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.
INDUSTRIAL IMPACT, MEDICAL
Businesswire | April 24, 2023
Guardant Health, Inc. a leading precision oncology company, is pleased to announce that it will present data from two studies highlighting the performance of its blood-based screening technology to detect early-stage colorectal cancer (CRC), including the acceptance of the ECLIPSE study as a late-breaking abstract, at Digestive Disease Week (DDW) taking place in Chicago, Illinois on May 6-9, 2023.
“Despite multiple screening modalities for colorectal cancer, screening rates remain stubbornly low leaving many cancers undetected. A blood test that can accurately detect CRC represents a significant opportunity to overcome some of the known challenges with patient adherence,” said AmirAli Talasaz, Guardant Health co-CEO. “We are thrilled that DDW has accepted the ECLIPSE study, one of the largest studies of its kind, as a late-breaking abstract, and we look forward to sharing this new data with the healthcare community at this year’s meeting.”
“The need for a convenient, less invasive way to screen for CRC is great, and an accurate blood test can play a critical role in improving screening rates, especially for those reluctant to get screened,” said Daniel Chung, MD, gastroenterologist at Massachusetts General Hospital and Professor of Medicine at Harvard Medical School. “I look forward to presenting at DDW and sharing new data from the ECLIPSE Study on the performance and potential of the Shield blood test to help improve adherence to screening."
About Guardant Health
Guardant Health is a leading precision oncology company focused on helping conquer cancer globally through use of its proprietary tests, vast data sets and advanced analytics. The Guardant Health oncology platform leverages capabilities to drive commercial adoption, improve patient clinical outcomes and lower healthcare costs across all stages of the cancer care continuum. Guardant Health has commercially launched Guardant360®, Guardant360 CDx, Guardant360 TissueNext™, Guardant360 Response™, and GuardantINFINITY™ tests for advanced-stage cancer patients, and Guardant Reveal™ for early-stage cancer patients. The Guardant Health screening portfolio, including the commercially launched Shield™ test, aims to address the needs of individuals eligible for cancer screening.
Premier Research | March 14, 2023
Premier Research has partnered with InSilicoTrials, a leader in using artificial intelligence (AI) and computational modeling and simulation (CM&S) to speed up the development of new therapies and medical devices. Premier Research helps biotech and device companies take their ideas from the idea stage to the commercialization stage. The aim of the partnership is to make it easier, faster, and safer for treatments for rare diseases to get approval from the government.
CM&S and AI have made it possible to use virtual patients in clinical trials in whole or in part, which the FDA approves of in some cases. In silico trials make it possible to create fake control or treatment arms, help figure out how to sign up patients, and predict the safety and effectiveness of new drugs and medical devices more accurately.
The use of synthetic control arms generated using in silico CM&S techniques represents a powerful tool for enabling non-feasible rare disease research, both preclinical and clinical research. In the last five years, Premier Research has been in charge of more than 240 rare disease studies for a wide range of indications. This shows that the company is committed to orphan drug development for a long time. Premier Research is continuing to put money into trials for rare diseases by partnering with InSilicoTrials.
One major advantage of in silico simulation is the ability to investigate 'what if' scenarios, particularly in rare diseases where a larger patient population does not exist. By using biological, molecular, or genetic data to describe rare diseases, in silico models can make synthetic control arms. This, along with Premier Research's expertise in clinical and preclinical development, execution, and regulatory aspects of small population trials, can shorten the time it takes to make effective treatments for rare diseases.
The partnership between Premier Research and InSilicoTrials will focus on smarter in silico study design, leading to a more effective pre-clinical review of trial design parameters and faster submission-ready studies. CEO of InSilicoTrials Luca Emili says that combining modeling and simulation with AI is the best way to improve the R&D process in drug development. This is because it allows sponsors to speed up the development of new treatments, make medical products safer, and lower R&D costs.
About Premier Research
Premier Research is a global company that does clinical research, product development, and consulting. It focuses on using new technologies to design and run smart studies and trials. With more than 2,500 employees in 75 countries, it has a lot of experience in certain therapeutic areas and uses. The company is committed to empowering its customers to meet the unmet needs of patients and offers great benefits and flexible working conditions.