Homology Medicines: An Interview With Chief Scientific Officer Albert Seymour

The last year has been a busy one for Bedford, Mass.-based Homology Medicines. In late March 2018, the company launched its initial public offering (IPO), raising an initial $144 million. It has since then built a manufacturing facility for its gene therapy products, and on April 4, 2019, received the go-ahead from the U.S. Food and Drug Administration (FDA) to begin its Phase I/II pheNIX clinical trial for HMI-102 for phenylketonuria (PKU). Homology was one of BioSpace’s NextGen “Class of 2017” Life Science Startups to Watch. Albert Seymour, Homology’s chief science officer, took time out to talk to BioSpace about the company and where it’s headed.

Spotlight

Allevi

Allevi is building tools to design and engineer life. Our goals are to cure disease, eliminate the organ waiting list, reverse climate change, and live on other planets. Our first products are 3D bioprinters, software, and bioinks that print 3D living tissues one layer at a time.

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MedTech

Next-Gen Gene Therapy to Counter Complex Diseases

Article | October 7, 2022

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.

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MedTech

Making Predictions by Digitizing Bioprocessing

Article | July 20, 2022

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

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MedTech

2022 U.S. Market Research Report with COVID-19 Forecasts2

Article | July 11, 2022

The global biotechnology market is expected to grow at a compound annual growth rate (CAGR) of 13.9 percent from 2022 to 2030, with a value estimated at USD 1,023.92 billion in 2021. The market is being propelled by strong government support in the form of initiatives aimed at modernizing the regulatory framework, improving approval processes and reimbursement policies, and standardizing clinical studies. The growing presence of personalized medicine and an increasing number of orphan drug formulations are opening up new avenues for biotechnology applications and driving the influx of emerging and innovative biotechnology companies, which is driving market revenue even further. The 2022 Biotech Research and Development Market Research Report is one of the most comprehensive and in-depth assessments of the industry in the United States, containing over 100 data sets spanning the years 2013 to 2026. This Kentley Insights report contains historical and forecasted market size, product lines, profitability, financial ratios, BCG matrix, state statistics, operating expense details, organizational breakdown, consolidation analysis, employee productivity, price inflation, pay bands for the top 20 industry jobs, trend analysis and forecasts on companies, locations, employees, payroll, and much more. Companies in the Biotech Research and Development industry are primarily engaged in biotechnology research and experimental development. Biotechnology research and development entails the investigation of the use of microorganisms and cellular and bimolecular processes to create or modify living or non-living materials. This biotechnology research and development may result in the development of new biotechnology processes or prototypes of new or genetically altered products that can be replicated, used, or implemented by various industries. This report was created using the findings of extensive business surveys and econometrics. The professionals follow reports with accurate and apt information on market sizing, benchmarking, strategic planning, due diligence, cost-cutting, planning, understanding industry dynamics, forecasting, streamlining, gap analysis, and other ana

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

Article | April 17, 2020

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

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Spotlight

Allevi

Allevi is building tools to design and engineer life. Our goals are to cure disease, eliminate the organ waiting list, reverse climate change, and live on other planets. Our first products are 3D bioprinters, software, and bioinks that print 3D living tissues one layer at a time.

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Cell and Gene Therapy, Diagnostics

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

Globenewswire | July 04, 2023

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

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Cell and Gene Therapy, AI

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

Businesswire | June 05, 2023

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

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Cell and Gene Therapy

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

PR Newswire | May 12, 2023

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

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Cell and Gene Therapy, Diagnostics

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

Globenewswire | July 04, 2023

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

Read More

Cell and Gene Therapy, AI

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

Businesswire | June 05, 2023

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

Read More

Cell and Gene Therapy

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

PR Newswire | May 12, 2023

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

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