The challenges of hit-to-lead in drug discovery

VICTORIA REES | October 7, 2019 | 75 views

Researchers can narrow down their search for drug candidates using hit-to-lead techniques that reveal which compounds are most active against a target. Our ability to generate biological data libraries far exceeds our ability to test them,3 meaning that there is an abundance of available data with no use, as of yet.

Spotlight

Paragon Bioservices, Inc.

Paragon Bioservices is an industry-leading, private-equity backed contract development and manufacturing organization (CDMO) whose focus is the development and manufacturing of cutting-edge biopharmaceuticals. Paragon’s aim is to build strong client partnerships with the world’s best biotech and pharma companies, focusing on transformative technologies, including gene therapies (AAV), next-generation vaccines, oncology immunotherapies (oncolytic viruses and CAR-T cell therapies), therapeutic protein, and other complex biologics.

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MEDTECH

Top 10 biotech IPOs in 2019

Article | July 16, 2022

The big question at the start of 2019 was whether the IPO window would stay open for biotech companies, particularly those seeking to pull off ever-larger IPOs at increasingly earlier stages of development. The short answer is yes—kind of. Here’s the long answer: In the words of Renaissance Capital, the IPO market had “a mostly good year.” The total number of deals fell to 159 from 192 the year before, but technology and healthcare companies were standout performers. The latter—which include biotech, medtech and diagnostics companies—led the pack, making up 43% of all IPOs in 2019. By Renaissance’s count, seven companies went public at valuations exceeding $1 billion, up from five the year before

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MEDTECH

Cell Out? Lysate-Based Expression an Option for Personalized Meds

Article | July 11, 2022

Cell-free expression (CFE) is the practice of making a protein without using a living cell. In contrast with cell line-based methods, production is achieved using a fluid containing biological components extracted from a cell, i.e., a lysate. CFE offers potential advantages for biopharma according to Philip Probert, PhD, a senior scientist at the Centre for Process Innovation in the U.K.

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RESEARCH

Closing bacterial genomes from the human gut microbiome using long-read sequencing

Article | July 11, 2022

In our lab, we focus on the impact of the gut microbiome on human health and disease. To evaluate this relationship, it’s important to understand the particular functions that different bacteria have. As bacteria are able to exchange, duplicate, and rearrange their genes in ways that directly affect their phenotypes, complete bacterial genomes assembled directly from human samples are essential to understand the strain variation and potential functions of the bacteria we host. Advances in the microbiome space have allowed for the de novo assembly of microbial genomes directly from metagenomes via short-read sequencing, assembly of reads into contigs, and binning of contigs into putative genome drafts. This is advantageous because it allows us to discover microbes without culturing them, directly from human samples and without reference databases. In the past year, there have been a number of tour de force efforts to broadly characterize the human gut microbiota through the creation of such metagenome-assembled genomes (MAGs)[1–4]. These works have produced hundreds of thousands of microbial genomes that vastly increase our understanding of the human gut. However, challenges in the assembly of short reads has limited our ability to correctly assemble repeated genomic elements and place them into genomic context. Thus, existing MAGs are often fragmented and do not include mobile genetic elements, 16S rRNA sequences, and other elements that are repeated or have high identity within and across bacterial genomes.

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Selexis Cell Line Development Strategies

Article | February 11, 2020

In today’s biotechnology landscape, to be competitive, meet regulations, and achieve market demands, “we must apply Bioprocessing 4.0,” said Igor Fisch, PhD, CEO, Selexis. In fact, in the last decade, “Selexis has evolved from cloning by limiting dilution to automated cell selection to nanofluidic chips and from monoclonality assessment by statistical calculation to proprietary bioinformatic analysis,” he added. Single-use processing systems are an expanding part of the biomanufacturing world; as such, they are a major component of Bioprocessing 4.0. “At Selexis, we use single use throughout our cell line development workflow. Currently, we have incorporated single-use automated bioprocessing systems such as ambr® and the Beacon® optofluidic platform for accelerated cell line development. By using these systems and optimizing our parameters, we were able to achieve high titers in shake flasks. Additionally, the Beacon systems integrate miniaturized cell culture with high-throughput liquid handling automation and cell imaging. This allows us to control, adjust, and monitor programs at the same time,” noted Fisch.

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Spotlight

Paragon Bioservices, Inc.

