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Overcoming the barriers to cell and gene therapies

ATMPs are varied and complex, ranging from potential one-off curative treatments to treatments tailored to a specific individual. As such, they differ significantly from ‘traditional’ medicinal treatments, something which has been recognised by regulators in Europe: the European Parliament introduced ATMPs as a new class in 2007 and that same year saw the establishment of the European Medicines Agency’s Committee for Advanced Therapies (CAT) to address the specific demands of this growing class of medicines.

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Phage Biocontrol, Inc

Phage Biocontrol offers a green, non-toxic solution to the remediation of microbial induced problems in pipes, containers, tanks, pits, reservoirs and more by utilizing bacteriophage, natural bacterial predators.

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MedTech

How to Choose a Reliable Biotech Clinical Trial Management System?

Article | October 7, 2022

Introduction The medical and life-science industries are experiencing a robust transformation with the increasing prevalence of various types of diseases, including infectious diseases, chronic disorders, and acute conditions around the world. As a result, a significant rise in demand for more effective therapeutic drugs and bionics is being witnessed, leading to a swift increase in the number of clinical trials. For a successful trial, it is important for biotech companies to ensure the data submitted to regulatory bodies regarding clinical trials is accurate, reliable, and definitive from an ethical point of view. A reliable clinical trial management system plays a vital role in collecting, monitoring, and managing clinical data. The availability of high-quality clinical data also helps clinical research institutions make efficient treatment decisions and provide proper patient care. Hence, a number of biotech companies and research organizations are focusing on leveraging innovative clinical trial management solutions to handle a large amount of data, particularly in multi-center trials, and generate reliable, high-quality, and statistically sound data from clinical trials. However, selecting the most appropriate and reliable clinical trial management system is vital for the clinical trial's success. Let's see some of the steps that will assist these firms in choosing the right CTMS. Key Steps for Selecting Right Biotech Clinical Trial Management System Prioritize Study Needs Considering and prioritizing study needs is a crucial step in choosing the most reliable clinical trial management system for biotech companies. Prioritizing helps them to identify a solution that improves the study's quality and removes uncertainty for researchers when faced with difficult choices. Hence, biotech and life-science organizations should choose a clinical trial system that is simple to use, well-organized, and suitably designed to minimize the number of clicks required to complete a task. Select CTMS with Multiple Integrations Integrated clinical trial management systems provide the best value for the companies’ funds as they guarantee the smooth functioning of research protocols. In addition, integrations are necessary to fully understand the importance and advantages of clinical trial management software for ensuring smooth transitions between site management and data collection. Biotech and clinical research should look for CTMS platforms that can integrate with electronic medical record (EMR) platforms and clinical research process content (CRPC) billing grids. This will allow them to use the same billing designations and ensure compliance while minimizing the need for duplicate processes. Ensure System Compliance and Security Clinical research organizations need to adhere to a plethora of complex regulations in order to ensure compliance with one of the most challenging environments of principles, which is information security and privacy. Security and system compliance are vital aspects of choosing the right CTMS solutions for biotech firms as they assist in building trust and form a part of the system’s duties. While selecting CTMS systems, it is essential for companies engaged in clinical research to ensure that these platforms are able to configure both, group and individual permissions, along with having a data backup and recovery plan for hosted systems. This will allow companies to assess the privacy and security implications of research and anticipate complications that may arise in each phase of the project. Assess the Scalability Choosing a scalable CTMS that can accommodate various types of fluctuations and expansions enables biotech and clinical firms to quickly adapt to fast-changing trends and demand spikes while reducing maintenance costs and enhancing user agility. As scalability also means secure and expanded data storage, these businesses should instead use SaaS solutions than manually manage an ever-growing collection of hard drives. The right CTMS ensures accommodating the firm’s availability requirements without incurring the capital costs associated with expanding a physical infrastructure. The Closing Thought A well-executed and successful clinical trial involves multiple stages and processes. Several quality controls and stringent adherence to regulations are essential for the steps, along with efficient cross-departmental processes and procedures. Incorporating the right CTMS paves the way for paperless data collection, regulatory filing, and fiscal management tools for biotech researchers and administrative personnel.

