New gene variation which causes motor neurone disease discovered in novel biological pathway

Medical Xpress | February 26, 2019

Scientists have discovered a new gene variation that causes motor neuron disease (MND) in a novel biological pathway that until now hasn't been linked with neurodegeneration. The findings for the pioneering study, conducted by the team of researchers from the Sheffield Institute for Translational Neuroscience (SITraN) and the NIHR Sheffield Biomedical Research Centre (BRC), could potentially help to identify completely new ways of treating MND which currently affects over 5,000 people in the UK. MND, also known as amyotrophic lateral sclerosis (ALS), is a devastating neurogenerative disorder that affects the nerves—motor neurones—that form the connection between the brain and the muscles. The messages from these nerves gradually stop reaching the muscles, causing them to weaken, stiffen and eventually waste. The progressive disease affects a persons' ability to walk, talk, eat and breathe.

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Bacteriophage (phage) therapy, i.e., the use of viruses that infect bacteria as antimicrobial agents, is a promising alternative to conventional antibiotics. Indeed, resistance to antibiotics has become a major public health problem after decades of extensive usage. However, one of the main questions regarding phage therapy is the possible rapid emergence of phage-resistant bacterial variants, which could impede favorable treatment outcomes.

Spotlight

Bacteriophage (phage) therapy, i.e., the use of viruses that infect bacteria as antimicrobial agents, is a promising alternative to conventional antibiotics. Indeed, resistance to antibiotics has become a major public health problem after decades of extensive usage. However, one of the main questions regarding phage therapy is the possible rapid emergence of phage-resistant bacterial variants, which could impede favorable treatment outcomes.

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INDUSTRIAL IMPACT

Ginkgo Bioworks Completes Acquisition of Zymergen

Ginkgo Bioworks | October 20, 2022

Today, Ginkgo Bioworks the leading horizontal platform for cell programming, and Zymergen announced that Ginkgo has completed its previously announced acquisition of Zymergen. The acquisition is expected to significantly enhance Ginkgo's platform by integrating strong automation and software capabilities as well as a wealth of experience across diverse biological engineering approaches. "Today marks an important step in our long-term growth as we complete the Zymergen acquisition and welcome their world-class team to Ginkgo. We are excited to integrate Zymergen's capabilities into our platform and explore new and expanded partnerships and opportunities for their diverse array of product concepts currently under development." Jason Kelly, CEO and co-founder of Ginkgo Bioworks Under the terms of the merger agreement entered into on July 24, 2022, Zymergen stockholders received, for each share of Zymergen common stock, 0.9179 shares of Ginkgo Class A common stock. Zymergen shares will no longer be traded on Nasdaq. Ginkgo Class A common stock will continue to trade on NYSE under the ticker symbol DNA. About Ginkgo Bioworks Ginkgo is building a platform to enable customers to program cells as easily as we can program computers. The company's platform is enabling biotechnology applications across diverse markets, from food and agriculture to industrial chemicals to pharmaceuticals. Ginkgo has also actively supported a number of COVID-19 response efforts, including K-12 pooled testing, vaccine manufacturing optimization and therapeutics discovery. About Zymergen Zymergen is a biotech company that designs and produces molecules, microbes and materials for diverse end markets. Zymergen partners with nature to make better products, a better way, for a better world.

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INDUSTRIAL IMPACT

MycoWorks Secures Strategic Investment from GM Ventures to Develop Fine Mycelium Materials for Sustainable Automotive Interiors

MycoWorks | October 19, 2022

Biotechnology company MycoWorks announced today its newest investor GM Ventures, the investment arm of General Motors Co. and their long-term agreement to co-develop Fine Mycelium™ materials for potential use in a range of applications within automotive design. MycoWorks' collaboration with GM marks the exploration of its entry into one of the largest end-use markets for leather and demonstrates the significant opportunity to create more sustainable materials for the automotive space. MycoWorks' breakthrough Fine Mycelium technology engineers mycelium, the infinitely renewable root structure of mushrooms, to craft a range of natural, customizable leather alternatives that match the performance of the highest quality animal leather with lower environmental impact. The company's platform has enabled a new class of premium, non-animal materials which could have the opportunity to support GM's continued progress toward its vision of a world with zero crashes, zero emissions and zero congestion. Animal-free and chrome-free, Fine Mycelium also has superior strength at low thicknesses, offering a compelling pathway to efficiency and weight reduction in car interiors while still meeting the most stringent performance, aesthetic, and environmental requirements of the automotive space. "Working with General Motors to introduce Fine Mycelium to the automotive industry is an enormous step forward in the global new materials revolution. MycoWorks's collaboration with GM is a first for us outside of the fashion industry, demonstrating the enormous potential and applications for Fine Mycelium." Matt Scullin, CEO of MycoWorks "Our strategic investment in MycoWorks aims to advance the development of sustainable automotive materials," said Wade Sheffer, managing director of GM Ventures. "This collaboration will help facilitate R&D efforts and build more sustainable alternatives for our designers." In August 2022, following an oversubscribed $125M Series C funding round, MycoWorks broke ground on its first full-scale production facility, which will enable initial mass-production volumes of several million square feet of Fine Mycelium materials per year, dramatically increase production and partnership capacity, and service Fine Mycelium to a wider range of industries and companies. About MycoWorks In 2013, co-founders Philip Ross and Sophia Wang formed MycoWorks, a San Francisco-based biomaterials company dedicated to bringing new mycelium materials to the world. MycoWorks' patented Fine Mycelium technology, an advanced manufacturing platform and breakthrough in materials science, engineers mycelium during growth to form proprietary, interlocking cellular structures for unparalleled strength and durability. About General Motors General Motors is a global company focused on advancing an all-electric future that is inclusive and accessible to all. At the heart of this strategy is the Ultium battery platform, which powers everything from mass-market to high-performance vehicles. General Motors, its subsidiaries and its joint venture entities sell vehicles under the Chevrolet, Buick, GMC, Cadillac, Baojun and Wuling brands.

