Cryopreservation Freezing Solutions

Cryopreservation is designed to minimize damage to cells. For most eukaryotic samples whether bovine sperm, heirloom beer yeast, umbilical cord blood, or genetically engineered CAR-T cells—this means maintaining them cold enough so their biochemical and biophysical processes are effectively halted. But just as important for healthy, viable cells is how they get from 37°C to below about -130°C, as well as how they’re brought out of stasis and reconstituted back to a functional state.

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We are leading the gene therapy revolution. Our integrated product platforms encompass gene therapy, cancer immunotherapy and gene editing – providing us with the potential to treat, and hopefully cure, a broad range of serious diseases.

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Research

Next-Gen Genetics Cancer Therapies Creating Investment Prospects

Article | July 11, 2022

Genetic therapeutics such as genetic engineering and gene therapy are increasingly emerging as one of the most influential and transformed biotechnological solutions around the globe in recent times. These genetic solutions are being assessed across various medical domains, including cancer treatment, neurology, oncology, and ophthalmology. Citing the trend, the genetics industry is estimated to experience a tsunami of approvals, with over 1,000 cell and gene therapy clinical trials currently underway and over 900 companies worldwide focusing on these cutting-edge therapies. Growing Cancer Encourages Advancements in Genetic Technologies With the surging cases of cancers such as leukemias, carcinomas, lymphomas, and others, patients worldwide are increasing their spending on adopting novel therapeutic solutions for non-recurring treatment of the disease, such as gene therapy, genetic engineering, T-cell therapy, and gene editing. As per a study by the Fight Cancer Organization, spending on the treatment of cancer increased to $200.7 billion, and the amount is anticipated to exceed $245 billion by the end of 2030. Growing revenue prospects are encouraging biotechnology and biopharmaceutical companies to develop novel genetic solutions for cancer treatment. For instance, Bristol-Myers Squibb K.K., a Japanese pharmaceutical company, introduced a B-cell maturation antigen (BCMA)-directed chimeric antigen receptor (CAR) T cell immunotherapy, Abecma, for the treatment of relapsed or refractory (R/R) multiple myeloma in 2022. Amid a New Market: Genetics Will Attract Massive Investments Despite several developments and technological advancements, genetics is still considered to be in a nascent stage, providing significant prospects for growth to the companies that are already operating in the domain. Genetics solutions such as gene therapies, gene editing, and T-cell immunotherapy are emerging as highly active treatments across various medical fields, resulting in increasing research and development activities across the domain, drawing significant attention from investors. Given the potential of genetic treatments and the focus on finding new ways to treat cancer and other related diseases, it's easy to understand why companies are investing in the domain. For instance, Pfizer has recently announced an investment of around $800 million to construct development facilities supporting gene therapy manufacturing from initial preclinical research through final commercial-scale production. Due to these advancements, cell and gene therapies are forecast to grow from $4 billion annually to more than $45 billion, exhibiting growth at a 63% CAGR. The Future of Genetics Though there is a significant rise in advancement in genetic technologies and developments, the number of approved genetic treatments remains extremely small. However, with gene transfer and CRISPR solutions emerging as new modalities for cancer treatment, the start-up companies will attract a growing amount and proportion of private and public investments. This is expected present a tremendous opportunity for biopharma and biotechnology investors to help fund and benefit from the medical industry's shift from traditional treatments to cutting-edge genetic therapeutics in the coming years.

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Medical

How to Choose a Reliable Biotech Clinical Trial Management System?