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MedTech

Making Predictions by Digitizing Bioprocessing

Article | July 16, 2022

With advances in data analytics and machine learning, the move from descriptive and diagnostic analytics to predictive and prescriptive analytics and controls—allowing us to better forecast and understand what will happen and thus optimize process outcomes—is not only feasible but inevitable, according to Bonnie Shum, principal engineer, pharma technical innovation, technology & manufacturing sciences and technology at Genentech. “Well-trained artificial intelligence systems can help drive better decision making and how data is analyzed from drug discovery to process development and to manufacturing processes,” she says. Those advances, though, only really matter when they improve the lives of patients. That’s exactly what Shum expects. “The convergence of digital transformation and operational/processing changes will be critical for the facilities of the future and meeting the needs of our patients,” she continues. “Digital solutions may one day provide fully automated bioprocessing, eliminating manual intervention and enabling us to anticipate potential process deviations to prevent process failures, leading to real-time release and thus faster access for patients.” To turn Bioprocessing 4.0 into a production line for precision healthcare, real-time release and quickly manufacturing personalized medicines will be critical. Adding digitization and advanced analytics wherever possible will drive those improvements. In fact, many of these improvements, especially moving from descriptive to predictive bioprocessing, depend on more digitization.

Read More
Medical

2 Small-Cap Biotech Stocks You Haven't Heard of, But Should Know About

Article | August 16, 2022

With everything that's going on with the COVID-19 pandemic, many healthcare companies have grabbed plenty of spotlight during these challenging times. At the same time, a number of otherwise promising businesses have slipped under the radar. That's especially true for small-cap biotech stocks that aren't actively involved in developing tests, vaccines or treatments for COVID-19. Vaccine developers, protective equipment producers, and healthcare service providers are all attracting plenty of attention during this pandemic, but there are just as many promising biotech stocks that aren't involved in these areas. Here are two such companies that you might have missed, but they deserve a spot on your watch list.

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MedTech

Next-Gen Genetics Cancer Therapies Creating Investment Prospects

Article | July 5, 2022

Genetic therapeutics such as genetic engineering and gene therapy are increasingly emerging as one of the most influential and transformed biotechnological solutions around the globe in recent times. These genetic solutions are being assessed across various medical domains, including cancer treatment, neurology, oncology, and ophthalmology. Citing the trend, the genetics industry is estimated to experience a tsunami of approvals, with over 1,000 cell and gene therapy clinical trials currently underway and over 900 companies worldwide focusing on these cutting-edge therapies. Growing Cancer Encourages Advancements in Genetic Technologies With the surging cases of cancers such as leukemias, carcinomas, lymphomas, and others, patients worldwide are increasing their spending on adopting novel therapeutic solutions for non-recurring treatment of the disease, such as gene therapy, genetic engineering, T-cell therapy, and gene editing. As per a study by the Fight Cancer Organization, spending on the treatment of cancer increased to $200.7 billion, and the amount is anticipated to exceed $245 billion by the end of 2030. Growing revenue prospects are encouraging biotechnology and biopharmaceutical companies to develop novel genetic solutions for cancer treatment. For instance, Bristol-Myers Squibb K.K., a Japanese pharmaceutical company, introduced a B-cell maturation antigen (BCMA)-directed chimeric antigen receptor (CAR) T cell immunotherapy, Abecma, for the treatment of relapsed or refractory (R/R) multiple myeloma in 2022. Amid a New Market: Genetics Will Attract Massive Investments Despite several developments and technological advancements, genetics is still considered to be in a nascent stage, providing significant prospects for growth to the companies that are already operating in the domain. Genetics solutions such as gene therapies, gene editing, and T-cell immunotherapy are emerging as highly active treatments across various medical fields, resulting in increasing research and development activities across the domain, drawing significant attention from investors. Given the potential of genetic treatments and the focus on finding new ways to treat cancer and other related diseases, it's easy to understand why companies are investing in the domain. For instance, Pfizer has recently announced an investment of around $800 million to construct development facilities supporting gene therapy manufacturing from initial preclinical research through final commercial-scale production. Due to these advancements, cell and gene therapies are forecast to grow from $4 billion annually to more than $45 billion, exhibiting growth at a 63% CAGR. The Future of Genetics Though there is a significant rise in advancement in genetic technologies and developments, the number of approved genetic treatments remains extremely small. However, with gene transfer and CRISPR solutions emerging as new modalities for cancer treatment, the start-up companies will attract a growing amount and proportion of private and public investments. This is expected present a tremendous opportunity for biopharma and biotechnology investors to help fund and benefit from the medical industry's shift from traditional treatments to cutting-edge genetic therapeutics in the coming years.

Read More
MedTech

How to Choose a Reliable Biotech Clinical Trial Management System?

