BioTech 365

DECEMBER 5, 2020

Long-Term Data for bluebird bio’s betibeglogene autotemcel (beti-cel) Gene Therapy Show Patients Across Ages and ?-thalassemia

Genotype 40

Genotype Gene Therapy Gene 40

Worldwide Clinical Trials

AUGUST 14, 2024

FLT3 mutations seen in acute myeloid leukemia (AML) and BCR-ABL1 fusion gene seen in chronic myeloid leukemia (CML) are two examples. Understand Resistance Mechanisms : Biomarkers can help identify mechanisms of resistance to treatments, as mutations in certain genes can alter the effectiveness of targeted therapies.

Genetics 195

Genetics Protein Gene Genotype 195

pharmaphorum

DECEMBER 9, 2020

bluebird bio has presented long-term data from its Zinteglo one-time gene therapy for the blood disorder beta-thalassaemia, as the company continues talks with payers in Europe to bring the ultra-pricey treatment to market. The post Bluebird trumpets long-term data from beta-thalassaemia gene therapy appeared first on.

Gene Therapy 59

Gene Therapy Gene Genotype Marketing 59

BioTech 365

SEPTEMBER 10, 2020

Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: GTEx Consortium releases fresh insights into how DNA differences govern gene expression.Scientists from the Genotype-Tissue Expression (GTEx) project, a National Institutes of Health-funded consortium including researchers from (..)

Gene Expression 40

Gene Expression DNA Gene Genotype 40

Worldwide Clinical Trials

DECEMBER 12, 2022

In some cases, the gene associated with a patient’s condition may not yet be discovered or included on the test ordered and the interpretation of genetic variants may change over time as new information is uncovered. single gene testing vs comprehensive panel), the reportable range (e.g.,

Clinical Trials 130

Clinical Trials Clinical Trials Genetics Trials 130

XTalks

SEPTEMBER 28, 2020

The test is based on the detection of actionable genes – genes with known driver mutations that can be targeted by an approved therapy (or investigational therapies in clinical trials). The panel of genes in the test only include known NSCLC-associated genes, making the test specific to the disease. Personalized Approach.

Gene 105

Gene DNA Life Science Genotype 105

XTalks

JANUARY 21, 2025

Using cervical or self-collected vaginal specimens, the test works with the BD Viper LT and BD COR systems to detect individual genotypes, such as HPV 16, 18 and 31, and grouped results for other types. Companion diagnostic for Pfizers hemophilia B gene therapy Beqvez (idanacogene elaparvovec-dzkt).

FDA Approval 105

FDA Approval Manufacturing Development RNA 105

XTalks

APRIL 15, 2021

From a total of 30 single-nucleotide polymorphisms (SNPs) in genes previously identified as being associated with blood pressure, many had a relatively neutral effect on the risk of coronary artery disease. Figure 2: Using drug-target MR to validate sympathetic nervous system genes involved in heart failure.

Development 98

Development Drugs Genome Genomics 98

Pharmaceutical Technology

DECEMBER 15, 2022

Therefore, there is also a significant need for new therapies that target the underlying cause of CFD that may offer a cure for patients living with severe disease, or for patients with genotypes such as dysfibrinogenemia and hypodysfibrinogenemia, who are often ineligible for treatment with HFCs.

Marketing 130

Marketing Production Genotype Development 130

Bioengineer

JANUARY 16, 2024

In patients with T790M mutant NSCLC receiving osimertinib, 63% experienced loss of T790M at progression, commonly linked to histological transdifferentiation, KRAS mutations, or gene fusions. In the same study, those with T790M loss had a shorter treatment duration (6.1 months vs. 15.2 months, p = 0.01).

Genome 119

Genome Genomics Protein In-Vitro 119

XTalks

NOVEMBER 10, 2020

How many genes need to be scanned in order to determine the presence of a genetic disease? HS: Due to the heterogeneity of the rare disease population, there are many relevant gene lists, and the genomic data analysis of these patients covers almost 2,000 different genetic targets.

Genome 98

Genome Genomics Medicine Genetic Disease 98

pharmaphorum

JANUARY 24, 2023

Pharma has used this to begin creating targeted treatments, such as gene therapies , which are able to hone in on gene mutations that cause health issues and eradicate or replace the faulty gene. Scientists are able to study the aggregated, de-identified genetics of these individuals.

Genetics 98

Genetics Drugs Genome Project Biobanking 98

pharmaphorum

JANUARY 16, 2023

The study by a team at the Universities of East Anglia and Edinburgh shows that HRT use is associated with better memory, cognition, and larger brain volumes in later life among women carrying the APOE4 gene, a well-established risk factor for Alzheimer’s.