Paragon Bioservices is an industry-leading, private-equity backed contract development and manufacturing organization (CDMO) whose focus is the development and manufacturing of cutting-edge biopharmaceuticals. Paragon’s aim is to build strong client partnerships with the world’s best biotech and pharma companies, focusing on transformative technologies, including gene therapies (AAV), next-generation vaccines, oncology immunotherapies (oncolytic viruses and CAR-T cell therapies), therapeutic protein, and other complex biologics.

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INDUSTRIAL IMPACT, MEDICAL

Omega Therapeutics Announces Clinical Supply Agreement to Evaluate the Combination of OTX-2002 and Atezolizumab in Hepatocellular Carcinoma

Globenewswire | March 31, 2023

Omega Therapeutics, Inc. a clinical-stage biotechnology company pioneering the development of a new class of programmable epigenetic mRNA medicines, today announced a clinical supply agreement with Roche to evaluate OTX-2002, its lead candidate in development for the treatment of MYC-driven hepatocellular carcinoma (HCC), in combination with Roche’s anti-PD-L1 therapy, atezolizumab, as part of Omega’s Phase 1/2 MYCHELANGELO™ I clinical trial. “This agreement with Roche represents continued execution of our clinical trial strategy and the next step toward realizing the transformative potential of OTX-2002, a first-in-class epigenomic controller designed to pre-transcriptionally downregulate the MYC oncogene, a historically undruggable target,” said Mahesh Karande, President and Chief Executive Officer of Omega Therapeutics. “In preclinical studies, OTX-2002 demonstrated synergistic antitumor activity with existing standard of care therapies for HCC, including anti-PD-1 and anti-PD-L1 immune checkpoint inhibitors, with minimal impact on safety and tolerability. Through the combination of two orthogonal treatments, OTX-2002 and atezolizumab, a leading anti-PD-L1 therapy, we aim to simultaneously disrupt multiple drivers of cancer progression with the goal of improving treatment outcomes. With Roche’s support and partnership, we look forward to assessing the ability of this novel combination approach to enhance antitumor immune response in patients with advanced HCC.” The ongoing Phase 1/2 MYCHELANGELO I trial is designed to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary antitumor activity of OTX-2002 as a monotherapy (Part 1) and in combination with standard of care therapies in patients with relapsed or refractory HCC and other solid tumor types known for association with the MYC oncogene. Preliminary data from the Phase 1 monotherapy dose escalation portion of the study are anticipated in 2023. Under the terms of this agreement, Roche will supply atezolizumab and Omega will evaluate the combination as part of the overall conduct of the trial. About Omega Therapeutics Omega Therapeutics, founded by Flagship Pioneering, is a clinical-stage biotechnology company pioneering the development of a new class of programmable epigenetic mRNA medicines. The Company's OMEGA platform harnesses the power of epigenetics, the mechanism that controls gene expression and every aspect of an organism's life from cell genesis, growth, and differentiation to cell death. Using a suite of technologies, paired with Omega's process of systematic, rational, and integrative drug design, the OMEGA platform enables control of fundamental epigenetic processes to correct the root cause of disease by returning aberrant gene expression to a normal range without altering native nucleic acid sequences. Omega's modular and programmable mRNA medicines, Omega Epigenomic Controllers™(OECs), target specific epigenomic loci within insulated genomic domains, EpiZips, from amongst thousands of unique, mapped, and validated genome-wide DNA sequences, with high specificity to durably tune single or multiple genes to treat and cure diseases through unprecedented precision epigenomic control. Omega’s approach enables pre-transcriptional control of most human genes including historically undruggable, intractable, and difficult to treat targets. Omega’s pipeline of OEC candidates spans a range of disease areas, including oncology, regenerative medicine, multigenic diseases including immunology, and select monogenic diseases.are Diseases and Orphan Drugs from the Accreditation Commission for Health Care. PANTHERx is now a five-time winner of the prestigious MMIT Patient Choice Award, including the 2022 honor. PANTHERx is headquartered in Pittsburgh, licensed in all 50 states, and holds accreditations from URAC, NABP, and ACHC.

