Bioengineer

JANUARY 16, 2024

This mutation hinders the drug’s binding to the mutant EGFR protein. Osimertinib, however, can covalently bind to the T790M and cysteine-797 (C797) residue at the protein’s ATP binding site, overcoming resistance mechanisms. T790M loss has been linked to earlier resistance and poorer survival in other cohorts.

Genome 119

Genome Genomics Protein In-Vitro 119

XTalks

OCTOBER 20, 2023

Clinical-stage genome editing company Intellia Therapeutics has received clearance from the US Food and Drug Administration (FDA) for its Investigational New Drug (IND) application to start a pivotal phase III trial of NTLA-2001 for the treatment of transthyretin (ATTR) amyloidosis with cardiomyopathy.

In-Vivo 52

In-Vivo Gene Editing Trials Gene 52

Drug Discovery World

MARCH 9, 2023

This includes the measurement of plasma kallikrein protein levels and clinical activity as determined by HAE attack rate measures. The post FDA clears in vivo gene editing therapy for trials in US appeared first on Drug Discovery World (DDW).

In-Vivo 52

In-Vivo Gene Editing Gene Trials 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

JUNE 28, 2021

The first ever clinical data with a CRISPR/Cas9 drug used to edit the genomes of cells within the body has yielded impressive results in patients with ATTR amyloidosis, a life-threatening rare disease. . Intellia and Regeneron’s phase 1 trial looked at patients who had nerve damage (polyneuropathy) as a result of the disease.

In-Vivo 92

In-Vivo Gene Editing Trials Drugs 92

Drug Discovery World

NOVEMBER 14, 2023

A Phase Ib trial has shown low-density lipoprotein cholesterol (LDL-C) reductions up to 55% and blood PCSK9 protein reductions up to 84% after a single infusion of Verve Therapeutics’ Verve-101. Since the mutation only affects a single gene, it makes it a prime candidate for genome editing treatments.

In-Vivo 52

In-Vivo Gene Editing Gene Genome 52

World of DTC Marketing

DECEMBER 29, 2020

Both the Pfizer and Moderna vaccines copied RNA sequence from the virus genome and found a way to manufacture it at scale with high-level processes and quality control. Another consideration is that while in traditional vaccine development, clinical trials are carried out in sequence. 1455NO-HEALTH-CORONAVIRUS_VACCINES_PFIZER_O_.

Vaccination 307

Vaccination Vaccine Development Clinical Trials 307

pharmaphorum

OCTOBER 20, 2021

A software platform developed by Altoida that uses augmented reality and artificial intelligence to diagnose neurological diseases in their earliest stages is being tested in a five-year trial involving thousands of patients.

Trials 76

Trials Genome Genomics Bioinformatics 76

Pharmaceutical Technology

JULY 5, 2022

Researchers at the UNC School of Medicine have discovered that hepatitis A virus (HAV) replication needs particular interactions between the human protein ZCCHC14 and TENT4 poly(A) polymerases, a group of enzymes. HAV usually hijacks TENT4 and utilises it for the replication of its own genome.

Protein 130

Protein RNA Genome Genomics 130

Pharmaceutical Technology

JANUARY 25, 2023

PXL770 is a new, first-in-class direct activator of adenosine monophosphate-activated protein kinase (AMPK). PXL065 is a new deuterium-stabilised R-stereoisomer of pioglitazone that exerts effects through several non-genomic pathways that are engaged by thiazolidinedione molecules.

Drugs 130

Drugs Genome Genomics Medicine 130

WCG Clinical

NOVEMBER 17, 2023

This change expanded the definition of HGT research to include research using nucleic acids that are able to replicate, be transcribed, translated into protein, and/or integrate into the host genome. If implemented, these changes would broaden the definition of HGT to include new gene editing and genome-modified products.

Genome 52

Genome Genomics Gene Editing Genetic Engineering 52

Drug Discovery World

MAY 30, 2024

Dr Pollock is the Chief Investigator of the LEGACY03 clinical trial, a study aiming to investigate lymph nodes and vaccination responses across age groups. RA: Could you tell us about the LEGACY03 trial? It is these lymph nodes which help to make proteins called antibodies that protect from infection.

Vaccination 59

Vaccination Vaccine Scientist Clinical Trials 59

XTalks

NOVEMBER 7, 2022

These tumor-derived entities are used to derive genomic and proteomic data. Gleich, MD, FACS, Senior Vice President, Medical Department, and Dr. Christopher Huth, PhD, Senior Clinical Trial Manager, Clinical Trial Management. Liquid Biopsy Use in Oncology Clinical Trials.

Clinical Trials 98

Clinical Trials Clinical Trials Trials FDA Approval 98

pharmaphorum

SEPTEMBER 16, 2022

A mere six months ago Verily launched the study with Sosei Heptares – a global leader in GPCR structure-based drug design – with an aim to “prioritise protein targets for therapeutic targeting in immune-mediated disease”. It also bodes well for improving clinical trial success rates. What, then, is the solution?

Development 126

Development Research Drugs Clinical Trials 126

Drug Discovery World

APRIL 30, 2024

Its job is to carry coding information that is essential to the translation and processing of functional proteins. This is essential to its use as a therapeutic agent and gives the technology a vast versatility making it suitable to treating a wide range of diseases – especially those that have high protein expression.

RNA 52

RNA Protein Vaccination Vaccine 52

Drug Discovery World

JUNE 23, 2023

Elevidys addresses the root genetic cause of Duchenne – mutations in the dystrophin gene that result in the lack of dystrophin protein – by delivering a gene that codes for a shortened form of dystrophin to muscle cells known as Elevidys micro-dystrophin.

