OTL-104 / Kyowa Kirin - LARVOL DELTA

Haematopoietic stem cell gene therapy as a treatment for NOD2-deficient severe Crohn’s Disease (ESGCT 2024) - "Gene expression ontology analysis of gut tissue from transplanted mice confirms that NOD2-dependent innate immune activation, intestinal tissue remodeling and immune homeostasis signaling is fully corrected to levels observed in WT mice. These results confirm the impact of NOD2 deficiency in primary immune activation which drives disease induction and pathology in Crohn’s Disease and demonstrates the therapeutic potential of OTL-104 HSC-GT to achieve long-term correction of severe disease in NOD2-CD."

Gene therapy • Crohn's disease • Gastroenterology • Gastrointestinal Disorder • Gene Therapies • Immunology • Infectious Disease • Inflammatory Bowel Disease • Primary Immunodeficiency • CD34 • CXCL1 • IL6 • NLRC5

Orchard Therapeutics Announces Multiple Data Presentations and Publications (GlobeNewswire) - "Ten presentations (three oral and seven posters) from across its hematopoietic stem cell (HSC) gene therapy portfolio will be featured at the European Society of Gene and Cell Therapy (ESGCT) 31st Annual Congress taking place October 22-25...Featured data include several accepted abstracts and encore presentations detailing clinical, biochemical and other functional outcomes from across the company’s commercial- and clinical-stage neurometabolic portfolio in metachromatic leukodystrophy (MLD), the Hurler subtype of mucopolysaccharidosis type I (MPS-IH), and mucopolysaccharidosis type IIIA (MPS-IIIA), also known as Sanfilippo syndrome type A....An oral presentation of preclinical proof-of-concept data showing the therapeutic potential of OTL-104 for NOD2-deficient Crohn’s disease, a severe and treatment-refractory form of the condition; A poster presentation outlining the potential of HSC gene therapy as a mechanism to deliver therapeutic antibodies..."

Clinical • Preclinical • Crohn's disease • Hurler Syndrome • Immunology • Inflammatory Bowel Disease • Metabolic Disorders • Multiple Sclerosis

EPOSTER: HEMATOPOIETIC STEM CELL GENE THERAPY AS A TREATMENT FOR SEVERE CROHN’S DISEASE ASSOCIATED WITH PATHOGENIC NOD2 GENETIC VARIANTS (DDW 2024) - "Key to our therapeutic approach histopathological analysis of intestinal lamina propria from transplanted mice shows a normal biodistribution and physiological NOD gene expression in tissue resident cells. These results confirm the impact of NOD deficiency in primary immune activation and demonstrates the therapeutic potential of OTL-104 HSC-GT for long-term correction of NOD -associated CD."

Gene therapy • Crohn's disease • Gastroenterology • Gastrointestinal Disorder • Gene Therapies • Hematological Disorders • Immunology • Infectious Disease • Inflammatory Bowel Disease • Primary Immunodeficiency • Transplantation • CD34 • CXCL1 • CXCL8 • IL10 • IL6 • NLRC5

Orchard Therapeutics Announces Multiple Presentations at ASGCT 2024 (GlobeNewswire) - "Orchard Therapeutics,...announced four oral and four poster presentations from across its hematopoietic stem cell (HSC) gene therapy platform will be featured at the 27th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) taking place May 7-11, 2024, in Baltimore....In addition, Orchard Therapeutics will give an invited oral presentation highlighting the potential of OTL-104, a pre-clinical HSC gene therapy developed by its in-house research team, to address a severe and treatment refractory form of Crohn’s disease."

Preclinical • Crohn's disease • Immunology • Inflammatory Bowel Disease

Haematopoietic stem cell gene therapy as a treatment for severe Crohn’s Disease associated with pathogenic NOD2 genetic variants (ECCO-IBD 2024) - "Key to our therapeutic approach, histopathological analysis of intestinal lamina propria from transplanted mice shows a normal biodistribution and physiological NOD2 gene expression in tissue resident cells. Conclusion These results confirm the impact of NOD2 deficiency in primary immune activation and demonstrates the therapeutic potential of OTL-104 HSC-GT for long-term correction of NOD2-deficient CD."

