Carbon Biosciences to Present Four Times at ASGCT 27th Annual Meeting

Carbon Biosciences, a biotechnology firm focused on developing genetic treatments for pulmonary and cardiac conditions, will present significant advancements at the American Society of Gene and Cell Therapy's (ASGCT) 27th Annual Meeting from May 7-11, 2024. The company's presentations will highlight their pioneering work with novel viral vectors, specifically the CBN-1000 and CBN-1100, which offer promising new approaches for gene therapy.

In a notable oral presentation at the Presidential Symposium, Carbon Biosciences will unveil CGT-001, a gene therapy candidate for Cystic Fibrosis (CF). This therapy is based on the CBN-1000 vector, a non-AAV parvovirus designed to deliver genes surpassing the capacity of traditional adeno-associated viruses (AAV). The CBN-1000 vector shows excellent transgene expression and tolerability in nonhuman primates' lungs and airways when administered via nebulization. CGT-001 demonstrates significant expression of the full-length cystic fibrosis transmembrane conductance regulator (CFTR) gene and functional correction in bronchial epithelial cells from CF patients, representing a significant step forward for those unresponsive to current treatments.

Dr. Marc Abrams, Chief Scientific Officer at Carbon Biosciences, emphasized the potential of their PAVE platform, which allows for the systemic or localized delivery of large therapeutic targets without provoking liver targeting or prior human exposure. This platform could revolutionize gene therapy by leveraging existing AAV knowledge while exceeding its limitations.

The company will also present three posters detailing their progress with the PAVE platform and novel viral vectors. The first poster features the systemic administration of CBN-1100, another non-AAV parvovirus vector, in rodents and nonhuman primates. This vector showcased robust gene expression in heart cells and avoided liver expression, indicating a potentially safer and more specific treatment for cardiac conditions compared to AAV-based therapies.

The second poster explores the distinctive properties of CBN-1100, which can package up to 5.9 kilobytes of genetic material, over 1 kilobyte more than AAV vectors. It demonstrated good tolerance in nonhuman primates at high doses, with no significant immune responses or liver enzyme elevations, affirming its potential for safe and effective cardiac gene therapy.

The third poster discusses the development and manufacturing process of CBN-1000. Carbon Biosciences has established a robust suspension-based triple transfection process for producing this vector, achieving high levels of full capsids and productivity akin to AAV-based methods. This development supports scalable manufacturing and precise analytical characterization, crucial for advancing gene therapy candidates into clinical stages.

In summary, Carbon Biosciences is making noteworthy strides in gene therapy for pulmonary and cardiac diseases through innovative vector technology. Their presentations at the ASGCT meeting underscore the potential of their non-AAV parvovirus-based vectors, CBN-1000 and CBN-1100, in addressing the limitations of current gene therapies. The company anticipates nominating multiple pre-clinical development candidates by the end of 2024, further solidifying their commitment to advancing genetic medicine and improving patient outcomes.