MEDICAL, DIAGNOSTICS

Premier Research and InSilicoTrials Partner for Rare Disease Therapy Development

Premier Research | March 14, 2023 | Read time : 02:00 min

Premier Research and InSilico

Premier Research has partnered with InSilicoTrials, a leader in using artificial intelligence (AI) and computational modeling and simulation (CM&S) to speed up the development of new therapies and medical devices. Premier Research helps biotech and device companies take their ideas from the idea stage to the commercialization stage. The aim of the partnership is to make it easier, faster, and safer for treatments for rare diseases to get approval from the government.

CM&S and AI have made it possible to use virtual patients in clinical trials in whole or in part, which the FDA approves of in some cases. In silico trials make it possible to create fake control or treatment arms, help figure out how to sign up patients, and predict the safety and effectiveness of new drugs and medical devices more accurately.

The use of synthetic control arms generated using in silico CM&S techniques represents a powerful tool for enabling non-feasible rare disease research, both preclinical and clinical research. In the last five years, Premier Research has been in charge of more than 240 rare disease studies for a wide range of indications. This shows that the company is committed to orphan drug development for a long time. Premier Research is continuing to put money into trials for rare diseases by partnering with InSilicoTrials.

One major advantage of in silico simulation is the ability to investigate 'what if' scenarios, particularly in rare diseases where a larger patient population does not exist. By using biological, molecular, or genetic data to describe rare diseases, in silico models can make synthetic control arms. This, along with Premier Research's expertise in clinical and preclinical development, execution, and regulatory aspects of small population trials, can shorten the time it takes to make effective treatments for rare diseases.

The partnership between Premier Research and InSilicoTrials will focus on smarter in silico study design, leading to a more effective pre-clinical review of trial design parameters and faster submission-ready studies. CEO of InSilicoTrials Luca Emili says that combining modeling and simulation with AI is the best way to improve the R&D process in drug development. This is because it allows sponsors to speed up the development of new treatments, make medical products safer, and lower R&D costs.

About Premier Research

Premier Research is a global company that does clinical research, product development, and consulting. It focuses on using new technologies to design and run smart studies and trials. With more than 2,500 employees in 75 countries, it has a lot of experience in certain therapeutic areas and uses. The company is committed to empowering its customers to meet the unmet needs of patients and offers great benefits and flexible working conditions. 

Spotlight

Implementation of a metabolite-based cell density<br /> estimation and evaluation of harvest strategies for<br /> Lentivirus production with HEK293T cells

Spotlight

Implementation of a metabolite-based cell density<br /> estimation and evaluation of harvest strategies for<br /> Lentivirus production with HEK293T cells

Related News

CELL AND GENE THERAPY, INDUSTRY OUTLOOK

Adaptive Biotechnologies Announces FDA Acceptance of Genentech’s Investigational New Drug Application for First Neoantigen-Directed T-Cell Therapy

Globenewswire | May 10, 2023

Adaptive Biotechnologies Corporation a commercial stage biotechnology company that aims to translate the genetics of the adaptive immune system into clinical products to detect and treat disease, announced that the U.S. Food and Drug Administration has accepted an investigational new drug (IND) application submitted by its collaborator, Genentech, a member of the Roche Group, for a T-cell receptor (TCR) based T-Cell Therapy. This is the first TCR-based therapeutic product candidate to advance into clinical development based on Adaptive’s collaboration with Genentech in oncology. “This IND acceptance reaffirms the value of our immune medicine platform and Adaptive’s ability to identify and characterize clinical grade, therapeutic T-cell receptors, which is the cornerstone of our drug discovery capabilities,” said Chad Robins, chief executive officer and co-founder, Adaptive Biotechnologies. “We look forward to supporting Genentech’s world-class team of scientists and drug developers to advance this potentially life-saving therapy into the clinic for patients with solid tumors.” Under the terms of Adaptive and Genentech’s collaboration agreement, Genentech has responsibility for clinical, regulatory and commercialization efforts for any T-Cell Therapy product candidate. Adaptive reiterates its full year revenue guidance in the range of $205 to $215 million. About Adaptive Biotechnologies Adaptive Biotechnologies is a commercial-stage biotechnology company focused on harnessing the inherent biology of the adaptive immune system to transform the diagnosis and treatment of disease. We believe the adaptive immune system is nature’s most finely tuned diagnostic and therapeutic for most diseases, but the inability to decode it has prevented the medical community from fully leveraging its capabilities. Our proprietary immune medicine platform reveals and translates the massive genetics of the adaptive immune system with scale, precision and speed. We apply our platform to partner with biopharmaceutical companies, inform drug development, and develop clinical diagnostics across our two business areas: Minimal Residual Disease (MRD) and Immune Medicine. Our commercial products and clinical pipeline enable the diagnosis, monitoring, and treatment of diseases such as cancer, autoimmune disorders, and infectious diseases. Our goal is to develop and commercialize immune-driven clinical products tailored to each individual patient.