Article | August 16, 2022

Introduction The medical and life-science industries are experiencing a robust transformation with the increasing prevalence of various types of diseases, including infectious diseases, chronic disorders, and acute conditions around the world. As a result, a significant rise in demand for more effective therapeutic drugs and bionics is being witnessed, leading to a swift increase in the number of clinical trials. For a successful trial, it is important for biotech companies to ensure the data submitted to regulatory bodies regarding clinical trials is accurate, reliable, and definitive from an ethical point of view. A reliable clinical trial management system plays a vital role in collecting, monitoring, and managing clinical data. The availability of high-quality clinical data also helps clinical research institutions make efficient treatment decisions and provide proper patient care. Hence, a number of biotech companies and research organizations are focusing on leveraging innovative clinical trial management solutions to handle a large amount of data, particularly in multi-center trials, and generate reliable, high-quality, and statistically sound data from clinical trials. However, selecting the most appropriate and reliable clinical trial management system is vital for the clinical trial's success. Let's see some of the steps that will assist these firms in choosing the right CTMS. Key Steps for Selecting Right Biotech Clinical Trial Management System Prioritize Study Needs Considering and prioritizing study needs is a crucial step in choosing the most reliable clinical trial management system for biotech companies. Prioritizing helps them to identify a solution that improves the study's quality and removes uncertainty for researchers when faced with difficult choices. Hence, biotech and life-science organizations should choose a clinical trial system that is simple to use, well-organized, and suitably designed to minimize the number of clicks required to complete a task. Select CTMS with Multiple Integrations Integrated clinical trial management systems provide the best value for the companies’ funds as they guarantee the smooth functioning of research protocols. In addition, integrations are necessary to fully understand the importance and advantages of clinical trial management software for ensuring smooth transitions between site management and data collection. Biotech and clinical research should look for CTMS platforms that can integrate with electronic medical record (EMR) platforms and clinical research process content (CRPC) billing grids. This will allow them to use the same billing designations and ensure compliance while minimizing the need for duplicate processes. Ensure System Compliance and Security Clinical research organizations need to adhere to a plethora of complex regulations in order to ensure compliance with one of the most challenging environments of principles, which is information security and privacy. Security and system compliance are vital aspects of choosing the right CTMS solutions for biotech firms as they assist in building trust and form a part of the system’s duties. While selecting CTMS systems, it is essential for companies engaged in clinical research to ensure that these platforms are able to configure both, group and individual permissions, along with having a data backup and recovery plan for hosted systems. This will allow companies to assess the privacy and security implications of research and anticipate complications that may arise in each phase of the project. Assess the Scalability Choosing a scalable CTMS that can accommodate various types of fluctuations and expansions enables biotech and clinical firms to quickly adapt to fast-changing trends and demand spikes while reducing maintenance costs and enhancing user agility. As scalability also means secure and expanded data storage, these businesses should instead use SaaS solutions than manually manage an ever-growing collection of hard drives. The right CTMS ensures accommodating the firm’s availability requirements without incurring the capital costs associated with expanding a physical infrastructure. The Closing Thought A well-executed and successful clinical trial involves multiple stages and processes. Several quality controls and stringent adherence to regulations are essential for the steps, along with efficient cross-departmental processes and procedures. Incorporating the right CTMS paves the way for paperless data collection, regulatory filing, and fiscal management tools for biotech researchers and administrative personnel.

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Medical

Data Analytics: A Groundbreaking Technology in Biotech

Article | July 14, 2022

Biotechnology is a vast discipline of biology that employs diverse biological systems to create solutions that can significantly alter the ways in which they operate across various domains. That said, biotechnology is not a new notion. It has existed for millennia, with ancient civilizations using its earliest incarnations to cultivate crops and create alcoholic beverages. Today, the biotechnology industry has developed by leaps and bounds and has amassed a vast quantity of scientific data through study and research. Given the importance of data in the biotechnology business, it is not difficult to understand why biotech companies utilize data analytics. Modern data analytics tools have made it possible for researchers in the biotech industry to build predictive analytics models and gain knowledge about the most efficient approaches to accomplish their desired goals and objectives. Data analytics is increasingly being adopted by biotech businesses to better understand their industry and foresee any problems down the road. How is Data Analytics Revolutionizing Fields in Biotechnology? Today's business and scientific fields greatly benefit from data. Without the analysis of vast information libraries that provide new insights and enable new innovations, no industry can really advance. Being highly reliant on big data analytics, biotech is not an exception in this regard. With the tools and methods that help scientists systematize their findings and speed up their research for better and safer results, data analytics is making deeper inroads into the biotechnology industry. It is emerging as a crucial link between knowledge and information and is extensively being used for purposes other than just examining the information that is already available. The following are a few of the cutting-edge biotechnology applications of data analytics Genomics and Disease Treatment Pharmaceutical Drug Discovery Drug Recycling and Safety Agriculture and Agri-products Environmental Damage Mitigation Data Analytics Possibilities in Biotechnology With data analytics becoming an integral part of how biotech businesses operate, biotechnologists and related stakeholders need to understand its emergence and crucial role. Data analytics has opened new frontiers in the realm of biotechnology. Thanks to developments in data analytics, research and development activities that once took years may now be accomplished in a matter of months. Also, now scientists have access to biological, social, and environmental insights that can be exploited to create more effective and sustainable products. By understanding the importance of data-related tools and techniques applications, biotech companies are aiming to invest in the popularizing technology to stay updated in the fast-paced biotechnology industry.

