EdiGene 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.

BEIJING & WALTHAM, Mass.--(BUSINESS WIRE)-- EdiGene, Inc., a global, clinical-stage company focused on translating gene-editing technologies into transformative genetic medicines for patients with significant unmet medical needs, announced it had completed the last patient dosing in Phase I clinical trial of ET-01, its investigational gene-editing hematopoietic stem cell therapy for transfusion dependent β-thalassemia (TDT). “The dosing of the 8th and last patient in our multi-center Phase I study is another important milestone in our efforts to bring a potentially one-time cure for patients with TDT,” said Dong Wei, Ph.D., CEO of EdiGene. “We are deeply grateful for the patients and their families, as well as investigators whose participation and dedication have played a critical role in advancing the Phase I study. The study outcome will shed important insight on the safety and efficacy pro ET-01 in this particular patient population, and when at an appropriate time in 2023, we plan to discuss with regulators on Phase II clinical study strategy and design, bringing ET-01 one step closer to patients with TDT in China.” β-thalassemia is a hereditary hemolytic anemia disease. The current standard treatment for TDT requires lifelong packed red blood cell transfusions and iron chelation therapy. ET-01 is designed to achieve a functional cure with a single administration of CRISPR/Cas9 gene-modified autologous hematopoietic stem and progenitor cells. The Phase I clinical trial is a multicenter, open-label, single-arm study to assess the safety and efficacy of a single dose of ET-01 in TDT patients with eight subjects enrolled. EdiGene announced the activation of investigational sites and the first patient enrollment in September 2021. About ET-01 ET-01 is an autologous CD34+ hematopoietic stem/progenitor cell with the erythroid-specific enhancer of the BCL11A gene modified by CRISPR/Cas9. It is the first gene-editing experimental therapy and the first hematopoietic stem cell experimental therapy with IND application approval by China National Medical Products Administration (NMPA). About EdiGene, Inc. EdiGene Inc. is a global, clinical-stage company focusing on translating gene-editing technologies into transformative genetic medicines for patients with significant unmet medical needs. Anchored by its proprietary technologies of gene editing, bioinformatics, and high throughput genome-editing screening, EdiGene advances the pipeline on therapeutic platforms of LEAPERTM-based in vivo RNA base editing, ex vivo gene-editing hematopoietic stem cells, and ex vivo gene-editing T cells for allogeneic CAR-T. Founded in 2015, EdiGene is headquartered in Beijing, with offices in Guangzhou and Shanghai, China, and Waltham, Massachusetts, USA. More information can be found at https://www.EdiGene.com. View source version on businesswire.com: https://www.businesswire.com/news/home/20221118005126/en/

BEIJING & WALTHAM, Mass.--( BUSINESS WIRE )--EdiGene, Inc., a global, clinical-stage company focused on translating its gene-editing technologies into transformative genetic medicines for patients with significant unmet medical needs, announced its subsidiary, EdiGene Biotechnology USA, has moved into a new Research & Development Center in Waltham, Mass. to advance proprietary LEAPER™ technology into transformative in vivo RNA editing therapies with an initial focus on ophthalmology and the central nervous system. LEAPER™ (Leveraging endogenous ADAR for programmable editing of RNA) was developed in a lab at Peking University led by Professor Wensheng Wei, the Scientific Founder of EdiGene, and was first reported in Nature Biotechnology in 2019. In February 2022, Professor Wei’s lab published a research article in Nature Biotechnology on an upgraded version of LEAPER™ that shows significantly improved efficiency and fidelity of RNA editing in vitro and in vivo . “Moving into our new R&D Center is a significant milestone for EdiGene. It marks our continued efforts to advance the development of and accelerate the translation of our exciting RNA editing technology at the global level," said Dong Wei, Ph.D., CEO of EdiGene. “With a strong foundation in RNA editing and translational expertise, we are committed to developing genetic medicines to address the unmet medical needs for patients around the world." “We have been building on our R&D efforts in the greater Boston area with a growing team responsible for drug discovery and development, AAV process development, bioinformatics and clinical development for almost two years,” said Bo Zhang, Ph.D., Head of EdiGene’s U.S. subsidiary. “We are excited to move into our new site to accommodate our rapidly expanding workforce and laboratory operations. By leveraging our unique platform, our team of talented scientists and professionals are creating innovative therapies through internal R&D efforts and external partnerships.” Earlier this year, EdiGene expanded its management team with the appointment of Syed Mahmood, M.D. as Chief Medical Officer. Dr. Mahmood is a physician-scientist with a proven track record of leadership and experience spanning more than 15 years in all facets of drug development, including translational medicine, clinical development, and medical affairs in the United States in global pharmaceutical and biotechnology companies, including Intensity Therapeutics, Novartis, GlaxoSmithKline, Daiichi Sankyo and GE Healthcare. “I am thrilled to join EdiGene because of the potential of its exciting technology portfolio and the critical development stage of the lead programs,” said Syed Mahmood M.D., Chief Medical Officer of EdiGene. “Our new R&D Center represents a new chapter for EdiGene. It was built not only to meet our current needs, but also to scale with our business as we continue to grow. We are at the cusp of advancing LEAPER™ lead candidates to the clinic in the near future and developing potential therapies for patients with serious unmet needs.” In terms of LEAPER™-based in vivo RNA editing therapeutic development, its proof of concept in multiple in vitro and in vivo model systems was presented at the RNA Editing Summit and the annual TIDES USA Conference in 2022. EdiGene established a research collaboration with the University of Wisconsin-Madison in November 2021. Located in the Winter Street office park in the premier suburban Boston office market of Waltham, EdiGene’s 14,000 sq. ft. space holds state-of-the-art laboratories integrated with office space. The opening ceremony hosted representatives from patient advocacy groups, academic and research institutions, industry peers and partners, and investors who share our sense of urgency to meet the medical demand for innovative therapies. About EdiGene, Inc. EdiGene Inc. is a global, clinical-stage company focused on translating gene-editing technologies into transformative genetic medicines for patients with significant unmet medical needs. Anchored by its proprietary technologies of gene editing, bioinformatics, and high throughput genome-editing screening, EdiGene advances the pipeline on therapeutic platforms of LEAPER™-based in vivo RNA base editing, ex vivo gene-editing hematopoietic stem cells, and ex vivo gene-editing T cells for allogeneic CAR-T. Founded in 2015, EdiGene is headquartered in Beijing, with offices in Guangzhou and Shanghai, China, and Waltham, Massachusetts, USA. More information can be found at https://www.EdiGene.com .