Nanoparticles for RNA Vaccine Delivery Help Stop Melanoma Tumor Growth

Medgadget | October 01, 2019

RNA vaccines are a promising way to harness the immune system to fight cancers and infections. Some are currently in clinical trials, but the technology is still in its early stages. A major impediment to the effectiveness of RNA vaccines is the difficulty in getting them into the correct immune cells to produce the desired proteins and guaranteeing that the immune system will respond with sufficient force. To help with this, chemists at MIT have created a range of lipid nanoparticles that can be used as delivery vesicles for RNA vaccines. These nanoparticles have already been shown to improve target protein production and increase the effectiveness of the immune system in fighting melanoma tumors in mice. “One of the key discoveries of this paper is that you can build RNA delivery lipids that can also activate the immune system in important ways,” said Daniel Anderson, the senior author of the study, in an MIT press release. RNA vaccines have to penetrate antigen-presenting cells to induce them to produce a protein that will signal T cells and other immune cells to attack a target cell or tissue involved in disease. The MIT researchers synthesized thousands of lipid nanoparticles and picked out ones that had the desired chemical features, specifically a cyclic structure at the tip of the nanoparticles that seems to activate the stimulator of interferon genes (STING) signaling pathway. When it is active, STING makes cells produce cytokines that make T cells do their signature tricks.

Spotlight

THE WORLD'S MOST COMMON DISEASES. The challenge is unprecedented -- a 25% reduction by 2025 in premature deaths from noncommunicable diseases.More than 14 million people between the ages of 30 and 70 die each year from non communicable diseases (NCDs).Eighty five per cent live in developing countries.

Spotlight

THE WORLD'S MOST COMMON DISEASES. The challenge is unprecedented -- a 25% reduction by 2025 in premature deaths from noncommunicable diseases.More than 14 million people between the ages of 30 and 70 die each year from non communicable diseases (NCDs).Eighty five per cent live in developing countries.

Related News

PHARMA TECH

BlackRock and QLS Advisors Partner Through BlackRock Systematic to Invest in Biomedical Innovation

