Entrada Therapeutics Appoints John F. Crowley to Board of Directors

Entrada Therapeutics, a biotechnology company dedicated to transforming the treatment of devastating diseases through the intracellular delivery of biologics, today announced the appointment of John F. Crowley as an independent board member to the Company's Board of Directors.

Spotlight

Charm ATP Detection

Adenosine triphosphate (ATP) is the energy source of all living cells. It has gained popularity as a testing source to determine sanitation or hygiene levels since associated testing methods deliver significantly faster results than plating. Monitoring sanitation is essential in preventative programs in dairy, food & beverage, feed & grain, water, hospitals and many other industries.

OTHER ARTICLES
Medical

Expansion of BioPharma: Opportunities and Investments

Article | August 16, 2022

Biopharmaceutical innovations are among the most ingenious and refined achievements of modern medical science. New concepts, techniques, and therapies are emerging, such as the cell therapy Provenge, which can be used to treat cancer, and gene therapies, which provide even more amazing promises of disease remission and regenerative medicine. In addition, the COVID-19 pandemic has caused a huge boom in the pharmaceutical industry. This is because more and more attention is being paid to increasing manufacturing capacity and starting new research on drug development. Biopharma: Leading the Way in the Pharma Sector In the past couple of years, the biopharmaceutical sector has deepened its roots across the medical and pharmaceutical industries, on account of the transformation of pharmaceutical companies towards biotechnology, creating opportunities for growth. Also, growing advancements in technologies such as 3D bioprinting, biosensors, and gene editing, along with the integration of advanced artificial intelligence and virtual and augmented reality are estimated to further create prospects for growth. According to a study, the biopharmaceutical sector makes nearly $163 billion around the world and grows by more than 8% each year, which is twice as fast as the traditional pharma sector. Massive Investments Directed Towards Biopharma Investing in biotech research and development (R&D) has yielded better returns than the pharma industry average. Hence, a number of pharmaceutical companies are shifting their presence toward biopharma to capitalize on the upcoming opportunities by investing in and expanding their biotechnology infrastructure. For instance, Thermo Fisher Scientific Inc., an American manufacturer of scientific instrumentation, reagents and consumables, and software services, announced an investment of $97 million to expand its bioanalytical laboratory operations into three new locations in the U.S. With this investment, the company will add 150,000 square feet of scientific workspace and install the most advanced drug development technologies to produce life-changing medicines for patients in need.

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Research

Immunology: A New Frontier in Medical Science

Article | July 11, 2022

Introduction Recent developments in the bioengineering of monoclonal antibodies (mAbs) have revolutionized the treatment of numerous rheumatic and immunological disorders. Currently, several immunological disorders are successfully being targeted and treated using innovative medical techniques such as immunotherapy. Leading companies are increasingly investing in research activities to expand the usage and application of immunology for the treatment of various infectious diseases, including multiple sclerosis, inflammatory bowel disorders, lupus, and psoriasis, leading companies are increasingly investing in research activities. Today, the efforts of researchers in immunology, with a long history of study and research, have borne fruit, as bioengineered mAbs are now being employed in clinical practices. Accelerating Investments: Paving the Way for Immunology The increasing prevalence of infectious diseases, cancer, and immune-mediated inflammatory disorders (IMIDs) is raising the need for more precise classification and an in-depth understanding of the pathology underlying these ailments. Numerous leaders in the biotechnology domain are thus focusing on undertaking numerous strategies, such as new facility launches and collaborations, to address the need by finding deeper inroads into immunology and its use in disease treatments. For instance, in 2022, the University of Texas MD Anderson Cancer Center announced the launch of a visionary research and innovation hub, the James P. Allison Institute, to find new roads in immunotherapy, develop new treatments, and foster groundbreaking science. These developments will result in better diagnosis through the use of selective biomarkers, and early detection of fatal diseases and their treatment, which will prevent complications from happening. Also, the identification of high-risk populations through a deeper understanding of genetic and environmental factors can assist in the prevention of disease through immunotherapy. The Way Forward Immunology has led to the development of biotechnology, making it possible to develop novel drugs and vaccines, as well as diagnostic tests, that can be used to prevent, diagnose, and treat a wide range of autoimmune, infectious, and cancerous diseases. With the rapid advancement in technology and the integration of artificial intelligence, immunology is finding its way into an array of domains and industries, encompassing several research areas including medicine, pharmaceuticals, agriculture, and space. Today, not only researchers but also leading biotech and pharmaceutical companies have recognized that conventional therapies with pharmaceutical and chemical products are being replaced by products derived from immunology. This is because they work well for health problems, are environmentally friendly, and are also emerging as a wealth-generating business in the medical field.