Article | October 7, 2022

Introduction The medical and life-science industries are experiencing a robust transformation with the increasing prevalence of various types of diseases, including infectious diseases, chronic disorders, and acute conditions around the world. As a result, a significant rise in demand for more effective therapeutic drugs and bionics is being witnessed, leading to a swift increase in the number of clinical trials. For a successful trial, it is important for biotech companies to ensure the data submitted to regulatory bodies regarding clinical trials is accurate, reliable, and definitive from an ethical point of view. A reliable clinical trial management system plays a vital role in collecting, monitoring, and managing clinical data. The availability of high-quality clinical data also helps clinical research institutions make efficient treatment decisions and provide proper patient care. Hence, a number of biotech companies and research organizations are focusing on leveraging innovative clinical trial management solutions to handle a large amount of data, particularly in multi-center trials, and generate reliable, high-quality, and statistically sound data from clinical trials. However, selecting the most appropriate and reliable clinical trial management system is vital for the clinical trial's success. Let's see some of the steps that will assist these firms in choosing the right CTMS. Key Steps for Selecting Right Biotech Clinical Trial Management System Prioritize Study Needs Considering and prioritizing study needs is a crucial step in choosing the most reliable clinical trial management system for biotech companies. Prioritizing helps them to identify a solution that improves the study's quality and removes uncertainty for researchers when faced with difficult choices. Hence, biotech and life-science organizations should choose a clinical trial system that is simple to use, well-organized, and suitably designed to minimize the number of clicks required to complete a task. Select CTMS with Multiple Integrations Integrated clinical trial management systems provide the best value for the companies’ funds as they guarantee the smooth functioning of research protocols. In addition, integrations are necessary to fully understand the importance and advantages of clinical trial management software for ensuring smooth transitions between site management and data collection. Biotech and clinical research should look for CTMS platforms that can integrate with electronic medical record (EMR) platforms and clinical research process content (CRPC) billing grids. This will allow them to use the same billing designations and ensure compliance while minimizing the need for duplicate processes. Ensure System Compliance and Security Clinical research organizations need to adhere to a plethora of complex regulations in order to ensure compliance with one of the most challenging environments of principles, which is information security and privacy. Security and system compliance are vital aspects of choosing the right CTMS solutions for biotech firms as they assist in building trust and form a part of the system’s duties. While selecting CTMS systems, it is essential for companies engaged in clinical research to ensure that these platforms are able to configure both, group and individual permissions, along with having a data backup and recovery plan for hosted systems. This will allow companies to assess the privacy and security implications of research and anticipate complications that may arise in each phase of the project. Assess the Scalability Choosing a scalable CTMS that can accommodate various types of fluctuations and expansions enables biotech and clinical firms to quickly adapt to fast-changing trends and demand spikes while reducing maintenance costs and enhancing user agility. As scalability also means secure and expanded data storage, these businesses should instead use SaaS solutions than manually manage an ever-growing collection of hard drives. The right CTMS ensures accommodating the firm’s availability requirements without incurring the capital costs associated with expanding a physical infrastructure. The Closing Thought A well-executed and successful clinical trial involves multiple stages and processes. Several quality controls and stringent adherence to regulations are essential for the steps, along with efficient cross-departmental processes and procedures. Incorporating the right CTMS paves the way for paperless data collection, regulatory filing, and fiscal management tools for biotech researchers and administrative personnel.

Read More
MedTech

Immunology: A New Frontier in Medical Science

Article | July 16, 2022

With advances in data analytics and machine learning, the move from descriptive and diagnostic analytics to predictive and prescriptive analytics and controls—allowing us to better forecast and understand what will happen and thus optimize process outcomes—is not only feasible but inevitable, according to Bonnie Shum, principal engineer, pharma technical innovation, technology & manufacturing sciences and technology at Genentech. “Well-trained artificial intelligence systems can help drive better decision making and how data is analyzed from drug discovery to process development and to manufacturing processes,” she says. Those advances, though, only really matter when they improve the lives of patients. That’s exactly what Shum expects. “The convergence of digital transformation and operational/processing changes will be critical for the facilities of the future and meeting the needs of our patients,” she continues. “Digital solutions may one day provide fully automated bioprocessing, eliminating manual intervention and enabling us to anticipate potential process deviations to prevent process failures, leading to real-time release and thus faster access for patients.” To turn Bioprocessing 4.0 into a production line for precision healthcare, real-time release and quickly manufacturing personalized medicines will be critical. Adding digitization and advanced analytics wherever possible will drive those improvements. In fact, many of these improvements, especially moving from descriptive to predictive bioprocessing, depend on more digitization.

