Next-Gen Gene Therapy to Counter Complex Diseases

Kutubkhan Bohari | July 13, 2022 | 844 views | Read Time : 1 min

Next-Gen Gene
Gene therapy has historically been used to treat disorders with in-depth knowledge caused by a single genetic mutation. Thanks to the introduction of new generation technologies, the potential of gene therapy is expanding tAo treat diseases that were previously untreatable.

Evolution of Gene Therapy

One of the major success stories of the twenty-first century has been gene therapy. However, it has not been the same in the past. The field's journey to this point has been long and mostly difficult, with both tragedy and triumph along the way.

Initially, genetic disorders were thought to be untreatable and permanently carved into the genomes of individuals unfortunate enough to be born with them. But due to the constant technological advancement and research activities, gene therapy now has the potential to treat various genetic mutation-causing diseases with its ability to insert a new copy and replace faulty genes.


Gene Therapy is Finding New Roads in the Medical Sector

Gene therapy can help researchers treat a variety of conditions that fall under the general heading of epilepsy, instead of only focusing on a particular kind of disorder brought on by a genetic mutation. Following are some of the domains transformed by gene therapy.

Neurology – Gene therapy can be used for the treatment of seizures by directly injecting it into the area causing an uncontrolled electrical disturbance in the brain. Furthermore, by using DNA sequences known as promoters, gene therapy can be restricted to specific neurons within that area.

Ophthalmology – Genetic conditions such as blindness can be caused due to the mutation of any gene out of over 200 and resulting in progressive vision loss in children. With advanced gene therapies such as optogenetics, lost photoreceptor function can be transferred to the retinal cells, which are responsible for relaying visual information to the brain. This might give patients the ability to navigate in an unknown environment with a certain level of autonomy.


The Future of Gene Therapy

The news surrounding gene therapy has been largely favorable over the past few years, with treatment after treatment obtaining regulatory approvals, successful clinical trials, and garnering significant funds to begin development. With more than 1,000 clinical trials presently underway, the long-awaited gene therapy revolution might finally be here.

Spotlight

XBiotech USA, Inc.

XBiotech has established a current good manufacturing practices (cGMP) program to commercialize its lead product candidate. The Company has purchased land and designed a commercial-scale plant with 25,000L total bioreactor capacity. Construction of the facility is planned to commence in 4th quarter 2011..

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How to Choose a Reliable Biotech Clinical Trial Management System?

Article | July 11, 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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Top 3 Biotech Clinical Data Management Trends to Watch in 2022

