Ensysce, a California Based Biotech Company With a Mission to Solve the Opioid Crisis, Exploring a Treatment for COVID-19

TheDailyTimes | April 15, 2020

Ensysce Biosciences announced that it is exploring the opportunity to initiate a clinical trial of oral Nafamostat, an ingredient from its opioid overdose protection platform, as a treatment for COVID-19. The goal is to evaluate if nafamostat mesylate (Nafamostat), a synthetic protease inhibitor will be effective in preventing or reducing the lethal outcome of COVID-19 infection. Currently, there are no approved drugs to manage infection with SARS-CoV-2 and nafamostat has shown promise in several studies against Middle East Respiratory Syndrome (MERS) and more recently SARS-CoV-2. Nafamostat was found to be a potent inhibitor that blocks the viral entry into host cells. Ensysce is planning a Phase 1/2 trial to examine an oral solution of nafamostat, a route of administration that has not been explored for this drug.

Spotlight

We have just begun to scratch the surface of linking genes to specific genetic diseases. We’re starting to see drugs be approved to treat these rare and unusual diseases based on the genetics involved, and at Alger we have invested up and down the entire genomics food chain.

Spotlight

We have just begun to scratch the surface of linking genes to specific genetic diseases. We’re starting to see drugs be approved to treat these rare and unusual diseases based on the genetics involved, and at Alger we have invested up and down the entire genomics food chain.

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MEDICAL

Be Bio and Resilience Announce Strategic Collaboration to Manufacture Engineered B Cells, a New Class of Cellular Medicines

Be Bio | May 20, 2022

Be Biopharma, Inc. (“Be Bio”) and National Resilience, Inc. (Resilience) today announced a strategic collaboration to advance initial programs in Be Bio’s rare disease pipeline. Be Bio’s proprietary engineered B Cell Medicines (BeCM) platform is harnessing the power of the human B cell to create a new class of autologous and allogeneic cellular medicines that durably and redosably produce therapeutic proteins in vivo without toxic pre-conditioning. The two companies are investing to drive innovation and reliability in cell therapy manufacturing, a critical success factor for broad and meaningful patient impact. As part of this unique partnership, Resilience will dedicate personnel solely to produce and supply Good Manufacturing Practices (GMP)-grade viral vector and cell therapy drug product for the initial programs in Be Bio’s rare disease pipeline. Through a creative cost and risk-sharing model, Resilience will be responsible for manufacturing costs and receive potential future milestones and royalties. Over the past year, Be Bio has built a strong foundation with our BeCM platform, pipeline, team, and recent $130 million financing. Manufacturing is critical to rapidly progress our BeCMs to the clinic and we have built non-GMP manufacturing capabilities in our Cambridge facility. This deal allows us to drive GMP manufacturing with an outstanding partner, and in a capital efficient manner, Resilience’s broad manufacturing capabilities, strong collaborative spirit and dedicated resources to our platform, make them an ideal partner for our BeCM programs.” Joanne Smith-Farrell, Ph.D., Chief Executive Officer at Be Bio. Resilience will lead clinical GMP manufacturing of both the viral vector and the cell therapy drug product for Be Bio’s initial rare disease programs to support first-in-human clinical trials. Resilience will apply its pioneering bioprocessing solutions and network of cell therapy sites, including facilities in Research Triangle Park, NC, Philadelphia, PA, Waltham, MA and Marlborough, MA to conduct the work. This collaboration shows our excitement for the promising science of Be Bio’s proprietary BeCM platforms, and our confidence in their expertise to deliver transformative cell therapies, By working alongside Be Bio early in the drug development process, we aim to accelerate the development of their B cell medicines with the potential to unlock a pipeline of product candidates across a variety of serious diseases.” Rahul Singhvi, Sc.D., Chief Executive Officer of Resilience. About B Cells – A New Class of Cellular Medicines Imagine what could “Be?” In nature, a single B cell engrafts in the bone marrow and can produce thousands of proteins per second at constant levels over decades. B cells are nature’s exquisite medicine factories, manufacturing proteins to fight disease and maintain health. Unleashing the power of B cells is driving a new class of cellular medicines – Engineered B Cell Medicines (BeCM). BeCMs have the potential to be durable, allogeneic, redosable and administered without toxic conditioning. The promise of BeCMs could transform therapeutic biologics with broad application — across protein classes, patient populations and therapeutic areas. About Be Biopharma Be Biopharma (“Be Bio”) is pioneering Engineered B Cell Medicines (BeCM) to dramatically improve patients’ lives who are living with cancer, rare diseases and other serious conditions. With eyes locked on the patient, our team of purpose-driven scientists, technologists, manufacturing experts and business builders collaborate to create a bold new class of cell therapies. Be Bio was founded in October 2020 by Longwood Fund and B cell engineering pioneers David Rawlings, M.D., and Richard James, Ph.D. from Seattle Children’s Research Institute. Be Bio is backed by ARCH Venture Partners, Atlas Venture, RA Capital Management, Alta Partners, Longwood Fund, Bristol Myers Squibb and Takeda Ventures. Since our founding, Be Bio’s investors have committed over $180 million to enable the Company to re-imagine medicine based on the power of B cell therapy. About Resilience Resilience is a technology-focused biomanufacturing company dedicated to broadening access to complex medicines. Founded in 2020, the company is building a sustainable network of high-tech, end-to-end manufacturing solutions to ensure the treatments of today and tomorrow can be made quickly, safely, and at scale. Resilience seeks to free its partners to focus on the discoveries that improve patients’ lives by continuously advancing the science of biopharmaceutical manufacturing and development.

