XTalks

JUNE 18, 2024

These platforms — Bobcat mRNATM, controllable self-replicating RNA (c-srRNA) and ZSCAN4 delivered by an RNA virus — represent the forefront of genetic therapy, harnessing the power of mRNA to combat diseases at their genetic roots.

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Protein RNA Genetics Clinical Trials 52

XTalks

AUGUST 5, 2020

In recent webinars by Genuity Science, formerly known as WuXi NextCODE, experts from the biotech and pharma industries spoke about leveraging the power of single cell RNA sequencing platforms and solutions in conjunction with machine learning technologies such as AI in cell biology and disease research. The Power of Single Cell Technology.

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Gene Expression Development RNA Drugs 112

Drug Discovery World

JANUARY 23, 2023

Following the release of Clarivate’s report ‘RNA Technology Companies to Watch’, DDW’s Diana Spencer speaks to Strand Therapeutics’ Jacob Becraft and HAYA Therapeutics’ Samir Ounzain to find out how this technology could revolutionise disease treatment.

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RNA Genome Genomics Engineer 98

XTalks

NOVEMBER 30, 2023

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. The noteworthy candidate, EBT-101, is designed to eliminate HIV proviral DNA using CRISPR-Cas9 along with two guide RNAs (gRNAs).

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FDA Approval Gene Therapy Clinical Trials Clinical Trials 111

The Pharma Data

MARCH 23, 2022

Polymerase is a viral protein that directs how Ebola virus replicates its genome as it infects new hosts. Back in 2018, researchers tested a broad-spectrum antiviral candidate called remdesivir/VEKLURY, which acts as a nucleotide decoy to get incorporated into the viral RNA genome and stop viral polymerase.

Protein 52

Protein Genome Genomics Research 52

XTalks

MARCH 8, 2022

With exciting new applications being developed in the areas of gene therapy, CRISPR and RNA editing, the contributions of women to these fields was highlighted in a piece we published last year on women scientists who paved the way forward in genetics.

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Life Science Gene Therapy DNA Vaccination 105

Roots Analysis

OCTOBER 30, 2023

In the past few years, Next Generation RNA therapeutics have emerged as one of the key therapeutic modalities in the modern healthcare industry. These RNA based therapeutics play a crucial role in protein production and regulation of gene functions.

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RNA Vaccination Vaccine Development 40

Delveinsight

FEBRUARY 25, 2021

Beam Therapeutics has taken over a startup whose technology could proffer the biotech’s genetic medicines to more tissues in the body, widening the potential to approach more diseases. Cambridge, Massachusetts-based Beam aims to make more precise edits with genetic medicines, which employ base-editing.

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DNA In-Vivo Genetics Medicine 52

Drug Discovery World

SEPTEMBER 14, 2023

Moreover, in July 2023, AstraZeneca and Vaxess Technologies commenced a collaboration for the evaluation of a novel RNA-based pandemic influenza prototype vaccine in patch format. In July 2023, AstraZeneca and Ionis Pharmaceuticals expanded their existing collaboration on eplontersen to also include Latin America.

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Drugs Vaccination Vaccine Medicine 59

Drug Discovery World

OCTOBER 16, 2023

A company within this ecosystem is STORM Therapeutics, a clinical stage biotechnology company creating novel small molecule therapies that inhibit RNA modifying enzymes (RME) for use in oncology and other diseases. We also recently opened the Scientific Centre of Excellence for Genomic Insight at our Cambridge site.”

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Drugs Life Science Gene Therapy Genome 75

The Pharma Data

JANUARY 17, 2021

n-Lorem selects patients based on their genetic mutation and the organ affected rather than their specific diseases. “We That’s not the case with RNA-targeted drugs. They’re basically the same,” but each one uses a particular nucleotide to bind to specific genetic sequence. We’re the middleman,” he said.