Article | July 16, 2022

Introduction The administration of medical records and data has advanced significantly during the past few decades. Clinical data management, which was once only a small subset of biotech research organizations, has now developed into a mission-critical, specialized unit. In the late 1990s, electronic data capture (EDC) began to alter the traditional function of clinical data management. After that, the data configuration and management of data queries for the EDC system fell under the purview of clinical data management services. Today, clinical data management is not only responsible for managing the clinical data configuration and data queries but also developing and implementing data administration plans, ensuring data accuracy and completeness, and maintaining optimum data security. In recent years, as digital technologies have gained acceptance around the globe, data has become a vital aspect in decision-making across numerous industries, and the life sciences and biotechnology sectors are no exception. Using data has provided granular insights to biotech organizations, assisting them in creating breakthroughs in drug development and medical research and signifying the importance of clinical trial management systems in these medical verticals. The Biggest Biotech Clinical Data Management Trends to Know About Today The future of clinical data management is contingent upon the implementation of systems and regulations. It is imperative for all organizations participating in a medical or life science trial to have transparent rules in place for sharing and retaining patient data. Also, there is a need to have a standardized format for maintaining these records and documents related to trials. This assists biotech organizations in reducing the chances of ambiguity regarding who owns what kind of data or paperwork at any given time. Over the past couple of years, the focus of the life science and biotechnology industries has shifted towards developing more effective medications and therapies, implementing personalized treatment, and finding cures for diseases such as cancer and AIDS. In response to this, a substantial rise in the number of clinical trials is being witnessed globally. As the number of clinical trials continues to accelerate, the spending on these trials rises as well. In response to this, the worldwide cost of conducting clinical trials is anticipated to reach US$ 49.80 billion in 2022. With the transition of the world from traditional to digital, medical professionals and biotech businesses are increasingly shifting towards adopting high-tech and reliable clinical trial management systems for various applications, starting from diagnosis and clinical trials to patient data documentation. But, what are the future trends in biotechnology clinical data management? Let’s discuss. Cloud-Based Clinical Metadata Repositories Automation is emerging as a new frontier in the biotech clinical data management domain, along with other innovative technologies such as artificial intelligence and machine learning. Because of this, life science establishments are witnessing a huge shift from paper-based documentation toward data-based documentation, which is creating mountains of research, compliance, and clinical data. The growing demand for new and more effective medications and drugs is augmenting the need to expedite clinical trials. This is resulting in an increased number of initiatives aimed at optimizing clinical trial processes to prepare and launch successful trials. However, pharmaceutical and biotechnology laboratories are encountering several challenges in collecting, managing, and analyzing metadata due to its complexities. So, what is the best solution to this problem? The answer to this is cloud-based clinical metadata repositories. Clinical research facilities are leveraging advanced, all-in-one, cloud-based clinical metadata repositories to assist them in centralizing and managing metadata; increasing metadata quality, consistency, and accuracy; and speeding up clinical trial management, documentation, and compliance processes. Shift Towards Digital Solutions Electronic Case Report Form Adequate research and accurate data are crucial for a clinical trial to succeed. Whether developing new drugs, medication, or therapies; conducting life science research; or studying the latest clinical trial systems, it is best to use electronic solutions as it reduces the room for mistakes during the transition of clinical data from paper-based format. Realizing this, biotech organizations are shifting towards using electronic case report forms to speed up record retrieval, improve record security, and cut down on operational costs associated with running clinical trials. The electronic case report form assists in lowering the failure rate of the clinical trial, enhancing efficiency, and optimizing security along with improving clinical trial documentation and productivity, further driving its adoption in the medical space. Electronic Clinical Outcome Assessment Electronic clinical outcome assessment is surfacing as one of the fast-growing future trends in biotechnology. It allows clinical trial facilities to automate data entry and improve the reliability of the collected information. The technology enables clinical trial institutions to automatically record patient-provided information about side effects, symptoms, drug timing, and other aspects during the clinical trial for increased precision. It also helps these institutions analyze the results of medication or therapy in clinical trials and lets clinical researchers use medical technologies like biosensor-enabled devices, self-service applications, and medical wearables for evaluation. Hence, biotech clinical facilities are increasingly deploying advanced electronic clinical outcome