Cell and Gene Therapy, Medical
prnewswire | August 25, 2023
Synthego, a leading provider of industrialized genome editing solutions that accelerate discovery to clinic journey, and bit.bio, the company coding human cells for novel cures, today announced a strategic partnership to build a platform centered on the implementation of synthetic circuitry in cells for therapeutic application.
bit.bio is building a pipeline of cell therapies focused on areas of unmet clinical need. The partnership will apply Synthego's expertise in gene editing to bit.bio's opti-oxTM precision cell programming technology, with the aim of discovering and developing advanced genetic engineering solutions for cell therapies.
Synthego will rapidly design, build, test and deliver an end-to-end optimized multiplexed genetic editing strategy. The implementation of the multiplexed opti-ox engineering approach will reduce the number of engineering steps required, with the potential of shortening clinical cell engineering and development timelines by up to 10 months.
"bit.bio is the leading synthetic biology company focussed on human cells. We are thrilled to deepen our partnership with bit.bio to facilitate a leap forward in the development of synthetic biology-based cell therapies," said Paul Dabrowski, CEO at Synthego. "By coordinating our respective technologies, expertise and resources, we will unlock new opportunities for addressing critical challenges in the field by advancing the R&D cycle of innovative cell therapies."
"Synthego are experts when it comes to industrialized cell engineering," stated Mark Kotter, CEO at bit.bio. "Teaming up with Synthego will boost our genetic engineering capabilities. We expect efficiency gains across bit.bio's platform which will allow us and our partners to deliver transformative therapies more rapidly to patients in need."
Synthego is a genome engineering company that enables the acceleration of life science research and development in the pursuit of improved human health. The company leverages machine learning, automation, and gene editing to build platforms and products for science at scale. With its foundations in engineering disciplines, the company's platform technologies vertically integrate proprietary hardware, software, bioinformatics, chemistries, and molecular biology to advance basic research, target validation, and clinical trials. With its technologies cited in hundreds of peer-reviewed publications and utilized by thousands of commercial and academic researchers and therapeutic drug developers, Synthego is at the forefront of innovation, enabling the next generation of medicines by delivering genome editing at an unprecedented scale.
bit.bio is a synthetic biology company focused on human cells that is advancing medicine (UN SDG9) and enabling curative treatments (UN SDG3). The company does this by industrializing the manufacture of human cells and making them more accessible. The company was spun out of the University of Cambridge in 2016 and has since raised approximately $200m from investors such as Arch Ventures, Foresite Capital, Milky Way, Charles River Laboratories, National Resilience, Tencent, Verition Fund and Puhua Capital.
bit.bio's opti-ox™ precision cell programming and manufacturing technology enables conversion of induced pluripotent stem cells (iPSCs) into any desired human cell type in a single step. This can be achieved within days and at industrial scale, while maintaining exceptional purity and unparalleled consistency.
Our discovery platform extends this approach to any desired cell type by identifying the transcription factor combinations that define cell states (including identity, cell subtype identity, maturity) using high throughput screens and advanced data analysis. We believe that opti-ox can revolutionize regenerative medicine similarly to how CRISPR is unlocking gene therapy.
bit.bio's cell therapy pipeline is focused on serious diseases that currently lack effective treatments. Our preclinical research areas include liver, immunology and metabolic disease, and we have a collaboration with BlueRock Therapeutics (Bayer AG) to create therapeutics for autoimmune and inflammatory disorders. In addition, our extensive ioCells™ research cell product portfolio, which includes wild type and disease model cells, is opening up new possibilities for studying human biology and developing new medicines in both research and high throughput and high content drug discovery.
Businesswire | June 29, 2023
Alloy Therapeutics, a biotechnology ecosystem company, announced the launch of its novel AntiClastic™ Antisense Oligonucleotide (AntiClastic ASO) platform, which employs optimally created spatial conformation nucleic acids that further enhance the drug-like properties of antisense. Alloy has exclusively licensed IP underlying the AntiClastic ASO platform from Sudhir Agrawal’s Arnay Sciences LLC.
Antisense therapeutics hold promise for reaching many undruggable intracellular targets at the nucleic acid level but have been limited due to therapeutic index and delivery challenges. Alloy’s AntiClastic ASO platform combines improvements in the primary sequence with a novel spatial conformation of nucleic acid drugs. The resultant AntiClastic ASO candidates have improved potency, promote the delivery of antisense to target RNA, and minimize interactions with unintended RNA targets and the inflammatory responses—for a superior therapeutic index. This platform is broadly applicable to treat diseases by targeting genes expressed in the central nervous system (CNS), liver, muscles, ocular, and more.