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Wisconsin biotech companies could play key roles in long-term economic recovery from COVID-19 pandemic

Article | April 19, 2020

Whether it’s called a modern “Manhattan Project” or a medical moon shot, the concept of long-term economic recovery rests on how confident people are they won’t risk serious illness by venturing forth in public again. Wisconsin stands to be a significant part of such an undertaking, whatever it’s called. The shorter-term debate is well under way over the gradual lifting of COVID-19 emergency rules, such as the now-extended “safer-at-home” order in Wisconsin. At least a dozen states, including regional coalitions on the East and West coasts, are exploring next steps as they seek to balance responses to the virus with calls for reopening the economy, at least, in part. Wisconsin’s ability to shape longer-term responses will come from private and public resources, which range from companies engaged in production of diagnostics.

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We are leading the gene therapy revolution. Our integrated product platforms encompass gene therapy, cancer immunotherapy and gene editing – providing us with the potential to treat, and hopefully cure, a broad range of serious diseases.

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Personalis and ClearNote Health Announce Partnership to Advance Epigenomic Technology

Personalis, Inc. | February 05, 2024

Personalis, Inc. a leader in advanced genomics for cancer, and ClearNote Health, Inc., a pioneer in epigenomic technologies, today unveiled an alliance through which Personalis will expand its pharmaceutical service offerings by distributing ClearNote’s cutting-edge epigenomic 5-hydroxymethylcytosine (5hmC) platform. “We have built a unique pharma channel based on our industry-leading tissue and MRD assays and ClearNote Health’s blood-based epigenomic approach is complementary to our offering as it helps our customers deepen their understanding of a patient’s response to immunotherapies,” said Chris Hall, CEO and President of Personalis. “Commercial partnerships such as this broaden our portfolio and are expected to appeal to a wide range of biopharma customers and accelerate our revenue growth.” Echoing this sentiment, Dave Mullarkey, CEO of ClearNote Health, remarked, “Partnering with Personalis presents an excellent opportunity to bring our 5hmC technology to the forefront of cancer research. This alliance is a testament to the synergy between our two companies, enabling us to expand our reach and significantly impact the biopharmaceutical industry. Together, we can accelerate the development of personalized therapies and make a real difference in the lives of patients.” ClearNote Health’s Epigenomics Platform represents a groundbreaking advance in cancer detection, offering real-time insights into disease-specific pathways. By tracking changes in 5hmC levels coupled with artificial intelligence-based analytical methods, the platform can detect cancer earlier, monitor disease progression, understand mechanisms of resistance, and identify promising drug targets and biomarkers. These insights are invaluable for optimizing drug development programs and delivering more effective treatments to patients. The Epigenomics Platform identifies changes in gene activation and gene regulation by labeling specific changes in the 5hmC landscape from plasma-derived cell-free DNA. This rich biological information, as part of clinical trials, enables the monitoring of cancer therapies in real time and contributes to an understanding of drug resistance mechanisms. The partnership marks a pivotal moment in cancer research, leveraging the strengths of both companies to offer unparalleled solutions in the biopharmaceutical industry. About ClearNote Heath, Inc. ClearNote Health is a cancer detection company focused on enabling people at risk for high-mortality cancers to live longer, healthier lives. Utilizing a standard blood draw, the company applies its proprietary epigenomic platform, combining biology and artificial intelligence, to identify DNA-based changes in biology as cancer develops. With lead programs in non-invasive early detection of pancreatic and ovarian cancers in patients at the highest risk for these diseases, ClearNote Health identifies cancers before they progress and when patients are most likely to benefit from treatment. ClearNote Health’s first commercially available test is the Avantect™ Pancreatic Cancer Test, which detects the presence of pancreatic cancer signals in patients at high risk of the disease, including those recently diagnosed with Type 2 diabetes. ClearNote Health is headquartered in San Diego, with additional presence in the San Francisco Bay area and internationally. The company’s CLIA- and CAP-accredited laboratory is located in San Diego, Calif. About Personalis, Inc. At Personalis, we are transforming the active management of cancer through breakthrough personalized testing. We aim to drive a new paradigm for cancer management, guiding care from biopsy through the life of the patient. Our highly sensitive assays combine tumor-and-normal profiling with proprietary algorithms to deliver advanced insights even as cancer evolves over time. Our products are designed to detect minimal residual disease (MRD) and recurrence at the earliest time points, enable the selection of targeted therapies based on ultra-comprehensive genomic profiling, and enhance biomarker strategy for drug development. Personalis is based in Fremont, California.