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INDUSTRIAL IMPACT, MEDICAL

Elemental Machines partners with System-c bioprocess to bring its IoT hardware solutions to the European biopharmaceutical industry

Prnewswire | March 30, 2023

Elemental Machines, developer of the Intelligent Operations Platform, has entered into a partnership with System-c bioprocess as an authorized reseller of Elemental Machines' hardware solutions, servicing the biopharmaceutical industry in France. The partnership will allow Elemental Machines to expand its footprint further in European markets. System-c serves as a partner and distributor for benchmark brands that cover a range of bioprocessing products from screening to filtration and beyond. Under the terms of the agreement, System-c will be an authorized reseller and distributor of Elemental Machines' suite of IoT-enabled sensors, which operators can use to collect and unify data across their biopharmaceutical facilities. "We are excited to partner with System-c and leverage their expertise in bringing innovative technology solutions to their bio-pharma customers,"said Dan Petkanas, Director of Global Alliances at Elemental Machines. "Together we will be able to deliver much needed tools to support and speed drug discovery." "We are thrilled to announce our partnership with Elemental Machines," said Yannick Carfantan, CEO of System-c. "Elemental Machines' leading technology platform for optimizing laboratory operations is going to give our company a new boost in our business development, not only for the French market but across Europe. Their measurement, information, alerting and data recovery technology opens new horizons for the control of laboratory equipment and R&D, pilot, and production departments enabling peace of mind for a number of actors in the biotech and biopharma industries. A winner for sure!" About System-c bioprocess As a partner and distributor for benchmark brands and continually searching for new innovative technologies, our product range covers all stages of the bioprocesses: from screening to filtration of the final product. Our solutions are always evolving in parallel with the new developments in life sciences, as well as the requirements for quality, safety and process productivity. Established in 1988 System-c bioprocess has been created to guarantee their customers complete, easy-to-use and customizable solutions to ensure productivity, reliability, and continuous traceability for all bioprocesses. Experts in innovative solutions and technologies for cell culture and fermentation processes, we have been constantly working with researchers, technicians and engineers from the pharmaceutical, biotechnology and agrifood industries for more than 30 years. About Elemental Machines Elemental Machines' Intelligent Operations Platform combines the best of IoT technology with purpose-built software solutions to deliver actionable intel to operators in the life sciences and byond. The Platform simplifies, optimizes, and augments operations by connecting the physical and digital infrastructure. Science and technology leaders trust Elemental Machines' ecosystem of hardware and software to deliver actionable insights that accelerate their pace of innovation.

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INDUSTRIAL IMPACT, MEDICAL

Bicycle Therapeutics Announces a Strategic Collaboration with Novartis to Discover, Develop and Commercialize Bicycle® Radio-Conjugates

Businesswire | March 29, 2023

Bicycle Therapeutics plc a biotechnology company pioneering a new and differentiated class of therapeutics based on its proprietary bicyclic peptide (Bicycle®) technology, today announced that it has entered into a strategic collaboration agreement with Novartis to develop, manufacture and commercialize Bicycle® radio-conjugates (BRCs) for multiple agreed upon oncology targets. “This collaboration builds on the groundbreaking clinical work we have been doing in the toxin conjugate field and provides new and additional validation for this unique technology,” said Kevin Lee, Ph.D., Chief Executive Officer of Bicycle Therapeutics. “We look forward to working closely with Novartis to pioneer the discovery and development of potential new cutting-edge radiopharmaceutical cancer treatments based on Bicycles. We believe the properties of Bicycles make them well suited for the development of precision guided radiopharmaceuticals and represents the next leg in the application of our proprietary discovery platform in oncology.” Under the terms of the agreement, Bicycle will utilize its proprietary phage platform to discover Bicycles to be developed into BRCs. Novartis will be responsible for further development, manufacture and commercialization of the BRCs. Novartis will fund all pre-clinical and clinical development and commercialization activities. Bicycle will receive a $50 million upfront payment and is eligible for development and commercial-based milestone payments totaling up to $1.7 billion. Bicycle will also be eligible to receive tiered royalties on Bicycle-based medicines commercialized by Novartis. About Bicycle Therapeutics Bicycle Therapeutics is a clinical-stage biopharmaceutical company developing a novel class of medicines, referred to as Bicycles, for diseases that are underserved by existing therapeutics. Bicycles are fully synthetic short peptides constrained with small molecule scaffolds to form two loops that stabilize their structural geometry. This constraint facilitates target binding with high affinity and selectivity, making Bicycles attractive candidates for drug development. Bicycle is evaluating BT5528, a second-generation Bicycle Toxin Conjugate targeting EphA2; BT8009, a second-generation BTC targeting Nectin-4, a well-validated tumor antigen; and BT7480, a Bicycle TICA™ targeting Nectin-4 and agonizing CD137, in company-sponsored Phase I/II trials. In addition, BT1718, a BTC that targets MT1-MMP, is being investigated in an ongoing Phase I/IIa clinical trial sponsored by the Cancer Research UK Centre for Drug Development. Bicycle is headquartered in Cambridge, UK, with many key functions and members of its leadership team located in Cambridge, MA.

