Drug delivery system would allow tuberculosis patients to switch from daily to monthly doses

Medical Xpress | March 13, 2019

Tuberculosis is one of the world's deadliest infectious diseases: One-third of the world's population is infected with TB, and more than 1 million people die from the disease every year. One reason TB is so pervasive is that treatment requires a six-month course of daily antibiotics, which is difficult for about half of all patients to adhere to, especially in rural areas with limited access to medical facilities. To help overcome that, a team of researchers led by MIT has devised a new way to deliver antibiotics, which they hope will make it easier to cure more patients and reduce health care costs. Using this new approach, a coiled wire loaded with antibiotics is inserted into the patient's stomach through a nasogastric tube. Once in the stomach, the device slowly releases antibiotics over one month, eliminating the need for patients to take pills every day.

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Our Accounting and Finance team has published an overview of the accounting and finance challenges facing the Biopharmaceutical industry and interrelated Biotechnology organizations.

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Our Accounting and Finance team has published an overview of the accounting and finance challenges facing the Biopharmaceutical industry and interrelated Biotechnology organizations.

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INDUSTRIAL IMPACT

Laronde Attracts $440M in First External Financing to Further Advance Endless RNA™ Platform

Laronde | August 30, 2021

Laronde, the company pioneering Endless RNA™ a novel, engineered form of RNA that can be programmed to express therapeutic proteins inside the body, today announced that it has raised $440 Million in a Series B Financing to advance the development of its eRNA platform and a broad pipeline of programs across a number of therapeutic categories. The financing round included the company's founder, Flagship Pioneering, along with funds and accounts advised by T. Rowe Price Associates, Inc., Invus, Canada Pension Plan Investment Board (CPP Investments), Fidelity Management & Research Company, funds and accounts managed by BlackRock, and Federated Hermes Kaufmann Funds, among others. "Endless RNA represents a whole new approach to making medicines and treating disease," said Diego Miralles, M.D., Chief Executive Officer of Laronde and CEO-Partner at Flagship Pioneering. "Laronde is creating a new class of drugs that can be programmed to persistently express proteins in the body, is redosable, and can be administered through simple delivery mechanisms, resulting in highly tunable protein levels. The therapeutic possibilities enabled by eRNA are vast with the potential to greatly improve global human health. Having assembled such a knowledgeable and committed group of investors gives us the ability to not only advance this powerful technology platform but also build a transformative company to support our bold vision." "Over the past few years, we have seen how programmable platforms like translatable mRNA can rapidly bring forward new medicines," said Avak Kahvejian, Ph.D., Co-Founder and Board Member of Laronde, and General Partner, Flagship Pioneering. "eRNA solves many of the limitations of other drug modalities and unlocks new opportunities for creating first- and best-in-class therapeutics. Since launch, we continue to advance the science of eRNA and have been able to precisely demonstrate a controlled expression of secreted peptides, proteins, antibodies, and vaccine constructs in vivo through the use of an array of delivery options, including subcutaneous administration. Given the programmability of the platform and its unique pharmacology, we anticipate accelerated development timelines and a higher rate of program success than traditional biotech programs." "The field of biotech is at the leading edge of technological progress, challenging us to think beyond incremental advances and to take big leaps – Endless RNA is one of those big leaps," said Noubar Afeyan, Ph.D., Co-founder and Chairman of the Board of Laronde and Founder and Chief Executive Officer of Flagship Pioneering. "We are delighted that investors well familiar with the power and potential of Flagship's bioplatforms are fueling Laronde as we scale the company and the platform to realize the potential of this powerful new class of medicines." About Endless RNA™ Ribonucleic Acids, or RNAs, play a significant role in biology. Among their functions, they provide the "code" that instructs cells to produce or regulate proteins, the building blocks of life and important regulators of disease processes. Because RNAs provide coded instructions to cells, they can be programmed to produce specific effects. This approach has been validated by the successful development of several important RNA-based medicines, but RNA's potential as a drug development modality has just begun. Endless RNA™, or eRNA, was invented at Flagship Labs and is a new class of synthetic, closed-loop RNA. Because eRNA has no free ends, it is not recognized by the immune system and is very stable, enabling a long duration of protein expression. In addition, eRNA can serve protein-coding and non-protein-coding functions, and its protein translation capabilities are completely modular switching an eRNA "protein sequence cassette" enables the expression of a different protein or multiple proteins that can be tuned as needed on an application-by-application basis. About Laronde Laronde is pioneering a platform that offers a completely novel way of modulating human biology. Endless RNA™, invented at Flagship Labs, is a uniquely engineered RNA that can be programmed to express diverse proteins inside the body. It is persistent, non-immunogenic, allows for repeat dosing, and offers flexibility in formulation and delivery. Laronde was founded in 2017 by Flagship Labs, the innovation foundry of Flagship Pioneering. The company is rapidly scaling to support the parallel development of multiple programs across many disease areas. About Flagship Pioneering Flagship Pioneering conceives, creates, resources, and develops first-in-category bioplatform companies to transform human health and sustainability. Since its launch in 2000, the firm has, through its Flagship Labs unit, applied its unique hypothesis-driven innovation process to originate and foster more than 100 scientific ventures, resulting in over $130 billion in aggregate value. To date, Flagship has deployed over $2.5 billion in capital toward the founding and growth of its pioneering companies alongside more than $19 billion of follow-on investments from other institutions. The current Flagship ecosystem comprises 41 transformative companies, including Denali Therapeutics , Indigo Ag, Moderna , Rubius Therapeutics , Sana Biotechnology , Seres Therapeutics , and Valo Health.

