BioPharma Reporter

JUNE 19, 2023

Abu Dhabi’s Department of Health has collaborated with Mass General Brigham's International Center for Genetic Disease (iCGD).

Genetic Disease 97

Genetic Disease Genome Genomics Genetics 97

STAT News

DECEMBER 12, 2022

LONDON — England is launching a pilot program sequencing the genomes of up to 100,000 newborns to see if such a strategy can speed up the detection of genetic illnesses. The Newborn Genomes Programme will scan DNA for mutations that can cause some 200 conditions.

Genome 98

Genome Genomics Genetic Disease Genetics 98

Drug Discovery World

NOVEMBER 7, 2022

Genomic healthcare data is critical to identify disease risk, ancestry, traits and response to medicines and aids in the development of new targeted therapies – precision medicines. In April 2003, after its launch in October 1990, the project was completed, generating the first sequence of the human genome. The origins .

Genome 98

Genome Genomics Development Genetics 98

STAT News

FEBRUARY 22, 2023

The Cambridge biotech company announced Wednesday that it will partner with Life Edit Therapeutics to develop potentially permanent treatments for rare genetic diseases and other conditions. Life Edit is the North Carolina subsidiary of ElevateBio, a cell and gene therapy manufacturing firm in Waltham.

Gene Editing 126

Gene Editing Gene Genetic Disease Gene Therapy 126

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. . It also detected potential disease-causing variants in a further 3.9% Context . The study .

Genome 52

Genome Genomics Genetics Genetic Disease 52

Drug Discovery World

MARCH 21, 2023

UK researchers, from the Wellcome Sanger Institute and collaborators, have used human stem cells and neurons to investigate what features influence how well CRISPR activation works for different sets of genes. The post New research begins to write the rules on CRISPR activation appeared first on Drug Discovery World (DDW).

Research 52

Research Gene Gene Editing Genome 52

Drug Discovery World

DECEMBER 5, 2022

Scientists have unlocked key insights into virus evolution, revealing new information that could help develop treatments for a wide variety of genetic diseases. . The research focuses on a group of small, ubiquitous viruses called ‘parvoviruses’ (from the Latin word ‘ parvus’ meaning ‘small’, ‘puny’ or ‘unimportant’).

Gene Therapy 52

Gene Therapy Gene Genetic Disease Genetics 52

Drug Discovery World

DECEMBER 5, 2022

A new partnership between industry and three rare disease foundations has formed with the goal of accelerating the development of precision drugs to treat rare neurodevelopmental disorders. . Understanding rare diseases remains a significant challenge.

Development 52

Development Genome Genomics Drugs 52

Drug Discovery World

APRIL 17, 2024

DS: CAMP4 leverages research from co-founder Richard Young on how regulatory RNA (regRNA) molecules control the expression of genes. Recently, research published by Dr Richard (Rick) Young (Whitehead Institute) describes the key mechanism of how these regRNAs regulate gene expression. What diseases are you currently targeting?

Gene Expression 59

Gene Expression Gene Genetic Disease RNA 59

Pharmaceutical Technology

FEBRUARY 23, 2023

Moderna has entered a strategic research and development partnership with ElevateBio’s Life Edit Therapeutics to discover and develop new in-vivo mRNA gene editing therapies. Under the deal, both companies will partner on the research and preclinical studies, which will be funded by Moderna.

Gene Editing 147

Gene Editing Gene In-Vivo Gene Therapy 147

pharmaphorum

OCTOBER 4, 2022

AstraZeneca’s rare disease firm Alexion is set to expand its genomic medicine portfolio with the acquisition of gene editing specialist LogicBio Therapeutics, in a deal worth approximately $68 million. per share, a rare 660% premium on LogicBio’s share price. .

Gene Editing 64

Gene Editing Gene Genome Genomics 64

Drug Discovery World

MARCH 24, 2023

Israeli researchers have developed a drug that is effective in treating a rare genetic muscle disease. The team demonstrated that the disease is caused by a mutation in the gene encoding HMG CoA reductase, inhibiting the enzyme’s activity.

Research 52

Research Genetics Gene Licensing 52

pharmaphorum

SEPTEMBER 9, 2020

Geneticist Dr Charles Steward has spent his career studying the human genome – but his work became much more personal when his children were diagnosed with severe neurological diseases. Charlie told pharmaphorum how his search for a genetic cause has led him to straddle the divide between scientist and patient advocate.

Genome 102

Genome Genomics Genetics Genome Project 102

Pharmaceutical Technology

MAY 8, 2023

Innovation S-curve for the pharmaceutical industry Zinc-finger nucleases is a key innovation area in pharmaceutical Zinc finger nucleases (ZFNs) are tools used in genetic engineering to cleave a chosen genomic sequence. ZFNs are also used to create a new generation of genetic disease models called isogenic human disease models.

Genetics 130

Genetics DNA Genetic Engineering Antibody 130

pharmaphorum

SEPTEMBER 9, 2020

Geneticist Dr Charles Steward has spent his career studying the human genome – but his work became much more personal when his children were diagnosed with severe neurological diseases. Charlie told pharmaphorum how his search for a genetic cause has led him to straddle the divide between scientist and patient advocate.

Genome 80

Genome Genomics Genetics Genome Project 80

Roots Analysis

FEBRUARY 21, 2022

Since the introduction of a gene sequencing method by Frederick Sanger in 1977, the field of genomic data collection and analysis has evolved significantly. Advancement in DNA sequencing technologies have resulted in noteworthy developments in various healthcare-related research fields, such as diagnostics and personalized medicine.