assessment systems to ensure adherence to protocols and regulations. Clinical Trial Customization The success of a new drug is determined by numerous factors other than its effectiveness, safety, and creativity of its developers, such as a successful clinical trial. Each clinical trial involves a number of decision-making points, and one wrong choice in any of these aspects can jeopardize the success of the entire endeavor. A crucial component of making well-informed decisions is data management, which is a part of clinical study as a whole. Clinical trial customization is emerging as one of the most prominent biotech clinical trial management trends. Every clinical trial is unique and needs a tailored approach to be successful. With the emergence of the trend of personalized treatment around the globe, biotech and pharmaceutical organizations are adopting innovative customized clinical trial management solutions to accelerate the pace of clinical trials and approvals. This is giving clinical researchers innovative ways to come up with new medicines for patients and streamline the clinical data as per the requirements for faster approvals. What Are the Key Clinical Data Management Challenges Faced by Biotech Companies? Groundbreaking medical interventions are of no use without reliable, accurate, and extensive clinical trial data. Without the data, biotech and pharmaceutical companies will not be able to provide the assurance of safety and efficacy required to bring the medication to market. Regulatory bodies such as the Food and Drug Administration (FDA), the Medicines and Healthcare Products Regulatory Agency (MHRA), and others are putting stricter rules in place to ensure the quality of clinical data. In addition, the fast-changing clinical development environment is creating more obstacles for biotech and medical spaces to ensure the accuracy, standard, and completeness of the clinical trial data. Hence, clinical teams are spending valuable time cleaning up data instead of analyzing it. Time spent trying to figure out issues with clinical trial data is detrimental and expensive but also mission-critical. This is because a small issue in the data can lead to numerous consequences, from small delays to calamitous setbacks, making it necessary to rerun clinical trials. This problem will only get more challenging to address as the volume of data and the types of data sources continue to grow. Here are some of the major clinical data management challenges that biotech firms encounter Standardization of Clinical Metadata Stringent Regulatory Compliance Increased Clinical Trial Complexity Mid-Study Changes Why Are Clinical Data Management Systems Garnering Popularity in the Biotech Industry? With the changing regulatory and clinical landscape, biotech and pharmaceutical companies are facing several obstacles in the management of clinical data and clinical trials. In addition, regulatory agencies are moving toward integrated electronic systems, which is making it more and more important for clinical laboratories to change the format of their submissions. Because of this, several biotech clinical labs are focusing on adopting innovative laboratory solutions, such as biotech clinical data management systems, to meet the need for standardized data inputs and replace all manual ways of working with electronic systems. A clinical data management system establishes the framework for error-free data collection and high-quality data submission, resulting in speedier drug discovery and shorter time-to-market. These solutions are gaining huge traction among biotech and pharmaceutical companies, owing to their ability to effectively manage clinical data, accelerate clinical trials, and ensure compliance. Let’s see some of the features of biotech clinical data management software that are most sought after by life-science companies Controlled, standardized data repository. Centralized data analysis and administration. Reduced operational expenditures for clinical data processes. Enhanced process effectiveness. Superior submission quality Compliance with predefined standards. Clinical Data Management Systems: The Future The role of clinical data management systems is evolving at a rapid pace as the life science and medical industries continue to incorporate digital solutions for diverse operations. These systems are being used in a variety of biotech clinical settings, ranging from clinical data compliance to data science and analytics, to help them analyze large and growing volumes of clinical data. Hence, a number of high-tech medical companies are aiming at integrating innovative technologies, such as artificial intelligence and machine learning, into clinical data management software to automate clinical data management tasks, improve clinical data submission, and enhance data quality. These new biotech clinical management technologies are anticipated to help life science laboratories gain a better understanding of diseases and speed up clinical trials in the coming years. FAQ What is a clinical data management system? A clinical data management system (CDMS) is a tool used in clinical research to track, record, and manage clinical trial data across medical establishments such as biotech laboratories. What are the key functions of the biotech clinical data management system? Some of the key functions of biotech clinical data management system are Documentation of Protocols and Regulations Patient Recruitment Real-time Clinical Study Analytics Reporting Investigator Relationship Management Electronic Visit Report Why is a clinical data management system needed for clinical trials today? A clinical data management system helps shorten the time from drug development to marketing by assisting in the collection of high-quality, statistically sound, and accurate data from clinical trials.

