Medical Xpress

MAY 16, 2023

Sometimes scientists arrive at new findings and discoveries by chance. They study gene variation and mutations that cause rare genetic diseases. A mutation is a permanent change in the genetic material. A mutation is a permanent change in the genetic material.

In-Vitro 98

In-Vitro In-Vivo Genetic Disease Genetics 98

STAT News

MARCH 1, 2023

Tessera Therapeutics chief scientist Michael Holmes had just finished presenting a much-anticipated peek at a technology the company had previously said could “ revolutionize genetic medicine ” and “cure nearly any genetic disease.”

Gene Editing 119

Gene Editing Genetic Disease Genome Genomics 119

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…

Genome 111

Genome Genomics Gene Editing Genetic Disease 111

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

Scienmag

SEPTEMBER 29, 2021

JUPITER, FL – The American Chemical Society announced that its prestigious 2022 Nobel Laureate Signature Award for Graduate Education in Chemistry honors a student from the Skaggs Graduate School of Chemical and Biological Sciences at Scripps Research and her faculty advisor, for their pioneering work developing RNA-targeting medications for genetic (..)

RNA 77

RNA Genetic Disease Scientist Genetics 77

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. . It was led by scientists at the MRC-University of Glasgow Centre for Virus Research (CVR) and University of Massachusetts Chan Medical School. .

Gene Therapy 52

Gene Therapy Gene Genetic Disease Genetics 52

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.

Gene Editing 58

Gene Editing DNA Gene Development 58

Drug Discovery World

APRIL 17, 2024

CAMP4 is exploiting these regulatory interactions to specifically control the expression of genes tied to disease. So, regRNAs haven’t been exploited previously because scientists have only recently understood their role in regulating gene expression. What diseases are you currently targeting? Can you give some examples?

Gene Expression 59

Gene Expression Gene Genetic Disease RNA 59

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.

Protein 42

Protein Genetic Disease Genetics Scientist 42

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.

Genome 98

Genome Genomics Development Genetics 98

Drug Discovery World

NOVEMBER 7, 2022

In 2018, the UK’s department of health announced an NHS Genomic Medicine Service, which allows patients with rare diseases to have their entire genetic code read in the hope of providing a much-needed diagnosis. That said, some people with rare genetic diseases remain without a molecular diagnosis after their genome is analysed. . “We

Genome 52

Genome Genomics Genetics Genetic Disease 52

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. By protecting the mRNA in a microdroplet of lipids, scientists were able to inject the mice intravenously with the therapy and target their liver cells.

Research 59

Research Gene Therapy RNA Gene 59

Drug Discovery World

JUNE 19, 2023

Cystic fibrosis is a progressive genetic disease that affects the lungs, pancreas and other organs. AdBio Partners’ mission is to support founding scientists in the development of new therapeutic approaches,” said Alain Huriez, Managing Partner of AdBio Partners.

Development 52

Development Life Science In-Vivo In-Vitro 52

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?

Genetics 52

Genetics Protein Genetic Disease Clinical Trials 52

Drug Discovery World

JANUARY 24, 2023

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

Development 52

Development Clinical Trials Clinical Trials Drugs 52

Pharma Mirror

APRIL 21, 2024

This represents an increasingly promising field of precision medicine, holding significant prospects for preventing and treating numerous challenging or genetic diseases. Scientists have been striving to optimize mRNA stability, immunogenicity, translation efficiency, and delivery systems to achieve efficient and safe mRNA delivery.

In-Vitro 130

In-Vitro Drugs Genetic Disease RNA 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 83

Genome Genomics Genetics Genome Project 83

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 66

Genome Genomics Genetics Genome Project 66

Delveinsight

FEBRUARY 11, 2021

The scientists analyzed samples from patients with the most common pancreatic cancer, pancreatic ductal adenocarcinoma (PDAC). The observations published in Science Immunology could inform the development of therapies that target IL-17 signaling for chronic inflammatory diseases as per the researchers.

Gene Therapy 52

Gene Therapy Gene Immune Response In-Vivo 52

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.

DNA 52

DNA Scientist Genetic Disease Research 52

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.

FDA Approval 64

FDA Approval Genotype Clinical Trials Clinical Trials 64

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.

Genome 98

Genome Genomics Medicine Genetic Disease 98

XTalks

SEPTEMBER 29, 2021

Some scientists and researchers have been visiting their workplaces during the pandemic. Scientists and researchers have already begun experiencing and living the hybrid model of work that many companies in all fields are now exploring.

Life Science 122

Life Science HR Scientist Genetic Disease 122

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.

In-Vitro 52

In-Vitro FDA Approval Manufacturing Licensing 52

XTalks

OCTOBER 1, 2020

Leaving academia for industry is a route that academic scientists and professors did not traditionally pursue in the past due to perceived differences in skillsets, experiences and even associated stigmas. WeiQi Lin, MD, PhD, executive vice president of research & development and principal scientist, DURECT Corporation.

Scientist 52

Scientist In-Vitro Research Life Science 52

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

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.

RNA 52

RNA Protein Vaccination Vaccine 52

pharmaphorum

JANUARY 27, 2023

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. As an overall term, ‘patient centricity’ itself has undergone a transformation, as well. “We We know that there are 3.5

Clinical Trials 96

Clinical Trials Clinical Trials Trials Development 96

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.

DNA 98

DNA Gene Gene Silencing Genome 98

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. Megan Thomas caught up with those planning to attend this year.

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

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

The Pharma Data

OCTOBER 15, 2020

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. Prior to joining Inozyme, Sabbagh served as the head of rare renal and musculoskeletal diseases research at Sanofi.

In-Vivo 52

In-Vivo Business Development Manufacturing Development 52

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. It can be sedating, which may be undesirable in patients with MS.

Drugs 59

Drugs Gene Clinical Trials Clinical Trials 59

The Pharma Data

NOVEMBER 25, 2020

Ongoing research was put on the backburner and scientists began to focus on understanding the virus and assessing what medications could be used against it. The industry, along with scientists from various government agencies and academic institutions joined together in a united front against the global pandemic.

Vaccination 52

Vaccination Vaccine Genetic Disease Genetics 52