CTFR gene (Johns Hopkins University)

- Oral presentation at the Presidential Symposium highlighting Carbon's pulmonary-targeting vector, CBN-1000, capable of delivering the full-length CFTR gene for the treatment of Cystic Fibrosis - - Poster presentations underscore key advancements utilizing Carbon's vector engine, PAVE; CBN vectors effectively shown to de-target the liver while demonstrating tropism in the heart and lung; supported by scalable manufacturing and analytical platforms - - Company remains on track to nominate multiple pre-clinical development candidates before end of 2024 - WALTHAM, Mass., May 2, 2024 /PRNewswire/ -- Carbon Biosciences, a preclinical stage biotechnology company developing genetic medicines for the treatment of pulmonary and cardiac diseases, announced that it has been selected for an oral presentation at the Presidential Symposium during the American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting taking place from May 7-11, 2024, in Baltimore, MD. The presentation will highlight the development of CGT-001, a potential gene therapy for the treatment of Cystic Fibrosis (CF), which utilizes the novel pulmonary targeting vector, CBN-1000, capable of delivering genes beyond the capacity of AAV. In addition, the Company will be featuring three poster presentations detailing new preclinical data, and manufacturing process development and analytical characterization for two of its novel viral gene therapy vectors. "We are pleased to present data at ASGCT demonstrating the potential of the PAVE platform to expand the reach of gene therapy, while simultaneously benefiting from existing AAV knowledge and infrastructure," said, Marc Abrams, Ph.D., Chief Scientific Officer at Carbon Biosciences. "PAVE vectors enable local or systemic delivery of well-validated therapeutic targets that exceed the cargo capacity of existing AAV vectors, without unwanted liver targeting or prior human exposure." Key insights from Carbon Biosciences' presentations at ASGCT: CBN-1000: CBN-1000, a non-AAV parvovirus-based gene therapy vector, demonstrates excellent tolerability and transgene expression throughout the lung and airway in nonhuman primates after nebulization. Powered by the CBN-1000 vector, CGT-001, in development for CF patients not amenable to approved therapies, demonstrates strong expression of full-length cystic fibrosis transmembrane conductance regulator (CFTR) and functional correction in bronchial epithelial cells derived from CF patients, the gold-standard model for functional assessment in CF. Carbon has developed a robust suspension-based triple transfection process for the manufacture of a non-AAV parvovirus-based gene therapy candidate, CGT-001, resulting in high levels of full capsids and productivity similar to AAV-based processes. CBN-1100: CBN-1100 vector demonstrates transgene packaging of up to 5.9 kb, representing >1 kb more capacity than AAV vectors. Systemic administration of CBN-1100 in rodents and nonhuman primates results in robust transgene expression in cardiomyocytes. The complete absence of expression in the liver suggests the potential for a differentiated specificity and safety profile compared to AAV-based vectors for cardiac gene therapy. CBN-1100 is well-tolerated in nonhuman primates at dose levels up to 1.1e14/kg (the highest dose tested), with no significant cytokine response and low liver enzyme elevation, consistent with a lack of liver exposure. Detail for the oral presentation: Abstract Title: A Recombinant Human Bocavirus Vector Delivers Therapeutic Levels of CTFR to Cystic Fibrosis Primary Human Airway Cells and Transduces Airway Epithelium In Vivo (Abstract #3) Oral Session: Presidential Symposium Presenting Author: Jeff Moffit, PhD, DABT Date & Time: Wednesday May 08, 2024, at 10:15am-12:00pm EDT Details for the poster presentations: Abstract Title: Systemic Administration of CBN-1100, a New Recombinant Parvovirus Vector with a Non-AAV Capsid, Shows Favorable Biodistribution and Reduced Liver Tropism in Nonhuman Primates (Abstract #606) Poster Session: Wednesday Posters: Heart, Lung, and Kidney Diseases Presenting Author: Samantha Smith, PhD Date: Wednesday May 08, 2024 Abstract Title: CBN-1100: A New and Differentiated Non-AAV Parvovirus Vector Designed to Overcome Current Gene Therapy Limitations (Abstract #463) Poster Session: Wednesday Posters: AAV Vectors - Virology and Vectorology Presenting Author: Sebastian Aguirre, PhD Date: Wednesday May 08, 2024 Abstract Title: Manufacturing Process Development and Analytical Characterization of CBN-1000, a New Viral Vector Derived from Human Bocavirus (Abstract #1844) Poster Session: Friday Posters: Vector Product Engineering, Development, and Manufacturing Presenting Author: Alfred G. Tamayo, PhD Date: Friday May 10, 2024 About Carbon Biosciences: Carbon Biosciences is a team of passionate drug developers extending the reach of genetic medicines for the treatment of devastating diseases. We combine the genetic diversity and potency of viruses that have evolved over millions of years with the recent clinical and manufacturing advances in the gene therapy field. Our proprietary platform, PAVE, has the potential advantages of exquisite tissue specificity, larger payloads and durable treatment strategies across multiple modalities. Our goal: realize the power of natural viral evolution and deliver on the promise of genetic medicines. SOURCE Carbon Biosciences