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The Drug Controller General of India (DCGI) has added in-vitro diagnostic (IVD) medical devices including those for diagnosis of Covid-19, ribonucleic acid (RNA) and deoxyribonucleic acid (DNA) extraction kits, among others into the Class C risk category under the Medical Devices Rules (MDR), 2017.
The deal will see Polyplus join the German life science group’s portfolio allowing the latter to leverage expertise in transfection reagents and plasmid DNA for gene therapy. to complete an in-house plasmid DNA facility in Marburg, Germany. “In In February 2023, the mRNA specialist BioNTech invested $43.8m
Pharma companies are always talking about moving ‘beyond the pill’, and Pfizer’s new brand identity embodies that – it’s decades old pill-like logo has been replaced with a DNA double helix that it says reflects its commitment to breakthrough science. Encoded within a successful emblem is a company’s DNA – its history, its future.
It is a comprehensive term which encompasses a large variety of therapy products including viral and bacterial vectors, plasmid DNA, human gene editing technology, and patient-specific cellular gene therapy. For example, it is beneficial to administer RNA treatments for some lung diseases directly to the disease site through inhalation.
Only a few weeks into the new year, the prospect of getting a successful advanced HIV vaccine shrank after the discontinuation of yet another late-stage trial. This marks the second time one of Janssen’s HIV vaccines failed after another showed disappointing results in the Phase IIb Imbokodo trial in August 2021.
Whether it’s for a treatment for a chronic ambulatory condition, precision medicine or cell and gene therapy, there is a massive uptick in clinical trial complexity. It’s important to make sure that with this increase in clinical trial complexity, we don’t make our trials overly burdensome to sites or patients,” says Markham.
Verve Therapeutics has started dosing patients in a phase 1b trial of its in vivo gene-editing drug for high cholesterol, designed to permanently switch off the PCSK9 gene with a one-shot treatment. Through base editing, drugs replace single nucleotide in the DNA strand with another, without making double-strand breaks (DSBs) in the gene.
Startup Rome Therapeutics has raised $77 million in second-round financing to help mine sequences of DNA – which were dismissed for years as ‘junk’ – for hidden treasure. Much of this repeatome is thought to derive from viruses that embedded in our DNA millions of years ago.
In a clinical trial, participants demonstrated a 98% success rate after six months post implantation, with arteries widened successfully and no stent fractures observed. The Shield test provides a non-invasive approach to colorectal cancer screening by analyzing blood for key DNA changes linked to cancer. Photo courtesy of BD.
Viral vectors have traditionally been used in gene editing where the shells of viruses carry the template DNA into cells, but this method has its disadvantages. The CRISPR-Cas9 system works by having “spacer” sequences transcribed into short RNA sequences that can guide the system to the matching target in the DNA sequence of a cell.
Liquid biopsy tests in oncology involve isolating entities such as circulating tumor cells (CTC), circulating tumor DNA (ctDNA) and tumor-derived exosomes. Gleich, MD, FACS, Senior Vice President, Medical Department, and Dr. Christopher Huth, PhD, Senior Clinical Trial Manager, Clinical Trial Management.
The RNA Revolution: From mRNA Vaccines to RNA Editing. The age of RNA is officially here, and it’s here to stay as more than a passing life science trend. RNA technology is not new nor has its potential been surprising. RNA in the Making. So why did this perceived RNA ‘revolution’ take so long?
The pandemic has had a lasting impact on the ability of sites to participate in trials, particularly in the US. We need to consider how to use diverse populations in gene therapy trials. But it’s important we strive to find ways to include diverse patients so trial data can be representative of the wider population.
Measuring RNA, DNA quickly. Clinical Trials – Identifying the right candidate for the trial based on history and disease conditions, and additional attributes, overlaying with infection rates, demographics, and ethnicity to represent the most impacted. .
The small-molecule PIKfyve inhibitor – called VRG50635 – has been administered to the first subject in the phase 1 trial involving healthy volunteers, according to the San Francisco-based biotech, which was founded in 2015 by Alice Zhang and Jason Chen.