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Genetics Clinical Trials Clinical Trials Drugs 52

The Pharma Data

OCTOBER 19, 2020

By leveraging the comprehensive genomic, transcriptomic and proteomic profiling available through the Caris Molecular Intelligence® platform, physicians from Winship will be able to further prioritize therapeutic options and determine which clinical trial opportunities may benefit their patients. Brille , Vice Chairman of Caris Life Sciences.

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Life Science Clinical Trials Clinical Trials RNA 52

Drug Discovery World

AUGUST 1, 2023

1 August was first chosen as World RNA Day in 2018 as a play on AUG (adenine, uracil and guanine), a triple sequence of RNA (called a codon) that initiates protein synthesis by the cell. Though some genetic mutations are harmful, others can be protective.

RNA 52

RNA Drugs Gene Silencing Vaccination 52

Drug Discovery World

JUNE 27, 2024

SeekRNA uses a programmable ribonucleic acid (RNA) strand that can directly identify sites for insertion in genetic sequences, simplifying the editing process and reducing errors. Using the accuracy from this insertion sequence family, seekRNA can be modified to any genomic sequence and insert the new DNA in a precise orientation.

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Gene Editing Gene DNA Genetic Engineering 59

Drug Discovery World

JULY 11, 2023

Now, with the growth of publicly available genomics, transcriptomics, and proteomics databases, the ability to quickly carry out large-scale DNA, RNA, and protein screenings, and the availability of massive sets of de-identified patient data, the amount of high-value, analysable data has reached enormous proportions.

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Big Data Development Life Science Drugs 40

XTalks

DECEMBER 13, 2023

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. Alpine Immune Sciences has recently announced an augmentation in its R&D investment.

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Genetics Vaccination Vaccine DNA 111

Drug Discovery World

OCTOBER 4, 2022

As it stands, there are currently 19 gene therapies approved for clinical use, alongside 18 RNA therapies and 59 non-genetically modified cell therapies, according to the Q2 report by the American Society of Cell & Gene Therapy. In the UK, six universities received £4.25

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Gene Therapy Drugs Gene Manufacturing 52

pharmaphorum

FEBRUARY 3, 2021

These treatments are examples of patient-specific, engineered chimeric antigen receptor (CAR)-T cell therapies that harness the immune system to target and eliminate cancer cells. Thus, the availability of universal, “off-the-shelf” engineered T cells presents an attractive alternative.

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Gene Editing Gene DNA Engineer 96

Roots Analysis

MARCH 16, 2023

It is important in the process of protein synthesis because mRNA is responsible for transferring genetic information from DNA to ribosomes, which then decodes the genetic information into a protein. Messenger ribonucleic acid (mRNA) is a single-stranded molecule that is complementary to a gene’s DNA.

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Vaccination Vaccine DNA Protein 52

Drug Discovery World

SEPTEMBER 6, 2022

The mysteries of cancer are starting to yield to the range of advanced therapies available and cell therapies – especially engineered cell therapies – are at the leading edge of that progress.” – Carlo Russo, CEO of Genenta.? . “In Research into genomic medicines is only at its infancy. New indications .

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Gene Therapy Gene Manufacturing Medicine 52

Drug Discovery World

DECEMBER 15, 2022

Gene editing tools such as zinc finger nucleases, transcription activator-like effector nucleases (TALEN) and clustered regularly interspaced short palindromic repeat (CRISPR) nucleases have been heralded for their enormous potential to treat diseases and genetic disorders. In one example, Choi et al.

Gene Editing 52

Gene Editing Gene In-Vivo Genome 52

XTalks

AUGUST 29, 2022

CRISPR is notable for engineering living cells, allowing scientists to edit, turn off, delete, or replace genes in a cell’s genome. It is difficult to control where the viral vectors insert genes in the genome, and it is difficult to manufacture large quantities of clinical-grade viral vectors.