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Medical

Jnana Therapeutics Announces Positive Clinical Proof of Concept Achieved with JNT-517, a Potential First-in-Class Oral Treatment for PKU

Jnana Therapeutics, Inc. | February 02, 2024

Jnana Therapeutics, a clinical-stage biotechnology company leveraging its next-generation chemoproteomics platform to discover medicines for challenging-to-drug targets, today announced positive, statistically significant interim results from its ongoing clinical study of JNT-517 in individuals with phenylketonuria (PKU). JNT-517, a small molecule inhibitor of the phenylalanine (Phe) transporter SLC6A19, is being evaluated as a potential first-in-class oral treatment for PKU across all ages and genotypes. On the basis of these positive interim results, Jnana has adapted the Phase 1b trial design to support the potential for accelerated progression of JNT-517. “There is an urgent need for an oral, safe, and efficacious therapy for the more than 60% of individuals with PKU not currently on therapy. Across the spectrum of mild to severe disease, our results demonstrate a robust, sustained reduction in blood Phe levels, the registrational endpoint for PKU, giving us high confidence in the path forward for JNT-517,” said George Vratsanos, M.D., Chief Medical Officer and Head of R&D at Jnana Therapeutics. “We are also encouraged by this validation of the power of our RAPID platform to discover small molecules with compelling clinical benefit against challenging-to-drug targets.” JNT-517 is being studied in a randomized, double-blind, placebo-controlled trial in individuals with mild to severe PKU. Following a 28-day screening period focused predominantly on ensuring an average blood Phe level of >600µM, study participants were randomized with no run-in period to 75mg of JNT-517 twice daily (BID) or placebo. The planned interim analysis was based on 13 participants, eight dosed with JNT-517 and five dosed with placebo over 28 days, and demonstrated the following results JNT-517 led to a statistically significant (p=0.0019 vs. placebo) mean blood Phe reduction from baseline of 51%, measured per-protocol at day 28. A high response rate was seen where seven of eight (88%) treated participants achieved >30% reduction in blood Phe from baseline; five of eight (63%) achieved >45% reduction; and two of eight (25%) achieved >65% reduction. A robust response was seen across participants treated with JNT-517 irrespective of baseline blood Phe levels, which ranged from 593µM to 1,526µM with a mean of 1,124µM. A rapid onset of effect was observed with significant blood Phe reduction achieved within seven days after commencing dosing, which was sustained through the full 28 days of dosing. JNT-517 was safe and well tolerated with no serious adverse events and no clinically significant changes in laboratory parameters, consistent with the safety profile demonstrated in the Phase 1a healthy volunteer study. "JNT-517 represents a completely new therapeutic approach that could transform the current treatment paradigm in PKU, in particular for individuals with severe, or classical, PKU where there is the highest unmet medical need,” said Cary O. Harding, M.D., study investigator and Professor of Molecular and Medical Genetics at Oregon Health and Science University School of Medicine. “I am encouraged by the clinical results to date and look forward to working with Jnana and the PKU community to continue to advance this program.” Based on these interim results, Jnana has adapted the protocol of the ongoing trial to include dose exploration. Jnana expects topline data from the second dose cohort in mid-2024 and plans to submit full data from the two dose cohorts for presentation at a scientific meeting in the second half of 2024. Jnana anticipates the company will engage regulators in the second half of 2024 and seek to advance JNT-517 directly into a pivotal Phase 3 study in the first half of 2025. JNT-517 Phase 1b Clinical Trial The ongoing clinical program includes a randomized, double-blind, placebo-controlled trial evaluating the safety, tolerability, pharmacokinetics, and effect on blood and urinary Phe of JNT-517 dosed over a four-week period in individuals diagnosed with PKU. The study dosed its first participant with PKU in August 2023 and is enrolling individuals aged 18 to 65 at clinical sites in the United States and Australia. For more information about the study, please see clinicaltrials.gov (NCT05781399). About JNT-517 JNT-517 is a selective small molecule inhibitor of the Phe transporter SLC6A19 that has the potential to be a first-in-class oral therapy used to treat any person with PKU, regardless of age or genotype. JNT-517 acts at a novel, cryptic allosteric site to block kidney reabsorption of Phe and offers a promising new approach to reduce blood Phe levels. The U.S. Food and Drug Administration granted JNT-517 Rare Pediatric Disease Designation in late 2022. About PKU PKU is a rare inherited metabolic disorder caused by a deficiency of the enzyme phenylalanine hydroxylase (PAH). This enzyme is required for the breakdown of phenylalanine (Phe), an amino acid found in all protein-containing foods. When PAH is deficient or defective, Phe accumulates to abnormally high levels in the blood. If left untreated, toxic levels of Phe in the blood can result in progressive and severe neurological impairment and neuropsychological complications. The SLC transporter SLC6A19 is responsible for kidney reabsorption of Phe back into the bloodstream, and the inhibition of SLC6A19 offers a novel, oral approach for the treatment of PKU by facilitating urinary excretion of excess Phe. About Jnana Therapeutics Jnana Therapeutics is a clinical-stage biotechnology company leveraging its next-generation RAPID chemoproteomics platform to discover medicines for highly validated, challenging-to-drug targets to treat diseases with high unmet needs. Jnana is focused on developing first- and best-in-class therapies to treat a wide range of diseases, including rare diseases and immune-mediated diseases. Jnana’s wholly owned lead program, JNT-517, which targets an allosteric site on the phenylalanine transporter SLC6A19, is a potential first-in-class oral approach for the treatment of PKU, a rare genetic metabolic disease. Located in Boston, Jnana brings together scientific leaders in small molecule drug discovery and development, a highly experienced management team, and the backing of leading life science investors Bain Capital Life Sciences, RA Capital Management, Polaris Partners, Versant Ventures, Avalon Ventures, Pfizer Ventures, and AbbVie Ventures.

