Drug Discovery World

NOVEMBER 7, 2022

A new study led by Medical Research Council-funded researchers from UCL has found that tailoring the analysis of whole genome sequencing to individual patients could double the diagnostic rates of rare diseases. . Consequently, the UK has established itself at the forefront of diagnostic whole genome sequencing. Context .

Genome 52

Genome Genomics Genetics Genetic Disease 52

pharmaphorum

SEPTEMBER 14, 2021

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. Most drug discovery programmes target the roughly 2% of the human genome which encodes for protein.

DNA 52

DNA Immune Response Genome Genomics 52

Drug Discovery World

MARCH 27, 2023

Genomics is driving a revolution in drug development and, in the long run, the state of public healthcare. The first draft human genome took a decade to create, and 13 years to complete. Advances in sequencing are leading to scientific breakthroughs in preventative care, faster diagnoses, and proactive medical interventions.

Genome 52

Genome Genomics Research Scientist 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. 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.

Gene Editing 98

Gene Editing DNA Gene Genome 98

Pharmaceutical Technology

FEBRUARY 15, 2023

Within the emerging innovation stage, cell therapy for ocular disorders, coronavirus vaccine components, and DNA polymerase compositions are disruptive technologies that are in the early stages of application and should be tracked closely. Nucleases are enzymes that hydrolytically cleave the phosphodiester backbone of DNA.

Gene Splicing 130

Gene Splicing Gene Recombinant DNA Technology DNA 130

XTalks

MAY 23, 2024

Eterovic brings much knowledge and experience to the table, having led the development of a new Oncology Diagnostic program at Eurofins Viracor that integrates the latest genomic profiling techniques. Eterovic highlights that the financial and logistical barriers to comprehensive genomic profiling are high but not impossible to overcome.

Genetics 114

Genetics Research Genome Genomics 114

The Pharma Data

OCTOBER 26, 2021

The AVENIO Tumor Tissue Comprehensive Genomic Profiling (CGP) Kit is the first jointly-developed product that brings together the expertise and reach of Roche with Foundation Medicine’s pioneering leadership in genomic science. Cancer is a disease of the genome and treatment no longer depends solely on the tissue of origin.

Genome 52

Genome Genomics Research Medicine 52

Drug Discovery World

NOVEMBER 8, 2023

Deepika Khedekar , Associate Centralized Clinical Lead at IQVIA, explains how artificial intelligence could make clinical trials more accurate, accessible and standardised. Tapping into the capabilities of AI One reason AI may accelerate clinical trials is its ability to rapidly process and analyse vast amounts of data.

Clinical Trials 72

Clinical Trials Clinical Trials Trials Drugs 72

pharmaphorum

JULY 13, 2022

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.

Gene Editing 97

Gene Editing Trials Drugs Gene 97

Drug Discovery World

DECEMBER 15, 2022

These platforms all induce double strand cuts in the chromosomal DNA that can be sealed by the cell, leading to a specific gene disruption, or resulting in a new target site for inserting DNA segments. Early phase clinical trials for gene editing therapies. Gene editing challenges and potential solutions.

Gene Editing 52

Gene Editing Gene In-Vivo Genome 52

Drug Discovery World

JULY 22, 2022

Junk DNA could lead to cancer, study says . Non-coding or ‘junk’ DNA could contribute to the development of certain cancers due to how it disrupts the replication of the genome, scientists have discovered.?? . US launches clinical trial for pandemic preparation .

DNA 52

DNA Drugs Clinical Trials Clinical Trials 52

XTalks

NOVEMBER 7, 2022

Liquid biopsy tests in oncology involve isolating entities such as circulating tumor cells (CTC), circulating tumor DNA (ctDNA) and tumor-derived exosomes. These tumor-derived entities are used to derive genomic and proteomic data. Liquid Biopsy Use in Oncology Clinical Trials.

Clinical Trials 98

Clinical Trials Clinical Trials Trials FDA Approval 98

Drug Discovery World

JANUARY 17, 2024

Brett Swansiger, Chief Commercial Officer at ANGLE discusses the importance of liquid biopsies in precision medicine trials for cancer. Furthermore, oncology clinical trials are frequently challenged by low enrolment rates, failure to achieve primary endpoints, study design complexity and limited funding 5.

