects: iPSCs are being used in wildlife conservation and de-extinction projects. For example, Colossal Biosciences is using iPSC technology in an effort to achieve woolly mammoth de-extinction. Key Topics Covered: 1. REPORT OVERVIEW 2. INTRODUCTION 3. CURRENT STATUS OF IPSC INDUSTRY 3.1 Progress made in Autologous Cell Therapy using iPSCs 3.2 Allogeneic iPSC-based Cell Therapies 3.3 Share of iPSC-based Research within the Overall Stem Cell Industry 3.4 Major Focus Areas of iPSC Companies 3.5 Commercially Available iPSC-derived Cell Types 3.6 Relative use of iPSC-derived Cell Types in Toxicology Testing Assays 3.7 iPSC-derived Cell Types used in Clinical Trials 3.8 Currently Available iPSC Technologies Nucleofector Technology Opti-ox Technology MOGRIFY Technology Transcription Factor-based iPSC Differentiation Technology Flowfect Technology Technology for Mass Production of Platelets SynFire Technology 4. HISTORY OF INDUCED PLURIPOTENT STEM CELLS (IPSCS) 5. RESEARCH PUBLICATIONS ON IPSCS 6. IPSC: PATENT LANDSCAPE ANALYSIS 6.1 iPSC Patent Applications by Jurisdiction 6.2 iPSC Patent Applicants 6.3 Inventors of iPSC Patents 6.4 iPSC Patent Owners 6.5 Legal Status of iPSC Patents 7. IPSC: CLINICAL TRIAL LANDSCAPE 7.1 Number of iPSC Clinical Trials 7.2 Recruitment Status of iPSC Clinical Trials 7.3 iPSC Clinical Trials Study Designs 7.4 Therapeutic & Non-Therapeutic iPSC Clinical Trials 7.5 iPSC-based Trials by Phase of Study 7.6 iPSC Clinical Trials by Funder Type 7.7 Geographic Distribution of iPSC-based Clinical Trials 7.8 Promising iPSC Product Candidates CYP-001, CYP-004 & CYP-006 from Cynata Therapeutics BioVAT-HF from Repairon GmbH HS-001 from Heartseed CNTY-101 from Century Therapeutics FT-576 & FT-819 from Fate Therapeutics RPE from National Eye Institute QN-019a from Qihan Biotech iPSC-CL from Heartworks, Inc. 7.9 Companies having Preclinical iPSC Assets Aspen Neuroscience Ryne Biotech Bluerock Therapeutics Vita Therapeutics Hopstem Biotechnology Res Nova Bio, Inc. Cytovia Therapeutics Hebecell Corporation Sana Biotechnology SCG Cell Therapy Pte Cytomed Shoreline Biosciences Neukio Biotherapeutics Exacis Biotherapeutics CellOrigin Biotech 8. M&A, COLLABORATIONS & FUNDING ACTIVITIES IN IPSC SECTOR 8.1 Mergers and Acquisitions (M&A) Sector Century Therapeutics & Clade Therapeutics Evotech & Rigenerand Fujifilm Corporation & Atara Biotherapeutics Catalent & RheinCell Therapeutics Axol Biosciences & Censo Biotechnologies Bayer AG & Bluerock Therapeutics Pluriomix & Axiogenesis 8.2 Partnership/Collaboration & Licensing Deals in iPSC Sector Shinobi Therapeutics & Panasonic SCG Cell Therapy and A*STAR Charles River Laboratories & Pluristyx, Inc. Pluristyx, Inc. & National Resilience, Inc. University of Texas & GeneCure Heartseed, Inc. & Undisclosed Biotech Bluerock Therapeutics & Bit.bio Applied Stem Cell, Inc. & CIRM Resolution Therapeutics & OmniaBio, Inc. REPROCELL, Inc. & CIRM REPROCELL, Inc. & BioBridge Global Elevate Bio & CIRM Evotec & Sernova Evotec & Almiral Quell Therapeutics & Cellistic MDimmune & YiPSCELL Edigene & Neukio Biotherapeutics Matricelf & Ramot Evotec & Boehringer Ingelheim