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MEDICAL

Felix Biotechnology Announces Initiation of CYPHY Phase 1/2 study at Yale for Lead Asset

Felix Biotechnology | January 08, 2021

Felix Biotechnology reported the inception of CYPHY, a Phase 1/2 examiner started single focus preliminary at Yale University for focused phage treatment YPT-01 in the therapy of constant P. aeruginosa diseases in cystic fibrosis. This twofold visually impaired, fake treatment controlled study (NCT 04684641) will evaluate the wellbeing and adequacy of YPT-01 added to standard antimicrobial treatment in 36 patients. CYPHY will likewise evaluate the capacity of YPT-01 to lessen the harmfulness and anti-infection obstruction of P. aeruginosa, improving patient results and re-empowering utilization of conventional anti-infection agents against multi-drug safe strains. The lead specialist for this investigation, Dr. Jon Koff, Associate Professor and Director of Yale's Adult Cystic Fibrosis Program, is supported by an academic grant from the Cystic Fibrosis Foundation. "This is a fantastic opportunity to show how effective phage therapy can be when deployed in an evolutionary framework. We know that pathogens evolve resistance to any antibiotic or therapy we use, so our approach turns that to our advantage," said Dr. Paul Turner, Professor of Ecology and Evolutionary Biology, co-inventor of YPT-01, and co-founder of Felix Biotechnology. "By targeting phage to mechanisms of virulence, we ensure that if pathogens evolve resistance to phage, they lose traits that make them effective pathogens, putting them in an evolutionary Catch-22."

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CELL AND GENE THERAPY

2seventy bio Announces Expanded Collaboration Agreement With Novo Nordisk to Continue Development of in vivo Gene Editing Approach

