ASCO: With FDA review nearing, Epizyme shares updated sarcoma data

fiercebiotech | June 03, 2019

Epizyme has posted updated data from the phase 2 trial it hopes will secure tazemetostat approval in epithelioid sarcoma. The data feature a slight uptick in objective responses offset by rises in the rates of some adverse events.Massachusetts-based Epizyme filed for authorization of tazemetostat last week and arrived at the American Society of Clinical Oncology (ASCO) annual meeting armed with data it hopes will persuade the FDA to approve the EZH2 inhibitor. The ASCO data set features one more month of follow-up than the results shared by Epizyme at a previous cancer conference.

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MEDTECH

Insempra Announces Strategic Investment in Solena Materials

Insempra, | July 26, 2022

Insempra, a biology-powered company enabling businesses to make superior products in partnership with nature, today announces a strategic investment in Solena Materials Ltd a synthetic biology company. Solena, a spin-out from Imperial College London, develops synthetic proteins for high-performance clothing fibers. The investment will allow Insempra, formerly Origin.Bio, to accelerate its strategy of harnessing new technologies to advance biological production processes, creating naturally superior products to drive the regenerative revolution. Solena will be a major part of Insempra’s platform to deliver high-performance, intrinsically sustainable ingredients for a broad array of industries. Solena is using computational design to develop new classes of synthetic proteins to produce high-performance clothing fibers, which can absorb large amounts of kinetic energy. Insempra will accelerate the development and production of these synthetic proteins on an industrial scale, offering better, biobased solutions to the petrochemically sourced, non-biodegradable materials or fibers extracted from nature or animals, such as silk, currently used in the textile and clothing industries. This technology also reduces other environmental impacts such as the rise of microplastics in water bodies coming from washing petrochemically-sourced textiles. “We are hugely excited by this investment in Solena, which will help to accelerate our market-first approach to develop superior, intrinsically sustainable ingredients. We look forward to fast-tracking Solena’s development and production of their unique synthetic proteins to develop customized, high-performance fibers for a variety of applications.” Jens Klein, founder and CEO of Insempra and CEO of Solena Materials Dr James MacDonald, inventor, co-founder and CTO of Solena Materials, added: “The team and I are very excited to make this technology a huge success.” Professor Paul Freemont of Imperial College London, added: “This investment from Insempra recognizes the potential of our technology to revolutionize high-performance fabrics, and their supply chains. Together, we can harness our synthetic biology capabilities to develop, produce and manufacture a new class of superior, more sustainable fiber technology.” Insempra, a co-founder of Solena, will be the sole contributing shareholder in Solena, which is based at Imperial College’s Translation & Innovation Hub (I-HUB) in White City, London. Solena’s Board will be comprised of Dr James MacDonald of Solena, Professor Paul Freemont and Professor Milo Shaffer of Imperial College London, and Jens Klein and Andreas Heyl of Insempra. About Insempra Insempra is a biology-powered company enabling businesses to make superior products with nature. We combine bioscience and technology to grow matter for new and better products. For too long, businesses have relied on chemical industrialization processes and petrochemicals, depleting our planet’s limited resources. That’s why we are committed to drive the regenerative revolution to manufacture at scale in collaboration with nature. Led by a hand-picked team of biologists, technologists and entrepreneurs with rebel hearts, Insempra is here to create a new school of thought and collective action. The time to restore the balance between people and the planet is now. We don't wait for change. We do what needs to be done to grow a better future. About Solena Materials Solena is a protein materials design company. We use world-leading computational design, machine learning, and automation to accelerate the development of bespoke materials targeted at multiple sectors, from fashion apparel to medical textiles. Our new protein-based fibers will replace materials extracted from nature such as silk and petrochemically-derived materials, to create a new world of biodegradable, functional and sustainable smart materials for consumers, industry, and the planet.

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CELL AND GENE THERAPY

GenScript and Gladstone-UCSF Institute of Genomic Immunology Collaborate on Groundbreaking Non-Viral Cell Therapy Study