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Top 10 biotech IPOs in 2019

Article | July 20, 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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Cell Out? Lysate-Based Expression an Option for Personalized Meds

Article | February 18, 2020

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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ElevateBio BaseCamp Unveils its LentiPeakTM Lentiviral Vector Platform

ElevateBio, LLC | November 07, 2022

ElevateBio, LLC (ElevateBio), a technology-driven company focused on powering transformative cell and gene therapies, announced the unveiling of ElevateBio BaseCamp’s proprietary LentiPeakTM lentiviral vector platform. LentiPeak is a serum-free, suspension-based, scalable production platform that has demonstrated high volumetric productivity of therapeutically relevant vector yields that meet regulatory guidelines. LentiPeak will enable efficient transition for cell and gene therapies from preclinical stage through clinical development and commercialization with accelerated timelines and reduced manufacturing costs.The LentiPeak platform leverages ElevateBio BaseCamp’s end-to-end process and analytical development expertise and scientific leadership and builds upon the company’s current Good Manufacturing Practice (cGMP) manufacturing capabilities for research, clinical and commercial cell and gene therapies, and regenerative medicines. “As a 30-year veteran of the cell and gene therapy industry, I’ve witnessed the immense advancement of lentiviral vector technology from its infancy to the current landscape with six FDA-approved lentiviral vector-derived cell and gene therapies, The LentiPeak platform is made possible by ElevateBio’s strong scientific foundation, industry-leading expertise, and the investments we’ve made into viral vector production. Our pioneering work continues to be the basis for our continued innovation to drive many more therapies through development to commercialization for our partners.” -Mitchell Finer, Ph.D., Co-founder and President of R&D, ElevateBio ElevateBio BaseCamp’s LentiPeak platform offers a robust and scalable platform process for development and cGMP manufacturing of lentiviral vectors. The LentiPeak platform utilizes a serum-free suspension process capable of producing clinically relevant lentiviral vectors with high titers ranging from 1x10^8 TU/ml to 1x10^9 TU/ml, suitable to meet various therapeutic program needs from preclinical scale to commercial scale supply. The LentiPeak platform can also be seamlessly integrated into cell therapy programs with ElevateBio’s ability to conduct both vector and cell production activities. By leveraging the aligned process unit operations, materials, analytics, and manufacturing expertise at ElevateBio BaseCamp, the LentiPeak platform will simplify the product development and commercialization lifecycle, reducing complexity and ensuring comparability between early-stage and pivotal trials. “Our team of industry-leading experts have developed next-generation platform-based processes that are designed to address lentivirus manufacturing challenges head-on. With our partners’ needs in mind, our proprietary LentiPeak platform has demonstrated leading vector titers with high potency, and process scalability to help our partners de-risk vector production, right from the start, At ElevateBio BaseCamp, we are ever committed to simplifying the complex cell and gene therapy landscape to enable our partners to rapidly progress their therapeutic programs and make these therapies more accessible to patients in need.” -Andrew Sandford, President, ElevateBio BaseCamp About ElevateBio: ElevateBio is a technology-driven company built to power the development of transformative cell and gene therapies today and for many decades to come. The company has assembled industry-leading talent, built state-of-the-art facilities, and integrated diverse technology platforms, including gene editing, induced pluripotent stem cells (iPSCs), and protein, vector, and cellular engineering, necessary to drive innovation and commercialization of cellular and genetic medicines. In addition, ElevateBio BaseCamp in Waltham, MA, is a purpose-built facility offering process innovation, process sciences, and current Good Manufacturing Practice (cGMP) manufacturing capabilities. It was designed to support diverse cell and gene therapy products, including autologous, allogeneic, and regenerative medicine cell products and viral vector manufacturing capabilities. Through BaseCamp and its enabling technologies, ElevateBio is focused on growing its collaborations with industry partners while also developing its own portfolio of cellular and genetic medicines. ElevateBio's team of scientists, drug developers, and company builders are redefining what it means to be a technology company in the world of drug development, blurring the line between technology and healthcare.

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Lysando AG and Amicogen Inc. Sign General Development Agreement

Lysando AG and Amicogen Inc. | October 19, 2022

Lysando AG market leader in the field of antimicrobial proteins has signed a general development agreement with Amicogen Inc., a publicly traded biotechnology company and CDMO headquartered in South Korea. In August last year, Lysando and Amicogen entered into a strategic partnership backed up by a mutual participation model. This collaboration has since been further intensified, now culminating in aforementioned general development agreement. "Amicogen is our first choice for process development and scale-up of any new Artilysin®. This important step in our partnership with Amicogen will help our customers to significantly shorten the time-to-market of Artilysin®-powered products." Count Markus Matuschka de Greiffenclau, Chairman of the Board of Directors of Lysando AG Amicogen is an established and professional partner enabling Lysando to develop its own projects to market-readiness at maximum speed, thus further accelerating Lysando’s entry into the pharmaceutical industry. Ongoing projects targeting indications such as urinary tract infections or neurodermatitis are progressing well. About Amicogen Inc. Amicogen Inc is a CDMO and biotechnology company based in Jinju, South Korea. Amicogen is a leader in the development and production of specialty enzymes and proteins for the biopharmaceutical, food, cosmetics and other industries. One of Amicogen's core competencies is the use of new technologies such as directed gene evolution. The heart of the business is a novel approach that uses biocatalysts to replace chemical with enzymatic processes. These biocatalysts provide an environmentally friendly manufacturing process for downstream industries reducing emissions, simplifying production processes and increasing productivity. About Lysando AG Lysando AG is the market leader for antimicrobial proteins, so-called Artilysin®s. They can effectively eliminate problem-causing bacteria without associated high risk of resistance formation and microbial disbalances. Artilysin®s constitute an innovative, proprietary and environmentally friendly technology with a wide range of applications: in human and veterinary medicine, but for example also in cosmetics. Lysando AG's research facility is Lysando Innovations Lab GmbH located in Regensburg's BioPark. BioPark Regensburg fosters companies in biotechnology, medical technology, diagnostics & analytics as well as the local healthcare management.