Medicine 59

Medicine DNA Genome Genomics 59

Bioengineer

JANUARY 16, 2024

Analysis of circulating tumor (ct) DNA at the time of treatment discontinuation or progression showed that 49% of patients in the AURA3 trial lacked the T790M mutation. not reached) in genomic analysis of post-progression samples from patients receiving second-line osimertinib. months, 95% CI 1.3,

Genome 119

Genome Genomics Protein In-Vitro 119

Drug Discovery World

APRIL 18, 2023

A combination of the PARP inhibitor olaparib (Lynparza) and the investigational ATR inhibitor ceralasertib showed clinical benefit in paediatric patients with solid tumours exhibiting DNA replication stress and/or DNA repair deficiencies, according to new data.

DNA 52

DNA Genome Genomics HR 52

Drug Discovery World

JULY 18, 2023

Rolf Turk , Senior Manager, Genomics Medicine at Integrated DNA Technologies, examines how CRISPR is being used to enhance cancer therapies. These methods offer safer CAR T-based therapies, laying the foundation for clinical trials and the rapid development of a new generation of CAR T cells for both autologous and allogeneic use.

DNA 98

DNA Genome Genomics RNA 98

Drug Discovery World

DECEMBER 13, 2022

Boston Children’s Hospital researchers will use HiFi whole-genome sequencing (HiFi WGS) for the purpose of investigating genetic and epigenetic variants associated with rare paediatric diseases. The study will also explore epigenetic variation using the ability of HiFi WGS to measure DNA methylation in every sequencing run. .

Genetics 52

Genetics Research Genome Genomics 52

Drug Discovery World

OCTOBER 19, 2022

Yapan Bio has expanded its capabilities with a new process development facility at Genome Valley, Hyderabad, India, which has enhanced its ability to support end-to-end development and manufacturing of RNA, DNA and gene therapy products starting from plasmids. .

Development 52

Development Gene Therapy RNA DNA 52

Pharmaceutical Technology

OCTOBER 26, 2022

Gene therapy uses DNA to manipulate cells and correct defective genes, whereas cell therapy is the infusion or transplantation of cells into a patient. New doors were opened in the 2000s with the development of genome editing, such as CRISPR-Cas9, which introduces molecular tools to change existing DNA.

Gene Therapy 130

Gene Therapy Gene Marketing Manufacturing 130

pharmaphorum

JANUARY 18, 2023

The DNA of these so-called ‘genetic superheroes’ may contain clues about how to treat severe disease. Fast forward to today and, while many large-scale genomics studies still lack the infrastructure and consent policies to enable recontact, this is starting to change. Giving participants something in return.

Genetic Disease 81

Genetic Disease Genetics Research Genome 81

Worldwide Clinical Trials

JUNE 15, 2022

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.

Gene Therapy 130

Gene Therapy Gene Clinical Trials Clinical Trials 130

Drug Discovery World

MARCH 6, 2024

Guiding neurological drug discovery and development to success The 20 years since the Human Genome Project has seen transformational advances in the molecular understanding of cancers and rare genetic diseases, leading to genetically informed, personalised selection of therapies and massively improved outcomes for many patients.

Genome 64

Genome Genomics Clinical Trials Clinical Trials 64

Drug Discovery World

MARCH 26, 2024

More importantly, it also highlighted that significant challenges remain in selecting the right therapies for the right patients despite the progress made through genomic testing and implementation of circulating tumour (ct) DNA analyses. Results of that Phase I trial are expected to be presented later this year.

RNA 64

RNA In-Vivo In-Vitro DNA 64

pharmaphorum

JANUARY 5, 2023

and OriCiro Genomics K.K. 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. Biotech Moderna, Inc.

Gene Sequencing 80

Gene Sequencing DNA Vaccination Vaccine 80

pharmaphorum

MARCH 10, 2021

Known as Zynteglo in Europe where it is approved to treat the rare blood disorder beta-thalassemia, the gene therapy has been given the all-clear by bluebird’s scientists after reviewing the case of AML that emerged in a trial of the drug in sickle cell disease. bluebird bio’s Philip Gregory.