BlackRock and QLS Advisors | July 19, 2022

BlackRock and QLS Advisors LLC, a life sciences technology and advisory firm, have announced a partnership through BlackRock Systematic to develop data-driven investment solutions to help accelerate biomedical innovation. The partnership will leverage the firms’ combined Research and Development and investment experience, directing funding toward public and private companies that have the potential to create transformative therapies while seeking to earn attractive investment returns for clients. Biomedicine is at an inflection point: breakthrough therapies—with the potential to extend longevity and improve quality of life—are emerging at an ever-increasing pace. Funding these therapeutic development programs has the potential to deliver attractive investment returns, but as biomedicine has grown more complex, so have the risks to investors. QLS has developed proprietary technology for estimating the probability of success of clinical trials, a key driver of financial performance, and systematically incorporating these forecasts into fundamental valuation models and portfolio construction algorithms. Their approach applies machine learning techniques to a multitude of predictive factors including therapeutic characteristics, clinical trial design features, disease-specific characteristics, and drug company track records. This methodology, coupled with BlackRock Systematic’s global platform, proprietary analytics, and more than 35 years1 of data-driven investment experience, presents a differentiated approach to investing in life sciences companies. “BlackRock’s partnership with QLS applies sophisticated quantitative investment technologies to the intersection of finance, academia, and life sciences. QLS brings an industry-leading approach to investing in life sciences that complements the work that BlackRock Systematic is doing in our actively managed portfolios. We expect this will create compelling, differentiated solutions that can deliver attractive returns for our clients while providing funding for life-changing therapies.” Rich Kushel, Head of the Portfolio Management Group BlackRock Andrew Lo, QLS Co-founder and Professor of Finance at the MIT Sloan School of Management commented: “We founded QLS to improve the way financial resources are allocated to emerging therapies, with the goal of lowering the cost of capital in this important sector. With BlackRock, we gain a partner who is a recognized leader in data-driven investing and shares our passion for delivering investment returns with positive outcomes for society. We’re thrilled and honored to join forces with a firm that has such a strong and trusted global brand, broad network, and access to world-class investment resources.” BlackRock and QLS have also assembled a Scientific Advisory Council comprising renowned academics, medical doctors, and inventors. The Council will support sourcing, due diligence, and value creation through the members’ vast networks and decades of experience as industry leaders in their respective fields. The Council is expected to include Dr. Robert Langer, Institute Professor, MIT Department of Chemical Engineering, and founder of over 40 biotechnology companies including Moderna. Ms. Lita Nelsen, retired director of the MIT Technology Licensing Office; now consultant in technology licensing. Dr. Larry Norton, Senior Vice President, Norna S. Sarofim Chair in Clinical Oncology and Medical Director of the Evelyn H. Lauder Breast Center at Memorial Sloan Kettering Cancer Center, Past President of the American Society of Clinical Oncology, ASCO David Karnofsky Awardee, FASCO, FAACR. Dr. Richard H. Scheller, Chairman of Research and Development BridgeBio. Board of Directors BridgeBio, Alector, 23andMe, DICE and others. Former Executive Vice President Research and Early Development Genentech and former HHMI Investigator and Professor Stanford University Medical School. Dr. Phillip A. Sharp, Institute Professor, MIT Department of Biology, geneticist and molecular biologist who co-discovered RNA splicing, co-founded Biogen and Alnylam, and the recipient of the 1993 Nobel Prize in Physiology or Medicine. About BlackRock Systematic BlackRock’s systematic investment platform brings together BlackRock’s community of systematic investors across equity, fixed income, and factor-based strategies. Systematic portfolios combine cutting-edge technology, scientific research, and human insight in the relentless pursuit of investment performance. With over 35 years of experience and nearly 200 professionals across the globe managing over US$200 bn in client assets as of March 31, 2022, BlackRock Systematic partners with clients to deliver quantitative alpha-seeking and factor-based investment solutions for the whole portfolio. About QLS Advisors LLC QLS Advisors LLC is a life sciences technology and advisory company based in Cambridge, MA, dedicated to fostering biomedical innovation. QLS employs a unique blend of fundamental and quantitative tools to help clients manage risk, assess reward, and develop investment and financing strategies for portfolios of healthcare-related assets.

Read More

PHARMA TECH

Cambrex Completes First Phase of $30 Million Capacity Expansion Project in High Point, North Carolina

Cambrex | September 05, 2022

Cambrex today announced the completion of the first phase of its $30 million investment in its small molecule active pharmaceutical ingredient manufacturing facility in High Point, North Carolina. The newly constructed space adds analytical and chemical development laboratories totaling 30,000 square feet and provides future workspace for 85 analytical and chemical development scientists. These laboratories will support the development of APIs to be manufactured in the facility's current clinical manufacturing area, as well as the future expanded clinical manufacturing and commercial manufacturing suites. "With the growing number of therapies in clinical development and trend toward more targeted therapeutics, including orphan drugs, this expansion is preparing Cambrex to support the growing demand for small-scale API manufacturing. Our expansion goes beyond providing capacity – it provides state-of-the-art technology and laboratory equipment, an energy efficient infrastructure, and an ideal space for our scientific experts to work side-by-side with our clients." Tom Loewald, CEO, Cambrex Phase 2 of the project is ongoing and will approximately double the facility's manufacturing capacity with the addition of clinical and commercial manufacturing suites with reactors up to 2,000 liters. The new commercial area will provide an ideal scale for the manufacturing of orphan drugs and niche therapies, while larger volume products developed at the facility can be manufactured commercially at one of Cambrex's larger scale facilities in Iowa and Sweden. This expansion follows Cambrex's 2021 investment in the Cambrex High Point continuous flow R&D center of excellence, including expanded capabilities for continuous flow process development, manufacturing, and scale up. Cambrex continues to expand its capabilities and capacity across its North American and European network to meet the growing demand for outsourced product development and manufacturing services. About Cambrex Cambrex is a leading global contract development and manufacturing organization that provides drug substance, drug product, and analytical services across the entire drug lifecycle. With over 40 years of experience and a growing team of over 2,300 experts servicing global clients from North America and Europe, Cambrex is a trusted partner in branded and generic markets for API and finished dosage form development and manufacturing. Cambrex offers a range of specialized drug substance technologies and capabilities, including biocatalysis, continuous flow, controlled substances, solid-state science, material characterization, stability storage, and highly potent APIs. In addition, Cambrex can support conventional dosage forms, including oral solids, semi-solids, and liquids, and has the expertise to manufacture specialty dosage forms such as modified-release, fixed-dose combination, pediatric, bi-layer tablets, stick packs, topicals, controlled substances, sterile, and non-sterile ointments.