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AbCellera and Rallybio Announce Strategic Alliance to Discover, Develop, and Commercialize Novel Antibody-Based Therapeutics for Rare Diseases

Rallybio | December 02, 2022

AbCellera and Rallybio Corporation announced that they have entered into a strategic alliance to discover, develop, and commercialize novel antibody-based therapeutics for rare diseases. This multi-year, multi-target collaboration will combine AbCellera’s antibody discovery engine with Rallybio’s clinical and commercial expertise in rare diseases to identify optimal clinical candidates and ultimately deliver therapies to patients. “It’s clear that we need to accelerate drug development for rare diseases to address the enormous unmet medical need for these patients. Rallybio’s team has decades of experience in sourcing, identifying, and evaluating therapeutic targets. By bringing together Rallybio’s deep expertise in rare diseases with AbCellera’s integrated technology for delivering clinical leads with unmatched precision and speed, this partnership creates a unique advantage that we believe will deliver new and transformative medicines to patients.” Carl Hansen, Ph.D., founder and CEO of AbCellera “AbCellera’s clinically validated discovery engine will strengthen our efforts in identifying novel rare disease candidates to add to our existing pipeline,” said Martin Mackay, Ph.D., Chief Executive Officer of Rallybio. “We are excited to establish this partnership and look forward to working closely with our colleagues at AbCellera as we build novel medicines together.” Under the terms of the agreement, AbCellera and Rallybio will co-develop up to five rare disease therapeutic targets, which will be chosen together by both companies. The collaboration will allow Rallybio to add product candidates to its existing pipeline and also provides the option for AbCellera to conduct process development and clinical manufacturing activities. The partnership’s first program will focus on addressing the significant unmet therapeutic needs of patients with rare metabolic diseases. About AbCellera Biologics Inc. AbCellera is a technology company that searches, decodes, and analyzes natural immune systems to find antibodies that its partners can develop into drugs to prevent and treat disease. AbCellera partners with drug developers of all sizes, from large pharmaceutical to small biotechnology companies, empowering them to move quickly, reduce cost, and tackle the toughest problems in drug development. About Rallybio Rallybio is a clinical-stage biotechnology company committed to identifying and accelerating the development of life-transforming therapies for patients with severe and rare diseases. Since its launch in January 2018, Rallybio has built a portfolio of promising product candidates, which are now in development to address rare diseases in the areas of hematology, immuno-inflammation, maternal fetal health, and metabolic disorders. The Company’s mission is being advanced by a team of highly experienced biopharma industry leaders with extensive research, development, and rare disease expertise. Rallybio is headquartered in New Haven, Connecticut, with an additional facility at the University of Connecticut’s Technology Incubation Program in Farmington, Connecticut.

