BioPharma Reporter

JUNE 19, 2023

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

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Genetic Disease Genome Genomics Genetics 97

Scienmag

OCTOBER 27, 2020

In the world of rare genetic diseases, exome and genome sequencing are two powerful tools used to make a diagnosis. A recent addition to the toolkit, RNA sequencing, has been demonstrated to help researchers narrow down disease candidate variants identified first on exome and genome sequencing.

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Genetic Disease RNA Genetics Genome 69

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.

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Genome Genomics Genetic Disease 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.

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Gene Editing Gene Genetic Disease Research 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 .

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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).

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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.

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Gene Editing Gene In-Vivo Gene Therapy 147

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’).

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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.

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Development Genome Genomics Drugs 52

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. .

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Gene Editing Gene Genome Genomics 63

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.

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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

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.

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Marketing Gene Sequencing DNA Genome 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.

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Genome Genomics Genetics Genome Project 85

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.

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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 71

Genome Genomics Genetics Genome Project 71

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.

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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.

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Genetic Disease Genetics Genome Genomics 75

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.

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Gene Therapy Gene Genetics Genetic Disease 52

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.

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The Pharma Data

JANUARY 17, 2021

The Undiagnosed Disease Network (a consortium of tertiary care institutions for that purpose) directs a large number of patients to n-Lorem, but most come from physicians. Genetic diseases are vastly more common and more complex than we used to think.”. It has a three-year runway.

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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.

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Gene Therapy Gene Trials Clinical Trials 95

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.

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Development Drugs Genome Genomics 98

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.

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DNA Gene Gene Silencing Genome 98

Drug Discovery World

MARCH 6, 2024

To find new ways of diagnosing and treating complex diseases we first must understand the mechanisms underpinning their key pathological drivers, how these relate to different patient subgroups, and which drugs might be useful in ameliorating their effects – this is the basis of precision neuroscience.

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Genome Genomics Clinical Trials Clinical Trials 64

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.

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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.

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Gene Editing DNA Gene Genome 98

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

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.

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RNA Protein 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 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 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.”

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Drug Discovery World

FEBRUARY 28, 2023

Finally, dosing and timing may be controlled simply by adjusting the amount of mRNA delivered to the cells, rather than relying on inducible systems that are integrated into the genome. She is passionate about advancing research in the pursuit of healthier lives for all.

Genetics 52

Genetics Genetic Analysis Vaccination Vaccine 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.

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Gene Editing DNA Gene Genetics 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.

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