How to Solve the Manufacturing Bottleneck in Gene Therapy?

NICOLE FAUST | October 21, 2019 | 4 views

With over $4B raised in the first half of 2019 and approximately 370 clinical trials underway, the global gene therapy sector is booming. Recent years have seen a steady increase in the number of trials and the amount of money invested. As therapies shift to more common diseases and larger patient numbers, new manufacturing solutions are needed. Labiotech spoke to CEVEC Pharmaceuticals about recent trends and new standards in gene therapy manufacturing.

Spotlight

ITR Laboratories Canada Inc.

TR Canada provides preclinical testing services for the pharmaceutical and biotechnology industries. Since our founding in 1989 we have grown into a mid-sized Contract Research Organization (CRO).

OTHER ARTICLES
INDUSTRIAL IMPACT

Advancement in Genomics Accelerating its Penetration into Precision Health

Article | January 20, 2021

Genomics is an interdisciplinary field of biology emphasizing the structure, editing, evolution, function, and mapping of genomes. It is creating deeper inroads across the precision health domain with the increasing introduction of advanced technologies such as quantum simulation, next-generation sequencing (NGS), and precise genome manipulation. As precision health focuses on providing the proper intervention to the right patient at the right time, genomics increasingly finds applications in human and pathogen genome sequencing in clinical and research spaces. Rising Hereditary Diseases Burden Paving the Way for Genomics in Precision Health In the last few years, a significant surge in the prevalence of diseases and ailments such as diabetes, obesity, baldness, and others has been witnessed across the globe. A history of family members with chronic diseases, such as cancer, diabetes, high blood pressure, hearing issues, and heart disease, can sometimes continue into the next generation. Hence, the study of genes is extensively being conducted for predicting health risks and early treatment of these diseases. It also finds use in CRISPR-based diagnostics and the preparation of precision medication for the individual. In addition, ongoing advancements in genomics are making it possible to identify different genetic traits that persuade people to more widespread diseases and health problems. The Emergence of Genomics Improves Disease Understanding Genomics refers to the study of the complete genetic makeup of a cell or organism. Increasing scientific research in the area substantially contributes to increasing knowledge about the human genome and assists in improving the ability to understand disease etiology, risk, diagnosis, treatment, and prevention. On account of these improvements, innovative genomic technologies and tools are being developed to enable better precision health not only for the individual but for various regional populations as well. The Way Forward With growing preference for personalized medicine and an increasing need for more accurate pathogen detection and diagnostics, genomics is gaining huge popularity across the precision health domain. Also, increasing research activities for developing novel high-precision therapeutics and rising importance of gene study in the prevention, diagnosis, and management of infectious and genetic diseases will further pave the way for genomics in the forthcoming years.

Read More

Top 10 biotech IPOs in 2019

Article | September 13, 2019

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

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

Article | September 13, 2019

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

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

Article | February 12, 2020

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

Spotlight

ITR Laboratories Canada Inc.

TR Canada provides preclinical testing services for the pharmaceutical and biotechnology industries. Since our founding in 1989 we have grown into a mid-sized Contract Research Organization (CRO).

Related News

CELL AND GENE THERAPY

Antengene Announces Clinical Trial Collaboration with BeiGene to Evaluate Selinexor in Combination with Tislelizumab in T and NK-Cell Lymphoma

Antengene | June 28, 2022

Antengene Corporation Limited ("Antengene" SEHK: 6996.HK), a leading innovative, commercial-stage global biopharmaceutical company dedicated to discovering, developing and commercializing first-in-class and/or best-in-class therapeutics in hematology and oncology, today announced that it has entered into a clinical trial collaboration with BeiGene to evaluate the safety, pharmacokinetics, pharmacodynamics and preliminary efficacy of selinexor in combination with BeiGene's anti-PD-1 checkpoint inhibitor, tislelizumab. This multi-center, open-label Phase I/II trial will evaluate the investigational combination as a potential treatment option for patients with T and NK-cell lymphoma. We are delighted to partner with BeiGene, a company that strives for innovation and excellence, and is committed to developing best-in-class or first-in-class anti-cancer therapies for patients across the globe. These qualities are very similar to those of our vision at Antengene, We look forward to advancing the combination of selinexor and tislelizumab to clinical development. With good data we will be able to bring this treatment regimen to patients with T and NK-cell lymphoma, diseases that are endemic in Asia but underserved by current therapies." Dr. Jay Mei, Antengene's Founder, Chairman and CEO. Tislelizumab is a PD-1 inhibitor designed to help aid the body's immune cells to detect and fight tumors. Tislelizumab, a humanized monoclonal antibody, is specifically designed to minimize binding to FcγR on macrophages. In pre-clinical studies, binding to FcγR on macrophages has been shown to compromise the anti-tumor activity of PD-1 antibodies through activation of antibody-dependent macrophage-mediated killing of T effector cells. About T and NK-Cell Lymphoma T and NK-cell lymphoma is a set of heterogeneous diseases, accounting for 25-30% of Non-Hodgkin Lymphoma (NHL) cases in China and only about 10% in USA and Europe. There has been little improvement in the past decade when compared to B-cell Non-Hodgkin Lymphoma (B-NHL) as 5-year overall survival rate was only 30% in most common subtypes[1]. The unmet medical needs remain as agents with new mechanism of action to be explored and possibility to improve the treatment paradigm for the disease. About the SINE Compounds Selective Inhibitor of Nuclear Export (SINE) compounds are inhibitors of the major nuclear export protein Exportin 1 (XPO1). Currently, there are three oral SINE compounds, ATG-010 (selinexor), ATG-016 (eltanexor), and ATG-527 (verdinexor), under clinical development. Antengene has obtained exclusive development and commercialisation rights from Karyopharm Therapeutics Inc. (Nasdaq: KPTI) to these three compounds in certain APAC markets.

