Laboratory Information Management System for Biotech Labs: Significance & Benefits

Kutubkhan Bohari | August 16, 2022 | 1297 views | Read Time : 02:00 min

Laboratory Information
If you have ever visited the testing laboratory of a large biotechnology company, you will be aware that managing the laboratory's operations single-handedly is no easy task. The greater the size of a lab, the more research and testing activities it must accommodate.

A variety of diagnostic tests are prescribed for patients in order to detect various diseases. For example, it may include blood glucose testing for diabetics, lipid panel, or liver panel tests for evaluating cardiac risk and liver function, cultures for diagnosing infections, thyroid function tests, and others.

Laboratory management solutions such as laboratory information management systems (LIMS) and other software play a significant role in managing various operational data at biotech laboratories. It is one of the important types of software developed to address the data management and regulatory challenges of laboratories. The software enhances the operational efficiency of biotech labs by streamlining workflows, proper record-keeping, and eradicating the need for manually maintaining data.

What Are the Benefits of Laboratory Information Management Software in Biotechnology?
As the trends of digitization and technology continue to create deeper inroads into the biotechnology sector, a significant rise in the adoption of innovative medical software solutions, such as LIMS, is being witnessed for managing research data, testing reports, and post-research results globally. Here are a few reasons that are encouraging biotech facilities to adopt LIMS solutions

Real-Time Data Collection and Tracking
Previously, collecting and transporting samples was a tedious and time-consuming task. However, the adoption of LIMS with innovative tracking modules has made the job easier. The real-time sample tracking feature of LIMS has made it possible for personnel to collect the research data in real-time and manage and control the workflow with a few mouse clicks on the screen.

Increase Revenue
LIMS makes it possible to test workflows while giving users complete control over the testing process. A laboratory is able to collect data, schedule equipment maintenance or upgrades, enhance operational efficiency, and maintain a lower overhead with the help of the LIMS, thereby increasing revenue.

Streamlined Workflow
With its completion monitoring, LIMS speeds up laboratory workflows and keeps track of information. It assigns tasks to the specialist along with keeping a real-time track of the status and completion of each task. LIMS is integrated into the laboratory using lab information, which ultimately speeds up internal processes and streamlines the workflow.  

Automatic Data Exchange
LIMS solutions store data in a centralized database. Automated transfer of data between departments and organizations is one of the major features of LIMS. Through its automated information exchange feature, LIMS improves internal operations, decreases the reporting time for data sharing, and assists in faster decision-making.

Final Thoughts
As the healthcare sector continues to ride the wave of digital transformation, biotech laboratories are emphasizing adopting newer technologies to keep up with the changes.

Citing this trend, laboratory information management systems are becoming crucial for biotech and medical organizations for maintaining research data, instant reporting, and managing confidential, inventory, and financial data with centralized data storage.

Spotlight

Insig

In early 2015, we realized that there was a wealth of medical knowledge online but no intelligent interpretation or curation of it. We aimed to solve this by creating an Artificial Intelligence engine that could bring a patient to diagnosis based on their current medical state, symptoms and past medical history. In our pursuit we analyzed over 3 million patient records in engineering an AI model that could lead a naive patient right to diagnosis through simple questioning.

OTHER ARTICLES
MEDICAL

Top 10 biotech IPOs in 2019

Article | August 2, 2021

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

Read More
MEDICAL

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

Article | July 14, 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.

Read More
MEDICAL

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

Article | June 22, 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.

Read More

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.

Read More

Spotlight

Insig

In early 2015, we realized that there was a wealth of medical knowledge online but no intelligent interpretation or curation of it. We aimed to solve this by creating an Artificial Intelligence engine that could bring a patient to diagnosis based on their current medical state, symptoms and past medical history. In our pursuit we analyzed over 3 million patient records in engineering an AI model that could lead a naive patient right to diagnosis through simple questioning.