Pluristyx, Pancella & Implant Therapeutics Century Therapeutics & Bristol Myers Squibb Fujifilm Cellular Dynamics & Pheno Vista Biosciences Metrion Biosciences & Bioqube Ventures Cytovia Therapeutics & Cellectis Exacis Biotherapeutics & CCRM Cynata Therapeutics & Fujifilm Corporation Bone Therapeutics & Implant Therapeutics REPROCELL & TEXCELL Jacobio & Herbecell NeuCyte & KIF1A.ORG Kite & Shoreline Biosciences Neuropath Therapeutics & Hopstem Biotechnology Allele Biotech & Cellatoz Bluerock Therapeutics, Fujifilm Cellular Dynamics & Opsis Therapeutics Newcells & Takeda Biocentriq & Kytopen Fujifilm Cellular Dynamics & Sana Biotechnology Evotec & Medical Center Hamburg-Eppdorf (UKE) NeuCyte & Seaver Autism Center for Research and Treatment Cytovia Therapeutics & National Cancer Institute Mogrify & MRC Laboratory of Molecular Biology 8.3 Venture Capital Funding in iPSC Sector Asgard Therapeutics Kenai Therapeutics Pluristyx Fujifilm Cellular Dynamics Mogrify Ltd. Heartseed, Inc. Elevate Bio Aspen Neurosciences Axol Biosciences Thyas, Co. Ltd Synthego Cellino Biotech, Inc Curi Bio Ncardia Evotec SE bit.bio Clade Therapeutics Shoreline Biosciences Kytopen Cytovia Therapeutics & CytoLynx TreeFrog Therapeutics HebeCell Corporation Neukio Biotherapeutics Stemson Therapeutics Vita Therapeutics Century Therapeutics Heartseed Mogrify Metrion Biosciences Elevate Bio Vita Therapeutics 9. GENERATION OF INDUCED PLURIPOTENT STEM CELLS (IPSCS) 9.1 OSKM Cocktail 9.2 Pluripotency-Associated Transcription Factors and their Functions 9.3 Delivery of Reprogramming Factors 9.3.1 Integrating Systems 9.3.2 Non-Integrative Delivery Systems 9.3.3 Comparison of Delivery Methods 9.4 Genome Editing Technologies in iPSC Generation 9.5 Available iPSC Lines and their applications 10. HUMAN IPSC BANKING 10.1 Major Biobanks Storing iPSCs & iPSC Lines 10.1.1 RIKEN 10.1.2 WiCell 10.1.3 Fujifilm Cellular Dynamics, Inc. 10.1.4 Sampled 10.1.5 Coriell Institute for Medical Research 10.1.6 European Bank for Induced Pluripotent Stem Cells (EBiSC) 10.2 Cell Sources for iPSC Banks 10.3 Reprogramming Methods in iPSC Banks 10.4 Ownership and Investments made in iPSC Banks 11. BIOMEDICAL APPLICATIONS OF IPSCs 11.1 iPSCs in Basic Research 11.2 Applications of iPSCs in Drug Discovery 11.3 Applications of iPSCs in Toxicology Studies 11.4 Applications of iPSCs in Disease Modeling 11.5 Applications of iPSCs in Cell-Based Therapies 11.6 Other Novel Applications of iPSCs 12. MARKET ANALYSIS 12.1 Global Market for iPSCs by Geography 12.2 Global Market for iPSCs by Technology 12.3 Global Market for iPSCs by Biomedical Application 12.4 Global Market for iPSCs by Derived Cell Type 12.5 Market Drivers 12.5.1 Current Drivers Impacting the iPSC Market Place 12.6 Market Restraints 13. COMPANY PROFILES For more information about this report visit https://www.researchandmarkets.com/r/pf57qx About ResearchAndMarkets.com ResearchAndMarkets.com is the world's leading source for international market research reports and market data. We provide you with the latest data on international and regional markets, key industries, the top companies, new products and the latest trends.