Gene Therapy 52

Gene Therapy Gene Trials Gene Expression 52

XTalks

NOVEMBER 30, 2023

The safety and efficacy of Cabenuva were established through two randomized, open-label, controlled clinical trials involving 1,182 HIV-infected adults who were virologically suppressed (HIV-1 RNA less than 50 copies/mL) before initiating Cabenuva treatment. The Phase I trial for HB-500 is scheduled to start in the first half of 2024.

FDA Approval 111

FDA Approval Gene Therapy Clinical Trials Clinical Trials 111

pharmaphorum

JANUARY 29, 2021

Genome editing is an exciting but still nascent field, and companies in the area face as many obstacles as they do opportunities. ZFPs can be engineered to make zinc finger nucleases, or ZFNs, which are proteins that can be used to edit genomes by knocking select genes in or out to specifically modify DNA sequences.

Gene Editing 142

Gene Editing Gene Gene Therapy In-Vivo 142

pharmaphorum

FEBRUARY 3, 2021

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.

Gene Editing 92

Gene Editing Gene DNA Engineer 92

Drug Discovery World

OCTOBER 6, 2022

Jarret Glasscock , PhD, CEO of Cofactor Genomics explains how diagnostics are emerging as the key to ensuring the right patients get matched to the right therapy, at the right time. . In the cancer space alone over the last five years, clinical trials have increased 500% for new drugs and new drug combinations.

Medicine 52

Medicine Clinical Trials Clinical Trials Genome 52

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).

Medicine 52

Medicine Genome Genomics Bioinformatics 52

The Pharma Data

APRIL 8, 2021

Leadership in DNA damage response demonstrated in multiple presentations for AZD5305, a next-generation PARP1 selective inhibitor. Data for AZD5305 will demonstrate how the next wave of DNA damage response medicines can build on the success of PARP inhibitors, potentially allowing patients to stay on treatment longer.

DNA 52

DNA Medicine Genome Genomics 52

XTalks

DECEMBER 20, 2023

This includes analyzing how drug combinations may impact individual patients or groups of patients before even entering a clinical trial. With a staggering 90 percent of drugs failing in clinical trials, AI has the potential to help improve these statistics. These companies are at various stages of research and clinical trials.

Life Science 119

Life Science Gene Editing Development Clinical Trials 119

The Pharma Data

NOVEMBER 17, 2021

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.

Scientist 52

Scientist RNA DNA Genetics 52

Cloudbyz

JUNE 26, 2023

Clinical Trial Design and Optimization: Generative AI can aid in the design and optimization of clinical trials. By simulating patient populations, generating synthetic trial data, and optimizing trial protocols, generative models can enhance the efficiency and effectiveness of clinical trials.

Clinical Research 77

Clinical Research Research Clinical Trials Clinical Trials 77

XTalks

DECEMBER 13, 2023

The company’s sustained investment in R&D has led to a rapid expansion of its pipeline in 2023, with notable milestones such as four infectious disease vaccines in Phase III trials, including the recently submitted respiratory syncytial virus (RSV) vaccine for regulatory approval. million, marking a significant increase from the $56.1

Genetics 111

Genetics Vaccination Vaccine DNA 111

Drug Discovery World

DECEMBER 29, 2022

Recombinant DNA technology and its impact on modern drug discovery . What can we learn from the genome? . Now is the time for the transformation of clinical trials . Is genomic data the new frontier in drug discovery? . Sitting Down With . Catch up on the Sitting Down With podcast episodes released in 2022: .

Recombinant DNA Technology 52

Recombinant DNA Technology Genome Genomics DNA 52

pharmaphorum

OCTOBER 27, 2022

Discussion was centred around ESMO 2022, where Dr Ray-Coquard presented collated data from a landmark five-year follow-up of the AstraZeneca/MSD PAOLA-1 phase 3 trial, the results from which suggested a clinically meaningful improvement in overall survival rates of certain patients with ovarian cancer. Furthermore, 65.5%

Trials 71

Trials DNA Genome Genomics 71