Read More
Medical

Laboratory Information Management System for Biotech Labs: Significance & Benefits

Article | August 16, 2022

With everything that's going on with the COVID-19 pandemic, many healthcare companies have grabbed plenty of spotlight during these challenging times. At the same time, a number of otherwise promising businesses have slipped under the radar. That's especially true for small-cap biotech stocks that aren't actively involved in developing tests, vaccines or treatments for COVID-19. Vaccine developers, protective equipment producers, and healthcare service providers are all attracting plenty of attention during this pandemic, but there are just as many promising biotech stocks that aren't involved in these areas. Here are two such companies that you might have missed, but they deserve a spot on your watch list.

Read More
MedTech

Next-Gen Genetics Cancer Therapies Creating Investment Prospects

Article | July 5, 2022

Genetic therapeutics such as genetic engineering and gene therapy are increasingly emerging as one of the most influential and transformed biotechnological solutions around the globe in recent times. These genetic solutions are being assessed across various medical domains, including cancer treatment, neurology, oncology, and ophthalmology. Citing the trend, the genetics industry is estimated to experience a tsunami of approvals, with over 1,000 cell and gene therapy clinical trials currently underway and over 900 companies worldwide focusing on these cutting-edge therapies. Growing Cancer Encourages Advancements in Genetic Technologies With the surging cases of cancers such as leukemias, carcinomas, lymphomas, and others, patients worldwide are increasing their spending on adopting novel therapeutic solutions for non-recurring treatment of the disease, such as gene therapy, genetic engineering, T-cell therapy, and gene editing. As per a study by the Fight Cancer Organization, spending on the treatment of cancer increased to $200.7 billion, and the amount is anticipated to exceed $245 billion by the end of 2030. Growing revenue prospects are encouraging biotechnology and biopharmaceutical companies to develop novel genetic solutions for cancer treatment. For instance, Bristol-Myers Squibb K.K., a Japanese pharmaceutical company, introduced a B-cell maturation antigen (BCMA)-directed chimeric antigen receptor (CAR) T cell immunotherapy, Abecma, for the treatment of relapsed or refractory (R/R) multiple myeloma in 2022. Amid a New Market: Genetics Will Attract Massive Investments Despite several developments and technological advancements, genetics is still considered to be in a nascent stage, providing significant prospects for growth to the companies that are already operating in the domain. Genetics solutions such as gene therapies, gene editing, and T-cell immunotherapy are emerging as highly active treatments across various medical fields, resulting in increasing research and development activities across the domain, drawing significant attention from investors. Given the potential of genetic treatments and the focus on finding new ways to treat cancer and other related diseases, it's easy to understand why companies are investing in the domain. For instance, Pfizer has recently announced an investment of around $800 million to construct development facilities supporting gene therapy manufacturing from initial preclinical research through final commercial-scale production. Due to these advancements, cell and gene therapies are forecast to grow from $4 billion annually to more than $45 billion, exhibiting growth at a 63% CAGR. The Future of Genetics Though there is a significant rise in advancement in genetic technologies and developments, the number of approved genetic treatments remains extremely small. However, with gene transfer and CRISPR solutions emerging as new modalities for cancer treatment, the start-up companies will attract a growing amount and proportion of private and public investments. This is expected present a tremendous opportunity for biopharma and biotechnology investors to help fund and benefit from the medical industry's shift from traditional treatments to cutting-edge genetic therapeutics in the coming years.

Read More
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Spotlight

Phage Biocontrol, Inc

Phage Biocontrol offers a green, non-toxic solution to the remediation of microbial induced problems in pipes, containers, tanks, pits, reservoirs and more by utilizing bacteriophage, natural bacterial predators.

Related News

Cell and Gene Therapy, Diagnostics

NorthX Biologics, a leading Nordic development and manufacturing organisation announces acquisition of a biologics manufacturing unit from Valneva