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Twist Bioscience Expands Express Genes Rapid Synthesis Service

Twist Bioscience Corporation | January 30, 2024

Twist Bioscience Corporation a company enabling customers to succeed through its offering of high-quality synthetic DNA using its silicon platform, today announced the expansion of Twist Express Genes, a new leading gene synthesis service with an order to shipping turnaround starting at five business days1, to include larger DNA preparations up to 1 milligram. The Express Genes offering, initially launched in November 2023, now extends to larger midiprep (10μg to 100 μg) and maxiprep (100μg to 1mg) DNA preparations. As with Twist standard speed Clonal Genes, all Twist Express Genes are NGS-verified as clonally perfect. “Since the initial launch of our Express Genes service in November, we have received positive and enthusiastic feedback from existing customers on our consistent and rapid turnaround time. By expanding Express Genes to include midiprep and maxiprep, we can now offer rapid gene synthesis at all gene prep scales, enabling large scale experimentation for pharmaceutical, biotechnology, academic and industrial chemical researchers,” said Emily M. Leproust, Ph.D., CEO and co-founder of Twist Bioscience. “With the expanded offering of Twist Express Genes, more researchers can gain access to Twist genes fast and at scale, including those using alternative providers and those cloning their own genes. The ability to order and receive synthetic DNA fast and at scale means more time for cutting-edge research, more make-test-learn cycles, and an accelerated journey to game-changing discoveries.” Twist leverages its ability to miniaturize the chemical reaction to create DNA using its silicon-based DNA synthesis platform along with expertise, software, optimized processes and the expanded layout of its Wilsonville facility to deliver Clonal Genes and Gene Fragments at scale and with rapid turnaround times. Twist’s Express Genes are offered with dynamic pricing, which reflects market demand and manufacturing capacity in a responsive premium price. Customers are able to quickly and easily place orders for Express Genes through Twist’s ecommerce platform. Twist Express Genes Twist Express Genes are 0.3kb to 5.0kb in length. Due to scale enabled by Twist’s platform, orders of any size both small and large can be filled, and as with standard speed Twist Clonal Genes, Twist Express Genes are NGS sequence-verified as one hundred percent accurate. Twist Express Genes can be cloned into catalog vectors or custom vectors so that customers can move right to experimentation. They can also be shipped in customers’ preferred delivery formats, including tubes and plates. For more information and product specifications click here. Current turnaround times for some Twist Bioscience products Express Genes1 (50ng-10µg): starting at 5 business days, now for up to 10µg Standard Clonal Genes (50ng-10µg): starting at 10 business days Express Genes1 (10µg-1mg): starting at 8 business days Standard Clonal Genes (10µg-1mg): starting at 13 business days 1 Express Clonal Genes ship in 5-7 business days. DNA prep sizes including 10 μg - 100 μg, and 100 μg - 1 mg may incur an additional 3-5 days for synthesis. Orders placed outside of the US will incur additional delivery turnaround time. Express Genes orders that require new custom vector onboarding will incur an additional 1-2 weeks for processing. 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.