Read More

CELL AND GENE THERAPY, INDUSTRIAL IMPACT

Fulgent Genetics Launches Mission Bio Platform to Broaden Accessibility of Single-Cell Multi-Omics for Drug Development and Clinical Research

Prnewswire | April 12, 2023

Mission Bio, Inc., the pioneer in high-throughput single-cell DNA and multi-omics analysis, announced that it has entered into an agreement with Fulgent Genetics a technology-based company with a well-established clinical diagnostic business and a therapeutic development business. Fulgent Genetics is now able to offer single-cell multi-omics on the Tapestri Platform on its fully customizable menu of services, including whole genome, whole exome, RNA sequencing, tumor profiling, methylation sequencing, liquid biopsy, single cell sequencing, spatial biology, and pathology services to support its growing pharma client demands. Additionally, Fulgent Genetics will evaluate the Tapestri Platform for applications in clinical development to streamline the drug development and approval process. Over the last ten to fifteen years, there has been a paradigm shift in clinical trials towards precision oncology by using biomarkers to select or enrich trial cohorts. The Tapestri Platform's ability to do single-cell analysis of genotype and phenotype from the same cell can provide the sensitivity and resolution for biomarker discovery unattainable by current bulk methods. Single-cell resolution can reveal treatment resistance mechanisms that may help to better stratify patients in clinical trials. While on treatment, residual levels of disease can be detected with the high sensitivity of single-cell analysis. Characterizing clonal heterogeneity and tracking clonal evolution during treatment can potentially guide adaptive and rational combination therapies for better patient stratification and better clinical trial outcomes. "The increasing adoption of the Tapestri Platform for translational research and clinical development by many cancer centers and global pharma companies demonstrates the necessity of single-cell multi-omics," said Dr. Hanlin (Harry) Gao M.D., Ph.D., D.A.B.M.G., F.A.C.M.G., Chief Scientific Officer of Fulgent Genetics. "The relationship between Fulgent Genetics and Mission Bio will make it easier and faster for biopharmaceutical customers to generate data for more precise clinical trials." "Fulgent Genetics' full scope, end-to-end service and comprehensive genomic portfolio is the perfect one-stop shop for pharma customers," said Todd Druley, M.D., Ph.D., Chief Medical Officer of Mission Bio. "While some pharma customers can and still do partner with us through our internal Pharma Assay Development (PAD) services, this agreement provides another option for them to leverage the single-cell multi-omics expertise and full suite of solutions offered by Fulgent Genetics, particularly as they complete the validation of the Tapestri Platform in their CAP CLIA lab in the near future." Customers today have access to the Tapestri Platform and its full breadth of single-cell multi-omics and scMRD assays through Fulgent Genetics' labs. About Mission Bio Mission Bio is a life sciences company that accelerates discoveries and cures for a wide range of diseases by equipping researchers with the tools they need to better measure and predict our resistance and response to new therapies. Mission Bio's multi-omics approach improves time-to-market for new therapeutics, including innovative cell and gene therapies that provide new pathways to health. Founded in 2014, Mission Bio has secured investment from Novo Growth, Cota Capital, Agilent Technologies, Mayfield Fund, and others. The company's Tapestri Platform gives researchers around the globe the power to interrogate every molecule in a cell together, providing a comprehensive understanding of activity from a single sample. Tapestri is the only commercialized multi-omics platform capable of analyzing DNA and protein simultaneously from the same sample at single-cell resolution.