“The AntiClastic format has the potential to transform the development of antisense therapeutics,” said Agrawal. “Alloy’s model of making pre-competitive discovery technologies widely available to drug developers through accessible terms and an ecosystem mindset means this technology can reach the widest possible audience of patients—rather than staying locked up under one company and accessible to a limited set of therapeutic programs.”
Drug developers can access the novel AntiClastic ASO format by collaborating on their desired target or by providing existing antisense sequences to be converted into AntiClastic molecules. All collaborative campaigns receive Alloy’s discovery support and preclinical data and leverage Alloy’s flexible research and partnership terms based on a shared success model.
In launching the AntiClastic ASO platform, Alloy is expanding into its third biologic modality, genetic medicines. The company started by making its ATX-Gx™ transgenic humanized mice platform for antibody discovery widely available to the global scientific community through campaigns with Alloy’s Antibody Discovery Services or for use in partner’s respective labs. The platform has since accrued more than 150 partners and has provided the framework for how Alloy makes pre-competitive technologies available to drug developers everywhere. It launched Keyway™ TCR Discovery to democratize technologies and expertise for developing soluble TCR therapeutics and TCR mimics against intracellular targets. Alloy’s genetic medicines group is led by Vinod Vathipadiekal, PhD, who has held research leadership roles across RNA and nucleic acid therapeutics programs at biotech and pharma companies. Over the last year, Alloy has generated promising data on applying the AntiClastic technology to multiple antisense drug candidates against many relevant targets, enabling discovery collaborations with partners.
“Alloy is executing successfully against an ambitious roadmap in applying the AntiClastic platform and has developed a powerful model of collaboration and innovation with premier inventors like Sudhir,” said Alloy President Piotr Bobrowicz, PhD. “In offering an advanced, novel set of discovery capabilities across modalities, we aim to provide the global scientific community with the tools and expertise needed to advance the best drugs, regardless of format.”
In conjunction with the launch of its AntiClastic ASO platform, Alloy is also revealing its genetic medicines scientific advisory board (SAB), a group of leading thinkers across immunology, RNA- and nucleic acid-based therapeutics, rare disease, and bioinformatics who will guide further development of the platform
Douglas Golenbock, MD—Neil and Margery Blacklow Chair in Infectious Diseases and Immunology at UMass Chan Medical School—and a pioneering researcher in innate immunity, Toll-like receptor (TLR) biology, and the inflammasome.
Frank Slack, PhD—Shields Warren Mallinckrodt Professor, Department of Pathology, and Director of the Institute for RNA Medicine at Beth Israel Deaconess Medical Center—who has spent decades researching and re-thinking small RNA gene regulation capabilities.
Malcolm MacCoss, PhD—Visiting Professor of Chemistry for Medicine at Oxford University—with decades of expertise in novel chemistries for nucleoside and nucleic acid-based therapeutic approaches through research roles at Merck and Schering Plough, as well as SAB or consultant positions at Idera, Gilead, UCB, and Sitryx, among others.
Matt Might, PhD—Professor and Director of the Hugh Kaul Precision Medicine Institute at the University of Alabama at Birmingham—whose expertise spans precision medicine leveraging novel bioinformatic techniques and developing therapies for rare diseases.
“We are grateful that the best minds and innovators in genetic and precision medicine and the disease areas that can benefit from it have coalesced around this exciting platform and new modality at Alloy,” said Errik Anderson, Alloy Therapeutics CEO and Founder. “Our model of co-creating pre-competitive technologies and making them as widely available as possible means more patients will be able to benefit from new, potent antisense therapeutics for previously intractable conditions.”
About Alloy Therapeutics
Alloy Therapeutics is a biotechnology ecosystem company empowering the global scientific community to make better medicines together. Through a community of partners across academia, biotech, and the largest biopharma, Alloy democratizes access to pre-competitive tools, technologies, services, and company creation capabilities that are foundational for discovering and developing therapeutic biologics across six modalities: antibodies, TCRs, genetic medicines, peptides, cell therapies, and drug delivery. Partners may access all current and future technologies through a discovery service relationship or for a flat annual fee through Alloy’s Innovation Subscriptions offering. As a reflection of Alloy’s relentless commitment to the scientific community, Alloy reinvests 100% of its revenue in innovation and access to innovation.