Related News

DIAGNOSTICS

PhenomeX Launches Beacon Quest™ Optofluidic System with Opto® T Cell Profiling Workflows

PRNewswire | June 06, 2023

PhenomeX Inc. the functional cell biology company, announced the launch of the Beacon Quest™ optofluidic system, with Opto® T Cell Profiling workflows for immunotherapy translational research and a platform price under $1 million USD, less than half of the higher-throughput Beacon system, enabling broader accessibility to the cutting-edge technology critical for next-generation precision medicine. The Opto T Cell Profiling workflows have enabled scientists to comprehensively profile single T cells to correlate polyfunctionality with cytotoxicity and recover those same cells for downstream analysis such as transcriptome and genome sequencing, revealing functional connections of phenotype with molecular mechanism. This depth of understanding of biology and response to therapeutics is not available on any other platform. Combined with the value-matched Beacon Quest system, more academic, non-profit, and government customers can now access the power of optofluidic and NanoPen® chamber technology and propriety single T Cell Profiling applications along with the optimum level of features to meet their research and budgetary needs. "At PhenomeX, we are excited to launch the Beacon Quest, as it supports our objective of offering scientists and researchers globally the ability to affordably access the main applications of the Beacon system, including antibody discovery, cell line development and single-cell functional multiomic T-cell profiling," said Siddhartha Kadia, Ph.D., chief executive officer of PhenomeX. "We look forward to partnering with and providing significant value in the academic research segments, particularly in immuno-oncology translational cancer centers and innovative cell and gene therapy development centers." Beacon Quest for T Cell Profiling Workflow Features Opto T Cell Profiling workflows on the Beacon Quest system enable multi-functional characterization of single T cells, including for development of efficacious immunotherapies and cell-based cancer therapeutics, that are ideally suited for academic, non-profit, and government customers. The Beacon Quest system enables a variety of assays to profile single T cells including detection of cytokine secretion, cytotoxicity, cell surface markers, transcriptome profiling and TCRSeq, with the potential to study growth characteristics and cell motility. The resulting analysis is used to identify cells with phenotypes of interest and guide recovery of the same cells for downstream assays, enabling researchers to gain a deeper understanding of the underlying biology by Investigating molecular mechanisms associated with polyfunctional T cells; Identifying desirable phenomes by correlating polyfunctionality with cytotoxicity; and Linking desirable phenotypes to T cell receptor sequences of the same cells. In addition to supporting the Opto T Cell Profiling workflows, Beacon Quest will enable customers to access the Opto B Discovery workflows and Opto Cell Line Development workflows. To further increase customer accessibility and affordability, PhenomeX will also offer reduced priced OptoSelect® chips (OptoSelect® Research chips) for Beacon Quest, helping academic customers accelerate the pace of scientific discovery for clinical research use applications. About PhenomeX Inc. PhenomeX is empowering scientists to leverage the full potential of each cell and drive the next era of functional cell biology that will advance human health. We enable scientists to reveal the most complete insights on cell function and obtain a full view of the behavior of each cell. Our unique suite of proven high-throughput tools and services offer unparalleled resolution and speed, accelerating the insights that are key to advancing discoveries that can profoundly improve the prevention and treatment of disease. Our award-winning platforms are used by researchers across the globe, including those at the top 15 global pharmaceutical companies and approximately 85% of leading U.S. comprehensive cancer centers.