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INDUSTRIAL IMPACT, MEDICAL

Omega Therapeutics Announces Clinical Supply Agreement to Evaluate the Combination of OTX-2002 and Atezolizumab in Hepatocellular Carcinoma

Globenewswire | March 31, 2023

Omega Therapeutics, Inc. a clinical-stage biotechnology company pioneering the development of a new class of programmable epigenetic mRNA medicines, today announced a clinical supply agreement with Roche to evaluate OTX-2002, its lead candidate in development for the treatment of MYC-driven hepatocellular carcinoma (HCC), in combination with Roche’s anti-PD-L1 therapy, atezolizumab, as part of Omega’s Phase 1/2 MYCHELANGELO™ I clinical trial. “This agreement with Roche represents continued execution of our clinical trial strategy and the next step toward realizing the transformative potential of OTX-2002, a first-in-class epigenomic controller designed to pre-transcriptionally downregulate the MYC oncogene, a historically undruggable target,” said Mahesh Karande, President and Chief Executive Officer of Omega Therapeutics. “In preclinical studies, OTX-2002 demonstrated synergistic antitumor activity with existing standard of care therapies for HCC, including anti-PD-1 and anti-PD-L1 immune checkpoint inhibitors, with minimal impact on safety and tolerability. Through the combination of two orthogonal treatments, OTX-2002 and atezolizumab, a leading anti-PD-L1 therapy, we aim to simultaneously disrupt multiple drivers of cancer progression with the goal of improving treatment outcomes. With Roche’s support and partnership, we look forward to assessing the ability of this novel combination approach to enhance antitumor immune response in patients with advanced HCC.” The ongoing Phase 1/2 MYCHELANGELO I trial is designed to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary antitumor activity of OTX-2002 as a monotherapy (Part 1) and in combination with standard of care therapies in patients with relapsed or refractory HCC and other solid tumor types known for association with the MYC oncogene. Preliminary data from the Phase 1 monotherapy dose escalation portion of the study are anticipated in 2023. Under the terms of this agreement, Roche will supply atezolizumab and Omega will evaluate the combination as part of the overall conduct of the trial. About Omega Therapeutics Omega Therapeutics, founded by Flagship Pioneering, is a clinical-stage biotechnology company pioneering the development of a new class of programmable epigenetic mRNA medicines. The Company's OMEGA platform harnesses the power of epigenetics, the mechanism that controls gene expression and every aspect of an organism's life from cell genesis, growth, and differentiation to cell death. Using a suite of technologies, paired with Omega's process of systematic, rational, and integrative drug design, the OMEGA platform enables control of fundamental epigenetic processes to correct the root cause of disease by returning aberrant gene expression to a normal range without altering native nucleic acid sequences. Omega's modular and programmable mRNA medicines, Omega Epigenomic Controllers™(OECs), target specific epigenomic loci within insulated genomic domains, EpiZips, from amongst thousands of unique, mapped, and validated genome-wide DNA sequences, with high specificity to durably tune single or multiple genes to treat and cure diseases through unprecedented precision epigenomic control. Omega’s approach enables pre-transcriptional control of most human genes including historically undruggable, intractable, and difficult to treat targets. Omega’s pipeline of OEC candidates spans a range of disease areas, including oncology, regenerative medicine, multigenic diseases including immunology, and select monogenic diseases.are Diseases and Orphan Drugs from the Accreditation Commission for Health Care. PANTHERx is now a five-time winner of the prestigious MMIT Patient Choice Award, including the 2022 honor. PANTHERx is headquartered in Pittsburgh, licensed in all 50 states, and holds accreditations from URAC, NABP, and ACHC.

Read More

INDUSTRIAL IMPACT, MEDICAL

Elemental Machines partners with System-c bioprocess to bring its IoT hardware solutions to the European biopharmaceutical industry