Gene therapy • Crohn's disease • Immunology • Inflammatory Bowel Disease • CD34 • CXCL1 • IL6 • NLRC5

HEMATOPOIETIC STEM CELL GENE THERAPY AS A NOVEL THERAPEUTIC APPROACH FOR SEVERE CROHN’S DISEASE ASSOCIATED WITH NOD2-DEFICIENCY (CCCongress 2024) - "Key to our therapeutic approach, histopathological analysis of intestinal lamina propria from transplanted mice shows a normal biodistribution and physiological NOD2 gene expression in tissue resident cells (Figure 1). These results confirm the impact of NOD2 deficiency in primary immune activation and demonstrates the therapeutic potential of OTL-104 HSC-GT for long-term correction of NOD2-deficient CD."

Gene therapy • Crohn's disease • Gastroenterology • Gastrointestinal Disorder • Gene Therapies • Hematological Disorders • Immunology • Infectious Disease • Inflammatory Bowel Disease • Pediatrics • Primary Immunodeficiency • Transplantation • CD34 • CXCL1 • CXCL8 • IL10 • IL6 • NLRC5 • TNFA

Orchard Therapeutics Reports Third Quarter 2023 Financial Results and Highlights Recent Business Accomplishments (GlobeNewswire) - "OTL-104 for NOD2-Crohn’s disease: Commence IND- and CTA-enabling studies by year end, ahead of a potential filing in 2025."

Preclinical • Crohn's disease • Immunology • Inflammatory Bowel Disease

HEMATOPOIETIC STEM CELL GENE THERAPY AS A NOVEL THERAPEUTIC APPROACH FOR SEVERE NOD2-DEFICIENT CROHN'S DISEASE (UEGW 2023) - "These results confirm the negative impact of NOD2 deficiency in primary immune activation and support the therapeutic potential of OTL-104 HSC-GT for long-term correction of NOD2-deficient CD."

Gene therapy • Crohn's disease • Gastroenterology • Gene Therapies • Hematological Disorders • Immunology • Infectious Disease • Inflammatory Bowel Disease • Pediatrics • Primary Immunodeficiency • Transplantation • CD34 • CTSG • CXCL1 • CXCL8 • IL10 • IL6 • NLRC5 • TNFA

Orchard Therapeutics Presents Data from Research Programs at ASGCT Demonstrating the Ability of HSC Gene Therapy to Address Larger Indications (GlobeNewswire) - "Orchard Therapeutics...today presented new data from the company’s hematopoietic stem cell (HSC) gene therapy research pipeline targeting larger indications at the ongoing 26th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) in Los Angeles. Presentations include preclinical proof-of-concept data from OTL-104, the company’s investigational HSC gene therapy for the treatment of nucleotide-binding oligomerization domain containing protein 2 (NOD2) Crohn’s disease, as well as in vivo preclinical data demonstrating the development of functional CAR-Treg cells from genetically engineered HSCs. Both programs were designed and developed in Orchard’s own research laboratories utilizing the company’s proprietary HSC gene therapy platform....The company expects to commence IND- and CTA-enabling studies in the second half of 2023, with a potential filing now anticipated in the first half of 2025."

IND • Preclinical • Crohn's disease • Immunology • Inflammatory Bowel Disease

Orchard Therapeutics Reports First Quarter 2023 Financial Results and Announces Initiation of Rolling Submission for Biologics License Application of OTL-200 for Metachromatic Leukodystrophy to U.S. FDA (Yahoo Finance) - "OTL-104 for NOD2-Crohn’s disease: Report pre-clinical PoC data at the upcoming ASGCT annual meeting and initiate IND / CTA-enabling activities."