Read More

MEDTECH, MEDICAL

Twist Bioscience Launches Portfolio of RNA Sequencing Tools

Businesswire | May 23, 2023

Twist Bioscience Corporation a company enabling customers to succeed through its offering of high-quality synthetic DNA using its silicon platform, today announced the launch of a portfolio of RNA sequencing tools, which includes the Twist RNA Exome, Twist RNA Library Prep Kit and the Twist Ribosomal RNA (rRNA) & Hemoglobin (Globin) Depletion Kit, as well as custom target enrichment capabilities for RNA and whole transcriptome sequencing. This comprehensive suite of RNA tools can enable targeted or whole transcriptome research across fields including precision medicine, biomarker discovery and immuno-oncology research. “Twist’s RNA sequencing portfolio builds on our established NGS capabilities in genomic sequencing and methylation detection and expands into gene expression analysis, opening a new and significant market for Twist,” said Emily M. Leproust, Ph.D., CEO and co-founder of Twist Bioscience. “This offering allows our customers to leverage Twist’s leading target enrichment platform to discover and resolve low abundance RNA transcripts in the human transcriptome. Our RNA sequencing workflows are designed specifically for challenging samples. By enriching relevant transcripts, we can enable researchers to get high-quality data from a range of sample types, including damaged RNA.” Unlike the genome, which is relatively static, the transcriptome changes over time in response to varying disease states and drug therapies. Gene expression levels also differ across tissues and cell types. To fully understand this dynamic quality of RNA, researchers often sequence RNA samples from the same source at multiple time points, incurring additional costs and resource burdens. Each component of the Twist RNA sequencing portfolio is purpose-built to maximize data quality, minimize workflow inefficiency, decrease wasted sequencing reads, and support comprehensive transcriptomic profiling across even low-quality and low-input samples common in oncology. The Twist RNA sequencing portfolio provides end-to-end workflows that can be used to study total RNA and measure the whole transcriptome, protein coding sequences of messenger RNA (mRNA) or custom targets. These products can also be integrated into customers’ existing workflows. Twist RNA sequencing tools enable minimal hands-on time and increase the scale of samples sequenced through utilizing automation. They are compatible with a wide range of sample inputs, including difficult or low-quality samples. RNA panel designs can also now incorporate Twist’s new exon-aware design algorithm, which reduces bias in detection and can provide an important tool for researchers studying rare gene transcripts that drive tumor biology. Twist Targeted RNA Sequencing Workflow Twist target enrichment for RNA leverages the performance, efficiency and sensitivity of Twist target enrichment for sequencing of RNA transcripts of interest across samples and species. It can be used to enrich relevant transcripts with the RNA Exome or in custom RNA panels. Twist RNA Exome The Twist RNA Exome with Twist’s exon-aware design algorithm enables targeted sequencing of the protein coding regions of the human transcriptome without bias toward specific combinations of exons. The Twist RNA Exome covers up to 99.9% of protein coding sequencing in Gencode and RefSeq databases. This coverage, along with the exon-aware design approach, could enable researchers to capture important transcripts including those in low input and degraded samples from formalin fixed paraffin embedded (FFPE) sections. Twist Whole Transcriptome Sequencing Workflow Twist Whole Transcriptome Sequencing can be used to discover novel transcripts, including low expressing genes and novel isoforms. It can be used with a variety of samples including whole blood, fresh and frozen tissue samples and low quality samples, such as FFPE samples. The Twist rRNA & Globin Depletion Kit can be used with this workflow to deplete rRNA and hemoglobin targets so that researchers can study the rest of the transcriptome. The full workflow can be completed in less than five hours with minimal hands-on time. About Twist Bioscience Corporation Twist Bioscience is a leading and rapidly growing synthetic biology and genomics company that has developed a disruptive DNA synthesis platform to industrialize the engineering of biology. The core of the platform is a proprietary technology that pioneers a new method of manufacturing synthetic DNA by “writing” DNA on a silicon chip. Twist is leveraging its unique technology to manufacture a broad range of synthetic DNA-based products, including synthetic genes, tools for next-generation sequencing (NGS) preparation, and antibody libraries for drug discovery and development. Twist is also pursuing longer-term opportunities in digital data storage in DNA and biologics drug discovery. Twist makes products for use across many industries including healthcare, industrial chemicals, agriculture and academic research.

Read More

MEDICAL, INDUSTRY OUTLOOK

REGENXBIO Announces NAV® Technology Platform Will Support Bespoke Gene Therapy Consortium's First Rare Disease Clinical Portfolio

