How to invest smartly in biotech?

PEKKA SIMULA | September 30, 2019 | 115 views

The subject line is of course provocative. Biotech investments are associated with high risks so, depending on your personal risk profile, it might never be smart for you to invest in biotech. On the other hand, any investment in the stock market is risky. Investing in the Nasdaq Biotechnology Index a decade ago turned your €5 into €20; however, in the past four years it has lost 20%. The big difference in biotech investments compared to more established businesses is that you need to assess a possible future success rather than consider existing cash flow and profit. You cannot calculate a price-earnings ratio if there are no earnings! Instead, one has to estimate the present value of something that may have a significant market potential if it makes it to the market.

Spotlight

AxoGen, Inc.

It's time to rethink nerve repair! AxoGen (NASDAQ: AXGN) is a medical products company with a portfolio of proprietary products and technologies for peripheral nerve reconstruction and regeneration. People suffer traumatic injuries or undergo surgeries that impact the function of their peripheral nerves daily. Damage may cause loss of function and feeling. AxoGen is bringing the science of nerve repair to life with its Avance® Nerve Graft which AxoGen believes is the first and only commercially available allograft for bridging nerve discontinuities, AxoGuard® Nerve Connector, a coaptation aid allowing for close approximation of severed nerves, and AxoGuard® Nerve Protector that protects nerves during the healing process.

OTHER ARTICLES
MEDICAL

Top 10 biotech IPOs in 2019

Article | August 16, 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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MEDTECH

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

Article | September 22, 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.

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MEDICAL

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

Article | July 14, 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.

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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.

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Spotlight

AxoGen, Inc.

It's time to rethink nerve repair! AxoGen (NASDAQ: AXGN) is a medical products company with a portfolio of proprietary products and technologies for peripheral nerve reconstruction and regeneration. People suffer traumatic injuries or undergo surgeries that impact the function of their peripheral nerves daily. Damage may cause loss of function and feeling. AxoGen is bringing the science of nerve repair to life with its Avance® Nerve Graft which AxoGen believes is the first and only commercially available allograft for bridging nerve discontinuities, AxoGuard® Nerve Connector, a coaptation aid allowing for close approximation of severed nerves, and AxoGuard® Nerve Protector that protects nerves during the healing process.

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MEDICAL

Matica Bio Launches MatiMax™ Cell Lines at BIO International Convention

Businesswire | June 07, 2023

Matica Biotechnology, Inc. a contract development and manufacturing organization specializing in the GMP production of cell and gene therapies, today unveiled the company’s new MatiMax™ cell lines at the BIO International Convention, taking place in Boston, MA.. Proprietary to Matica Bio, MatiMaxTM HEK293 and HEK293T cell lines have been rigorously optimized to enable. Faster doubling times – Reaching as low as 17 hours, which can significantly shorten processing timelines; 30% faster than industry standards Improved transfection efficiencies – Reducing the amount of DNA and transfection reagents required for production Increased titers – Supporting higher virus production at a lower cost “The current industry standard for media-based cell line development — typically focused on a limited set of factors, such as glucose, glutamine, nitrogen and ammonia — can potentially limit scalability, which is mission critical when selecting cell lines during the early stages of cell and gene therapy development to stay competitive,” said Michael Stewart, Matica Bio’s chief technology officer. “As a specialized CDMO, we are changing the rules of the game by incorporating a more holistic, multi-dimensional philosophy into our cell line platforms for cell and gene therapies, which incorporates some of the most advanced processes, analytical technologies and media analysis available. This fine tuning is absolutely necessary to reduce bottlenecks for any viral vector expression system, which, in turn, can improve yields and enable doubling times that are faster than other cell line service offerings on the market.”. MatiMaxTM is available for customers to use today, and comes in four different cell line options. HEK293: Adherent HEK293: Suspension HEK293T: Adherent HEK293T: Suspension “As advances in gene modified therapies continue to grow, Matica Bio is well-positioned to support the exploding number of cell and gene therapy candidates under development, which helps our clients reach the clinic faster with high-quality products,” said Andrew Arrage, Matica Bio’s Chief Commercial Officer. “We already have several clients currently using MatiMaxTM lines, which have immediate availability and attractive licensing terms. Our objective is to empower biopharmaceutical developers to achieve their clinical and commercial goals, and, ultimately, treat more patients.”. About Matica Biotechnology, Inc. Matica Biotechnology, Inc. is a contract development and manufacturing organization (CDMO) specializing in fully integrated cell and gene therapy manufacturing. The industry-leading experts at our state-of-the-art facility in College Station, Texas, deliver breakthrough therapies rapidly and safely through innovative manufacturing solutions.

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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.

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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.

Read More

MEDICAL

Matica Bio Launches MatiMax™ Cell Lines at BIO International Convention

Businesswire | June 07, 2023

Matica Biotechnology, Inc. a contract development and manufacturing organization specializing in the GMP production of cell and gene therapies, today unveiled the company’s new MatiMax™ cell lines at the BIO International Convention, taking place in Boston, MA.. Proprietary to Matica Bio, MatiMaxTM HEK293 and HEK293T cell lines have been rigorously optimized to enable. Faster doubling times – Reaching as low as 17 hours, which can significantly shorten processing timelines; 30% faster than industry standards Improved transfection efficiencies – Reducing the amount of DNA and transfection reagents required for production Increased titers – Supporting higher virus production at a lower cost “The current industry standard for media-based cell line development — typically focused on a limited set of factors, such as glucose, glutamine, nitrogen and ammonia — can potentially limit scalability, which is mission critical when selecting cell lines during the early stages of cell and gene therapy development to stay competitive,” said Michael Stewart, Matica Bio’s chief technology officer. “As a specialized CDMO, we are changing the rules of the game by incorporating a more holistic, multi-dimensional philosophy into our cell line platforms for cell and gene therapies, which incorporates some of the most advanced processes, analytical technologies and media analysis available. This fine tuning is absolutely necessary to reduce bottlenecks for any viral vector expression system, which, in turn, can improve yields and enable doubling times that are faster than other cell line service offerings on the market.”. MatiMaxTM is available for customers to use today, and comes in four different cell line options. HEK293: Adherent HEK293: Suspension HEK293T: Adherent HEK293T: Suspension “As advances in gene modified therapies continue to grow, Matica Bio is well-positioned to support the exploding number of cell and gene therapy candidates under development, which helps our clients reach the clinic faster with high-quality products,” said Andrew Arrage, Matica Bio’s Chief Commercial Officer. “We already have several clients currently using MatiMaxTM lines, which have immediate availability and attractive licensing terms. Our objective is to empower biopharmaceutical developers to achieve their clinical and commercial goals, and, ultimately, treat more patients.”. About Matica Biotechnology, Inc. Matica Biotechnology, Inc. is a contract development and manufacturing organization (CDMO) specializing in fully integrated cell and gene therapy manufacturing. The industry-leading experts at our state-of-the-art facility in College Station, Texas, deliver breakthrough therapies rapidly and safely through innovative manufacturing solutions.

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

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

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