The company develops mRNA therapeutics and vaccines , and the move will enable it to utilise the synthetic biology and enzyme tech of 2018-founded Japanese company OriCiro, which develops cell-free DNA synthesis and amplification technologies, and thereby expand Moderna’s portfolio.
Molecular Biomarkers : Encompass various molecules, such as RNA and metabolites, to reflect the physiological state of the cells and their disease pathways. The analysis of circulating tumor DNA (ctDNA) in a liquid biopsy can also permit genotyping and help monitor the effectiveness of chemotherapy.
Alnylam Pharmaceuticals, a leading RNA interference (RNAi) therapeutics biopharmaceutical company, announced it received approval from the US Food and Drug Administration (FDA) for its RNAi therapeutic Amvuttra (vutrisiran) for the treatment of the polyneuropathy of hereditary transthyretin-mediated (ATTR) amyloidosis in adults.
Over 26 weeks of Sunlenca combined with other antiretroviral drugs, 81 percent of participants achieved HIV RNA suppression, reaching levels low enough to be considered undetectable. Both trials concluded with maintained virologic suppression, and no clinically significant change from baseline in CD4+ cell counts was observed.
Messenger RNA vaccines contain nucleic acids that code for a specific protein, or target antigen, related to a virus or disease. Platforms designed to create RNA can also be easily adjusted to target a range of indications or mutated versions of the same virus. million in funding to support this work.
The alliance has also yielded a computer vision system to improve the accuracy and speed of immune response evaluations, and a platform that has improved the success rate and throughput of DNA/RNA synthesis. The post BioNTech buys UK-based AI startup InstaDeep in £562m deal appeared first on.
Cancerous cells and tumors release small fragments of DNA into the bloodstream called circulating tumour DNA (ctDNA). The researchers at the Cancer Research UK Cambridge Institute published this data from the LUng cancer CIrculating tumour DNA (LUCID) study in the journal Annals of Oncology.
While achieving the Nobel Prize spotlight would have been enough to impress, CRISPR-Cas9 gene editing is part of a growing list of technologies granted Investigational New Drug (IND) applications with early data from clinical trials supporting its safe use in edited cells re-introduced into a patient.
Mount Sinai researchers have developed a new model that uses DNA and RNA sequencing data from hundreds of patients to identify specific genes and genetic alterations responsible for never-before-defined subtypes of a blood cancer called multiple myeloma.
Related: Top 30 Pharma Companies in 2023: Statistics and Trends 1) Moderna Compound annual growth rate: 415 percent Moderna, headquartered in Massachusetts, is a prominent biotechnology company specializing in RNA therapeutics, particularly mRNA vaccines. In the third quarter of 2023, the company’s revenue surged to $63.7
Vaccibody, which is also entitled to tiered royalties on sales of any marketed products, will develop the DNA-based individualised neoantigen vaccines until the end of phase 1b. The Roche unit will also bear all costs for trials, filings, manufacturing and marketing after the completion of the phase 1b study.
An antisense drug developed by GlaxoSmithKline has been shown to suppress the hepatitis B virus (HBV) in a phase 2 trial, raising hopes of a “functional cure” for the widespread disease. Meanwhile, five of 12 previously-untreated patients had 2-log (90%) or a greater reduction in HBV DNA. billion in potential milestones.
The payload, Del Bourgo tell us, is the therapeutic DNA or RNA sequence that will cure or fix damaged cells. Whitelab Genomics operates a proprietary AI platform to accelerate the development of DNA and RNA-based therapies, also known as genomic therapies. “This is just the beginning; the potential is enormous,” he says.
13, 2021 — A single-shot COVID-19 vaccine from Johnson & Johnson has shown very strong results in early clinical trials, potentially providing a significant boost to U.S. The vaccine produced an immune response of all 805 clinical trial participants within two months of inoculation, according to results published Jan.
With over 300 active clinical trials, Winship researchers are dedicated to taking new cancer treatments from bench to bedside. cancer centers selected as a Lead Academic Participating Site for the NCI’s National Clinical Trials Network (NCTN). Winship is one of 32 U.S. MBA, FACP, Chairman of the Caris Precision Oncology Alliance.
