Computational Tool for Single-Cell Transcriptomics Reveals Gene Network Plasticity

genengnews | June 10, 2019

A team of investigators at the National Centre for Genomic Analysis (CNAG-CRG) in Barcelona has developed a new computational tool, based on the mathematical Graph theory, to infer global, large-scale regulatory networks, from healthy and pathological organs, such as those affected by diabetes or Alzheimer’s disease. Findings from the new study—published recently in Genome Biology through an article titled “Single-cell transcriptomics unveils gene regulatory network plasticity”—were able to pinpoint genes relevant to organ function and potential drivers of diseases. Although the knowledge we have about human cells and tissues has steadily increased over recent decades, many things remain unknown. For instance, cells exist in transient, dynamic states and understanding them is fundamental to decipher diseases and find cures. Classic techniques used in the lab to study cell types faced limitations and did not enable a finely detailed profile of cell function. To overcome this obstacle, the CNAG-CRG scientists developed this new tool based on their earlier work. “Our previously developed single-cell transcriptomic tools were very useful to discover unknown cell types,” explained lead study investigator Giovanni Iacono, PhD, senior postdoc researcher at the CNAG-CRG. “Those tools allowed us to describe new types and subtypes of cells, with their unique biological roles and hierarchical relationships.”

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Spotlight

ASSOCHAM strongly believes that it is important to strike the balance in population's ability to pay with their desire for access to modern care and technology. This requires innovation at all levels i.e. Pharmaceutical through product development business models, systems or services, Medical Devices through material/cost/selection based innovations, Diagnostics through improving the link between clinical needs and development of new tests, Machinery through adherence to global standards and packaging for avoiding counterfeiting and Financing through increasing public health expenditure and awareness on health insurance for minimizing out of pocket expenses.

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GenScript Receives FDA Emergency Use Authorization for cPass™ SARS-CoV-2 Neutralization Antibody Detection Kit

GenScript | November 10, 2020

GenScript USA Inc., the world's leading research reagent provider, announced today that the U.S. Food and Drug Administration (FDA) has granted Emergency Use Authorization (EUA) for the cPass™ SARS-CoV-2 Neutralization Antibody Detection Kit. This is the first commercially available test to specifically detect neutralizing antibodies without the use of live virus. Neutralizing antibodies, a subset of antibodies that are specifically able to block the ability of the virus to enter a cell, are widely recognized biomarkers of immunity. The test measures the presence of neutralizing antibodies in any sample, from those from patients recovering from COVID-19 or those vaccinated against SARS-CoV-2. The conventional method for measuring neutralizing antibodies requires testing to be performed in a high safety-level facility (BSL3) staffed with specialized personnel. Typically, results from the live virus assay are available in several days and due to the unpredictable nature of the live virus and live cells reagents used, these tests are also difficult to standardize. Results from different facilities may vary, even if performed using the same protocol. In contrast, the cPass™ kit utilizes pure proteins that can be produced in a more reproducible way. The detection of the presence of neutralizing antibodies can also be performed in most standard research or clinical diagnostic laboratories with short turnaround time (~1hr), making it broadly available and much more consistent between different facilities. Furthermore, a comparison of the direct clinical performance between the conventional live virus and the cPass™ assay shows results from both tests are strongly correlated.