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MedTech

Making Predictions by Digitizing Bioprocessing

Article | July 16, 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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Industrial Impact

AI and Biotechnology: The Future of Healthcare Industry

Article | January 20, 2021

Artificial intelligence has grasped the foundation in biotech. It can have the most innovative impact on biotechnology. AI has already established its presence in our day-to-day life. AI has made the existence of self-driving cars possible. Likewise, the benefits and quality that it can contribute to biotech can also be felt. With AI, bio technicians will be able to enhance virtual screening, overlook preliminary datasets from clinics, and decipher an enormous amount of information. It can also help in improving the medication process by gathering and analyzing every bit of information. The Significance of AI in Biotechnology In the past few years, the application of artificial intelligence in the biotechnology industry has shifted from being sci-fi to sci-fact. A vast number of biotech companies like Deep Genomics are adopting AI for making data-driven decisions and use analytics tools to work efficiently. Unlike the AI robots in sci-fi that are ready to take over the world. AI designed for biotech has been designed to solve certain problems or complete a bunch of tasks by using automated algorithms. The aim of AI technology for biotech is to collect insights along with hidden patterns from large amounts of data. All the different industries of biotech including agriculture, animal, medical, industrial, and bioinformatics are gradually being affected by artificial intelligence. Moreover, the biotech industry is realizing that AI enables them some of the important strength to their business, including: Expanding accessibility Cost-effectiveness Critical predictions Efficient decision-making Research centers like PwC have also estimated output of $15.7 trillion by 2030 solely with AI contribution in industries. A survey revealed that about 44% of life science experts are using AI for R&D activities, as well. Use of AI in Biotechnology Altering Biomedical and Clinical Data So far the most developed use of AI is its ability to read voluminous data records and interpret them. It can prove to be a life-save for bio technicians who would have to examine that much data from research publications by themselves for the validation of their hypothesis. With the help of AI, clinical studies of patients will also become easier as all the examination reports and prescriptions will be stored in one place for cross-reference. Furthermore, it will also help in blending and fetching data into usable formats for analysis. Test Result Prediction Through trial and error, AI along with machine learning can help in predicting the response of the patient to certain drugs to provide more effective outcomes. Drug Design & Discovery AI plays a vital role whether it’s designing a new molecule or identifying new biological targets. It helps in identifying and validating drugs. It reduces the cost and time spent on the entire drug trial process and reaches the market. Personalized Medications for Rare Diseases With the combination of body scan results, patients’ body and analytics, AI can also help in detecting dangerous diseases at an early stage. Improving Process of Manufacturing To improve the process of manufacturing in biotechnology, AI offers a wide range of opportunities. It controls quality, reduces wastage, improves useability, and minimizes the designing time. Moving Towards AI-Enhanced Biotech Future Ever since the concept of artificial intelligence has arrived, being curious by nature, humans have started working towards achieving this goal. It has been growing at a fast pace while showing unbelievable growth and achievements at times. In comparison to the traditional methods used in the biotechnology industry, AI-based methods seem more reliable and accurate. In the upcoming years, it will show its success by improving the quality of health people have. You can also develop your AI-based application or know more about it by taking IT consultations.

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Spotlight

Charm ATP Detection

Adenosine triphosphate (ATP) is the energy source of all living cells. It has gained popularity as a testing source to determine sanitation or hygiene levels since associated testing methods deliver significantly faster results than plating. Monitoring sanitation is essential in preventative programs in dairy, food & beverage, feed & grain, water, hospitals and many other industries.

Related News

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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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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