Genscript Biotech Corporation | September 23, 2022

GenScript USA Inc., the world's leading life-science research tools and services provider, is collaborating with researchers at the Gladstone-UCSF Institute of Genomic Immunology to advance the development of new, non-viral cell therapies that modify genomic sequences in the pursuit of more effective cell therapy products while limiting the cellular toxicity that is typically associated with previously available methods. A new study, published in Nature Biotechnology, details methods for achieving highly efficient non-viral knock-in using GenScript's GenExact™ single-strand DNA modified with Cas9-target sequences. This method achieved up to ˜40% knock-in efficiency in primary immune cell types. One powerful application of CRISPR/Cas genome editing technology involves the precise insertion of DNA sequences via the HDR pathway. Traditionally, researchers have relied on viral vectors to deliver DNA insertion templates used for gene therapy into cells. However, difficulties manufacturing large amounts of clinical-grade viral vectors have delayed getting cell therapies to patients. Additionally, viral vectors can insert genes at any location within the genome, leading to safety concerns. Previous research by the group at Gladstone and UCSF has shown that synthetic DNA templates can be delivered without the use of viral vectors, but high levels of double-stranded DNA can be toxic to cells, resulting in low efficiency. Efficiency can be improved using a modified version of the DNA templates that can bind to the Cas9 enzyme. However, additional work was required to improve the yield of successfully engineered cells and to make the process compatible with the manufacturing of future cell therapies. Those goals motivated the team's current study. In this study, the team tested modified GenExact ssDNA designed with Cas9-targeted sequences to determine if the gene-editing efficiency could be improved with lower toxicity. First, synthetic sgRNA was complexed with Cas9 protein to form a RNP complex. Then the RNP and ssDNA template were delivered into the cell via electroporation, enabling efficient gene editing. The team discovered that this combination of GenExact ssDNA with Cas9-targeted sequence offers up to 40% knock-in editing efficiency with minimal cellular toxicity. This approach can accelerate the development and manufacture of novel, high-yield non-viral gene therapies — and at lower cost. "We are very proud to have collaborated on this groundbreaking scale-up work by the talented team at Gladstone and UCSF. GenScript is excited for the opportunities that this high-yield cell-line engineering process will provide to our customers," said Ray Chen, Ph.D, president of GenScript Life Science Group. "We were very happy to partner with Genscript on critical experiments demonstrating high efficiency and yield of CAR knock-in cells at clinical scale. The long ssDNA produced by Genscript exceeded our expectations and helped us clearly demonstrate the potential for future therapeutic applications using these methods.", Brian Shy, MD, PhD, a former clinical fellow in the lab of Alex Marson, MD, PhD, director of the Gladstone-UCSF Institute of Genomic Immunology About GenScript Biotech Corporation GenScript Biotech Corporation is a global biotechnology group. Based on its leading gene synthesis technology, GenScript has developed four major platforms including the global cell therapy platform, the biologics contract development and manufacturing organization platform, the contract research organization platform, and the industrial synthesis product platform. GenScript was founded in New Jersey, USA in 2002 and listed on the Hong Kong Stock Exchange in 2015. GenScript's business operation spans over 100 countries and regions worldwide, with legal entities located in the USA, mainland China, Hong Kong, Japan, Singapore, the Netherlands, and Ireland. GenScript has provided premium, convenient, and reliable products and services for over 100,000 customers.

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CELL AND GENE THERAPY

Merck and Orna Therapeutics Collaborate to Advance Orna’s Next Generation of RNA Technology

Merck and Orna Therapeutics | August 17, 2022

Merck known as MSD outside the United States and Canada, and Orna Therapeutics, a biotechnology company pioneering a new investigational class of engineered circular RNA therapies, today announced a collaboration agreement to discover, develop, and commercialize multiple programs, including vaccines and therapeutics in the areas of infectious disease and oncology. Under the terms of the agreement, Merck will make an upfront payment to Orna of $150 million, which will be expensed by Merck in the third quarter of 2022 and included in non-GAAP results. In addition, Orna will be eligible to receive up to $3.5 billion in development, regulatory, and sales milestones associated with the progress of the multiple vaccine and therapeutic programs, as well as royalties on any approved products derived from the collaboration. Orna will retain rights to its oRNA-LNP technology platform and will continue to advance other wholly owned programs in areas such as oncology and genetic disease. Merck will also invest $100 million of equity in Orna’s recently completed Series B financing round. Orna’s proprietary oRNA technology creates circular RNAs from linear RNAs by self-circularization. oRNA molecules have been shown to have greater stability in vivo than linear mRNA and have the potential to produce larger quantities of therapeutic proteins inside the body. Newly synthesized oRNA molecules are more compactly packaged into custom lipid nanoparticles which Orna has engineered to target key tissues in the body. Preclinical data, including presentations at the 2022 American Society of Gene & Cell Therapy Annual Meeting, have demonstrated the potential of oRNA expression and delivery as an approach for further development in multiple areas, including vaccines and oncology therapeutics. “This broad strategic collaboration brings together Merck’s significant expertise in nucleic acid biology, clinical development, and manufacturing with Orna’s compelling circular RNA technology to explore the opportunity to develop a new generation of potential vaccines and therapeutics. We look forward to working with the talented scientific and technical teams at Orna." Fiona Marshall, senior vice president and head of discovery, preclinical and translational medicine at Merck Research Laboratories “We are thrilled to collaborate with Merck, a company committed to breakthrough science, which has recognized the potential our platform can bring to patients. Our oRNA technology plus novel delivery solutions are designed to unlock the full potential of RNA in therapeutics and vaccines,” said Tom Barnes, Chief Executive Officer of Orna. “The combined expertise of Orna and Merck aims to accelerate the development of RNA therapeutics for patients in need of better treatment options.” About Merck At Merck, known as MSD outside of the United States and Canada, we are unified around our purpose: We use the power of leading-edge science to save and improve lives around the world. For more than 130 years, we have brought hope to humanity through the development of important medicines and vaccines. We aspire to be the premier research-intensive biopharmaceutical company in the world – and today, we are at the forefront of research to deliver innovative health solutions that advance the prevention and treatment of diseases in people and animals. We foster a diverse and inclusive global workforce and operate responsibly every day to enable a safe, sustainable and healthy future for all people and communities. About Orna Therapeutics Orna Therapeutics was founded on groundbreaking research by Alex Wesselhoeft, Ph.D., and Daniel G. Anderson, Ph.D. from MIT and built by MPM Capital and BioImpact Capital, an investment management affiliate of MPM. Orna’s proprietary circular RNA (oRNA) is engineered as linear RNA that self-circularizes. By taking a line and turning it into a circle, oRNA exhibits numerous advantages over traditional linear mRNA therapies such as simplified production, increased protein expression, and a superior immunogenicity profile. With proprietary lipid nanoparticles including those from Orna’s joint venture with ReNAgade Therapeutics, an RNA delivery company, our technologies expand the possibilities of what RNA therapeutics can achieve.

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