Pharmaceutical Technology

APRIL 12, 2023

Repotrectinib, a tyrosine kinase inhibitor (TKI), is in an ongoing registrational Phase I/II trial (NCT03093116) for patients ROS1+ advanced non-small cell lung cancer (NSCLC) and NTRK+ advanced solid tumors, that are either TKI-naïve or TKI-pretreated. The ROS1 gene is altered in approximately in 1-2% of lung cancer patients.

Genome 130

Genome Genomics In-Vitro Gene 130

Worldwide Clinical Trials

DECEMBER 12, 2022

Genetic testing provides patients with a diagnosis for their illness, helps patients and family members to understand risks of developing new diseases, and can be used to support clinical trial advancement. Can prior genetic testing results be utilized in a trial, or should a new genetic sample be taken before participating?

Clinical Trials 130

Clinical Trials Clinical Trials Genetics Trials 130

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 DNA Recombinant DNA Technology 130

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 98

Gene Editing Trials Drugs In-Vivo 98

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

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 145

Gene Editing Gene Gene Therapy In-Vivo 145

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

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

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 98

Gene Editing Gene DNA Engineer 98

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

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

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

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 97

Genetic Disease Genetics Research Genome 97

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 97

Gene Sequencing DNA Vaccine Vaccination 97

XTalks

FEBRUARY 4, 2021

The power of leveraging clinical data to decipher disease mechanisms and fuel drug discovery has rapidly grown in the era of genomics and personalized medicine. Genuity Science focuses on population genomics as a tool to derive novel biological insights through partnerships with industry-leading pharmaceutical and biotech companies.

Research 98

Research Biobanking Drugs Genome 98

XTalks

SEPTEMBER 9, 2021

It was found that the gene being delivered through the therapy had integrated into the genomes of six of the seven mice that were administered the highest dose. Although a rare event that occurs at very low frequency, AAV vectors can integrate into genomes through homologous recombination.

Gene Therapy 105

Gene Therapy Gene Genome Genomics 105

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 94

Trials DNA Genome Genomics 94

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 Gene 111

XTalks

JANUARY 21, 2025

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.

FDA Approval 105

FDA Approval Manufacturing Development RNA 105

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 59

Gene Therapy Gene Trials Gene Expression 59

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 97

Clinical Research Research Clinical Trials Clinical Trials 97

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

XTalks

MAY 10, 2022

XTALKS WEBINAR: Harnessing Artificial Intelligence for Higher Quality Data in Preclinical Trials and Translational Research. Somatic variant classification and interpretation are the most time-consuming steps of tumor genomic profiling,” said the co-author of the study Marilyn M.

Bioinformatics 98

Bioinformatics Development Gene Genome 98

pharmaphorum

AUGUST 31, 2022

In recent trials, researchers found that when the gene therapy was injected into the spinal cords of mice with ALS that SynCav1 protected and preserved spinal cord motor neurons and extended the lifespan of the mice. The therapy candidate involves injecting an adeno-associated viral vector which carries synapsin-Caveolin-1 cDNA (SynCav1).

Gene Therapy 98

Gene Therapy Gene Gene Editing DNA 98

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 Vaccine Vaccination DNA 111

pharmaphorum

SEPTEMBER 13, 2022

Although clinical trials proved that chemicals could be used to treat cancer, the results of the study remained a closely guarded military secret until 1946. 2012 – The 100,000 Genomics Project begins. Unlocking the secrets of the human genome has intrigued investigators for centuries. 1956 – The rise of bone marrow transplants.

Genome Project 98

Genome Project Genome Genomics DNA 98

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

SEPTEMBER 2, 2021

It is known as the “guardian of the genome” given its central role in the DNA damage response. It senses damage to DNA and triggers protective mechanisms to try to repair it; if damage is extensive and beyond repair, it triggers death of the cell.

Protein 105

Protein DNA Antibody Genome 105

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

The Pharma Data

AUGUST 27, 2020

FoundationOne Liquid CDx analyses more than 300 cancer-related genes and multiple genomic signatures to help inform treatment decisions for all solid tumour cancers. FoundationOne Liquid CDx adds to Foundation Medicine’s genomic testing portfolio supporting Roche’s mission to deliver truly personalised healthcare.

FDA Approval 52

FDA Approval Genome Genomics Gene 52

pharmaphorum

APRIL 20, 2022

The potential of CRISPR technology has been a hot topic in the industry ever since it was first developed, but as trials progress further into the clinic, what therapeutic areas could be set to benefit? What this means is that it can edit virtually any segment of DNA, whilst being more precise and cheaper than existing editing tools.

Gene Editing 52

Gene Editing DNA Gene Genetics 52

The Pharma Data

DECEMBER 14, 2020

Many drugs fail in clinical trials because of their low blood brain barrier permeability. engine along with algorithms tuned to predict BBB permeability played an important role in helping determine which CNS cancers and which genomically-defined subtypes of CNS cancer should be prioritized for development. . Lantern’s A.I.

Drugs 52

Drugs Genome Genomics DNA 52

pharmaphorum

APRIL 14, 2021

But AZ thinks it can improve on Lynparza , which works by inhibiting poly (ADP-ribose) polymerase (PARP), part of the specialist DNA damage repair system that cancer cells with BRCA and similar mutations rely on to survive. O’Connor didn’t want to comment on whether AZD5305 could lead to an extension of the partnership with Merck & Co.

Drugs 59

Drugs DNA Genome Genomics 59

pharmaphorum

APRIL 27, 2021

Dosing can start once again in uniQure’s phase 3 trial of its haemophilia B gene therapy, after the FDA concluded that a case of liver cancer seen in the study was unlikely to be caused by the treatment. . The HOPE-B trial has now been cleared to restart dosing, and uniQure expects to report top-line results before the end of June.

Gene Therapy 52

Gene Therapy Gene Genome Genomics 52

The Pharma Data

SEPTEMBER 6, 2021

Genomic data from the phase II CUPISCO study in Cancer of Unknown Primary (CUP), an uncommon type of cancer with high unmet need, could support diagnostics and more personalised treatment plans. Tiragolumab. Real world data. Real-world Adjuvant Treatment Patterns and Survival Outcomes among early NSCLC US Patients. Real world data.

Genome 52

Genome Genomics HR Hormones 52