There currently are drugs that do just that but now they’re in clinical trials for metastatic breast cancer as well. There are antibody drugs targetting those cells that are already FDA approved and there are antibody drugs still in trials. An enzyme involved in DNA repair can be the key to making chemotherapy more effective.
Their technology is based on synthetic messenger RNA – short for ribose nucleic acid – which is a short transcript of a longer DNA code. Both Moderna and Pfizer’s shots aced the quickly convened clinical trials and became the first vaccines to make it to market less than a year after the pandemic began.
The Flu versus COVID-19: Virology and Vaccines SARS-CoV-2 versus Influenza Virology Viruses contain genetic material that can either be RNA or DNA. Both influenza and SARS-CoV-2 have RNA as their genetic material. An RNA genome enables the genetic material to mutate more rapidly.
The two-day conference will bring you high-quality insights and industry connections on the latest clinical trial candidates and a platform for exchanging ideas for tackling the biggest challenge: DELIVERY. Understand the regulatory and CMC environment surrounding oligonucleotide therapeutic development.
Even with the holidays among us, there were a number of clinical trial announcements. Vir Biotechnology and GlaxoSmithKline dosed the first patient in a new sub-trial of a Phase III study of monoclonal antibody VIR-7831 for hospitalized adults with COVID-19. Here’s a look. COVID-19-Related. Non-COVID-19-Related.
Gene switches can be regulatory proteins or specific DNA sequences that act to either switch on or off the expression of a gene. Basic Components of Gene Switch Gene switches are composed of noncoding DNA sequences and transcription factors.
The new study was conducted by doing DNA methylation profiling and RNA sequencing on 565 meningiomas. This enabled investigators to see what genes are expressed by the tumor and the level of expression, revealing a signature of the DNA. Choudhury A, Magill ST, Eaton CD et al. Nat Genet (2022). doi: 10.1038/s41588-022-01061-8.
The companies schedule to initiate a Phase 2 trial assessing combination therapy for both treatment-experienced and treatment-naïve people living with HBV. People in the trial with HBV treatment experience may also receive Gilead’s Vemlidy (tenofovir alafenamide fumarate, TAF). Gilead Sciences, Inc. and Vir Biotechnology, Inc.
Several LNP-based therapies are currently being evaluated in the clinical trials; majority of these are conducted for treating breast cancer, ovarian cancer and lung cancer. Medical Diagnostics: LNPs enable targeted imaging of biological tissues by using various imaging agents, such as contrast agents, fluorescent dyes and radionuclides.
In the ATLAS study, CABENUVA met the primary endpoint for noninferiority (the proportion of participants with plasma HIV-1 RNA ?50 50 copies per milliliter [c/mL] at Week 48), with a comparable number of patients receiving either CABENUVA or their daily current antiretroviral regimen (CAR) having an HIV-1 RNA level ?50
The company’s other two Phase 2 trials, Study 201 and 202, showed that the addition of VBR to NrtI therapy achieved a quicker, deeper level of viral suppression than with NrtI alone. It has been shown to be well-tolerated and statistically superior in antiviral activity in hepatitis B virus DNA suppression, compared to NrtI therapy alone. .
The use of engineered genetic materials in clinical trials is rapidly expanding, with potential applications for genetic vaccines, gene-modified cellular therapies, and gene therapies. Gene Delivery Systems Genetic material, in the form of DNA or RNA, does not easily enter cells without the aid of a delivery system.
Unveiling The Potential of mRNA Technology mRNA is a single-stranded molecule that relays the genetic instructions needed to make proteins from DNA in the cell nucleus to ribosomes. The COVID-19 mRNA vaccines from Pfizer / BioNTech and Moderna have entered supranational clinical trials with millions of doses delivered.
Nirmatrelvir is designed to inhibit viral replication at a stage known as proteolysis, which occurs before viral RNA replication. In preclinical studies, nirmatrelvir did not demonstrate evidence of mutagenic DNA interactions. No dosage adjustment is needed in patients with mild renal impairment.
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