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MEDTECH

ElevateBio BaseCamp Unveils its LentiPeakTM Lentiviral Vector Platform

ElevateBio, LLC | November 07, 2022

ElevateBio, LLC (ElevateBio), a technology-driven company focused on powering transformative cell and gene therapies, announced the unveiling of ElevateBio BaseCamp’s proprietary LentiPeakTM lentiviral vector platform. LentiPeak is a serum-free, suspension-based, scalable production platform that has demonstrated high volumetric productivity of therapeutically relevant vector yields that meet regulatory guidelines. LentiPeak will enable efficient transition for cell and gene therapies from preclinical stage through clinical development and commercialization with accelerated timelines and reduced manufacturing costs.The LentiPeak platform leverages ElevateBio BaseCamp’s end-to-end process and analytical development expertise and scientific leadership and builds upon the company’s current Good Manufacturing Practice (cGMP) manufacturing capabilities for research, clinical and commercial cell and gene therapies, and regenerative medicines. “As a 30-year veteran of the cell and gene therapy industry, I’ve witnessed the immense advancement of lentiviral vector technology from its infancy to the current landscape with six FDA-approved lentiviral vector-derived cell and gene therapies, The LentiPeak platform is made possible by ElevateBio’s strong scientific foundation, industry-leading expertise, and the investments we’ve made into viral vector production. Our pioneering work continues to be the basis for our continued innovation to drive many more therapies through development to commercialization for our partners.” -Mitchell Finer, Ph.D., Co-founder and President of R&D, ElevateBio ElevateBio BaseCamp’s LentiPeak platform offers a robust and scalable platform process for development and cGMP manufacturing of lentiviral vectors. The LentiPeak platform utilizes a serum-free suspension process capable of producing clinically relevant lentiviral vectors with high titers ranging from 1x10^8 TU/ml to 1x10^9 TU/ml, suitable to meet various therapeutic program needs from preclinical scale to commercial scale supply. The LentiPeak platform can also be seamlessly integrated into cell therapy programs with ElevateBio’s ability to conduct both vector and cell production activities. By leveraging the aligned process unit operations, materials, analytics, and manufacturing expertise at ElevateBio BaseCamp, the LentiPeak platform will simplify the product development and commercialization lifecycle, reducing complexity and ensuring comparability between early-stage and pivotal trials. “Our team of industry-leading experts have developed next-generation platform-based processes that are designed to address lentivirus manufacturing challenges head-on. With our partners’ needs in mind, our proprietary LentiPeak platform has demonstrated leading vector titers with high potency, and process scalability to help our partners de-risk vector production, right from the start, At ElevateBio BaseCamp, we are ever committed to simplifying the complex cell and gene therapy landscape to enable our partners to rapidly progress their therapeutic programs and make these therapies more accessible to patients in need.” -Andrew Sandford, President, ElevateBio BaseCamp About ElevateBio: ElevateBio is a technology-driven company built to power the development of transformative cell and gene therapies today and for many decades to come. The company has assembled industry-leading talent, built state-of-the-art facilities, and integrated diverse technology platforms, including gene editing, induced pluripotent stem cells (iPSCs), and protein, vector, and cellular engineering, necessary to drive innovation and commercialization of cellular and genetic medicines. In addition, ElevateBio BaseCamp in Waltham, MA, is a purpose-built facility offering process innovation, process sciences, and current Good Manufacturing Practice (cGMP) manufacturing capabilities. It was designed to support diverse cell and gene therapy products, including autologous, allogeneic, and regenerative medicine cell products and viral vector manufacturing capabilities. Through BaseCamp and its enabling technologies, ElevateBio is focused on growing its collaborations with industry partners while also developing its own portfolio of cellular and genetic medicines. ElevateBio's team of scientists, drug developers, and company builders are redefining what it means to be a technology company in the world of drug development, blurring the line between technology and healthcare.