PRNewswire | May 17, 2023

REGENXBIO Inc. announced that preclinical research in Mucopolysaccharidosis type IVA also known as Morquio syndrome, was selected for inclusion in the Foundation for the National Institutes of Health Accelerating Medicines Partnership® Bespoke Gene Therapy Consortium (AMP® BGTC), clinical trial portfolio. The Consortium brings together partners such as NIH and FDA, as well as partners from private and non-profit sectors. Sponsored by Nemours Children's Hospital, MPS IVA is one of eight programs selected as part of AMP® BGTC's first clinical portfolio to help accelerate the development of bespoke gene therapies, with the goal of streamlining the regulatory approval process. MPS IVA is a metabolic condition that primarily affects the skeleton, and is estimated to impact 1 in 200,000 to 300,000 individuals. "I am proud of our scientists who led this important clinical research work for REGENXBIO. Our mission to deliver the curative potential of gene therapy reflects not only patients that can be impacted by our clinical pipeline, but also all patients who should have the opportunity to be positively impacted by gene therapy," said Kenneth T. Mills, President and CEO REGENXBIO. "As a partner of the BGTC, REGENXBIO is pleased to see this program advancing important science into the clinic." Criteria for selection included the adequacy of the gene for insertion into an adeno-associated virus (AAV) vector, sufficient proof of concept and natural history data, the existence of an established disease model, a lack of available treatment and an overall readiness for entering into a clinical trial. REGENXBIO will donate licenses to NAV® AAV8 and NAV® AAV9 from its NAV® Technology Platform to enable AMP® BGTC's development of these programs aimed at addressing ultra rare diseases. Use of the NAV® Technology Platform will help address the goal of making gene therapy more accessible by creating a platform approach with standardized processes to deliver novel therapies for many different genetic disorders. These programs together further validate the versatility of NAV® vectors and will provide additional data that collectively drive the advancement of the AAV gene therapy field. About REGENXBIO Inc. REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. REGENXBIO's NAV Technology Platform, a proprietary adeno-associated virus (AAV) gene delivery platform, consists of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8 and AAV9. REGENXBIO and its third-party NAV Technology Platform Licensees are applying the NAV Technology Platform in the development of a broad pipeline of candidates, including late-stage and commercial programs, in multiple therapeutic areas. REGENXBIO is committed to a "5x'25" strategy to progress five AAV Therapeutics from our internal pipeline and licensed programs into pivotal-stage or commercial products by 2025.

Read More

DIAGNOSTICS

PhenomeX Launches Beacon Quest™ Optofluidic System with Opto® T Cell Profiling Workflows

PRNewswire | June 06, 2023

PhenomeX Inc. the functional cell biology company, announced the launch of the Beacon Quest™ optofluidic system, with Opto® T Cell Profiling workflows for immunotherapy translational research and a platform price under $1 million USD, less than half of the higher-throughput Beacon system, enabling broader accessibility to the cutting-edge technology critical for next-generation precision medicine. The Opto T Cell Profiling workflows have enabled scientists to comprehensively profile single T cells to correlate polyfunctionality with cytotoxicity and recover those same cells for downstream analysis such as transcriptome and genome sequencing, revealing functional connections of phenotype with molecular mechanism. This depth of understanding of biology and response to therapeutics is not available on any other platform. Combined with the value-matched Beacon Quest system, more academic, non-profit, and government customers can now access the power of optofluidic and NanoPen® chamber technology and propriety single T Cell Profiling applications along with the optimum level of features to meet their research and budgetary needs. "At PhenomeX, we are excited to launch the Beacon Quest, as it supports our objective of offering scientists and researchers globally the ability to affordably access the main applications of the Beacon system, including antibody discovery, cell line development and single-cell functional multiomic T-cell profiling," said Siddhartha Kadia, Ph.D., chief executive officer of PhenomeX. "We look forward to partnering with and providing significant value in the academic research segments, particularly in immuno-oncology translational cancer centers and innovative cell and gene therapy development centers." Beacon Quest for T Cell Profiling Workflow Features Opto T Cell Profiling workflows on the Beacon Quest system enable multi-functional characterization of single T cells, including for development of efficacious immunotherapies and cell-based cancer therapeutics, that are ideally suited for academic, non-profit, and government customers. The Beacon Quest system enables a variety of assays to profile single T cells including detection of cytokine secretion, cytotoxicity, cell surface markers, transcriptome profiling and TCRSeq, with the potential to study growth characteristics and cell motility. The resulting analysis is used to identify cells with phenotypes of interest and guide recovery of the same cells for downstream assays, enabling researchers to gain a deeper understanding of the underlying biology by Investigating molecular mechanisms associated with polyfunctional T cells; Identifying desirable phenomes by correlating polyfunctionality with cytotoxicity; and Linking desirable phenotypes to T cell receptor sequences of the same cells. In addition to supporting the Opto T Cell Profiling workflows, Beacon Quest will enable customers to access the Opto B Discovery workflows and Opto Cell Line Development workflows. To further increase customer accessibility and affordability, PhenomeX will also offer reduced priced OptoSelect® chips (OptoSelect® Research chips) for Beacon Quest, helping academic customers accelerate the pace of scientific discovery for clinical research use applications. About PhenomeX Inc. PhenomeX is empowering scientists to leverage the full potential of each cell and drive the next era of functional cell biology that will advance human health. We enable scientists to reveal the most complete insights on cell function and obtain a full view of the behavior of each cell. Our unique suite of proven high-throughput tools and services offer unparalleled resolution and speed, accelerating the insights that are key to advancing discoveries that can profoundly improve the prevention and treatment of disease. Our award-winning platforms are used by researchers across the globe, including those at the top 15 global pharmaceutical companies and approximately 85% of leading U.S. comprehensive cancer centers.