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Medical

Personalis and ClearNote Health Announce Partnership to Advance Epigenomic Technology

Personalis, Inc. | February 05, 2024

Personalis, Inc. a leader in advanced genomics for cancer, and ClearNote Health, Inc., a pioneer in epigenomic technologies, today unveiled an alliance through which Personalis will expand its pharmaceutical service offerings by distributing ClearNote’s cutting-edge epigenomic 5-hydroxymethylcytosine (5hmC) platform. “We have built a unique pharma channel based on our industry-leading tissue and MRD assays and ClearNote Health’s blood-based epigenomic approach is complementary to our offering as it helps our customers deepen their understanding of a patient’s response to immunotherapies,” said Chris Hall, CEO and President of Personalis. “Commercial partnerships such as this broaden our portfolio and are expected to appeal to a wide range of biopharma customers and accelerate our revenue growth.” Echoing this sentiment, Dave Mullarkey, CEO of ClearNote Health, remarked, “Partnering with Personalis presents an excellent opportunity to bring our 5hmC technology to the forefront of cancer research. This alliance is a testament to the synergy between our two companies, enabling us to expand our reach and significantly impact the biopharmaceutical industry. Together, we can accelerate the development of personalized therapies and make a real difference in the lives of patients.” ClearNote Health’s Epigenomics Platform represents a groundbreaking advance in cancer detection, offering real-time insights into disease-specific pathways. By tracking changes in 5hmC levels coupled with artificial intelligence-based analytical methods, the platform can detect cancer earlier, monitor disease progression, understand mechanisms of resistance, and identify promising drug targets and biomarkers. These insights are invaluable for optimizing drug development programs and delivering more effective treatments to patients. The Epigenomics Platform identifies changes in gene activation and gene regulation by labeling specific changes in the 5hmC landscape from plasma-derived cell-free DNA. This rich biological information, as part of clinical trials, enables the monitoring of cancer therapies in real time and contributes to an understanding of drug resistance mechanisms. The partnership marks a pivotal moment in cancer research, leveraging the strengths of both companies to offer unparalleled solutions in the biopharmaceutical industry. About ClearNote Heath, Inc. ClearNote Health is a cancer detection company focused on enabling people at risk for high-mortality cancers to live longer, healthier lives. Utilizing a standard blood draw, the company applies its proprietary epigenomic platform, combining biology and artificial intelligence, to identify DNA-based changes in biology as cancer develops. With lead programs in non-invasive early detection of pancreatic and ovarian cancers in patients at the highest risk for these diseases, ClearNote Health identifies cancers before they progress and when patients are most likely to benefit from treatment. ClearNote Health’s first commercially available test is the Avantect™ Pancreatic Cancer Test, which detects the presence of pancreatic cancer signals in patients at high risk of the disease, including those recently diagnosed with Type 2 diabetes. ClearNote Health is headquartered in San Diego, with additional presence in the San Francisco Bay area and internationally. The company’s CLIA- and CAP-accredited laboratory is located in San Diego, Calif. About Personalis, Inc. At Personalis, we are transforming the active management of cancer through breakthrough personalized testing. We aim to drive a new paradigm for cancer management, guiding care from biopsy through the life of the patient. Our highly sensitive assays combine tumor-and-normal profiling with proprietary algorithms to deliver advanced insights even as cancer evolves over time. Our products are designed to detect minimal residual disease (MRD) and recurrence at the earliest time points, enable the selection of targeted therapies based on ultra-comprehensive genomic profiling, and enhance biomarker strategy for drug development. Personalis is based in Fremont, California.