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MEDTECH

Lysando AG and Amicogen Inc. Sign General Development Agreement

Lysando AG and Amicogen Inc. | October 19, 2022

Lysando AG market leader in the field of antimicrobial proteins has signed a general development agreement with Amicogen Inc., a publicly traded biotechnology company and CDMO headquartered in South Korea. In August last year, Lysando and Amicogen entered into a strategic partnership backed up by a mutual participation model. This collaboration has since been further intensified, now culminating in aforementioned general development agreement. "Amicogen is our first choice for process development and scale-up of any new Artilysin®. This important step in our partnership with Amicogen will help our customers to significantly shorten the time-to-market of Artilysin®-powered products." Count Markus Matuschka de Greiffenclau, Chairman of the Board of Directors of Lysando AG Amicogen is an established and professional partner enabling Lysando to develop its own projects to market-readiness at maximum speed, thus further accelerating Lysando’s entry into the pharmaceutical industry. Ongoing projects targeting indications such as urinary tract infections or neurodermatitis are progressing well. About Amicogen Inc. Amicogen Inc is a CDMO and biotechnology company based in Jinju, South Korea. Amicogen is a leader in the development and production of specialty enzymes and proteins for the biopharmaceutical, food, cosmetics and other industries. One of Amicogen's core competencies is the use of new technologies such as directed gene evolution. The heart of the business is a novel approach that uses biocatalysts to replace chemical with enzymatic processes. These biocatalysts provide an environmentally friendly manufacturing process for downstream industries reducing emissions, simplifying production processes and increasing productivity. About Lysando AG Lysando AG is the market leader for antimicrobial proteins, so-called Artilysin®s. They can effectively eliminate problem-causing bacteria without associated high risk of resistance formation and microbial disbalances. Artilysin®s constitute an innovative, proprietary and environmentally friendly technology with a wide range of applications: in human and veterinary medicine, but for example also in cosmetics. Lysando AG's research facility is Lysando Innovations Lab GmbH located in Regensburg's BioPark. BioPark Regensburg fosters companies in biotechnology, medical technology, diagnostics & analytics as well as the local healthcare management.

Read More

INDUSTRIAL IMPACT

AbCellera and Rallybio Announce Strategic Alliance to Discover, Develop, and Commercialize Novel Antibody-Based Therapeutics for Rare Diseases

Rallybio | December 02, 2022

AbCellera and Rallybio Corporation announced that they have entered into a strategic alliance to discover, develop, and commercialize novel antibody-based therapeutics for rare diseases. This multi-year, multi-target collaboration will combine AbCellera’s antibody discovery engine with Rallybio’s clinical and commercial expertise in rare diseases to identify optimal clinical candidates and ultimately deliver therapies to patients. “It’s clear that we need to accelerate drug development for rare diseases to address the enormous unmet medical need for these patients. Rallybio’s team has decades of experience in sourcing, identifying, and evaluating therapeutic targets. By bringing together Rallybio’s deep expertise in rare diseases with AbCellera’s integrated technology for delivering clinical leads with unmatched precision and speed, this partnership creates a unique advantage that we believe will deliver new and transformative medicines to patients.” Carl Hansen, Ph.D., founder and CEO of AbCellera “AbCellera’s clinically validated discovery engine will strengthen our efforts in identifying novel rare disease candidates to add to our existing pipeline,” said Martin Mackay, Ph.D., Chief Executive Officer of Rallybio. “We are excited to establish this partnership and look forward to working closely with our colleagues at AbCellera as we build novel medicines together.” Under the terms of the agreement, AbCellera and Rallybio will co-develop up to five rare disease therapeutic targets, which will be chosen together by both companies. The collaboration will allow Rallybio to add product candidates to its existing pipeline and also provides the option for AbCellera to conduct process development and clinical manufacturing activities. The partnership’s first program will focus on addressing the significant unmet therapeutic needs of patients with rare metabolic diseases. About AbCellera Biologics Inc. AbCellera is a technology company that searches, decodes, and analyzes natural immune systems to find antibodies that its partners can develop into drugs to prevent and treat disease. AbCellera partners with drug developers of all sizes, from large pharmaceutical to small biotechnology companies, empowering them to move quickly, reduce cost, and tackle the toughest problems in drug development. About Rallybio Rallybio is a clinical-stage biotechnology company committed to identifying and accelerating the development of life-transforming therapies for patients with severe and rare diseases. Since its launch in January 2018, Rallybio has built a portfolio of promising product candidates, which are now in development to address rare diseases in the areas of hematology, immuno-inflammation, maternal fetal health, and metabolic disorders. The Company’s mission is being advanced by a team of highly experienced biopharma industry leaders with extensive research, development, and rare disease expertise. Rallybio is headquartered in New Haven, Connecticut, with an additional facility at the University of Connecticut’s Technology Incubation Program in Farmington, Connecticut.

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