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Gene Editing DNA Gene Genome 98

Drug Discovery World

JULY 26, 2023

With 100-fold the number of genes in the human genome 1 , this microbial collection is a rich genetic signature of clinical significance that we have only recently gained the tools to explore. Genome editing tools such as CRISPR may be employed to create “live biotherapeutics” (drugs composed of living bacteria).

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Medicine Genome Genomics Bioinformatics 52

The Pharma Data

JANUARY 19, 2021

In the paper, researchers from Johns Hopkins University and elsewhere found a natural long-form transactivating CRISPR RNA (tracr-L) in Streptococcus pyogenes that functions to downregulate its endogenous CRISPR-Cas9 system. But altering the tracr-L with genetic engineering to make it function more like a guide RNA increased CRISPR-Cas9 cuts.

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Bacteria Gene Editing Research RNA 52

Drug Discovery World

AUGUST 10, 2022

Finally, in 2017, after years of improving T cell engineering techniques, the first chimeric antigen receptor (CAR) T cell therapy received FDA approval. Cells from solid tumours are genetically heterogeneous both between and within tumours, making it challenging to identify a single, selective antigen target for treatment.

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Manufacturing Gene Genome Genomics 98

XTalks

MAY 4, 2021

Researchers at the University of California San Francisco (UCSF) and the Whitehead Institute have developed a novel CRISPR-based tool called “CRISPRoff” that can switch off genes in human cells through epigenetic editing without altering the genetic sequence itself. Epigenetic Editing with CRISPR.

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DNA Gene Gene Silencing Genome 98

Drug Discovery World

JANUARY 23, 2023

Increasing interest in CRISPR-Cas gene editing systems over recent years has delivered innovative developments in precision genome engineering technologies, holding enormous potential for applications across the healthcare sector. 2016/17 saw the emergence of this novel CRISPR-Cas9-based approach.

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Gene Editing Gene Engineer DNA 52

Drug Discovery World

NOVEMBER 15, 2022

The brainchild of multidisciplinary experts in computer science, physics, engineering, and biology, the field of synthetic biology has not only rapidly progressed in the last few decades but it’s also becoming more accessible and cost-effective. Synthetic libraries for antibody drug discovery .

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DNA Drugs Antibody Engineer 98

Drug Discovery World

FEBRUARY 6, 2023

A case in point is the emerging omics and other high-throughput laboratory technologies that allow simultaneous profiling of genetic alterations in combination with profiling thousands of molecules in plasma, cell populations, or single cells. Figure 2: Advanced data visualisation examples for transcriptomics data.

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Big Data RNA Bioinformatics Vaccination 52

XTalks

MAY 4, 2021

Researchers at the University of California San Francisco (UCSF) and the Whitehead Institute have developed a novel CRISPR-based tool called “CRISPRoff” that can switch off genes in human cells without editing the genetic sequence itself. These modifications regulate gene expression without altering the sequence or structure of DNA.

DNA 52

DNA Gene Gene Silencing Genome 52

Drug Discovery World

MAY 30, 2023

From antibody-drug conjugates and RNA, to CRISPR and CAR-T therapy, DDW asked the drug discovery industry what the most important development in oncology over the last five years has been. I strongly believe that the next frontier to further improve patient outcomes will be to favour an immune-prone tumour microenvironment (TME).

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Research Drugs Protein RNA 52

Drug Discovery World

JUNE 6, 2023

“We’re still seeing those at the meeting at the moment”, he said, and thinks they still hold great promise for patients the better we understand patient genetics. He said: “We saw patient transcriptomics from CRUK this week, particularly all the data coming out from the TRACERx study, looking at the evolution of lung cancer.

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Protein DNA RNA Research 52

XTalks

DECEMBER 24, 2020

From isolating SARS-CoV-2 in early January to sequencing its genome shortly thereafter and having a prototype vaccine against it within days, scientific process and progress have held steadfast throughout the pandemic. Infectious disease expert, epidemiologist and director of the Yale Institute for Global Health at Yale University, Saad B.

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