Hormones 75

Hormones Genotype Research Genetics 75

pharmaphorum

OCTOBER 9, 2020

The human history study, which is being funded by the foundation and its supporters, will feed into gene reviews, an international point-of-source for inherited disorders. The team hopes that this study’s scope will also shed light on the gene’s impact on the women who carry it in addition to an elaborative genotype-phenotype correlation.

Research 111

Research Genetics Genotype Nurses 111

pharmaphorum

AUGUST 11, 2022

Pharmacogenomics, the understanding of how genes impact an individual’s response to medications, provides a potential outlet to better optimised therapies. The program is aiming to genotype roughly 150,000 participants over the next few years to demonstrate the impact personalised testing can have on a large scale.

Genome 98

Genome Genomics Drugs Genetics 98

The Pharma Data

NOVEMBER 2, 2020

ONWARD is investigating Adial’s lead drug candidate, AD04, as a therapeutic agent for the treatment of Alcohol Use Disorder (AUD) in persons with certain target genotypes related to the serotonin transporter and receptor genes. Key ONWARD highlights include: All 25 planned investigative sites are active.

Trials 52

Trials Genotype Marketing Production 52

pharmaphorum

NOVEMBER 10, 2021

It recommends the National Library of Medicine, which has already published “considerable information” on resistance genes, genome sequences, antimicrobial susceptibility data, and bacterial genomes, establish an open-source, unified AMR database.

Medicine 105

Medicine Genome Genomics Genotype 105

XTalks

MARCH 3, 2021

MoCD Type A is an autosomal recessive, inborn error of metabolism caused by mutations in the molybdenum cofactor synthesis 1 gene. In the analysis involving 31 patients, 13 were treated with Nulibry and compared to 18 genotype-matched untreated patients from the history control group. What is Molybdenum Cofactor Deficiency Type A?

FDA Approval 64

FDA Approval Genotype Clinical Trials Clinical Trials 64

Roots Analysis

AUGUST 31, 2023

According to a study, around 20,000 genes are present in the human body, all of which interact with the nutrients in the food, either directly or indirectly. For instance, carbohydrates increase the expression of genes responsible for the synthesis of enzymes and transporters involved in their metabolism.

Genome 40

Genome Genomics Genetics Gene 40

Roots Analysis

AUGUST 12, 2024

Hybridization Tests – These tests utilize complementary DNA or RNA probes to detect and quantify specific genetic targets, enabling the identification of pathogens, genetic variations, and gene expression patterns. Such test can be used for gene expression profiling, genotyping and detecting chromosomal abnormalities.

Genetics 40

Genetics Gene Expression RNA DNA 40

XTalks

DECEMBER 12, 2023

In some areas, say Alzheimer’s disease, we have growing collections of imaging, gene, cerebrospinal fluid (CSF) and blood markers to follow and look at target engagement of our drugs,” says James Vornov, MD, PhD, Vice President of the Medical Department at Medpace.

Trials 111

Trials Clinical Trials Clinical Trials Genetics 111

The Pharma Data

NOVEMBER 18, 2020

such as the integrase gene and assay validation recommendations. The recommended specimen types for HIV drug resistance genotyping are plasma or dried blood spots. Dried blood spots should be used for HIV drug resistance surveys in which plasma cannot. be handled as per WHO recommendations.

Drugs 52

Drugs Genotype Medicine Development 52

The Pharma Data

DECEMBER 9, 2020

The Chinese biopharmaceutical industry is growing in leaps and bounds, but there is still a huge unmet need when it comes to getting patients access to the breakthrough therapeutic modalities and platforms like RNAi, cell and gene therapy and others. A solution could be at hand with Overland Pharmaceuticals.

In-Vivo 52

In-Vivo In-Vitro Antibody Development 52

Roots Analysis

APRIL 28, 2024

Absence of Biomarkers for Diagnosis: Due to the absence of genotype-phenotype correlations and prognostic markers, there have been challenges in the diagnosis and treatment of rare kidney diseases. Insufficient Model Organisms: Limitations of these model organisms includelong generation time and strain effects.

Genetics 40

Genetics Genotype Development Research 40

The Pharma Data

NOVEMBER 24, 2020

Management options to date were limited to hyperhydration, crystallization inhibitors and, in a minority of patients with a specific genotype, pyridoxine (vitamin B6). Until today, there were no approved pharmaceutical therapies for PH1.

FDA Approval 52

FDA Approval Production RNA Medicine 52