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CELL AND GENE THERAPY

Eiger BioPharmaceuticals Provides Update on Plans for Emergency Use Authorization Application Following FDA Feedback

Eiger BioPharmaceuticals, Inc. | October 06, 2022

Eiger BioPharmaceuticals, Inc. a commercial-stage biopharmaceutical company focused on the development of innovative therapies for hepatitis delta virus and other serious diseases, today announced that, following feedback from the U.S. Food and Drug Administration the company will not submit an emergency use authorization application of peginterferon lambda for the treatment of patients with mild-to-moderate COVID-19. Following Eiger's press release on September 6, 2022, the company submitted a pre-EUA meeting request to FDA, as well as additional morbidity and mortality outcomes data and analyses from the investigator-sponsored TOGETHER study. This included further statistical modeling and efficacy analyses of the study's primary and secondary endpoints and long-term follow-up data that the company believes continue to support the initial positive topline outcomes reported in March. In response, FDA denied the request for a pre-EUA meeting. Citing its concerns about the conduct of the TOGETHER study, FDA concluded that any authorization request based on these data is unlikely to meet the statutory criteria for issuance of an EUA in the current context of the pandemic. FDA suggested that, given peginterferon lambda's mechanism of action and the ongoing need for improved COVID-19 therapeutics, Eiger consider requesting an end-of-Phase 2 meeting to discuss a company-sponsored pivotal trial that could support an eventual Biologics License Application. Eiger is evaluating next steps for this program, in the U.S., as well as ex-U.S. emergency use authorization pathways and strategic options for continued development of peginterferon lambda for COVID-19 and other respiratory viral infections. "While we are disappointed that FDA will not consider an EUA application based on results generated from the TOGETHER study, we continue to have strong conviction in the potential of peginterferon lambda to confer a meaningful benefit for patients with COVID-19 and other respiratory viral infections. COVID-19 related deaths remain alarmingly high around the globe, including in the U.S. where, according to recent data from the Centers for Disease Control and Prevention, approximately 400 people die every day from this disease." David Cory, President and CEO, Eiger Eiger is advancing a late-stage pipeline of multiple FDA Breakthrough Therapy designated programs in Phase 3, including lonafarnib and peginterferon lambda for hepatitis delta virus (HDV) infection, and avexitide for congenital hyperinsulinism. The company expects to report topline data from D-LIVR, Eiger's landmark Phase 3 study of lonafarnib-based regimens for HDV, by year end. About TOGETHER Study TOGETHER is an independent multi-center, investigator-sponsored, randomized, placebo-controlled adaptive platform Phase 3 study evaluating therapeutics in newly diagnosed, high-risk, non-hospitalized patients with mild-to-moderate COVID-19. TOGETHER is the largest placebo-controlled study in COVID-19 and has evaluated 11 different therapeutic agents for non-hospitalized COVID-19 patients. The study was ongoing at the time the peginterferon lambda arm was added. The evaluation of peginterferon lambda versus placebo was the second largest study to date of a COVID-19 therapeutic of > 1,900 patients. Eligibility criteria required that all patients had laboratory-confirmed mild or moderate COVID-19 and were randomized within seven days of symptom onset. The study enrolled patients regardless of vaccination status or variant strain of SARS-CoV-2. The primary endpoint was a reduction in risk of clinical outcome comparing hospitalizations or emergency room visits greater than six hours after a single subcutaneous injection of peginterferon lambda versus placebo through Day 28. A key secondary endpoint was reduction in risk of hospitalizations or death in patients when dosed within three days of symptom onset. The TOGETHER study recruited from 12 sites in Brazil and 5 sites in Canada. About Peginterferon Lambda Peginterferon lambda is an investigational late-stage, first-in-class, type III interferon that stimulates immune responses that are critical for the development of host protection during viral infections and has been well-tolerated in clinical studies. Eiger is developing peginterferon lambda for the treatment of HDV infection. Peginterferon lambda has been administered to over 4,000 subjects in 28 clinical trials of HBV, HCV, HDV and COVID-19. Peginterferon lambda is an investigational agent and not yet approved for any indication. Eiger has received Orphan Designation by the U.S. Food and Drug Administration and European Medicines Agency, and Fast Track and Breakthrough Therapy Designation by FDA for peginterferon lambda in HDV. About Eiger Eiger is a commercial-stage biopharmaceutical company focused on the development of innovative therapies for hepatitis delta virus (HDV) and other serious diseases. The Eiger HDV platform includes two first-in-class therapies in Phase 3 that target critical host processes involved in viral replication.

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