Read More

INDUSTRY OUTLOOK

Magenta Therapeutics and Dianthus Therapeutics Announce Merger Agreement

Globenewswire | May 04, 2023

Magenta Therapeutics, Inc. and Dianthus Therapeutics, Inc. a privately-held, clinical-stage biotechnology company dedicated to advancing the next generation of antibody complement therapeutics, announced today that they have entered into a definitive merger agreement to combine the companies in an all-stock transaction. The combined company will focus on advancing Dianthus’ pipeline of next-generation complement inhibitors, including DNTH103 currently in a Phase 1 clinical trial. Upon completion of the merger, the combined company is expected to operate under the name Dianthus Therapeutics, Inc. and trade on the Nasdaq under the ticker symbol “DNTH”. In support of the merger, Dianthus has secured commitments for a $70 million private investment in its common stock and pre-funded warrants from a syndicate of healthcare investors led by Fidelity Management & Research Company, Catalio Capital Management, 5AM Ventures, Avidity Partners, Wedbush Healthcare Partners and founding investors Fairmount, Tellus BioVentures and Venrock Healthcare Capital Partners, that is expected to close immediately prior to completion of the merger. With the cash expected from both companies at closing and the proceeds of the concurrent private financing, the combined company is expected to have approximately $180 million of cash or cash equivalents immediately post-closing. The cash resources are intended to be used to advance Dianthus’ pipeline through multiple clinical data catalysts and is expected to fund operations into mid-2026. The merger and related financing are expected to close in the third quarter of 2023. “I’m delighted to announce this planned merger with Magenta, which comes at a pivotal moment in the evolution of our company as we advance our pipeline of next-generation complement therapeutics for people living with severe autoimmune diseases,” said Marino Garcia, President and Chief Executive Officer of Dianthus Therapeutics. “Gaining access to the public capital markets can enhance our financial strength and fuel our growth strategy, enabling us to unlock the full potential of our pipeline, including our plans to address multiple autoimmune disorders with our clinical-stage active C1s inhibitor, DNTH103.” “After a thorough exploration of our strategic alternatives, management and our Board of Directors believe the transaction with Dianthus Therapeutics will culminate in a successful outcome for our stockholders,” said Steve Mahoney, President, Chief Financial and Operating Officer of Magenta. “Dianthus has made rapid progress in developing and advancing DNTH103 into the clinic where it has the potential to be a transformative classical pathway inhibitor for severe autoimmune diseases. We are extremely grateful to our current and former employees who contributed to Magenta’s efforts to develop its programs and we now look forward to the combined company’s advancement on opportunities for value creation for patients.” Magenta previously announced a comprehensive review of strategic alternatives in February 2023 and has since completed winding down a majority of its activities and costs associated with its research and development initiatives, including the termination of its lease and the sale of key assets. erving as co-financial adviser and Goodwin Procter LLP is serving as legal counsel to Magenta Therapeutics. Jefferies, Evercore ISI, Guggenheim Securities and Raymond James are serving as the placement agents to Dianthus Therapeutics, and Gibson, Dunn & Crutcher LLP is serving as legal counsel to Dianthus Therapeutics. About Magenta Therapeutics Magenta Therapeutics is a clinical-stage biotechnology company developing medicines to bring the curative power of stem cell transplant to more patients with blood cancers, genetic diseases and autoimmune diseases. Magenta is combining leadership in stem cell biology and biotherapeutics development with clinical and regulatory expertise, a unique business model and broad networks in the stem cell transplant community to revolutionize immune reset for more patients.

Read More