INDUSTRY OUTLOOK
Globenewswire | May 29, 2023
Monte Rosa Therapeutics, Inc. a clinical-stage biotechnology company developing novel molecular glue degrader (MGD)-based medicines, announced its second development candidate, MRT-6160, a novel, potent, and selective molecular glue degrader of VAV1. The Company plans to file an Investigational New Drug (IND) application for MRT-6160 in the first half of 2024 and to develop the molecule as a potential treatment for autoimmune diseases.
“MRT-6160 is a potent, orally bioavailable MGD designed to degrade VAV1, an important protein involved in the signaling pathways of T and B cells. Our in vitro studies have shown that MRT-6160 selectively degrades VAV1 without detectable effects on other proteins. By targeting VAV1, MRT-6160 attenuates multiple aspects of T- and B-cell function and inhibits disease progression in established in vivo models of autoimmunity,” said Owen Wallace, Ph.D., Chief Scientific Officer of Monte Rosa. “The underlying biology and our preclinical data both demonstrate that MRT-6160 acts as an immune modulator, which has the potential to avoid the broad immune suppression seen with other approaches. We look forward to progressing our clinical plan developed with the goal of providing early insights into safety, PK and PD, and proof of concept regarding differentiated effects on key immunomodulatory signaling pathways.”
“Our goal centers on pioneering therapeutically meaningful new drugs for patients with serious diseases. By addressing VAV1, a validated but previously undruggable target, we believe we've created a potentially groundbreaking therapy for patients suffering from a range of serious autoimmune conditions, particularly those involving both T- and B cell-mediated autoimmunity,” said Markus Warmuth, M.D., CEO of Monte Rosa. “MRT-6160 is expected to be our second MGD to enter clinical trials, showcasing the continued productivity of our QuEEN™ platform. We anticipate significant progress and milestones in our portfolio in the upcoming year, including initial clinical data from our GSPT1 MGD, MRT-2359, in the second half of this year and filing of an IND application for MRT-6160 in the first half of next year.”
About Monte Rosa
Monte Rosa Therapeutics is a biotechnology company developing novel molecular glue degrader (MGD) medicines for patients with serious diseases such as oncology, autoimmune and inflammatory diseases. MGDs are small molecule protein degraders that employ the body’s natural mechanisms to selectively eliminate therapeutically relevant proteins. The Company’s QuEEN™ (Quantitative and Engineered Elimination of Neosubstrates) platform enables it to rapidly identify protein targets and design highly selective degraders by combining diverse libraries of proprietary MGDs with in-house proteomics, structural biology, AI/machine learning, and computational chemistry capabilities.
Read More
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."
About BenevolentAI
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.
About ALS
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.
Read More
INDUSTRIAL IMPACT, MEDICAL
prnewswire | April 04, 2023
Sensible Biotechnologies, an early stage biotechnology company, today announced a partnership to develop an in vivo microbial mRNA manufacturing platform with Ginkgo Bioworks which is building the leading platform for cell programming and biosecurity.
Today, the vast majority of mRNA used in vaccines, therapeutics and other applications is produced by in vitro transcription (IVT), a cell-free process, which is driven by purified enzymes. In conventional mRNA manufacturing, production runs are typically limited in reaction volume size, and the resulting mRNA needs to go through expensive purification processes to eliminate potentially harmful byproducts like dsRNA, which can cause adverse immune responses in patients. Moreover, it is difficult to produce high yields of certain kinds of mRNA in an IVT reaction. By contrast, the in vivo mRNA manufacturing method Sensible and Ginkgo are working to develop is designed to scale to upwards of 100,000L, with the goal of superior quality mRNA over traditional IVT, allowing for the production of mRNA molecules with increased length and expanding the potential of the mRNA platform to novel therapeutic modalities.
"mRNA technology has a potential to bring many life-saving therapeutics and vaccines, but its current, cell-free production represents one of the major bottlenecks. In vivo mRNA manufacturing could enable scalable mRNA manufacturing, which has long relied on production methods that face quality control challenges and are inherently difficult to scale," said Miroslav Gasparek, CEO at Sensible. "By working with Ginkgo, we aim to create a scalable commercial-grade manufacturing platform that produces mRNA of higher quality than is possible through in vitro expression and enable the advent of novel mRNA medicines."
v"As the market for mRNA continues to expand, biopharma companies are looking for more efficient and scalable production platforms to produce high-quality mRNA," said Austin Che, co-founder and Head of Strategy at Ginkgo Bioworks. "As Ginkgo deepens its commitment to the emerging field of nucleic acid therapeutics, we are excited to work with the team at Sensible Biotechnologies to optimize their microbial mRNA production platform with the goal of unleashing a new generation of mRNA products."
This partnership with Ginkgo builds on Ginkgo's existing pipeline of mRNA-related programs. Ginkgo worked with Moderna to support process optimization for raw materials used to make mRNA vaccines. Furthermore, through its partnership with Aldevron, Ginkgo optimized the production of vaccinia capping enzyme, an important component often required to manufacture mRNA vaccines and therapeutics. Most recently, Ginkgo announced the acquisition of Circularis, a biotechnology company with a proprietary circular RNA and promoter screening platform, and is actively engaged in improving circular RNA efficacy and manufacturing yields.
About Ginkgo Bioworks
Ginkgo Bioworks is the leading horizontal platform for cell programming, providing flexible, end-to-end services that solve challenges for organizations across diverse markets, from food and agriculture to pharmaceuticals to industrial and specialty chemicals. Ginkgo's biosecurity and public health unit, Concentric by Ginkgo, is building global infrastructure for biosecurity to empower governments, communities, and public health leaders to prevent, detect and respond to a wide variety of biological threats.
Read More