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RESEARCH

Chemistry42, an AI System from Insilico, has been Incorporated into UCB's Drug Discovery Programmes

Insilico Medicine | March 15, 2021

Insilico Medicine, an AI drug discovery company, reported that UCB will incorporate Insilico's Chemistry42™ into UCB's inward drug discovery pipeline. UCB's initial appropriation of Insilico Medicine's restrictive innovation will give UCB's researchers the capacity to plan novel hit compounds that fulfill various predefined boundaries quickly and smooth out lead enhancement. With the Chemistry42™ platform, UCB researchers will likewise lessen the endeavors and expenses commonly connected with the plan, testing and commercialization of new drugs. Chemistry42™ v1.0 will be modified and conveyed on UCB's cloud-based supercomputing infrastructure. Chemistry42™ is an adaptable, easy to understand programming platform that incorporates man-made brainpower (AI) strategies with the fields of therapeutic and computational science for the plan of novel little atoms with client characterized druglike physicochemical properties. The platform is an adaptable conveyed web application equipped for running various assignments in equal very quickly. Container coordination and work process the executives consider unsurprising equipment freethinker asset distribution and the execution on one or the other cloud or neighborhood HPC infrastructures. "UCB is one of the leading companies in small molecule drug discovery. It was their insight and approach at the forefront of this science that encouraged us to move into AI-powered chemistry over five years ago and it gives me great pleasure to see UCB among the launch partners of our Chemistry42 operating system," said Alex Zhavoronkov, Ph.D., CEO of Insilico Medicine. "Insilico Medicine has been very responsive in this quickly developing area of science and we are delighted to be one of the launch partners for Chemistry42," Jiye Shi, Global Head of Computer-Aided Drug Design at UCB said. "Our hope is that this platform will further enhance the digital transformation of our drug discovery capabilities for the benefit of patients worldwide." About Insilico Medicine Insilico Medicine develops software that leverages generative models, reinforcement learning (RL), and other modern machine learning techniques to generate new molecular structures with specific properties. Insilico Medicine also develops software for the generation of synthetic biological data, target identification, and the prediction of clinical trial outcomes. The company integrates two business models: providing AI-powered drug discovery services and software through its Pharma.AI platform (www.insilico.com/platform/) and developing its pipeline of preclinical programs. The preclinical program is the result of pursuing novel drug targets and novel molecules discovered through its platforms. Since its inception in 2014, Insilico Medicine has raised over $52 million and received multiple industry awards. About UCB UCB, Brussels, Belgium is a global biopharmaceutical company focused on the discovery and development of innovative medicines and solutions to transform the lives of people living with severe diseases of the immune system or of the central nervous system.

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CELL AND GENE THERAPY

Biotech Startup Mekonos raises oversubscribed $25 million round to overcome pharma industry's cell & gene therapy delivery hurdles

Mekonos, Inc. | November 10, 2021

Mekonos, a biotech company building the future of cell therapies on a chip, has raised $25M in an oversubscribed Series A round co-led by Reimagined Ventures, Fiscus Ventures, and PEAK6 Strategic Capital, a division of PEAK6 Investments. New institutional and strategic investors, including Section 32, Sands Capital, TDK Ventures, and the venture arm of Debiopharm, and previous investors, including Novartis Pharma AG (dRx Capital) and Elementum Ventures, also joined in the funding round that will support Mekonos' next stages in product development and commercialization. Mekonos has developed a novel chip technology platform that supports ex-vivo delivery of cell payloads at the single cell level. The platform allows for multi-payload physical delivery – multiplexing – into cells with repeatable and localized dose control for precise and scalable cell engineering. Demonstration pilot projects with top pharmaceutical and research labs have shown outstanding cell viability and uptake in delivery of CRISPR and other payloads into immune cells, stem cells, and other fragile cell types. "Mekonos' novel technology is solving a critical pain point in an area of cell and gene therapy that hasn't seen significant innovation in 20+ years," said Neil Tiwari, who is joining Mekonos' Board of Directors to represent the lead investors. "Mekonos has an exceptional leadership team equipped to further the impact of its technology and partnerships. The company is well-positioned to play a lead role in the significant and fast-growing addressable market for cell and gene therapy solutions that deliver accessible personalized medicine." "Mekonos is bringing truly disruptive cell engineering technology to reality. This investment aligns well with our aim to support the development of novel drug delivery systems and further explore the possibilities that digital platforms can bring to science and ultimately patients. We look forward to observing firsthand the improvements that Mekonos' plateform can bring to cell and gene therapeutics," Lambert Potin, Partnering Associate, Search and Evaluation, Debiopharm Mekonos will use the new capital to expand its MEMS, Microfluidics, Biology, and Business Development functions in order to advance its proprietary, integrated system-on-a-chip (SoC) for targeted ex vivo gene engineering. Jake Lesnik, previously an executive at System Biosciences, has also joined as VP of Business Development to expand partnerships and drive commercial strategy for Mekonos' market-leading cell engineering and therapeutics development platform. "announcement is a clear signal that Mekonos is building critical infrastructure to unlock personalized medicine at scale and enable cell engineering across life science verticals. We are proud and humbled to work with top tier venture and strategic investors, and to exceed our initial goals for this fundraising round," said Anil Narasimha, CEO and Co-founder of Mekonos. "We are excited to partner with leading investors who share our vision for developing a new era of cell engineering that can simplify individualized treatments across disease spaces." About Mekonos, Inc. Mekonos is an enabling technology company transforming synthetic biology and personalized medicine. The company's SoC merges innovations in MEMS, microfluidics, and chemistry for controlled and individualized molecular delivery in cells at scale. The company is headquartered in San Francisco, is backed by leading investors in both healthcare and technology, and is an alum of Berkeley Launch, Berkeley SkyDeck, Creative Destruction Lab, and named a 2018 Fierce 15 startup.

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