Liss' Lowdown #21

Preclinical Breakthrough of the month:

Diversifying Preclinical Research: AMSBIO's Role in Addressing Gender Discrepancies and Advancing Inclusivity

AMSBIO expands its offerings to support researchers in diversifying preclinical models, aiming to address gender gaps pervasive in research. Despite advancements in clinical trials, inclusivity in preclinical research remains overlooked, leading to critical knowledge gaps in treatment effects across populations. In a blog marking International Women's Day, AMSBIO proposes an intestinal organoid biobank to improve accessibility and highlights gender reporting disparities in COVID-19 vaccine development. As a global biospecimen provider, AMSBIO offers a diverse biorepository and custom tissue procurement service to meet specific research needs. Partnering with ScreenIn3D (S3D), AMSBIO leverages novel organoids and lab-on-a-chip technology to aid researchers in cost-effective high-throughput assays. CEO Alex Sim notes the FDA's 2023 legislation, mandating demographic requirements in clinical trials, and underscores AMSBIO's role in supporting research for societal benefit and the development of combination therapies.

Clinical Breakthrough of the month:

Promising Gene Therapy for Giant Axonal Neuropathy Shows Safety and Potential Benefits in NIH-led Trial

A gene therapy trial targeting giant axonal neuropathy (#GAN), a fatal childhood neurodegenerative disease, showed promising results in a study led by the The National Institutes of Health . The therapy, scAAV9/JeT-GAN, delivered functional copies of the defective GAN gene directly into spinal fluid. Results, published in the New England Journal of Medicine, indicated the treatment was well tolerated and slowed motor function decline in some patients. Notably, sensory nerve regeneration occurred in several cases. The trial, involving 14 children aged 6 to 14, revealed a favorable safety profile with few adverse events. The therapy's efficacy increased with higher doses, offering hope for GAN patients and potentially informing gene therapy for other diseases. Future research will explore treatment effectiveness in younger patients and those in earlier disease stages. Supported by various organizations, including NINDS and the National Institute of Arthritis and Musculoskeletal and Skin Diseases, this trial represents a collaborative effort to advance therapies for rare diseases.

Partnership of the month:

Strategic Partnership Enhances Drug Development: InnoSer and CR2O Set New Industry Standard

InnoSer and CR2O | Clinical CRO collaborate to provide a comprehensive drug development solution, bridging pre-clinical and clinical phases. This partnership aims to streamline the process, reduce costs, and mitigate risks, setting a new industry standard. Clients benefit from an integrated service spanning early-stage discovery to phase I-IV trials, with specialized offerings such as toxicology, pharmacology, and regulatory strategy. By combining expertise, the companies empower researchers with enhanced efficiency and regulatory insights, aligning with their commitment to innovation and patient care. Jan Bartels , CEO of InnoSer, emphasizes the partnership's focus on advancing patient care and excellence in research services. CR2O CEO Hadil Es-Sbai highlights the importance of integrating R&D processes to address industry fragmentation. Together, InnoSer and CR2O aim to offer validated drug development pathways, increasing client confidence and driving innovation in the sector. This strategic collaboration signifies a shift towards a more cohesive and efficient approach to drug development, benefitting both researchers and patients alike.