Marketing 52

Marketing Gene Sequencing DNA Genome 52

Drug Discovery World

FEBRUARY 27, 2023

However, the researchers urge caution as the majority of patients have not reached the age of four to five years where the most severe stages of disease progression typically present. Patients with MPS-IIIA have a mutation in the SGSH gene, causing them to lack an enzyme which normally breaks down large sugar molecules.

Gene Therapy 52

Gene Therapy Gene Genetics Genetic Disease 52

Medical Xpress

FEBRUARY 9, 2023

In a study appearing today in JAMA Network Open, Rady Children's Institute for Genomic Medicine (RCIGM) researchers found that the contribution of genetic diseases to infant deaths was higher than previously recognized.

Genetic Disease 75

Genetic Disease Genetics Genome Genomics 75

The Pharma Data

DECEMBER 13, 2020

which develops genome editing technologies to accelerate drug discovery and develop novel therapeutics for a broad range of diseases, today announced the appointment of Bo Zhang, Ph.D., Dr. Zhang has around 20 years of experience in research and drug development in both industry and academia in the US. 14, 2020 10:00 UTC.

Business Development 40

Business Development Development Gene Editing In-Vivo 40

Drug Discovery World

MAY 11, 2023

In The Evolving Role of the CRO, panellists showed that research partner organisations and their service provision to R&D companies is essential. However, she also presented thoughts in a technical update, Targeted Protein Degradation – Progress, Direction and Prospects.

Life Science 52

Life Science Genetics Vaccination Vaccine 52

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 It’s the cause of the mutation, not the name of the disease, that matters.”. They’re basically the same,” but each one uses a particular nucleotide to bind to specific genetic sequence.

Genetics 52

Genetics Clinical Trials Clinical Trials Drugs 52

XTalks

APRIL 24, 2023

Rare diseases can often be progressive, chronic and fatal. Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. Sadly, one-third of children with rare diseases die before their first birthday.

Gene Therapy 95

Gene Therapy Gene Trials Clinical Trials 95

XTalks

DECEMBER 13, 2023

In 2023, biotechnology and pharmaceutical companies leverage cutting-edge technology and significant investments in research and development (R&D) to continue pushing the boundaries of innovation in the healthcare sector, transcending the challenges posed by the pandemic.

Genetics 111

Genetics Vaccination Vaccine DNA 111

XTalks

APRIL 15, 2021

New advances in heart failure genomics are helping to address this challenge. Experts from Servier and Genuity Science recently spoke on a webinar about using genomics data to drive drug development in heart failure and identify new targets for novel therapeutics. Watch this on-demand webinar to hear from these experts.

Development 98

Development Drugs Genome Genomics 98

Drug Discovery World

MARCH 6, 2024

The development of effective new disease-modifying treatments in neurodegenerative and neuropsychiatric disorders has been hindered by their inherent genetic complexity, environmental influences, and clinical variability. This is comparatively easier to accomplish in these diseases because they are relatively monogenic.

Genome 64

Genome Genomics Clinical Trials Clinical Trials 64

Drug Discovery World

FEBRUARY 28, 2023

As we turn our focus to new potential applications and disease areas for the platform, scientists and companies must consider the potential life-saving impact on rare, inherited diseases, says Archana Gupta , PhD, staff scientist in genetic sciences at Thermo Fisher Scientific.

Genetics 52

Genetics Genetic Analysis Vaccination Vaccine 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 through epigenetic editing without altering the genetic sequence itself. The research was published earlier this month in the journal Cell.

DNA 98

DNA Gene Gene Silencing Genome 98

Drug Discovery World

APRIL 30, 2024

Messenger RNA (mRNA) has come into focus within the drug discovery and development as an exciting tool to deliver genetic information. Whilst research into mRNA has been ongoing for decade, interest has sparked in recent years and research has begun to expand into areas of vaccinology, infectious diseases and other indications.

RNA 52

RNA Protein Vaccination Vaccine 52

Drug Discovery World

OCTOBER 12, 2023

When it comes to the technologies driving drug discovery, she thinks that the ability to specifically edit regions of the genome using CRISPR/Cas9 is particularly revolutionary. With transformer and large language models (LLMs), AI is assisting bioinformaticians, as well as bench-based researchers.

Drugs 59

Drugs Life Science Genome Genomics 59

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. This technology has powerful implications for therapeutic uses, such as replacing mutated or disease-causing genes or increasing the activity of cancer-fighting cells.

Gene Editing 98

Gene Editing DNA Gene Genome 98

The Pharma Data

OCTOBER 27, 2020

.” The single-reaction panel targets five regions of the viral genome and provides excellent accuracy and sensitivity. “Most real time qPCR-based assays target only two or three targets on the viral genome.” “Most real time qPCR-based assays target only two or three targets on the viral genome.”

Genetic Analysis 52

Genetic Analysis Genome Genomics Reagent 52

pharmaphorum

APRIL 20, 2022

As is typical, the research built on work going back years prior, specifically to 1987 when the first CRISPR mechanism was identified in E. coli, and their research in turn formed the foundation for the CRISPR-based therapies being explored to create new treatment options. Why cancer? The pipeline.

Gene Editing 52

Gene Editing DNA Gene Genetics 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. The research was published earlier this month in the journal Cell. pyogenes dCas9.

DNA 52

DNA Gene Gene Silencing Genome 52

The Pharma Data

OCTOBER 28, 2020

The maternal-specific inheritance pattern of Angelman syndrome is due to genomic imprinting of UBE3A in neurons of the central nervous system, a naturally occurring phenomenon in which the maternal UBE3A allele is expressed and the paternal UBE3A is not. About Ultragenyx.

Protein 40

Protein Production Development Drugs 40