Globenewswire | July 04, 2023

NorthX Biologics (‘NorthX’), a leading Nordic development and manufacturing organisation with a focus on advanced biologics, CGT (cell and gene therapy) and vaccines, announced today the successful acquisition of the Stockholm-based Clinical Trial Manufacturing unit from Valneva Sweden, significantly expanding capabilities. The acquisition includes the transfer of a multi-purpose facility, situated in the Stockholm life science cluster, close to Karolinska University Hospital. In addition, 30 staff members who currently operate the facility will also join NorthX. The site and staff have a long history with extensive experience of serving both Valneva internally and also working with external customers on a contract development and manufacturing basis. With expertise in mammalian expression systems and viral vectors, the capabilities complement those of NorthX’s existing business of advanced microbial based manufacturing of proteins and plasmid DNA. The acquired unit excels in process development, scale up, GMP production, quality control analytics, and quality assurance/release and is capable of working with Biosafety Level (BSL) 2/2+ and BSL 3 organisms. With this expansion, NorthX enhances its capabilities and can offer comprehensive services to a wider range of clients globally. Janet Hoogstraate, currently Managing Director of Valneva Sweden, will join the NorthX team. She commented, “I am very proud when looking back at what we have achieved within the unit over the past years and look forward with great enthusiasm to build on NorthX’s position as the go-to manufacturer of advanced biologics in Northern Europe.” Helena Strigård, CEO of NorthX, said, “We are delighted to join forces with our new colleagues in Stockholm to bring new innovative treatments to tomorrow’s patients.” Thomas Eldered, Chairman of NorthX, commented, “This strategic move marks a significant milestone in our growth journey and strengthens NorthX as Sweden’s Innovation Hub. We are now able to work with ATMPs and advanced biologics, including process development and manufacture for clinical trials and commercial requirements.” ABOUT NORTHX BIOLOGICS NorthX Biologics develops and manufactures advanced biologics and has over 30 years of GMP production experience. The team provides process development and GMP manufacturing services with expertise in plasmid DNA, mRNA, proteins, cell therapy and other advanced biologics. Headquartered in the heart of Sweden, the team serves customers worldwide. In 2021 NorthX was recognised and appointed as the national innovation hub for GMP manufacture of advanced therapeutics and vaccines by the Swedish Government and Vinnova, Sweden's innovation agency. NorthX has the ambition to become a leading cell and gene therapy manufacturer and partner of choice for innovative drug development companies. For more information visit www.nxbio.com.

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Cell and Gene Therapy, AI

BenevolentAI Progresses BEN-34712 for the Potential Treatment of ALS into IND-Enabling Studies

Businesswire | June 05, 2023

BenevolentAI, a leader in the development of cutting-edge AI that accelerates biopharma discovery, announces the successful delivery of its pre-clinical candidate for the potential treatment of amyotrophic lateral sclerosis (ALS), BEN-34712. BEN-34712 is an oral, potent and selective brain penetrant RARɑβ (retinoic acid receptor alpha beta) biased agonist and will now enter investigational new drug (IND)-enabling studies. Impaired retinoic acid signalling has been shown to result in neuroinflammation, oxidative stress and mitochondrial dysfunction, all hallmarks of ALS. In preclinical studies conducted by the Company, BEN-34712 was neuroprotective in a patient-derived, disease-relevant in vitro motor neuron/iAstrocyte co-culture model, demonstrating significant efficacy in both sporadic and familial subtypes of ALS. In addition, BEN-34712 has demonstrated both central nervous system (CNS) target engagement and functional protective effects in the SOD1G93A mouse model of ALS after 50-day repeat dosing. BenevolentAI collaborated with the Sheffield Institute for Translational Neuroscience (SITraN) at the University of Sheffield on this programme, utilising their patient-derived motor neuron/iAstrocyte co-culture systems and in vivo model expertise. Anne Phelan, Chief Scientific Officer, BenevolentAI, said: “There remains a significant and urgent need for new and alternative therapies for patients with ALS. We are pleased by the promising advancement of our drug candidate, BEN-34712, towards clinical development, backed by the compelling preclinical data generated by our collaborators at SITraN.” Richard Mead, Senior Lecturer in Translational Neuroscience at SITraN, commented: "ALS patients suffering from this devastating neurodegenerative disease are in dire need of effective therapy, with the current standard of care options focusing on symptom management or offering limited clinical benefit. We believe BEN-34712 represents an exciting development in our research for a potential new treatment, particularly as it shows effectiveness in both the SOD1G93A mouse model system as well as familial and C9orf72 related ALS patient-derived cell models." About BenevolentAI BenevolentAI is a leading developer of advanced artificial intelligence technologies that unlock the value of multimodal data, surface novel insights, and accelerate biomedical discovery. Through the combined capabilities of its AI platform, its scientific expertise, and wet-lab facilities, the Company is developing an in-house drug pipeline of high-value assets. The Company is headquartered in London, with a research facility in Cambridge (UK) and a further office in New York. About ALS ALS is a progressive neurologic disorder characterised by the loss of cortical and spinal motor neurons, leading to the denervation of nerve endplates, axonal retraction and subsequent muscle atrophy. The average survival time following the initial diagnosis is around two-three years, and while there are drugs approved by the US FDA for ALS, they provide only modest benefits to patients, underwriting the urgent need for new and alternative therapies. About SITraN at the University of Sheffield The Sheffield Institute for Translational Neuroscience (SITraN) is an essential development in the fight against motor neurone disease and other common neurodegenerative disorders, including Parkinson's and dementia, as well as stroke and multiple sclerosis. SITraN has the potential to bring new treatments and new hope to patients and carers in the UK and worldwide, by significantly accelerating the pace of therapeutic development using technologies such as experimental modelling of disease, gene therapy and stem cell biology, gene expression profiling and bioinformatics analysis and modelling of the biological processes. Since its opening by Queen Elizabeth II in 2010, SITraN has grown immensely and developed into a leading global facility which is at the forefront of research and expertise.

