STAT News

MARCH 3, 2023

Next week, hundreds of scientists from around the world will convene in London for an international summit on genome editing. That technology, which enables scientists to easily excise, alter, or replace specific sections of DNA, was awarded the 2020 Nobel Prize for Chemistry. Continue to STAT+ to read the full story…

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Genome Genomics Gene Editing Genetic Disease 111

Scienmag

MAY 2, 2022

WINSTON-SALEM, NC – May 2, 2022 — Wake Forest Institute for Regenerative Medicine (WFIRM) scientists working on CRISPR/Cas9-mediated gene editing technology have developed a method to increase efficiency of editing while minimizing DNA deletion sizes, a key step toward developing gene editing therapies to treat genetic diseases.

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Drug Discovery World

APRIL 17, 2024

DDW’s Diana Spencer speaks to Josh Mandel-Brehm , CEO of Massachusetts-based biotech CAMP4, to understand the role regulatory RNAs play in gene expression and how the founders overcame the challenge of launching a business based on brand new science. What are the advantages compared to other gene modulating approaches?

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Drug Discovery World

JANUARY 11, 2024

Researchers have used messenger RNA (mRNA) to create an effective therapy for a rare liver disease in preclinical studies, demonstrating the technology’s potential therapeutic use in people. Successful industry/academia collaboration Fewer than 5% of rare diseases have approved therapies and most of these treatments use gene therapy.

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Drug Discovery World

NOVEMBER 7, 2022

WGS and Dante’s genomic reports will provide information about predisposition to genetic diseases, genetic carrier information and more. In April 2022, PacBio expanded a research collaboration with Children’s Mercy Kansas City in an effort to improve scientists’ understanding of the genetic underpinnings of rare diseases.

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Genome Genomics Development Genetics 98

Scienmag

FEBRUARY 3, 2021

Scientists illuminate the protein’s role in rare genetic diseases often diagnosed during infancy or childhood Scientists at Scripps Research have clarified the workings of a mysterious protein called G?o, o, which is one of the most abundant proteins in the brain and, when mutated, causes severe movement disorders.

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Protein Genetic Disease Genetics Scientist 42

Delveinsight

FEBRUARY 11, 2021

The scientists analyzed samples from patients with the most common pancreatic cancer, pancreatic ductal adenocarcinoma (PDAC). Avrobio gene therapy eradicates toxic substrate in Fabry patient. Late in 2019, a Fabry patient in phase 2 clinical trial of ex vivo lentiviral gene therapy AVR-RD-01 became the first patient dosed using Plato.

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Drug Discovery World

AUGUST 28, 2023

DS: What role does genetics play in autophagy? Could therapies like these treat rare genetic conditions? PH : Genetics plays an enormous role. There are many mutations in specific genes that have been shown to disrupt the autophagy process and lead to disease. Could it be possible to prevent genetic diseases?

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Drug Discovery World

JANUARY 24, 2023

DMD and FA are rare, degenerative, genetic diseases that affect movement and eventually lead to paralysis.

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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 87

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 76

XTalks

NOVEMBER 10, 2020

Expeditious and accurate diagnoses are necessary for patients to access healthcare services and treatment options for rare genetic diseases. Increasing the efficiency of case analysis and interpretation is essential to providing timely care for patients with genetic diseases.

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Genome Genomics Medicine Genetic Disease 98

XTalks

MARCH 3, 2021

The US Food and Drug Administration (FDA) has granted approval to Nulibry (fosdenopterin) for reducing the risk of death associated with molybdenum cofactor deficiency type A (MoCD Type A), making it the first treatment for the ultra-rare, genetic metabolic disorder. The approval was granted to BridgeBio Pharma, Inc.

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The Pharma Data

OCTOBER 18, 2020

Telomeres degrade and shorten with age and can become excessively damaged in certain genetic diseases, as well as from lifestyle factors such as smoking, poor diet, and chronic stress. Shortening of telomeres is associated with the symptoms of aging, heart disease, DNA damage and uncontrolled cell replication, which can lead to cancer.

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The Pharma Data

DECEMBER 20, 2020

Klisyri is a home-grown product discovered and characterized by Athenex scientists and developed from pre-IND to NDA by the Athenex team. CF is a rare, life-shortening genetic disease that affects the lungs, liver, GI tract, sinuses, sweat glands, pancreas and reproductive tract.

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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.

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Genetics Genetic Analysis Vaccination Vaccine 52

pharmaphorum

JANUARY 27, 2023

In the morning, I can be talking about gene therapy for hearing loss, for example, [and] in the afternoon, I can be talking about the treatment of a very specific tumour. million people in the UK that have a rare disease,” she continued, “and that’s where a lot of the innovation is, in more of these rare genetic diseases.

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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. It’s a great tool for controlling gene expression.”.

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XTalks

AUGUST 29, 2022

Now a common gene editing tool, the popularity of the CRISPR-Cas9 system has increased over the past decade. CRISPR is notable for engineering living cells, allowing scientists to edit, turn off, delete, or replace genes in a cell’s genome. From the Bench to Patient Therapeutics.

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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. Proteins perform myriad functions within the human body and scientists have estimated that there are at least 20,000 canonical proteins that we know about.

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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. These modifications regulate gene expression without altering the sequence or structure of DNA.

DNA 52

DNA Gene Gene Silencing Genome 52

Drug Discovery World

OCTOBER 12, 2023

New approaches and thought processes When it comes to diversity in drug discovery, this includes our scientists’ and pharma workforce’s approach to innovation which will help advance the industry. Dr Michelle Fraser is Head of Cell & Gene Therapy at global health science solutions company, Revvity.

Drugs 59

Drugs Life Science Genome Genomics 59

The Pharma Data

OCTOBER 15, 2020

Axovant – Kristin Vuori was named to the board of directors at Axovant Gene Therapies Ltd. F4 Pharma — In order to help F4 Pharma advance its COVID-19 asset FX06 and to provide strategic guidance, F4 Pharma has appointed a scientific advisory board consisting of clinicians and scientists. Additionally, Parag V.

In-Vivo 52

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Drug Discovery World

FEBRUARY 22, 2024

For diseases as serious and debilitating for patients and their families as leukodystrophies, learning about such advances in knowledge carries a great deal of hope, which IRCM warmly welcomes,” adds Dr Jean-François Côté, the IRCM’s President and Scientific Director.

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