Gilead climbs as coronavirus drug shows efficacy, starts trial

FierceBiotech | February 03, 2020

Gilead climbs as coronavirus drug shows efficacy, starts trial
Gilead has made progress in its effort to reposition failed Ebola drug remdesivir as a treatment for the coronavirus sweeping across China. In quick succession, it emerged that the nucleotide analogue prodrug appeared to help the first U.S. case of the virus and is set to undergo clinical testing in China, sending Gilead’s stock up by as much as 13% in premarket trading. Remdesivir was one of a clutch of drugs tested against Ebola, but it failed to match the efficacy of its rivals, leading its development against that virus to come to a halt. However, preclinical tests have suggested the drug is effective against murine hepatitis virus and Middle East respiratory syndrome coronavirus, leading Gilead to propose it as an answer to the outbreak that began in Wuhan, China. Gilead publicly floated the idea of using remdesivir to treat the coronavirus, dubbed 2019-nCoV, 10 days ago. Since then, the effort to advance the drug in the indication has gathered pace.

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BUSINESS INSIGHTS

Iktos and The University of Dundee, Drug Discovery Unit (DDU) Announce a Collaboration to Use AI for Drug Design and Retrosyntheis

Iktos | December 20, 2021

Iktos, a company specialized in Artificial Intelligence for new drug design, and The Drug Discovery Unit (DDU), School of Life Sciences, The University of Dundee, focused on the discovery of therapeutics for neglected diseases and the translation of novel biology through small molecule drug discovery, announced entering a collaboration to apply Iktos’s generative modelling artificial intelligence (AI) technology in one of DDU’s drug discovery programs. Under the agreement, Iktos will apply its de novo ligand and structure-based generative modelling technologies, its AI-based retrosynthesis analysis and planning tool Spaya™, and know-how complementing DDU’s drug discovery capabilities to expedite the identification of potential pre-clinical candidates and to identify additional novel chemical matter with suitable properties. The DDU was established in 2006 to respond to a lack of drug targets identified for neglected tropical diseases being translated into therapeutics for diseases impacting the poorest people in the world. The DDU translates basic science into lead compounds to validate putative drug targets, to use as tools to investigate disease pathways and, when appropriate, advance to pre-clinical drug candidates for multiple diseases, e.g. TB and cancer. Iktos’s AI technology, based on deep generative models, helps to bring new insights and directions into the drug discovery process based on a comprehensive data-driven chemical structure generation technology. This technology automatically designs virtual novel molecules with all of the characteristics of a successful drug molecule. This approach, validated through Iktos’s other collaborations, is a novel solution to one of the key challenges in drug design: rapid identification of molecules that simultaneously satisfy multiple parameters, such as potency, selectivity, safety, and project-specific properties. This approach uniquely enables the exploration of chemical space and produces innovative molecule designs with greater freedom to operate. Iktos has recently diversified its R&D efforts into the development of an AI technology for retrosynthesis. Identifying and selecting synthetic pathways is one of the most challenging and time-consuming tasks in synthetic and medicinal chemistry. Iktos has developed AI based retrosynthesis analysis and planning tool Spaya™ by harnessing the power of data-driven retrosynthesis algorithm for systematic exploration and prioritisation of synthetic routes for a desired compound in minutes. “We are thrilled to be working with Iktos to drive our projects towards drug candidates. Iktos AI platform combined with our own in-house computational and medicinal chemistry design teams provide a powerful combination for innovative new drug design.” Professor Paul Wyatt, Head of the DDU “We are thrilled and proud to join forces with DDU with the aim to help in the discovery of therapeutics for neglected diseases and the translation of novel biology through small molecule drug discovery,” commented Yann Gaston-Mathé, President and CEO of Iktos. “Pleased to have earned DDU’s trust, we are confident that together with DDU and their established R&D partners, we will be able to identify promising novel chemical matter and solve complex multiparametric optimisation problems. The feedback from DDU’s research team will be highly valuable as we improve our product offerings. Our strategy has always been to tackle challenging problems alongside our collaborators where we can demonstrate value generation for new and on-going drug discovery projects.” About DDU The Drug Discovery Unit (DDU) (est. 2006) within the School of Life Sciences at The University of Dundee is a fully integrated, Biotech-style, drug discovery centre working across multiple disease areas. The DDU collaborates with partners to translate world-class biology research into novel drug targets and candidate drugs to address unmet medical need. About Iktos Incorporated in October 2016, Iktos is a French start-up company specialized in the development of artificial intelligence solutions applied to chemical research, more specifically medicinal chemistry and new drug design. Iktos is developing a proprietary and innovative solution based on deep learning generative models, which enables, using existing data, to design molecules that are optimized in silico to meet all the success criteria of a small molecule discovery project. The use of Iktos technology enables major productivity gains in upstream pharmaceutical R&D. Iktos offers its technology both as professional services and as a SaaS software platform, Makya™. Iktos is also developing Spaya™, a synthesis planning software based upon Iktos’s proprietary AI technology for retrosynthesis.