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Cell and Gene Therapy

Mission Bio Develops Single-Cell Solution to Address Challenges in Genome Editing

PR Newswire | May 12, 2023

Mission Bio, the single-cell DNA and multi-omics company, announced today the Tapestri® Genome Editing Solution, an end-to-end product for genome editing analysis. The product will be previewed next week at the American Society of Gene and Cell Therapy Conference (ASGCT) 26th Annual Meeting. By enabling robust single-cell insights impacting both efficacy and safety, the solution will be a powerful analytical tool for developing the next generation of gene-edited therapies. The first CRISPR-modified therapy is now under regulatory review, and many similar cell-based therapies are expected to follow for multiple intractable diseases. However, genome editing can result in complex, heterogeneous mixtures of edits that make it challenging to apply a level of process control over genome-edited cell products. The Tapestri® Genome Editing Solution addresses these challenges by measuring gene editing outcomes at single-cell resolution, capturing the co-occurrence of on- and off-target edits, as well as the zygosity of edits, which conventional bulk analyses cannot. Additionally, this analysis can be completed within days by processing thousands of cells at a time without any prior selection, while conventional analytical methods require months for clonal outgrowth. An early iteration of the Tapestri® Genome Editing Solution is currently being tested by key genome editing researchers and leading cell therapy developers in academia and industry, who are providing vital feedback on the analysis. Mission Bio recently collaborated with the National Institute of Standards and Technology (NIST) in the Genome Editing Consortium, which provided qualified samples to collaborators to assess technologies that report variant size and frequency within a mixed cell population. Samantha Maragh, NIST Genome Editing Program Leader, will present results of the study at 12:00 p.m. PT on May 17 (Poster 533) at the ASGCT Annual Meeting. "We look forward to pulling back the curtain on our end-to-end Genome Editing Solution at ASGCT," said Todd Druley, MD, PhD, Chief Medical Officer at Mission Bio. "The data acquired under the Genome Editing Consortium further demonstrates the Tapestri® Platform's potential as a standard analysis tool within the genome editing community. Given the heterogeneous results of gene editing strategies, there is a great need to address both industry and regulatory genome editing concerns with a consistent and highly precise technology for accurately measuring gene editing outcomes, and our new offering will be a complete solution to do just that." About Mission Bio Mission Bio is a life sciences company that accelerates discoveries and cures for a wide range of diseases by equipping researchers with the tools they need to better measure and predict our resistance and response to new therapies. Mission Bio's multi-omics approach improves time-to-market for new therapeutics, including innovative cell and gene therapies that provide new pathways to health. Founded in 2014, Mission Bio has secured investment from Novo Growth, Cota Capital, Agilent Technologies, Mayfield Fund, and others. The company's Tapestri® Platform gives researchers around the globe the power to interrogate every molecule in a cell together, providing a comprehensive understanding of activity from a single sample. Tapestri® is the only commercialized multi-omics platform capable of analyzing DNA and protein simultaneously from the same sample at single-cell resolution. The Tapestri® Platform is being utilized by customers at leading research centers, pharmaceutical, and diagnostics companies worldwide to develop treatments and eventually cures for cancer.