Read More

MEDTECH, MEDICAL

Twist Bioscience Launches Portfolio of RNA Sequencing Tools

Businesswire | May 23, 2023

Twist Bioscience Corporation a company enabling customers to succeed through its offering of high-quality synthetic DNA using its silicon platform, today announced the launch of a portfolio of RNA sequencing tools, which includes the Twist RNA Exome, Twist RNA Library Prep Kit and the Twist Ribosomal RNA (rRNA) & Hemoglobin (Globin) Depletion Kit, as well as custom target enrichment capabilities for RNA and whole transcriptome sequencing. This comprehensive suite of RNA tools can enable targeted or whole transcriptome research across fields including precision medicine, biomarker discovery and immuno-oncology research. “Twist’s RNA sequencing portfolio builds on our established NGS capabilities in genomic sequencing and methylation detection and expands into gene expression analysis, opening a new and significant market for Twist,” said Emily M. Leproust, Ph.D., CEO and co-founder of Twist Bioscience. “This offering allows our customers to leverage Twist’s leading target enrichment platform to discover and resolve low abundance RNA transcripts in the human transcriptome. Our RNA sequencing workflows are designed specifically for challenging samples. By enriching relevant transcripts, we can enable researchers to get high-quality data from a range of sample types, including damaged RNA.” Unlike the genome, which is relatively static, the transcriptome changes over time in response to varying disease states and drug therapies. Gene expression levels also differ across tissues and cell types. To fully understand this dynamic quality of RNA, researchers often sequence RNA samples from the same source at multiple time points, incurring additional costs and resource burdens. Each component of the Twist RNA sequencing portfolio is purpose-built to maximize data quality, minimize workflow inefficiency, decrease wasted sequencing reads, and support comprehensive transcriptomic profiling across even low-quality and low-input samples common in oncology. The Twist RNA sequencing portfolio provides end-to-end workflows that can be used to study total RNA and measure the whole transcriptome, protein coding sequences of messenger RNA (mRNA) or custom targets. These products can also be integrated into customers’ existing workflows. Twist RNA sequencing tools enable minimal hands-on time and increase the scale of samples sequenced through utilizing automation. They are compatible with a wide range of sample inputs, including difficult or low-quality samples. RNA panel designs can also now incorporate Twist’s new exon-aware design algorithm, which reduces bias in detection and can provide an important tool for researchers studying rare gene transcripts that drive tumor biology. Twist Targeted RNA Sequencing Workflow Twist target enrichment for RNA leverages the performance, efficiency and sensitivity of Twist target enrichment for sequencing of RNA transcripts of interest across samples and species. It can be used to enrich relevant transcripts with the RNA Exome or in custom RNA panels. Twist RNA Exome The Twist RNA Exome with Twist’s exon-aware design algorithm enables targeted sequencing of the protein coding regions of the human transcriptome without bias toward specific combinations of exons. The Twist RNA Exome covers up to 99.9% of protein coding sequencing in Gencode and RefSeq databases. This coverage, along with the exon-aware design approach, could enable researchers to capture important transcripts including those in low input and degraded samples from formalin fixed paraffin embedded (FFPE) sections. Twist Whole Transcriptome Sequencing Workflow Twist Whole Transcriptome Sequencing can be used to discover novel transcripts, including low expressing genes and novel isoforms. It can be used with a variety of samples including whole blood, fresh and frozen tissue samples and low quality samples, such as FFPE samples. The Twist rRNA & Globin Depletion Kit can be used with this workflow to deplete rRNA and hemoglobin targets so that researchers can study the rest of the transcriptome. The full workflow can be completed in less than five hours with minimal hands-on time. About Twist Bioscience Corporation Twist Bioscience is a leading and rapidly growing synthetic biology and genomics company that has developed a disruptive DNA synthesis platform to industrialize the engineering of biology. The core of the platform is a proprietary technology that pioneers a new method of manufacturing synthetic DNA by “writing” DNA on a silicon chip. Twist is leveraging its unique technology to manufacture a broad range of synthetic DNA-based products, including synthetic genes, tools for next-generation sequencing (NGS) preparation, and antibody libraries for drug discovery and development. Twist is also pursuing longer-term opportunities in digital data storage in DNA and biologics drug discovery. Twist makes products for use across many industries including healthcare, industrial chemicals, agriculture and academic research.