Preclinical • Crohn's disease • Immunology • Inflammatory Bowel Disease

Hematopoietic Stem Cell Gene Therapy as a Novel Therapeutic Approach for Severe NOD2-Deficient Crohn's Disease (ASGCT 2023) - "Key to the success of our therapeutic approach, histopathological analysis of intestinal lamina propria from transplanted mice shows a normal biodistribution and physiological NOD2 gene expression in tissue resident cells (Figure 1). These results confirm the negative impact of NOD2 deficiency in primary immune activation and support the therapeutic potential of OTL-104 HSC-GT for long-term correction of NOD2-deficient CD."

Gene therapy • Crohn's disease • Gastroenterology • Gastrointestinal Disorder • Gene Therapies • Hematological Disorders • Immunology • Infectious Disease • Inflammatory Bowel Disease • Pediatrics • Primary Immunodeficiency • Transplantation • CD34 • CTSG • CXCL1 • CXCL8 • IL10 • IL6 • NLRC5 • TNFA

Orchard Therapeutics Highlights Recent Progress Across HSC Gene Therapy Portfolio and Outlines Key 2023 Milestones (GlobeNewswire) - "OTL-104 for NOD2-Crohn’s disease: Report pre-clinical PoC data in the first half of 2023 and initiate IND-enabling activities in advance of a planned 2024 filing."

Preclinical • Crohn's disease • Immunology • Inflammatory Bowel Disease

Orchard Therapeutics Reports Third Quarter 2022 Financial Results and Reviews Recent Business Highlights (GlobeNewswire) - "Report preclinical POC data for the OTL-104 program in NOD2 Crohn’s disease in early 2023 in advance of IND-enabling studies and an IND submission in 2024."

IND • Preclinical • Crohn's disease • Immunology • Inflammatory Bowel Disease

Orchard Therapeutics Reports First Quarter 2022 Financial Results and Highlights Recent Commercial Progress (GlobeNewswire) - "Orchard Therapeutics...announced recent business highlights along with its financial results for the quarter ended March 31, 2022....Report preclinical POC data for the OTL-104 program in NOD2 Crohn’s disease (NOD2-CD) by year end 2022 in advance of IND-enabling studies in 2023 and an IND submission in 2024."

IND • Preclinical • Crohn's disease • Immunology • Inflammatory Bowel Disease

Orchard Therapeutics Extends Runway into 2024, Focusing HSC Gene Therapy Platform Exclusively on Severe Neurometabolic Diseases and Research Platform (Businesswire) - "Orchard Therapeutics...today announced its intention to focus its hematopoietic stem cell (HSC) gene therapy platform exclusively on severe neurometabolic diseases and early research programs while also reporting its financial results for the quarter and year ended December 31, 2021....Report preclinical POC data for the OTL-104 program in NOD2 Crohn’s disease (NOD2-CD) by year end 2022 and file an IND in 2024..."

IND • Preclinical • Crohn's disease • Immunology • Inflammatory Bowel Disease

Orchard Therapeutics Outlines Differentiated Profile of Its HSC Gene Therapy Approach and Discusses Potential Future Applications at Virtual R&D Investor Event (GlobeNewswire) - "Orchard Therapeutics...will present on the company’s discovery and research efforts in hematopoietic stem cell (HSC) gene therapy, including an update on the OTL-104 program in development for NOD2 Crohn’s disease (NOD2-CD)...Restoration of NOD2 protein expression in murine and human stem cells can rescue a defective myeloid immune response to microbial peptides...Development of an experimental colitis induction model is now in progress for OTL-104 pre-clinical proof-of-concept studies. The promising data highlighted above supports the continued advancement of OTL-104 toward IND-enabling toxicology and biodistribution studies."

Preclinical • Crohn's disease • Inflammatory Bowel Disease • Ulcerative Colitis