Read More

PHARMA TECH

WuXi ATU Announces Licensing Agreement with Janssen for TESSA™ Technology

WuXi AppTec | August 12, 2022

WuXi Advanced Therapies a wholly owned subsidiary of WuXi AppTec, announced a licensing agreement with Janssen Biotech, Inc., one of the Janssen Pharmaceutical Companies of Johnson & Johnson. Under this agreement, WuXi ATU will license to Janssen its TESSA™ technology, a high-performance system that can produce 10 times more adeno-associated viral vectors than traditional AAV manufacturing systems. Janssen will also have access to work on WuXi ATU's proprietary clonal suspension HEK293 cell line. This agreement was facilitated by Johnson & Johnson Innovation. AAV vectors are commonly used for the delivery of gene therapies to patients due to their ability to transduce numerous cell and tissue types. WuXi ATU's TESSA™ technology responds to industry demand for large scale AAV manufacturing by producing higher quality AAV particles more efficiently. WuXi ATU has also successfully scaled up the TESSA™ technology to 200L; this achieved a 10-fold higher yield and a significantly higher percentage of full AAV capsids, greatly reducing overall AAV production costs compared to traditional plasmid-based AAV production systems. "We are honored that Janssen selected WuXi ATU's TESSA™ technology. We remain committed to improving the TESSA™ platform to produce faster and more cost-effective AAV products for patients." Dr. David Chang, Chief Executive Officer of WuXi Advanced Therapies As a Contract Testing, Development and Manufacturing Organization with global operations, WuXi ATU will continue to enhance its capability and capacity to help customers develop and deliver life-changing cell and gene therapies faster for patients in need. About WuXi Advanced Therapies (WuXi ATU) As the advanced therapies business unit of WuXi AppTec, WuXi Advanced Therapies is a Contract Testing, Development and Manufacturing Organization (CTDMO) that offers integrated platforms to transform the discovery, development, testing, manufacturing, and commercialization of cell and gene therapies. Our services and solutions accelerate time to market and support customer programs around the world. About WuXi AppTec As a global company with operations across Asia, Europe, and North America, WuXi AppTec provides a broad portfolio of R&D and manufacturing services that enable global pharmaceutical and healthcare industry to advance discoveries and deliver groundbreaking treatments to patients. Through its unique business models, WuXi AppTec's integrated, end-to-end services include chemistry drug CRDMO (Contract Research, Development and Manufacturing Organization), biology discovery, preclinical testing and clinical research services, cell and gene therapies CTDMO (Contract Testing, Development and Manufacturing Organization), helping customers improve the productivity of advancing healthcare products through cost-effective and efficient solutions. WuXi AppTec received AA ESG rating from MSCI in 2021 and its open-access platform is enabling more than 5,850 collaborators from over 30 countries to improve the health of those in need – and to realize the vision that "every drug can be made and every disease can be treated."

Read More