Read More

DIAGNOSTICS

ZIVO Bioscience Announce Joining Russell Microcap® Index Membership

Zivo | June 28, 2022

ZIVO Bioscience, Inc. is a U.S.-based R&D company dedicated to the study, development and commercialization of natural nutritional compounds and bioactive molecules derived from proprietary algal strains, announced joining the Russell Microcap® Index (^RUMIC) as a result of the Russell indexes annual reconstitution in 2022. Russell Microcap® Index membership that remains in place for a course of 12 months, means automatic inclusion in the appropriate value style and growth indexes. Membership for FTSE Russell's Russell indices is generally determined by market-capitalization rankings and style characteristics. A leader in global indices, FTSE Russell, offers cutting-edge analytics, benchmarking, and data solutions to investors all over the world. FTSE Russell creates thousands of indexes to measure and benchmark markets and asset classes for over 70 nations, covering 98 percent of the world's investable markets. It emphasizes on applying the strict industry norms in governance and index design and also embraces the IOSCO Principles. We are pleased to be added to the Russell Microcap® Index this year. Our inclusion reflects continued interest in our stock along with long- and near-term growth expectations as we evolve and execute on our business strategy, I also look forward to expanding our stock’s exposure to a broader investor audience, including more institutional investors, as a result of ZIVO’s inclusion.” John Payne, President and Chief Executive Officer of ZIVO Bioscience. Russell indexes are increasingly used by institutional investors and investment managers for index funds for benchmarking active investment strategies. Approximately US$12 trillion in assets are benchmarked against Russell’s US indexes.

Read More

DIAGNOSTICS

Zoetis to Complete Acquisition of Basepaws to Strengthen its Portfolio

Zoetis | June 27, 2022

Zoetis Inc. is the world's largest animal health company committed to providing medicine and vaccinations for pets and livestock. The company announced completing the acquisition of a privately held petcare genetics company, Basepaws, which offers early health risk assessments, genetic tests, and analytics to pet owners. The acquisition was first announced on June 7th. However, the financial terms of the purchase were not disclosed. Basepaws, founded by CEO Anna Skaya in 2017, provides genetic screening tools for the diagnosis of several pet diseases, health reports, and individualized breeds by identifying traits, potential hereditary conditions, and biomarkers. With the help of genetic insights from Basepaws, pet owners and doctors can better understand a pet's risk for the disease. This can result in more meaningful interactions and improve the chances of early identification and treatment of the disease. When we first met Basepaws CEO Anna Skaya and the team, we were immediately drawn to their passion for pets and the role that genetic testing and data analytics can have in advancing animal care – a purpose that drives us every day,The addition of Basepaws will enhance our portfolio in the precision animal health space and inform our future pipeline of petcare innovations. Working together, we can continue to provide veterinarians and pet owners with more comprehensive ways to proactively manage the health, wellness, and quality of care for their animals." Zoetis, CEO Kristin Peck.

Read More

CELL AND GENE THERAPY

Antengene Announces Clinical Trial Collaboration with BeiGene to Evaluate Selinexor in Combination with Tislelizumab in T and NK-Cell Lymphoma

