CELL AND GENE THERAPY, AI
Businesswire | June 05, 2023
BenevolentAI, a leader in the development of cutting-edge AI that accelerates biopharma discovery, announces the successful delivery of its pre-clinical candidate for the potential treatment of amyotrophic lateral sclerosis (ALS), BEN-34712.
BEN-34712 is an oral, potent and selective brain penetrant RARɑβ (retinoic acid receptor alpha beta) biased agonist and will now enter investigational new drug (IND)-enabling studies.
Impaired retinoic acid signalling has been shown to result in neuroinflammation, oxidative stress and mitochondrial dysfunction, all hallmarks of ALS. In preclinical studies conducted by the Company, BEN-34712 was neuroprotective in a patient-derived, disease-relevant in vitro motor neuron/iAstrocyte co-culture model, demonstrating significant efficacy in both sporadic and familial subtypes of ALS. In addition, BEN-34712 has demonstrated both central nervous system (CNS) target engagement and functional protective effects in the SOD1G93A mouse model of ALS after 50-day repeat dosing.
BenevolentAI collaborated with the Sheffield Institute for Translational Neuroscience (SITraN) at the University of Sheffield on this programme, utilising their patient-derived motor neuron/iAstrocyte co-culture systems and in vivo model expertise.
Anne Phelan, Chief Scientific Officer, BenevolentAI, said: “There remains a significant and urgent need for new and alternative therapies for patients with ALS. We are pleased by the promising advancement of our drug candidate, BEN-34712, towards clinical development, backed by the compelling preclinical data generated by our collaborators at SITraN.”
Richard Mead, Senior Lecturer in Translational Neuroscience at SITraN, commented: "ALS patients suffering from this devastating neurodegenerative disease are in dire need of effective therapy, with the current standard of care options focusing on symptom management or offering limited clinical benefit. We believe BEN-34712 represents an exciting development in our research for a potential new treatment, particularly as it shows effectiveness in both the SOD1G93A mouse model system as well as familial and C9orf72 related ALS patient-derived cell models."
BenevolentAI is a leading developer of advanced artificial intelligence technologies that unlock the value of multimodal data, surface novel insights, and accelerate biomedical discovery. Through the combined capabilities of its AI platform, its scientific expertise, and wet-lab facilities, the Company is developing an in-house drug pipeline of high-value assets. The Company is headquartered in London, with a research facility in Cambridge (UK) and a further office in New York.
ALS is a progressive neurologic disorder characterised by the loss of cortical and spinal motor neurons, leading to the denervation of nerve endplates, axonal retraction and subsequent muscle atrophy. The average survival time following the initial diagnosis is around two-three years, and while there are drugs approved by the US FDA for ALS, they provide only modest benefits to patients, underwriting the urgent need for new and alternative therapies.
About SITraN at the University of Sheffield
The Sheffield Institute for Translational Neuroscience (SITraN) is an essential development in the fight against motor neurone disease and other common neurodegenerative disorders, including Parkinson's and dementia, as well as stroke and multiple sclerosis. SITraN has the potential to bring new treatments and new hope to patients and carers in the UK and worldwide, by significantly accelerating the pace of therapeutic development using technologies such as experimental modelling of disease, gene therapy and stem cell biology, gene expression profiling and bioinformatics analysis and modelling of the biological processes. Since its opening by Queen Elizabeth II in 2010, SITraN has grown immensely and developed into a leading global facility which is at the forefront of research and expertise.
PRNewswire | June 02, 2023
During this year's AAPS National biotechnology conference, Navigate BioPharma Services, Inc., a Novartis subsidiary and a leading provider of innovative biomarker solutions for drug development, announced the launch of its new multiplex digital PCR assays for comprehensive quantification of multiple chimeric antigen receptor (CAR) T cells, a type of cancer immunotherapy that involves genetically modifying T cells to recognize and destroy certain cancers. These assays are based on the Qiagen QIAcuity digital PCR System, which offers a powerful yet easy-to-use digital PCR experience through its unique nanoplate system.
The new multiplex digital PCR assay enables simultaneous measurement of the number, function and persistence of CAR-T cells in clinical blood and bone marrow samples, without the need for a standard curve. It can detect as low as 0.01% of CAR-T cells among normal T cells, with a turnaround time of less than two hours. These assays can measure up to five different CAR-T cell markers in a single reaction, using specific probes for different CAR targets, to quantitate multiple CAR-domains, or combinations of commonly used therapeutic targets for CAR-T therapies. The assay can help clinicians monitor response or safety events during clinical trials to enable the development of new therapies.