2seventy bio, Inc | January 07, 2022

2seventy bio, Inc. announced that it has entered into an option and license agreement with Novo Nordisk for joint research and development of an in vivo gene editing treatment for hemophilia A. This agreement builds upon a successful existing multi-year research collaboration between the two companies. Under the terms of this agreement, Novo Nordisk will have the option to license 2seventy bio’s proprietary mRNA-based megaTAL™ technology for the development of a new treatment approach for hemophilia A patients with the goal of offering a sustained therapeutic effect. “This collaboration aims to develop the first direct in vivo application of our megaTAL technology. We are excited to explore this technology with Novo Nordisk, with the goal of developing a new potential therapeutic approach for patients with hemophilia A. Moreover, we view this work as further validation and a natural extension of our technology platform. Based on what we continue to learn, this technology may play a potential role in expanding our gene editing platform toward future in vivo and ex vivo applications that can enhance our oncology pipeline. We are also excited to announce a partnership between 2seventy bio and Genevant Sciences for the use of Genevant’s lipid nanoparticle platform in our collaboration with Novo Nordisk.” Philip Gregory, D. Phil., chief scientific officer, 2seventy bio The collaboration agreement with Novo Nordisk builds upon the original research collaboration signed between bluebird bio and Novo Nordisk in 2019, focused on identifying a development gene therapy candidate for people with hemophilia A. The collaboration utilizes 2seventy bio’s megaTAL technology that has the potential to provide a highly specific and efficient way to silence, edit, or insert genetic components. Hemophilia A is a genetic bleeding disorder resulting from defective Factor VIII. “We are excited to continue our partnership with 2seventy bio to jointly develop a next-generation in vivo genome editing treatment, with the ultimate ambition of offering people with hemophilia A lifetime free of factor replacement therapy,” said Karin Conde-Knape, senior vice president, Global Drug Discovery, Novo Nordisk. “This partnership reflects Novo Nordisk’s commitment to utilizing novel technology platforms to advance truly disease-modifying therapies for people with serious chronic diseases.” Under the terms of the agreement, Novo Nordisk will obtain the option to exclusively license 2seventy bio’s in vivo mRNA platform and gene editing technology for use in the treatment of patients with hemophilia A. 2seventy bio will receive an upfront payment of $5 million and is eligible for near-term milestone and option exercise payments of up to $35 million, in addition to development, regulatory, and commercial milestones, as well as a royalty on net sales. Novo Nordisk will be responsible for funding all research and development activities. Related to this collaboration, 2seventy bio has also entered into an agreement with Genevant Sciences for access to Genevant’s industry-leading LNP technology platform for use in 2seventy bio’s collaboration with Novo Nordisk for the treatment of patients with hemophilia A. 2seventy bio plans to use the Genevant LNP platform for efficient delivery of megaTAL mRNA to hepatocyte cells within the liver. “We are very pleased that 2seventy bio has entrusted Genevant and our LNP platform with delivery for its important gene editing program in hemophilia A,” said Pete Lutwyche, Ph.D., president and chief executive officer, Genevant Sciences Corporation. “Our scientists have been at the forefront of the LNP field for more than 20 years, and we are excited for our innovative technology to be used for this important application with great unmet need.” Under the terms of the agreement between 2seventy bio and Genevant, 2seventy bio obtained rights to license Genevant’s LNP technology for use with megaTAL mRNA products in the treatment of patients with hemophilia A. Genevant is eligible for upfront and near-term option exercise payments totaling $10 million, as well as development and commercialization milestones, and royalties in the mid-single digits on future product sales. About megaTALs MegaTALs are single-chain enzymes that combine the natural DNA recognition and cleavage processes of Homing Endonucleases with the modular DNA binding properties of transcription activator-like effectors. This protein fusion architecture allows the generation of highly specific and active nucleases in a compact format compatible with all current viral and non-viral cell delivery methods. About Novo Nordisk A/S Novo Nordisk is a leading global healthcare company, founded in 1923 and headquartered in Denmark. Our purpose is to drive change to defeat diabetes and other serious chronic diseases such as obesity and rare blood and endocrine disorders. We do so by pioneering scientific breakthroughs, expanding access to our medicines, and working to prevent and ultimately cure disease. Novo Nordisk employs about 47,000 people in 80 countries and markets its products in around 170 countries. About Genevant Sciences Genevant Sciences is a leading nucleic acid delivery company with world-class platforms, the industry’s most robust and expansive lipid nanoparticle patent estate, and decades of experience and expertise in nucleic acid drug delivery and development. The Company’s scientists have pioneered LNP delivery of nucleic acids for over 20 years, and the Company’s LNP platform, which has been studied across more than a dozen discrete product candidates and is the delivery technology behind the first and only approved RNAi-LNP (patisiran), enables a wide array of RNA-based applications, including vaccines, therapeutic protein production, and gene editing. About 2seventy bio Our name, 2seventy bio, reflects why we do what we do - TIME. Cancer rips time away and our goal is to work at the maximum speed of translating human thought into action – 270 miles per hour — to give the people we serve more time. We are building the leading immuno-oncology cell therapy company, focused on discovering new therapies and leveraging platform technologies that truly disrupt the cancer treatment landscape.

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