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PHARMACY MARKET

medical leverage, a communications company positions itself for 2022 growth

medical leverage | January 15, 2022

medical leverage, a communications company is pleased to announce the promotion of Justin Reed to VP of Finance, Alexandra Rivers to HR and Training Manager, and Janeanne Josephson to Associate Marketing Manager. These changes drive the company's mission to meet the challenges of today's unique market. With the additional hiring of several project managers to the team, ml is positioning itself well for growth in 2022. These promotions are all important shifts in responsibility that will ensure ml's service levels are enhanced during its expansion efforts. "Our 2022 expansion efforts started at the stroke of midnight on the new year, thus spring-boarding our talent acquisition initiatives. We continue to attract top-notch thinkers and doers by focusing on creating a culture where we offer improved operations, continued staff training, and product enhancements to best serve our current and future clients. As always, our expansion approach is guided by our core values and steered by our Executive Leadership Team." medical leverage's president, Dave Oury ABOUT medical leverage is a medical communications company that provides full-service solutions to pharmaceutical, biotech, biopharmaceutical, medical device, and medical diagnostic companies. We elevate healthcare communications through education by partnering strategically with life science companies and providers. For over twenty years, we have had the distinct honor of working with marketing, medical affairs, sales training, and market access teams within US and international companies. We know that behind every program there is a product and behind every healthcare professional there is a patient. We focus on our clients, so they can focus on healthcare professionals and, ultimately, their patients.

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PHARMA TECH

n-Lorem Foundation Partners with Alnylam to Expand Technology Access to RNAi Therapeutics for Patients with Nano-rare Diseases

n-Lorem a nonprofit Foundation | January 20, 2022

n-Lorem, a nonprofit Foundation, discovering and providing experimental antisense oligonucleotide medicines to nano-rare disease patients for free, for life, today announced a new partnership with Alnylam Pharmaceuticals Inc. “Discovering, developing and providing individualized medicines for nano-rare patients is a monumental task. At n-Lorem, using ASO technology, we take advantage of three decades of experience, unparalleled quality control and integrated safety databases that are used to select frequency and dosage for each individual ASO therapy. As we expand our highly selective and versatile model to other amendable technologies, it is important for us to maintain a high level of quality,” said Stanley T. Crooke, M.D., Ph.D., Founder, Chief Executive Officer and Chairman, n-Lorem Foundation. “Alnylam’s RNAi therapeutic technology is the perfect complement to Ionis’ ASO technology, both are well understood, proven drug discovery technologies. Adding Alnylam as a partner is another step in the creation of a broad network of partner stakeholders committed to treating nano-rare patients.” “We are pleased to partner with the n-Lorem Foundation in identifying opportunities to bring our novel RNAi technology platform to potentially help patients with nano-rare diseases. At Alnylam, we are committed to identifying pathways to expand access to our medicines and we applaud the work of the foundation in bringing together industry partners who are driven to provide options for patients that have little or no options for treatment,” Akshay Vaishnaw, M.D., Ph.D., President at Alnylam “Although we have the tools and expertise to understand nano-rare patients at a genetic level, the extreme rarity of these patients present insurmountable challenges to the current healthcare systems,” said Dr. Sarah Glass, Chief Development Officer, n-Lorem Foundation. “n-Lorem is addressing these challenges one nano-rare patient at a time and is supported through a consortium of well-renowned and diverse organizations worldwide. These include our biotechnology and pharmaceutical partners, including Ionis Pharmaceuticals, Biogen, Ultragenyx and today Alnylam; our clinical research partners, including Charles River Labs, Korea Institute of Toxicology and Parexel; our contract manufacturing partners, including ChemGenes, Cytiva and Nitto Avecia; and many other collaborations that make n-Lorem’s mission possible.” Alnylam is leading the translation of RNA interference into a whole new class of innovative medicines with the potential to transform the lives of people afflicted with rare genetic, cardio-metabolic, hepatic infectious, and central nervous system ocular diseases. Based on Nobel Prize-winning science, RNAi therapeutics represent a powerful, clinically validated approach for the treatment of a wide range of severe and debilitating diseases. Founded in 2002, Alnylam is delivering on a bold vision to turn scientific possibility into reality, with a robust RNAi therapeutics platform. Alnylam’s commercial RNAi therapeutic products are ONPATTRO®, GIVLAARI® (givosiran), and OXLUMO®, as well as Leqvio®, which is being developed and commercialized by Alnylam’s partner Novartis. Alnylam has a deep pipeline of investigational medicines, including six product candidates that are in late-stage development. Alnylam is executing on its “Alnylam P5x25” strategy to deliver transformative medicines in both rare and common diseases benefiting patients around the world through sustainable innovation and exceptional financial performance, resulting in a leading biotech profile. Alnylam is headquartered in Cambridge, MA. About n-Lorem n-Lorem Foundation is a non-profit organization established to apply the efficiency, versatility and specificity of antisense technology to charitably provide experimental antisense oligonucleotide medicines to treat nano-rare patients diagnosed with diseases that are the result of a single genetic defect unique to only one or very few individuals. Nano-rare patients describe a very small group of patients who, because of their small numbers, have few if any treatment options. n-Lorem Foundation was created to provide hope to these nano-rare patients by developing individualized ASO medicines, which are short strands of modified DNA that can specifically target the transcripts of a defective gene to correct the abnormality. The advantage of experimental ASO medicines is that they can be developed rapidly, inexpensively and are highly specific. To date, n-Lorem has assisted in the development and treatment of 14 nano-rare patients and received over 100 applications for treatment with more than 40 nano-rare patients approved. n-Lorem was founded by Stanley T. Crooke, M.D., Ph.D., former chairman and CEO of Ionis Pharmaceuticals, who founded Ionis Pharmaceuticals in 1989 and, through his vision and leadership, established the company as the leader in RNA-targeted therapeutics.