Read More

Cell and Gene Therapy, Diagnostics

NorthX Biologics, a leading Nordic development and manufacturing organisation announces acquisition of a biologics manufacturing unit from Valneva

Globenewswire | July 04, 2023

NorthX Biologics (‘NorthX’), a leading Nordic development and manufacturing organisation with a focus on advanced biologics, CGT (cell and gene therapy) and vaccines, announced today the successful acquisition of the Stockholm-based Clinical Trial Manufacturing unit from Valneva Sweden, significantly expanding capabilities. The acquisition includes the transfer of a multi-purpose facility, situated in the Stockholm life science cluster, close to Karolinska University Hospital. In addition, 30 staff members who currently operate the facility will also join NorthX. The site and staff have a long history with extensive experience of serving both Valneva internally and also working with external customers on a contract development and manufacturing basis. With expertise in mammalian expression systems and viral vectors, the capabilities complement those of NorthX’s existing business of advanced microbial based manufacturing of proteins and plasmid DNA. The acquired unit excels in process development, scale up, GMP production, quality control analytics, and quality assurance/release and is capable of working with Biosafety Level (BSL) 2/2+ and BSL 3 organisms. With this expansion, NorthX enhances its capabilities and can offer comprehensive services to a wider range of clients globally. Janet Hoogstraate, currently Managing Director of Valneva Sweden, will join the NorthX team. She commented, “I am very proud when looking back at what we have achieved within the unit over the past years and look forward with great enthusiasm to build on NorthX’s position as the go-to manufacturer of advanced biologics in Northern Europe.” Helena Strigård, CEO of NorthX, said, “We are delighted to join forces with our new colleagues in Stockholm to bring new innovative treatments to tomorrow’s patients.” Thomas Eldered, Chairman of NorthX, commented, “This strategic move marks a significant milestone in our growth journey and strengthens NorthX as Sweden’s Innovation Hub. We are now able to work with ATMPs and advanced biologics, including process development and manufacture for clinical trials and commercial requirements.” ABOUT NORTHX BIOLOGICS NorthX Biologics develops and manufactures advanced biologics and has over 30 years of GMP production experience. The team provides process development and GMP manufacturing services with expertise in plasmid DNA, mRNA, proteins, cell therapy and other advanced biologics. Headquartered in the heart of Sweden, the team serves customers worldwide. In 2021 NorthX was recognised and appointed as the national innovation hub for GMP manufacture of advanced therapeutics and vaccines by the Swedish Government and Vinnova, Sweden's innovation agency. NorthX has the ambition to become a leading cell and gene therapy manufacturer and partner of choice for innovative drug development companies. For more information visit www.nxbio.com.

Read More

Cell and Gene Therapy, AI

BenevolentAI Progresses BEN-34712 for the Potential Treatment of ALS into IND-Enabling Studies

Businesswire | June 05, 2023

BenevolentAI, a leader in the development of cutting-edge AI that accelerates biopharma discovery, announces the successful delivery of its pre-clinical candidate for the potential treatment of amyotrophic lateral sclerosis (ALS), BEN-34712. BEN-34712 is an oral, potent and selective brain penetrant RARɑβ (retinoic acid receptor alpha beta) biased agonist and will now enter investigational new drug (IND)-enabling studies. Impaired retinoic acid signalling has been shown to result in neuroinflammation, oxidative stress and mitochondrial dysfunction, all hallmarks of ALS. In preclinical studies conducted by the Company, BEN-34712 was neuroprotective in a patient-derived, disease-relevant in vitro motor neuron/iAstrocyte co-culture model, demonstrating significant efficacy in both sporadic and familial subtypes of ALS. In addition, BEN-34712 has demonstrated both central nervous system (CNS) target engagement and functional protective effects in the SOD1G93A mouse model of ALS after 50-day repeat dosing. BenevolentAI collaborated with the Sheffield Institute for Translational Neuroscience (SITraN) at the University of Sheffield on this programme, utilising their patient-derived motor neuron/iAstrocyte co-culture systems and in vivo model expertise. Anne Phelan, Chief Scientific Officer, BenevolentAI, said: “There remains a significant and urgent need for new and alternative therapies for patients with ALS. We are pleased by the promising advancement of our drug candidate, BEN-34712, towards clinical development, backed by the compelling preclinical data generated by our collaborators at SITraN.” Richard Mead, Senior Lecturer in Translational Neuroscience at SITraN, commented: "ALS patients suffering from this devastating neurodegenerative disease are in dire need of effective therapy, with the current standard of care options focusing on symptom management or offering limited clinical benefit. We believe BEN-34712 represents an exciting development in our research for a potential new treatment, particularly as it shows effectiveness in both the SOD1G93A mouse model system as well as familial and C9orf72 related ALS patient-derived cell models." About BenevolentAI BenevolentAI is a leading developer of advanced artificial intelligence technologies that unlock the value of multimodal data, surface novel insights, and accelerate biomedical discovery. Through the combined capabilities of its AI platform, its scientific expertise, and wet-lab facilities, the Company is developing an in-house drug pipeline of high-value assets. The Company is headquartered in London, with a research facility in Cambridge (UK) and a further office in New York. About ALS ALS is a progressive neurologic disorder characterised by the loss of cortical and spinal motor neurons, leading to the denervation of nerve endplates, axonal retraction and subsequent muscle atrophy. The average survival time following the initial diagnosis is around two-three years, and while there are drugs approved by the US FDA for ALS, they provide only modest benefits to patients, underwriting the urgent need for new and alternative therapies. About SITraN at the University of Sheffield The Sheffield Institute for Translational Neuroscience (SITraN) is an essential development in the fight against motor neurone disease and other common neurodegenerative disorders, including Parkinson's and dementia, as well as stroke and multiple sclerosis. SITraN has the potential to bring new treatments and new hope to patients and carers in the UK and worldwide, by significantly accelerating the pace of therapeutic development using technologies such as experimental modelling of disease, gene therapy and stem cell biology, gene expression profiling and bioinformatics analysis and modelling of the biological processes. Since its opening by Queen Elizabeth II in 2010, SITraN has grown immensely and developed into a leading global facility which is at the forefront of research and expertise.