Prnewswire | March 30, 2023

Elemental Machines, developer of the Intelligent Operations Platform, has entered into a partnership with System-c bioprocess as an authorized reseller of Elemental Machines' hardware solutions, servicing the biopharmaceutical industry in France. The partnership will allow Elemental Machines to expand its footprint further in European markets. System-c serves as a partner and distributor for benchmark brands that cover a range of bioprocessing products from screening to filtration and beyond. Under the terms of the agreement, System-c will be an authorized reseller and distributor of Elemental Machines' suite of IoT-enabled sensors, which operators can use to collect and unify data across their biopharmaceutical facilities. "We are excited to partner with System-c and leverage their expertise in bringing innovative technology solutions to their bio-pharma customers,"said Dan Petkanas, Director of Global Alliances at Elemental Machines. "Together we will be able to deliver much needed tools to support and speed drug discovery." "We are thrilled to announce our partnership with Elemental Machines," said Yannick Carfantan, CEO of System-c. "Elemental Machines' leading technology platform for optimizing laboratory operations is going to give our company a new boost in our business development, not only for the French market but across Europe. Their measurement, information, alerting and data recovery technology opens new horizons for the control of laboratory equipment and R&D, pilot, and production departments enabling peace of mind for a number of actors in the biotech and biopharma industries. A winner for sure!" About System-c bioprocess As a partner and distributor for benchmark brands and continually searching for new innovative technologies, our product range covers all stages of the bioprocesses: from screening to filtration of the final product. Our solutions are always evolving in parallel with the new developments in life sciences, as well as the requirements for quality, safety and process productivity. Established in 1988 System-c bioprocess has been created to guarantee their customers complete, easy-to-use and customizable solutions to ensure productivity, reliability, and continuous traceability for all bioprocesses. Experts in innovative solutions and technologies for cell culture and fermentation processes, we have been constantly working with researchers, technicians and engineers from the pharmaceutical, biotechnology and agrifood industries for more than 30 years. About Elemental Machines Elemental Machines' Intelligent Operations Platform combines the best of IoT technology with purpose-built software solutions to deliver actionable intel to operators in the life sciences and byond. The Platform simplifies, optimizes, and augments operations by connecting the physical and digital infrastructure. Science and technology leaders trust Elemental Machines' ecosystem of hardware and software to deliver actionable insights that accelerate their pace of innovation.

Read More

INDUSTRIAL IMPACT, MEDICAL

Bicycle Therapeutics Announces a Strategic Collaboration with Novartis to Discover, Develop and Commercialize Bicycle® Radio-Conjugates

Businesswire | March 29, 2023

Bicycle Therapeutics plc a biotechnology company pioneering a new and differentiated class of therapeutics based on its proprietary bicyclic peptide (Bicycle®) technology, today announced that it has entered into a strategic collaboration agreement with Novartis to develop, manufacture and commercialize Bicycle® radio-conjugates (BRCs) for multiple agreed upon oncology targets. “This collaboration builds on the groundbreaking clinical work we have been doing in the toxin conjugate field and provides new and additional validation for this unique technology,” said Kevin Lee, Ph.D., Chief Executive Officer of Bicycle Therapeutics. “We look forward to working closely with Novartis to pioneer the discovery and development of potential new cutting-edge radiopharmaceutical cancer treatments based on Bicycles. We believe the properties of Bicycles make them well suited for the development of precision guided radiopharmaceuticals and represents the next leg in the application of our proprietary discovery platform in oncology.” Under the terms of the agreement, Bicycle will utilize its proprietary phage platform to discover Bicycles to be developed into BRCs. Novartis will be responsible for further development, manufacture and commercialization of the BRCs. Novartis will fund all pre-clinical and clinical development and commercialization activities. Bicycle will receive a $50 million upfront payment and is eligible for development and commercial-based milestone payments totaling up to $1.7 billion. Bicycle will also be eligible to receive tiered royalties on Bicycle-based medicines commercialized by Novartis. About Bicycle Therapeutics Bicycle Therapeutics is a clinical-stage biopharmaceutical company developing a novel class of medicines, referred to as Bicycles, for diseases that are underserved by existing therapeutics. Bicycles are fully synthetic short peptides constrained with small molecule scaffolds to form two loops that stabilize their structural geometry. This constraint facilitates target binding with high affinity and selectivity, making Bicycles attractive candidates for drug development. Bicycle is evaluating BT5528, a second-generation Bicycle Toxin Conjugate targeting EphA2; BT8009, a second-generation BTC targeting Nectin-4, a well-validated tumor antigen; and BT7480, a Bicycle TICA™ targeting Nectin-4 and agonizing CD137, in company-sponsored Phase I/II trials. In addition, BT1718, a BTC that targets MT1-MMP, is being investigated in an ongoing Phase I/IIa clinical trial sponsored by the Cancer Research UK Centre for Drug Development. Bicycle is headquartered in Cambridge, UK, with many key functions and members of its leadership team located in Cambridge, MA.

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