Gene Therapy 97

Gene Therapy FDA Approval Gene Protein 97

Pharmaceutical Technology

FEBRUARY 15, 2023

They are engineered to cut specific genomic targets in order to modify the expression of single genes and proteins. Sangamo Therapeutics has a market cap of $560m and is primarily focused on developing genomic medicines in rare disease, neurology, oncology and autoimmune.

Gene Splicing 130

Gene Splicing Gene Recombinant DNA Technology DNA 130

Drug Discovery World

DECEMBER 15, 2022

Now, after nearly 35 years of research and more than a decade of preclinical progress, several different gene editing modalities are being tested in early phase clinical trials. Early phase clinical trials for gene editing therapies. Gene editing challenges and potential solutions.

Gene Editing 52

Gene Editing Gene In-Vivo Genome 52

pharmaphorum

JULY 20, 2022

Armed with a $100 million second-round financing, CAMP4 Therapeutics is preparing to start the first clinical trial of a drug targeting regulatory RNA (regRNA) molecules that can be used to fine-tune the expression of genes. ” The post CAMP4 raises $100m to take lead RNA drugs into clinic appeared first on.

RNA 52

RNA Genetic Disease Drugs Protein 52

Drug Discovery World

APRIL 18, 2023

The results from the Phase I portion of the Phase I/II AcSé-ESMART trial were presented at the AACR Annual Meeting 2023. “To Single-agent therapies targeting one mutated protein are often insufficient in paediatric patients, necessitating additional research into combination therapies and mechanisms of response.

DNA 52

DNA Genome Genomics HR 52

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

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

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

The Pharma Data

DECEMBER 17, 2020

Lupus causes the production of proteins called autoantibodies in the immune system, which attack tissues and organs, including the kidneys. LN, or inflammation of the kidneys, is a common system of systemic lupus erythematosus (SLE), one of the most prevalent autoimmune diseases. When Pigs Fly Allergy-friendly.

FDA Approval 52

FDA Approval Genome Genomics Production 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 142

Gene Editing Gene Gene Therapy In-Vivo 142

pharmaphorum

SEPTEMBER 14, 2021

The Cambridge, Massachusetts biotech is focusing specifically on the 60% of DNA sequences that repeat themselves hundreds or thousands of times in the genome, known as the ‘repeatome’, that could contain targets for cancer and autoimmune disorders.

DNA 52

DNA Immune Response Genome Genomics 52

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

Drug Discovery World

NOVEMBER 27, 2023

Cystic fibrosis mutations In people with certain types of mutations in the CFTR gene, the CFTR protein is not processed or folded normally within the cell, and this can prevent the CFTR protein from reaching the cell surface and functioning properly.

Protein 52

Protein Gene Medicine Gene Therapy 52

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

JANUARY 21, 2024

It is enabling computational protein design for vaccine immunogen discovery, and high-throughput and high-definition assays for quicker and more comprehensive characterisation of vaccine candidates and vaccine-elicited immunity. His recent research focuses on vaccines against influenza and other (re)emerging viruses.

Protein 52

Protein Vaccination Vaccine Development 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

XTalks

MARCH 7, 2024

Experts discuss the therapeutic landscape, treatment gaps, regulatory considerations and clinical trial strategies. A new workflow from BioSkryb Genomics merges extensive data from whole genome sequencing (WGS) and the focused insights gained from small, targeted panels that emphasize druggable targets.

Hormones 115

Hormones Genetics Genome Genomics 115

XTalks

DECEMBER 1, 2021

To ensure the new tests can identify Omicron based on its mutations, the companies assessed their tests against sequences in public databases of genomic data, such as GISAID and GenBank. Cue is also currently working with Google Cloud and genomics company Helix to study variants and share mutation sequencing data.

Gene 105

Gene Genome Genomics Genetics 105

Drug Discovery World

JANUARY 23, 2023

Strand’s mRNA therapeutics enable precise control of the location, timing, intensity and duration of therapeutic protein expression for improved efficacy and lower toxicity. Our team has been focused on engineering mRNA so that it’s only used to express cargo protein in certain cells regardless of where the molecule ends up in the body.

RNA 98

RNA Genome Genomics Engineer 98

The Pharma Data

MARCH 3, 2021

(Nasdaq: VIR) and GlaxoSmithKline plc (LSE/NYSE: GSK) today provided an update on the VIR-7831 (GSK4182136) arm of the National Institutes of Health’s (NIH) Accelerating COVID-19 Therapeutic Interventions and Vaccines (ACTIV) Program Phase 3 clinical trial.

Trials 52

Trials Antibody Vaccination Vaccine 52

The Pharma Data

JUNE 7, 2023

3,4,5 TROP2 is a protein expressed in more than 90% of NSCLC tumors. Results will be presented on June 6 during an oral presentation (#9004) at the American Society of Clinical Oncology Annual Meeting (#ASCO23). More than one million people are diagnosed with NSCLC at an advanced stage each year. Rahway, NJ, USA), an anti-PD-1 therapy.

Trials 40

Trials Antibody Development Genome 40

Drug Discovery World

APRIL 8, 2024

Aviv Regev, Head and Executive Vice President of Research and Early Development at Genentech, opened the session with a discussion of how advances in genomic sequencing are fostering the development of data-rich cell atlases and what that data could mean for new insights into cancer biology and novel therapeutics.

In-Vivo 52

In-Vivo In-Vitro Antibody Protein 52