"CAR-T therapy is a promising and innovative approach to treat cancer, but it requires accurate and reliable quantification of CAR-T cells for successful drug development," said Dr. Nathan Riccitelli, Associate Director of Innovative Technologies at Navigate BioPharma Services, Inc. "Our new proprietary multiplex digital PCR assay provides a comprehensive and robust solution for measuring CAR-T cells in clinical samples with high sensitivity, specificity and reproducibility."
Navigate BioPharma Services, Inc. has developed this assay for use in sponsors-led clinical trials. If interested, contact them about how this assay can be used in upcoming clinical trial studies.
Navigate Biopharma Services, Inc.
Navigate BioPharma Services, Inc., an independently operating subsidiary within the Novartis group of companies, is a leading provider of innovative biomarker and specialty bioanalytic solutions for clinical development. Over the past decade, Navigate BioPharma has built and developed its exclusive library of assays, methodologies, and biomarker solutions. Their extensive expertise offers unique, accurate, reliable, and cost-effective solutions for its customers and partners.
MEDTECH, INDUSTRY OUTLOOK
PRNewswire | June 01, 2023
Mission Bio, Inc., the pioneer in high-throughput single-cell DNA and multi-omics analysis, announced breakthrough improvements to the Tapestri® Platform and its core chemistry that enable highly confident detection of rare cells for a range of applications. With Tapestri® v3 chemistry, researchers can discover tiny numbers of single cells that, until now, easily escaped detection and influenced disease in invisible ways. At the same time, drug developers can use the new capabilities for a more complete understanding of their advanced therapies, potentially leading to safer, more effective medicines.
The new Tapestri® v3 chemistry increases the number of cells captured per sample by up to four times compared to the prior chemistry, a notable enhancement. With enhanced cell capture, the Tapestri® Platform can more reliably detect rare cells, opening incredible new possibilities like improving the assessment of measurable residual disease (MRD), a key metric used increasingly in clinical settings to estimate the risk of relapse with certain cancers.
In the case of MRD in hematological cancers, rare subclonal variants are commonly missed by bulk NGS due to its averaging effect, hindering the detection of relapse-driving clones that potentially offer new therapeutic targets. With Tapestri®'s expanded capabilities, new integrated multi-omics tools like the Tapestri® scMRD Assay for Acute Myeloid Leukemia (AML) will offer clinicians additional therapeutic insights, rather than providing a binary 'yes or no' answer to the presence of residual disease. A team of investigators from Memorial Sloan Kettering Cancer Center (MSK) using the assay has reported a high sensitivity of 0.01% limit of detection in data presented at Mission Bio's Tapestri® scMRD for AML Summit last year.
Tapestri®'s new capabilities also hold promise for cell and gene therapy developers looking to improve quality assessment throughout the therapy development process. Powered by Tapestri® v3 chemistry, the increase in cell throughput means Tapestri® Genome Editing Solution can measure gene editing outcomes at single-cell resolution – even for very low-frequency events like translocations, which can have significant effects on the safety of the therapy.
Tapestri® v3 is the latest example of Mission Bio's continued focus on customer-centric product and service development. The company has recently implemented additional quality control measurements including design and development, release, and documentation processes compliant with the ISO 9001 standard.
"Our customers' success is at the forefront of our mind," said Adam Abate, PhD, Co-founder and Interim Chief Executive Officer of Mission Bio. "Researchers and drug developers are demanding ever-greater sensitivity and highly robust products to effectively progress their research or advanced therapeutic program, and we are committed to serving our customers and helping them achieve their goals."
The new v3 reagents will be available for shipping starting in mid-June.
About Mission Bio
Mission Bio is a life sciences company that accelerates discoveries and cures for a wide range of diseases by equipping researchers with the tools they need to better measure and predict our resistance and response to new therapies. Mission Bio's multi-omics approach improves time-to-market for new therapeutics, including innovative cell and gene therapies that provide new pathways to health. Founded in 2014, Mission Bio has secured investment from Novo Growth, Cota Capital, Agilent Technologies, Mayfield Fund, and others.