Read More

MEDICAL, INDUSTRY OUTLOOK

REGENXBIO Announces NAV® Technology Platform Will Support Bespoke Gene Therapy Consortium's First Rare Disease Clinical Portfolio

PRNewswire | May 17, 2023

REGENXBIO Inc. announced that preclinical research in Mucopolysaccharidosis type IVA also known as Morquio syndrome, was selected for inclusion in the Foundation for the National Institutes of Health Accelerating Medicines Partnership® Bespoke Gene Therapy Consortium (AMP® BGTC), clinical trial portfolio. The Consortium brings together partners such as NIH and FDA, as well as partners from private and non-profit sectors. Sponsored by Nemours Children's Hospital, MPS IVA is one of eight programs selected as part of AMP® BGTC's first clinical portfolio to help accelerate the development of bespoke gene therapies, with the goal of streamlining the regulatory approval process. MPS IVA is a metabolic condition that primarily affects the skeleton, and is estimated to impact 1 in 200,000 to 300,000 individuals. "I am proud of our scientists who led this important clinical research work for REGENXBIO. Our mission to deliver the curative potential of gene therapy reflects not only patients that can be impacted by our clinical pipeline, but also all patients who should have the opportunity to be positively impacted by gene therapy," said Kenneth T. Mills, President and CEO REGENXBIO. "As a partner of the BGTC, REGENXBIO is pleased to see this program advancing important science into the clinic." Criteria for selection included the adequacy of the gene for insertion into an adeno-associated virus (AAV) vector, sufficient proof of concept and natural history data, the existence of an established disease model, a lack of available treatment and an overall readiness for entering into a clinical trial. REGENXBIO will donate licenses to NAV® AAV8 and NAV® AAV9 from its NAV® Technology Platform to enable AMP® BGTC's development of these programs aimed at addressing ultra rare diseases. Use of the NAV® Technology Platform will help address the goal of making gene therapy more accessible by creating a platform approach with standardized processes to deliver novel therapies for many different genetic disorders. These programs together further validate the versatility of NAV® vectors and will provide additional data that collectively drive the advancement of the AAV gene therapy field. About REGENXBIO Inc. REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. REGENXBIO's NAV Technology Platform, a proprietary adeno-associated virus (AAV) gene delivery platform, consists of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8 and AAV9. REGENXBIO and its third-party NAV Technology Platform Licensees are applying the NAV Technology Platform in the development of a broad pipeline of candidates, including late-stage and commercial programs, in multiple therapeutic areas. REGENXBIO is committed to a "5x'25" strategy to progress five AAV Therapeutics from our internal pipeline and licensed programs into pivotal-stage or commercial products by 2025.

Read More

Events