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Medical

Jnana Therapeutics Announces Positive Clinical Proof of Concept Achieved with JNT-517, a Potential First-in-Class Oral Treatment for PKU

Jnana Therapeutics, Inc. | February 02, 2024

Jnana Therapeutics, a clinical-stage biotechnology company leveraging its next-generation chemoproteomics platform to discover medicines for challenging-to-drug targets, today announced positive, statistically significant interim results from its ongoing clinical study of JNT-517 in individuals with phenylketonuria (PKU). JNT-517, a small molecule inhibitor of the phenylalanine (Phe) transporter SLC6A19, is being evaluated as a potential first-in-class oral treatment for PKU across all ages and genotypes. On the basis of these positive interim results, Jnana has adapted the Phase 1b trial design to support the potential for accelerated progression of JNT-517. “There is an urgent need for an oral, safe, and efficacious therapy for the more than 60% of individuals with PKU not currently on therapy. Across the spectrum of mild to severe disease, our results demonstrate a robust, sustained reduction in blood Phe levels, the registrational endpoint for PKU, giving us high confidence in the path forward for JNT-517,” said George Vratsanos, M.D., Chief Medical Officer and Head of R&D at Jnana Therapeutics. “We are also encouraged by this validation of the power of our RAPID platform to discover small molecules with compelling clinical benefit against challenging-to-drug targets.” JNT-517 is being studied in a randomized, double-blind, placebo-controlled trial in individuals with mild to severe PKU. Following a 28-day screening period focused predominantly on ensuring an average blood Phe level of >600µM, study participants were randomized with no run-in period to 75mg of JNT-517 twice daily (BID) or placebo. The planned interim analysis was based on 13 participants, eight dosed with JNT-517 and five dosed with placebo over 28 days, and demonstrated the following results JNT-517 led to a statistically significant (p=0.0019 vs. placebo) mean blood Phe reduction from baseline of 51%, measured per-protocol at day 28. A high response rate was seen where seven of eight (88%) treated participants achieved >30% reduction in blood Phe from baseline; five of eight (63%) achieved >45% reduction; and two of eight (25%) achieved >65% reduction. A robust response was seen across participants treated with JNT-517 irrespective of baseline blood Phe levels, which ranged from 593µM to 1,526µM with a mean of 1,124µM. A rapid onset of effect was observed with significant blood Phe reduction achieved within seven days after commencing dosing, which was sustained through the full 28 days of dosing. JNT-517 was safe and well tolerated with no serious adverse events and no clinically significant changes in laboratory parameters, consistent with the safety profile demonstrated in the Phase 1a healthy volunteer study. "JNT-517 represents a completely new therapeutic approach that could transform the current treatment paradigm in PKU, in particular for individuals with severe, or classical, PKU where there is the highest unmet medical need,” said Cary O. Harding, M.D., study investigator and Professor of Molecular and Medical Genetics at Oregon Health and Science University School of Medicine. “I am encouraged by the clinical results to date and look forward to working with Jnana and the PKU community to continue to advance this program.” Based on these interim results, Jnana has adapted the protocol of the ongoing trial to include dose exploration. Jnana expects topline data from the second dose cohort in mid-2024 and plans to submit full data from the two dose cohorts for presentation at a scientific meeting in the second half of 2024. Jnana anticipates the company will engage regulators in the second half of 2024 and seek to advance JNT-517 directly into a pivotal Phase 3 study in the first half of 2025. JNT-517 Phase 1b Clinical Trial The ongoing clinical program includes a randomized, double-blind, placebo-controlled trial evaluating the safety, tolerability, pharmacokinetics, and effect on blood and urinary Phe of JNT-517 dosed over a four-week period in individuals diagnosed with PKU. The study dosed its first participant with PKU in August 2023 and is enrolling individuals aged 18 to 65 at clinical sites in the United States and Australia. For more information about the study, please see clinicaltrials.gov (NCT05781399). About JNT-517 JNT-517 is a selective small molecule inhibitor of the Phe transporter SLC6A19 that has the potential to be a first-in-class oral therapy used to treat any person with PKU, regardless of age or genotype. JNT-517 acts at a novel, cryptic allosteric site to block kidney reabsorption of Phe and offers a promising new approach to reduce blood Phe levels. The U.S. Food and Drug Administration granted JNT-517 Rare Pediatric Disease Designation in late 2022. About PKU PKU is a rare inherited metabolic disorder caused by a deficiency of the enzyme phenylalanine hydroxylase (PAH). This enzyme is required for the breakdown of phenylalanine (Phe), an amino acid found in all protein-containing foods. When PAH is deficient or defective, Phe accumulates to abnormally high levels in the blood. If left untreated, toxic levels of Phe in the blood can result in progressive and severe neurological impairment and neuropsychological complications. The SLC transporter SLC6A19 is responsible for kidney reabsorption of Phe back into the bloodstream, and the inhibition of SLC6A19 offers a novel, oral approach for the treatment of PKU by facilitating urinary excretion of excess Phe. About Jnana Therapeutics Jnana Therapeutics is a clinical-stage biotechnology company leveraging its next-generation RAPID chemoproteomics platform to discover medicines for highly validated, challenging-to-drug targets to treat diseases with high unmet needs. Jnana is focused on developing first- and best-in-class therapies to treat a wide range of diseases, including rare diseases and immune-mediated diseases. Jnana’s wholly owned lead program, JNT-517, which targets an allosteric site on the phenylalanine transporter SLC6A19, is a potential first-in-class oral approach for the treatment of PKU, a rare genetic metabolic disease. Located in Boston, Jnana brings together scientific leaders in small molecule drug discovery and development, a highly experienced management team, and the backing of leading life science investors Bain Capital Life Sciences, RA Capital Management, Polaris Partners, Versant Ventures, Avalon Ventures, Pfizer Ventures, and AbbVie Ventures.

