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.

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Worldwide Clinical Trials

JANUARY 24, 2024

Much of what we discuss I covered in my presentation, “Leveraging Genetics to Support Rare Disease Clinical Trials,” at last year’s World Orphan Drug Congress (WODC) EU. Newborn Sequencing is a research initiative that allows for a baby’s full genome to be sequenced at birth, diagnosing their genetic diseases.

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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 (..)

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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.”

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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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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. .

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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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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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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?

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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.

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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. 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.

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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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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

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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. Such a precise prediction allows clinicians to run clinical trials more efficiently so that patients can access novel therapies quicker, and also help dose drugs more precisely.

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Drug Discovery World

AUGUST 28, 2023

We have a combination of talented and motivated young scientists along with experienced industry experts who have been through the process of drug research and development. Could it be possible to prevent genetic diseases? What advice do you have for other start-ups hoping to launch in a similar field?

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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.

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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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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.

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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.

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XTalks

MARCH 3, 2021

BridgeBio is dedicated to developing therapies for genetic diseases with unmet needs. The FDA reviewed Nulibry under Priority Review and received Orphan Drug Designation, Breakthrough Therapy Designation and Rare Pediatric Disease Designation from the agency. The approval was granted to BridgeBio Pharma, Inc.

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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.

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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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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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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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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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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

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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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Drug Discovery World

APRIL 30, 2024

mRNA exists in every cell within the human body and this, along with its role in the development of proteins, are the reasons why mRNA therapies have such potential in treating many diseases. We believe it can now do the same for rare diseases.” “We

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XTalks

MAY 4, 2021

While epigenetic therapies such as DNA methyltransferase (DNMT) inhibitors like azacitadine are used as hypomethylating agents in the treatment of diseases like myelodysplastic syndrome and sometimes in AML, CRISPRoff is the first molecular-based epigenetic tool that could modulate the epigenome to treat disease.

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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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XTalks

MAY 4, 2021

While epigenetic therapies such as DNA methyltransferase (DNMT) inhibitors like azacitadine are used as hypomethylating agents in the treatment of diseases like myelodysplastic syndrome and sometimes in AML, CRISPRoff is the first molecular-based epigenetic tool that could modulate the epigenome to treat disease.

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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.

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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.

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

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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.

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