Do schistosomes practice the art of host-manipulation?

Do schistosomes manipulate snails? A recent study looks at the altered phototropic behaviour of infected snails and how this may be a deliberate strategy from the schistosomes themselves. Parasites manipulating host behaviour is not a new story. There are lots of examples of parasites and parasitoids making their poor hosts do the most extraordinary things: grasshoppers purposefully jumping into water and drowning themselves, rats running towards cat urine smells, cockroaches walking into predator wasp lairs etc. This level of skilled “mind-control” would leave even our most talented illusionists agog with admiration and horror.

Spotlight

BioAtla LLC

BioAtla is a protein therapeutic company that uses discovery and evolution to develop safer and more potent drugs for the treatment of cancer.

OTHER ARTICLES
MedTech

Next-Gen Gene Therapy to Counter Complex Diseases

Article | July 13, 2022

Gene therapy has historically been used to treat disorders with in-depth knowledge caused by a single genetic mutation. Thanks to the introduction of new generation technologies, the potential of gene therapy is expanding tAo treat diseases that were previously untreatable. Evolution of Gene Therapy One of the major success stories of the twenty-first century has been gene therapy. However, it has not been the same in the past. The field's journey to this point has been long and mostly difficult, with both tragedy and triumph along the way. Initially, genetic disorders were thought to be untreatable and permanently carved into the genomes of individuals unfortunate enough to be born with them. But due to the constant technological advancement and research activities, gene therapy now has the potential to treat various genetic mutation-causing diseases with its ability to insert a new copy and replace faulty genes. Gene Therapy is Finding New Roads in the Medical Sector Gene therapy can help researchers treat a variety of conditions that fall under the general heading of epilepsy, instead of only focusing on a particular kind of disorder brought on by a genetic mutation. Following are some of the domains transformed by gene therapy. Neurology – Gene therapy can be used for the treatment of seizures by directly injecting it into the area causing an uncontrolled electrical disturbance in the brain. Furthermore, by using DNA sequences known as promoters, gene therapy can be restricted to specific neurons within that area. Ophthalmology – Genetic conditions such as blindness can be caused due to the mutation of any gene out of over 200 and resulting in progressive vision loss in children. With advanced gene therapies such as optogenetics, lost photoreceptor function can be transferred to the retinal cells, which are responsible for relaying visual information to the brain. This might give patients the ability to navigate in an unknown environment with a certain level of autonomy. The Future of Gene Therapy The news surrounding gene therapy has been largely favorable over the past few years, with treatment after treatment obtaining regulatory approvals, successful clinical trials, and garnering significant funds to begin development. With more than 1,000 clinical trials presently underway, the long-awaited gene therapy revolution might finally be here.

Read More
Research

5 Biotech Stocks Winning the Coronavirus Race

Article | July 11, 2022

There are quite a few companies that have found ways to grow their business during the ongoing COVID-19 pandemic. This is especially true for a number of biotechs now working on developing a potential treatment for, or vaccine against, the virus; shares of such companies have largely surged over the past couple of months. Although many of these treatments and vaccines are still have quite a way to go before they're widely available, it's still worth taking some time to look through what's going on in the COVID-19 space right now. Here are five biotech stocks that are leading the way when it comes to addressing COVID-19. Regeneron Pharmaceuticals (NASDAQ:REGN) wasn't among the initial wave of companies to announce a potential COVID-19 drug. However, investor excitement quickly sent shares surging when the company announced that its rheumatoid arthritis drug, Kevzara, could help treat COVID-19 patients.

Read More
MedTech

Better Purification and Recovery in Bioprocessing

Article | July 12, 2022

In the downstream portion of any bioprocess, one must pick through the dross before one can seize the gold the biotherapeutic that the bioprocess was always meant to generate. Unfortunately, the dross is both voluminous and various. And the biotherapeutic gold, unlike real gold, is corruptible. That is, it can suffer structural damage and activity loss. When discarding the dross and collecting the gold, bioprocessors must be efficient and gentle. They must, to the extent possible, eliminate contaminants and organic debris while ensuring that biotherapeutics avoid aggregation-inducing stresses and retain their integrity during purification and recovery. Anything less compromises purity and reduces yield. To purify and recover biotherapeutics efficiently and gently, bioprocessors must avail themselves of the most appropriate tools and techniques. Here, we talk with several experts about which tools and techniques can help bioprocessors overcome persistent challenges. Some of these experts also touch on new approaches that can help bioprocessors address emerging challenges.

Read More
MedTech

How to Choose a Reliable Biotech Clinical Trial Management System?

Article | October 7, 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.

Read More

Spotlight

BioAtla LLC

BioAtla is a protein therapeutic company that uses discovery and evolution to develop safer and more potent drugs for the treatment of cancer.

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.

Read More

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.

Read More

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.

Read More

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.

Read More

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.

Read More

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.

Read More

Events