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PHARMACY MARKET

KromaTiD Announces Launch of KromaTiD Pinpoint FISH TP53/CEP17 Kit for Synthetic Oligonucleotide-Based FISH Assay

KromaTiD Inc | January 14, 2022

KromaTiD is excited to announce early access to our KromaTiD Pinpoint FISH TP53/CEP17 Kit, our new product designed to supply the highest resolution, lowest background, and lowest limit of detection available. KromaTiD's Pinpoint FISH™ Kit, allows for better hybridization conditions and eliminating strand degradation steps. Pinpoint FISH™ allows researchers to expand beyond the capabilities of conventional FISH probes to detect smaller targets and design ultra-high specificity tests. Our technology is fully compatible with your standard FISH equipment/conditions and BAC probes and works with your established samples, workflow, and imaging system. "Single-cell genomic measurements from KromaTiD have unique capabilities to analyze and understand the underlying genomic drivers of human disease. Pinpoint FISH has an unmatched resolution, extraordinary ease of use, consistency from analysis to analysis, and an unprecedented ability to distinguish fine genomic structural variation" Dr. Tompkins continues, "While this is the first catalog product from KromaTiD, we have a ten-year history of building a custom assay for Pharma and Biotech companies based on the PPF and dGH platforms. As we begin to build out a comprehensive line of blood cancer measurement kits, we are delighted to bring Pinpoint FISH to a worldwide market". Pinpoint FISH™ is available now from KromaTiD for researchers' use in oligonucleotide-based FISH assays. Christopher Tompkins, KromaTiD's Chief Technology Officer About KromaTiD, Inc. KromaTiD is transforming the fields of genome engineering through the discovery and characterization of genomic structural changes that help leading gene editing and pharmaceutical companies advance therapies to market. KromaTiD offers a powerful suite of products and services for studying genomic rearrangements, custom assay development services and preclinical research support. KromaTiD's proprietary Pinpoint FISH™ and directional Genomic Hybridization platforms (dGHTM) have applications throughout genomics, supplying direct, definitive data on structural variations that no other technologies can provide. We provide the essential genomic structural context to sequence.

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Spotlight

Watch a tailored presentation by Dr. Pierre De Meyts, a world-reknown insulin expert, on the use of insulin in cell growth media. If you wish to obtain more details about the subjects presented in the showcase, you can read Dr. De Meyts articles.

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