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INDUSTRIAL IMPACT

Legend Biotech Announces Extension of PDUFA Date for Cilta-Cel

Cilta-cel | November 01, 2021

Legend Biotech Corporation (Legend Biotech), a global, clinical-stage biotechnology company developing and manufacturing novel therapies, today announced that the U.S. Food and Drug Administration has extended the Prescription Drug User Fee Act (PDUFA) target date for ciltacabtagene autoleucel (cilta-cel) to February 28, 2022. Cilta-cel is a BCMA-directed chimeric antigen receptor T cell (CAR-T) therapy being investigated for the treatment of adults with relapsed and/or refractory multiple myeloma. The Biologics License Application (BLA) was submitted by Legend Biotech’s collaboration partner Janssen Biotech, Inc. “We are working closely with Janssen and the FDA to facilitate an efficient and thorough review of the BLA for cilta-cel,Financial Officer at Legend Biotech.We remain confident that cilta-cel has shown great promise in patients with relapsed and refractory multiple myeloma, and we are focused on making this therapy available to them in the US as soon as possible.” Ying Huang, Ph.D., Chief Executive Officer and Chief The FDA notified Janssen on October 28, 2021 of the extension of the PDUFA date to allow sufficient time to review information recently submitted pertaining to an updated analytical method following an FDA information request. Legend and Janssen met with the FDA on November 1. No additional clinical data have been requested. About Cilta-cel Cilta-cel is an investigational chimeric antigen receptor T cell (CAR-T) therapy, formerly identified as JNJ-4528 in the U.S. and Europe and LCAR-B38M CAR-T cells in China, that is being studied in a comprehensive clinical development program for the treatment of patients with relapsed or refractory multiple myeloma and in earlier lines of treatment. The design consists of a structurally differentiated CAR-T with two BCMA-targeting single domain antibodies. In December 2017, Legend Biotech, Inc. entered into an exclusive worldwide license and collaboration agreement with Janssen Biotech, Inc. (Janssen) to develop and commercialize cilta-cel. In December 2020, Legend announced initiation of rolling submission of Biologics License Application to the FDA seeking approval of cilta-cel for the treatment of relapsed and/or refractory multiple Myeloma, which was accepted under Priority Review in May 2021. Cilta-cel was previously granted Breakthrough Therapy Designation (BTD) granted in the U.S. in December 2019, and Orphan Drug Designation in February 2019. About Multiple Myeloma Multiple myeloma is an incurable blood cancer that starts in the bone marrow and is characterized by an excessive proliferation of plasma cells.1 Although treatment may result in remission, unfortunately, patients will most likely relapse.2 Relapsed myeloma is when the disease has returned after a period of initial, partial or complete remission and does not meet the definition of being refractory.3 Refractory multiple myeloma is when a patient’s disease is non-responsive or progresses within 60 days of their last therapy.4,5 While some patients with multiple myeloma have no symptoms at all, most patients are diagnosed due to symptoms that can include bone problems, low blood counts, calcium elevation, kidney problems or infections.6 Patients who relapse after treatment with standard therapies, including protease inhibitors and immunomodulatory agents, have poor prognoses and few treatment options available.7 About Legend Biotech Legend Biotech is a global, clinical-stage biotechnology company dedicated to treating, and one day curing, life-threatening diseases. Headquartered in Somerset, New Jersey, we are developing advanced cell therapies across a diverse array of technology platforms, including autologous and allogenic chimeric antigen receptor T-cell, T-cell receptor (TCR-T), and natural killer (NK) cell-based immunotherapy. From our three R&D sites around the world, we apply these innovative technologies to pursue the discovery of safe, efficacious and cutting-edge therapeutics for patients worldwide. We are currently engaged in a strategic collaboration to develop and commercialize our lead product candidate, ciltacabtagene autoleucel, an investigational BCMA-targeted CAR-T cell therapy for patients living with multiple myeloma. Applications seeking approval of cilta-cel for the treatment of patients with RRMM are currently under regulatory review by several health authorities around the world, including the U.S. Food and Drug Administration and the European Medicines Agency. Cautionary Note Regarding Forward-Looking Statements Statements in this press release about future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, constitute “forward-looking statements” within the meaning of The Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements relating to the timing and outcome of regulatory reviews relating to cilta-cel, including the BLA being reviewed by the FDA. The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “will,” “would” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors. Legend Biotech’s expectations could be affected by, among other things, uncertainties involved in the development of new pharmaceutical products; unexpected clinical trial results, including as a result of additional analysis of existing clinical data or unexpected new clinical data; unexpected regulatory actions or delays, including requests for additional safety and/or efficacy data or analysis of data, or government regulation generally; as well as the other factors discussed in the “Risk Factors” section of Legend Biotech’s Annual Report on Form 20-F filed with the Securities and Exchange Commission on April 2, 2021. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those described in this presentation as anticipated, believed, estimated or expected. Legend Biotech specifically disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events or otherwise.

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MEDICAL

Moderna Announces Supply Agreement with UK Government for Additional 2 Million Doses of mRNA-1273, Moderna’s Vaccine Candidate Against Covid-19

Moderna | November 30, 2020

Moderna, Inc., a biotechnology organization pioneering messenger RNA (mRNA) therapeutics and vaccines to make new generation of transformative medicines for patients, today reported a supply agreement with the UK government for an extra 2 million doses of mRNA-1273, Moderna's vaccine candidate against COVID-19, to the United Kingdom starting in March 2021. The UK government has now made sure about 7 million dosages of mRNA-1273. This confirmation comes as the UK proceeds with its efforts to secure access to safe and effective COVID-19 vaccines by establishing a broad portfolio of the most promising vaccines. On November 16, Moderna reported that the autonomous, NIH-appointed Data Safety Monitoring Board (DSMB) for the Phase 3 investigation of mRNA-1273, its vaccine candidate against COVID-19, has informed Moderna that the trial has met the statistical criteria pre-specified in the study protocol for efficacy, with a vaccine efficacy of 94.5%. This study, known as the COVE study, enrolled more than 30,000 participants in the U.S. and is being conducted in collaboration with the National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health (NIH), and the Biomedical Advanced Research and Development Authority (BARDA), part of the Office of the Assistant Secretary for Preparedness and Response at the U.S. Department of Health and Human Services.

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