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Medical

Twist Bioscience Expands Express Genes Rapid Synthesis Service

Twist Bioscience Corporation | January 30, 2024

Twist Bioscience Corporation a company enabling customers to succeed through its offering of high-quality synthetic DNA using its silicon platform, today announced the expansion of Twist Express Genes, a new leading gene synthesis service with an order to shipping turnaround starting at five business days1, to include larger DNA preparations up to 1 milligram. The Express Genes offering, initially launched in November 2023, now extends to larger midiprep (10μg to 100 μg) and maxiprep (100μg to 1mg) DNA preparations. As with Twist standard speed Clonal Genes, all Twist Express Genes are NGS-verified as clonally perfect. “Since the initial launch of our Express Genes service in November, we have received positive and enthusiastic feedback from existing customers on our consistent and rapid turnaround time. By expanding Express Genes to include midiprep and maxiprep, we can now offer rapid gene synthesis at all gene prep scales, enabling large scale experimentation for pharmaceutical, biotechnology, academic and industrial chemical researchers,” said Emily M. Leproust, Ph.D., CEO and co-founder of Twist Bioscience. “With the expanded offering of Twist Express Genes, more researchers can gain access to Twist genes fast and at scale, including those using alternative providers and those cloning their own genes. The ability to order and receive synthetic DNA fast and at scale means more time for cutting-edge research, more make-test-learn cycles, and an accelerated journey to game-changing discoveries.” Twist leverages its ability to miniaturize the chemical reaction to create DNA using its silicon-based DNA synthesis platform along with expertise, software, optimized processes and the expanded layout of its Wilsonville facility to deliver Clonal Genes and Gene Fragments at scale and with rapid turnaround times. Twist’s Express Genes are offered with dynamic pricing, which reflects market demand and manufacturing capacity in a responsive premium price. Customers are able to quickly and easily place orders for Express Genes through Twist’s ecommerce platform. Twist Express Genes Twist Express Genes are 0.3kb to 5.0kb in length. Due to scale enabled by Twist’s platform, orders of any size both small and large can be filled, and as with standard speed Twist Clonal Genes, Twist Express Genes are NGS sequence-verified as one hundred percent accurate. Twist Express Genes can be cloned into catalog vectors or custom vectors so that customers can move right to experimentation. They can also be shipped in customers’ preferred delivery formats, including tubes and plates. For more information and product specifications click here. Current turnaround times for some Twist Bioscience products Express Genes1 (50ng-10µg): starting at 5 business days, now for up to 10µg Standard Clonal Genes (50ng-10µg): starting at 10 business days Express Genes1 (10µg-1mg): starting at 8 business days Standard Clonal Genes (10µg-1mg): starting at 13 business days 1 Express Clonal Genes ship in 5-7 business days. DNA prep sizes including 10 μg - 100 μg, and 100 μg - 1 mg may incur an additional 3-5 days for synthesis. Orders placed outside of the US will incur additional delivery turnaround time. Express Genes orders that require new custom vector onboarding will incur an additional 1-2 weeks for processing. 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.

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