About AntiClastic™ Antisense Oligonucleotides
AntiClastic™ Antisense Oligonucleotides (AntiClastic ASOs) are a novel therapeutic format exclusively available through Alloy Therapeutics collaborations that are designed to overcome potency and therapeutic index challenges that have historically limited the promise of antisense drugs. Sudhir Agrawal invented the core IP, which combines improvements in the primary sequence with a novel spatial conformation of nucleic acid drugs to promote the delivery of antisense to target RNA, mitigate the inflammatory response, and improve a drug’s therapeutic index. The resultant drug candidates have shown a significant increase in potency compared to gapmer antisense formats.
Businesswire | August 22, 2023
NanoString Technologies, Inc. a leading provider of life science tools for discovery and translational research,
The seminal research led by Alexander Loupy, MD, Ph.D., in cooperation with a global medical team, unravels the complex molecular characteristics of antibody-mediated rejection following the first and second-ever pig-to-human kidney transplants that occurred in 2021 at NYU Langone Transplant Institute. In an accompanying Lancet commentary, scientists praised the team “for laying the foundation for deeper assessment of xenoimmunology and establishing immunological research pathways to advance xenotransplantation.” They also commented on the importance of “state-of-the-art spatial techniques” to decipher transcriptional immune activation.
“We gained vital data and, for the first time ever, we were able to see what is happening immunologically in defined regions of the tissue,” said Dr. Loupy. “This data, enabled by NanoString’s platforms and an Allo - Xeno gene expression repository, allowed us to understand both the similarities and the novel aspects underpinning organ rejection in xenograft transplantation compared to human organ transplantation. NanoString’s nCounter and GeoMx instruments harmonized to create a holistic view of the immune response.” Researchers from the Paris Institute for Transplantation and Organ Regeneration, University of Paris, France’s National Institute of Health and Medical Research contributed to the study.
Xenotransplantation, the process of transplanting organs from animals to humans, is a crucial area of research dedicated to addressing the profound shortage of healthy human organs available for transplant patients. The groundbreaking experimental transplant surgeries performed by Dr. Robert Montgomery at NYU since 2021 involve patients in a state of brain death. Their families and ethical committees have consented to artificially maintain blood circulation during the transplants to advance scientific knowledge on xenotransplantation with the hope of offering more healthy organs for future patients.
The research team, including co-first authors of The Lancet study, Dr. Valentin Goutaudier and Dr. Alessia Giarraputto characterized the human immune response to the porcine kidney using bulk tissue transcriptome profiling on the nCounter Analysis System. Scientists used the nCounter Human Organ Transplant Panel, a process NanoString developed with the Banff International Classification Consortium. It is used by nearly one hundred global research institutions. The panel was adapted to xenotransplants by using bioinformatics-based pig and human gene sequences with homologies alignment. This analysis revealed a molecular architecture of antibody-mediated rejection, including interferon-gamma response, endothelial activation, macrophage activation, and injury repair response in the xenografts.
The GeoMx Digital Spatial Profiler was used next to isolate the immune response to specific regions of the kidney. The GeoMx Whole Transcriptome Assay revealed that antibody-mediated injury was mainly located in the glomeruli of xenografts, with significant enrichment of transcripts associated with monocytes, macrophages, neutrophils, and NK cells.
Drs. Loupy and Montgomery say their findings represent a treasure trove of new information for optimizing genetically modified pig models that may be useful in developing more advanced immunosuppressive treatments for future recipients of xenografts.
“An estimated 100,000 people are waiting for organ transplants in the United States. Thousands die on the wait list. Numbers like these drive us at NanoString to provide researchers with the tools they need to find innovative solutions to solve extraordinary medical challenges,” commented Brad Gray, President and CEO of NanoString. “We congratulate Dr. Loupy and his extended team on their findings and celebrate the on-going successful studies in humans.”
About NanoString Technologies, Inc.
NanoString Technologies, is a leader in spatial biology, offers an ecosystem of innovative discovery and translational research solutions, empowering our customers to map the universe of biology. The GeoMx® Digital Spatial Profiler is a flexible and consistent solution combining the power of whole tissue imaging with gene expression and protein data for spatial whole transcriptomics and proteomics. The CosMx™ Spatial Molecular Imager is a single-cell imaging platform powered by spatial multiomics enabling researchers to map single cells in their native environments to extract deep biological insights and novel discoveries from one experiment. The AtoMx™ Spatial Informatics Platform is a cloud-based informatics solution with advanced analytics and global collaboration capabilities, enabling powerful spatial biology insights anytime, anywhere. At the foundation of our research tools is our nCounter® Analysis System, which offers a secure way to easily profile the expression of hundreds of genes, proteins, miRNAs, or copy number variations, simultaneously with high sensitivity and precision.