Antengene | June 28, 2022

Antengene Corporation Limited ("Antengene" SEHK: 6996.HK), a leading innovative, commercial-stage global biopharmaceutical company dedicated to discovering, developing and commercializing first-in-class and/or best-in-class therapeutics in hematology and oncology, today announced that it has entered into a clinical trial collaboration with BeiGene to evaluate the safety, pharmacokinetics, pharmacodynamics and preliminary efficacy of selinexor in combination with BeiGene's anti-PD-1 checkpoint inhibitor, tislelizumab. This multi-center, open-label Phase I/II trial will evaluate the investigational combination as a potential treatment option for patients with T and NK-cell lymphoma. We are delighted to partner with BeiGene, a company that strives for innovation and excellence, and is committed to developing best-in-class or first-in-class anti-cancer therapies for patients across the globe. These qualities are very similar to those of our vision at Antengene, We look forward to advancing the combination of selinexor and tislelizumab to clinical development. With good data we will be able to bring this treatment regimen to patients with T and NK-cell lymphoma, diseases that are endemic in Asia but underserved by current therapies." Dr. Jay Mei, Antengene's Founder, Chairman and CEO. Tislelizumab is a PD-1 inhibitor designed to help aid the body's immune cells to detect and fight tumors. Tislelizumab, a humanized monoclonal antibody, is specifically designed to minimize binding to FcγR on macrophages. In pre-clinical studies, binding to FcγR on macrophages has been shown to compromise the anti-tumor activity of PD-1 antibodies through activation of antibody-dependent macrophage-mediated killing of T effector cells. About T and NK-Cell Lymphoma T and NK-cell lymphoma is a set of heterogeneous diseases, accounting for 25-30% of Non-Hodgkin Lymphoma (NHL) cases in China and only about 10% in USA and Europe. There has been little improvement in the past decade when compared to B-cell Non-Hodgkin Lymphoma (B-NHL) as 5-year overall survival rate was only 30% in most common subtypes[1]. The unmet medical needs remain as agents with new mechanism of action to be explored and possibility to improve the treatment paradigm for the disease. About the SINE Compounds Selective Inhibitor of Nuclear Export (SINE) compounds are inhibitors of the major nuclear export protein Exportin 1 (XPO1). Currently, there are three oral SINE compounds, ATG-010 (selinexor), ATG-016 (eltanexor), and ATG-527 (verdinexor), under clinical development. Antengene has obtained exclusive development and commercialisation rights from Karyopharm Therapeutics Inc. (Nasdaq: KPTI) to these three compounds in certain APAC markets.

Read More

DIAGNOSTICS

ZIVO Bioscience Announce Joining Russell Microcap® Index Membership

Zivo | June 28, 2022

ZIVO Bioscience, Inc. is a U.S.-based R&D company dedicated to the study, development and commercialization of natural nutritional compounds and bioactive molecules derived from proprietary algal strains, announced joining the Russell Microcap® Index (^RUMIC) as a result of the Russell indexes annual reconstitution in 2022. Russell Microcap® Index membership that remains in place for a course of 12 months, means automatic inclusion in the appropriate value style and growth indexes. Membership for FTSE Russell's Russell indices is generally determined by market-capitalization rankings and style characteristics. A leader in global indices, FTSE Russell, offers cutting-edge analytics, benchmarking, and data solutions to investors all over the world. FTSE Russell creates thousands of indexes to measure and benchmark markets and asset classes for over 70 nations, covering 98 percent of the world's investable markets. It emphasizes on applying the strict industry norms in governance and index design and also embraces the IOSCO Principles. We are pleased to be added to the Russell Microcap® Index this year. Our inclusion reflects continued interest in our stock along with long- and near-term growth expectations as we evolve and execute on our business strategy, I also look forward to expanding our stock’s exposure to a broader investor audience, including more institutional investors, as a result of ZIVO’s inclusion.” John Payne, President and Chief Executive Officer of ZIVO Bioscience. Russell indexes are increasingly used by institutional investors and investment managers for index funds for benchmarking active investment strategies. Approximately US$12 trillion in assets are benchmarked against Russell’s US indexes.

Read More

DIAGNOSTICS

Zoetis to Complete Acquisition of Basepaws to Strengthen its Portfolio

Zoetis | June 27, 2022

Zoetis Inc. is the world's largest animal health company committed to providing medicine and vaccinations for pets and livestock. The company announced completing the acquisition of a privately held petcare genetics company, Basepaws, which offers early health risk assessments, genetic tests, and analytics to pet owners. The acquisition was first announced on June 7th. However, the financial terms of the purchase were not disclosed. Basepaws, founded by CEO Anna Skaya in 2017, provides genetic screening tools for the diagnosis of several pet diseases, health reports, and individualized breeds by identifying traits, potential hereditary conditions, and biomarkers. With the help of genetic insights from Basepaws, pet owners and doctors can better understand a pet's risk for the disease. This can result in more meaningful interactions and improve the chances of early identification and treatment of the disease. When we first met Basepaws CEO Anna Skaya and the team, we were immediately drawn to their passion for pets and the role that genetic testing and data analytics can have in advancing animal care – a purpose that drives us every day,The addition of Basepaws will enhance our portfolio in the precision animal health space and inform our future pipeline of petcare innovations. Working together, we can continue to provide veterinarians and pet owners with more comprehensive ways to proactively manage the health, wellness, and quality of care for their animals." Zoetis, CEO Kristin Peck.

Read More

Events