Licensing agreement utilizing MilliporeSigma’s CRISPR genome-editing technology supports the advancement of Vita’s pipeline ‍Baltimore, MD, July 18, 2023 – Vita Therapeutics, Inc. today announced a licensing agreement between Vita and MilliporeSigma, the U.S. and Canada Life Science business of Merck KGaA, Darmstadt, Germany. Under the agreement, Vita will leverage MilliporeSigma’s foundational CRISPR patents to advance the development of its preclinical asset VTA-100 for the treatment of limb-girdle muscular dystrophy (LGMD2A). Financial and other terms of the agreement were not disclosed. “Our purpose is to impact life and health with science, empowering customers to deliver breakthroughs such as Vita's impactful, health-advancing research,” said Christopher Arnot, Director of Licensing and Business Development, Gene Editing, MilliporeSigma. “For people with limb-girdle muscular dystrophy, the calpain 3 (CAPN3) gene is mutated in satellite cells,” said Douglas Falk, MS, Chief Executive Officer at Vita Therapeutics. “MilliporeSigma’s proprietary CRISPR genome-editing technology will allow us to insert a functional copy of the gene into those cells, taking us an important step forward in advancing the development of VTA-100 as a potential cell therapy.” About Limb-Girdle Muscular Dystrophy ‍Limb-girdle muscular dystrophy (LGMD) is a group of disorders that cause weakness and wasting of muscles closest to the body (proximal muscles), specifically the muscles of the shoulders, upper arms, pelvic area, and thighs. The severity, age of onset, and disease progression of LGMD vary among the more than 30 known sub-types of this condition and may be inconsistent even within the same sub-type. As the atrophy and muscle weakness progresses, individuals with LGMD begin to have trouble lifting objects, walking, and climbing stairs, often requiring the use of assistive mobility devices. There is currently no cure for LGMD, with treatments limited to supportive therapies such as corticosteroids. About Vita Therapeutics ‍Vita Therapeutics is a biotechnology company developing state-of-the-art cellular therapeutics for the treatment of debilitating neuromuscular diseases and cancers. Vita Therapeutics uses induced pluripotent stem cell (iPSC) technology to engineer specific cell types designed to replace those that are defective in patients. The Company is progressing its lead program VTA-100 for the treatment of limb-girdle muscular dystrophy (LGMD2A) with the goal of filing Investigational New Drug Applications with the U.S. Food and Drug Administration in the next 18 months. Long term, the Company is developing its pipeline of cellular therapies following a dual development strategy beginning with autologous-derived cells before moving to a universal hypoimmunogenic cell line. Vita Therapeutics is currently working with numerous partners, including PanCella, Wyss Institute, and Johns Hopkins University, to advance their clinical programs. Learn more about the company at www.Vitatx.com. About the Life Science business of Merck KGaA, Darmstadt, Germany The Life Science business of Merck KGaA, Darmstadt, Germany, which operates as MilliporeSigma in the U.S. and Canada, has more than 28,000 employees and more than 55 total manufacturing and testing sites worldwide, with a portfolio of more than 300,000 products focused on scientific discovery, biomanufacturing and testing services. Merck KGaA, Darmstadt, Germany, a leading science and technology company, operates across healthcare, life science and electronics. More than 64,000 employees work to make a positive difference to millions of people’s lives every day by creating more joyful and sustainable ways to live. From providing products and services that accelerate drug development and manufacturing as well as discovering unique ways to treat the most challenging diseases to enabling the intelligence of devices – the company is everywhere. In 2022, Merck KGaA, Darmstadt, Germany, generated sales of € 22.2 billion in 66 countries. The company holds the global rights to the name and trademark “Merck” internationally. The only exceptions are the United States and Canada, where the business sectors of Merck KGaA, Darmstadt, Germany, operate as MilliporeSigma in life science, EMD Serono in healthcare and EMD Electronics in electronics. Since its founding in 1668, scientific exploration and responsible entrepreneurship have been key to the company’s technological and scientific advances. To this day, the founding family remains the majority owner of the publicly listed company. For more information about Merck KGaA, Darmstadt, Germany, visit  www.emdgroup.com. Follow MilliporeSigma on Twitter @MilliporeSigma, on Facebook @MilliporeSigma and on LinkedIn. All Merck KGaA, Darmstadt, Germany news releases are distributed by email at the same time they become available on the EMD Group website. In case you are a resident of the U.S. or Canada please go to www.emdgroup.com/subscribe to register again for your online subscription of this service as our newly introduced geo-targeting requires new links in the email. You may later change your selection or discontinue this service. ‍ # # # ‍ ‍Media Inquiries for Vita ‍Mike Beyer Sam Brown Inc. Healthcare Communications 312-961-2502 mikebeyer@sambrown.com Media Inquiries for MilliporeSigma ‍Livia Guadaim Millipore Sigma livia.guadaim@merckgroup.com ‍