Read More

Cell and Gene Therapy

Mission Bio Develops Single-Cell Solution to Address Challenges in Genome Editing

PR Newswire | May 12, 2023

Mission Bio, the single-cell DNA and multi-omics company, announced today the Tapestri® Genome Editing Solution, an end-to-end product for genome editing analysis. The product will be previewed next week at the American Society of Gene and Cell Therapy Conference (ASGCT) 26th Annual Meeting. By enabling robust single-cell insights impacting both efficacy and safety, the solution will be a powerful analytical tool for developing the next generation of gene-edited therapies. The first CRISPR-modified therapy is now under regulatory review, and many similar cell-based therapies are expected to follow for multiple intractable diseases. However, genome editing can result in complex, heterogeneous mixtures of edits that make it challenging to apply a level of process control over genome-edited cell products. The Tapestri® Genome Editing Solution addresses these challenges by measuring gene editing outcomes at single-cell resolution, capturing the co-occurrence of on- and off-target edits, as well as the zygosity of edits, which conventional bulk analyses cannot. Additionally, this analysis can be completed within days by processing thousands of cells at a time without any prior selection, while conventional analytical methods require months for clonal outgrowth. An early iteration of the Tapestri® Genome Editing Solution is currently being tested by key genome editing researchers and leading cell therapy developers in academia and industry, who are providing vital feedback on the analysis. Mission Bio recently collaborated with the National Institute of Standards and Technology (NIST) in the Genome Editing Consortium, which provided qualified samples to collaborators to assess technologies that report variant size and frequency within a mixed cell population. Samantha Maragh, NIST Genome Editing Program Leader, will present results of the study at 12:00 p.m. PT on May 17 (Poster 533) at the ASGCT Annual Meeting. "We look forward to pulling back the curtain on our end-to-end Genome Editing Solution at ASGCT," said Todd Druley, MD, PhD, Chief Medical Officer at Mission Bio. "The data acquired under the Genome Editing Consortium further demonstrates the Tapestri® Platform's potential as a standard analysis tool within the genome editing community. Given the heterogeneous results of gene editing strategies, there is a great need to address both industry and regulatory genome editing concerns with a consistent and highly precise technology for accurately measuring gene editing outcomes, and our new offering will be a complete solution to do just that." About Mission Bio Mission Bio is a life sciences company that accelerates discoveries and cures for a wide range of diseases by equipping researchers with the tools they need to better measure and predict our resistance and response to new therapies. Mission Bio's multi-omics approach improves time-to-market for new therapeutics, including innovative cell and gene therapies that provide new pathways to health. Founded in 2014, Mission Bio has secured investment from Novo Growth, Cota Capital, Agilent Technologies, Mayfield Fund, and others. The company's Tapestri® Platform gives researchers around the globe the power to interrogate every molecule in a cell together, providing a comprehensive understanding of activity from a single sample. Tapestri® is the only commercialized multi-omics platform capable of analyzing DNA and protein simultaneously from the same sample at single-cell resolution. The Tapestri® Platform is being utilized by customers at leading research centers, pharmaceutical, and diagnostics companies worldwide to develop treatments and eventually cures for cancer.

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