FALLS CHURCH, Va., May 15, 2023 /PRNewswire/ -- Today the Limb-Girdle Muscular Dystrophy (LGMD) Coalition, a partnership of six LGMD patient organizations, released the Voice of the Patient report summarizing outcomes from the September 2022 Externally-Led Patient Focused Drug Development Meeting (EL-PFDD). This meeting focused on LGMD subtypes LGMD2A/R1, LGMD2C/R5, LGMD2D/R3, LGMD2E/R4, LGMD2F/R6, and LGMD2i/R9. The goal of the report is to provide the U.S. Food and Drug Administration (FDA), clinicians, medical product developers, and academic researchers an opportunity to hear perspectives from individuals with LGMD on the health effects, daily impacts, treatment goals, and decision factors considered when seeking out or selecting a treatment. The meeting was conducted as a parallel effort to FDA's EL-PFDD initiative, a commitment under the fifth authorization of the Prescription Drug User Fee Act (PDUFA V) to more systematically gather patients' perspectives on their conditions and available therapies to treat their conditions. The meeting included 29 speakers and panelists who are living with LGMD2A/R1, LGMD2C/R5, LGMD2D/R3, LGMD2E/R4, LGMD2F/R6, and LGMD2I/R9, as well as caregivers of individuals living with these six subtypes. An additional 136 patients and family members responded to a live survey and over 250 written comments were submitted. In total, over 600 people attended the meeting, including over 27 FDA representatives, 48 drug company representatives, and over 400 LGMD patients and family participants. Common themes that emerged throughout the meeting included: (1) LGMDs are progressive diseases, but symptoms progress at different rates, and many individuals are severely affected; (2) People living with LGMDs urgently need better treatments; (3) LGMDs affect multiple body systems; (4) Individuals with LGMD experience discrimination and social exclusion, (5) Current treatments do not change the course of the diseases; (6) Many individuals are eager to participate in clinical trials. Six patient organizations, jointly referred to as the LGMD Coalition, partnered together to organize the event: Coalition to Cure Calpain 3, CureLGMD2i, the Kurt+Peter Foundation, the LGMD2D Foundation, the McColl-Lockwood Laboratory for Muscular Dystrophy Research, and the Speak Foundation. The Voice of the Patient report and a recording of the meeting are available at . "It is our hope that drug developers and regulators will read the report and incorporate patients' and caregivers' perspectives when developing and reviewing new therapies to best meet the needs and expectations of our community," shared Coalition to Cure Calpain 3 Scientific Director Jennifer Levy. The LGMD Coalition is appreciative for support from our sponsors, Sarepta Therapeutics, AskBio, Edgewise Therapeutics, ML BioSolutions, and Vita Therapeutics. "We wish to thank our many partners from advocacy and professional organizations, drug companies, federal agencies, and universities from across the world who attended our meeting," said Kathryn Bryant, founder of the Speak Foundation. "We are extremely grateful to all the individuals in the LGMD community who contributed to the meeting's success by watching online, providing oral and written testimony, and responding to the survey questions. We believe that their engagement in this process provided insights into these subtypes of LGMD which will help the research community develop effective therapies." About LGMD LGMD is a term used for a group of rare neuromuscular diseases which are inherited and cause muscle weakness and wasting. The muscles most affected are those closest to the body (proximal), specifically the muscles of the shoulders, upper arms, pelvic area, and thighs. More than 30 subtypes of LGMD exist. Together, LGMDs have an estimated prevalence of about 2 in every 100,000 individuals. There is currently no cure for any subtype of LGMD. About the LGMD Coalition The LGMD Coalition is a group of six 501(c)3 nonprofit organizations -- Coalition to Cure Calpain 3, CureLGMD2i, the Kurt+Peter Foundation, the LGMD2D Foundation, the McColl-Lockwood Laboratory for Muscular Dystrophy Research, and the Speak Foundation – that collaborated to host an LGMD EL-PFDD focused on subtypes LGMD2A/R1, LGMD2C/R5, LGMD2D/R3, LGMD2E/R4, LGMD2F/R6, and LGMD2I/R9. Learn more at . Contact: Jennifer Levy 3194009131 [email protected] SOURCE LGMD Coalition