Scienmag

JULY 31, 2021

Research from Wellesley College shows that despite being a clonal insect species, weevils use gene regulation to adapt to new food sources and pass down epigenetic changes to future generations Without the benefits of evolutionary genetic variation that accompany meiotic reproduction, how does an asexual invasive species adapt over time to a new environment (..)

Genetics 111

Genetics Gene Regulation Research 111

Pharmaceutical Technology

MAY 24, 2023

By Luisa Sterkel & Joana Loureiro , Tenthpin Consultants The promise and potential of cell and gene therapies (CGT) has emerged in the recent past and currently over 1.500 CGT are registered for clinical trials holding great hope for the treatment of challenging and uncurable diseases. plan a, plan b, … charter flight standby option).

Clinical Trials 246

Clinical Trials Clinical Trials Gene Therapy Clinical Supply Chain 246

BioPharma Reporter

NOVEMBER 19, 2024

PTC Therapeutics has received FDA approval for a gene therapy to treat AADC deficiency, a rare genetic disorder that impairs the normal function of neurons.

Gene Therapy 114

Gene Therapy FDA Approval Gene Genetics 114

Medical Xpress

FEBRUARY 13, 2023

UT Southwestern Medical Center researchers have mapped gene control elements in specialized cardiac cells responsible for coordinating heartbeats.

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Regulation Genetics Research Genome 75

XTalks

JULY 14, 2022

Every gene in a cell is expressed at exact levels due to complex gene regulatory networks. The connections between regulatory genes and their downstream targets have been mapped to some extent — they can be thought of as a subway or metro map, with connections existing between major “hubs.”

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Gene Regulation Protein Gene Editing 98

Pharmaceutical Technology

MAY 24, 2023

It can correct several disease elements associated with PKD, a rare, monogenic red blood cell disorder caused by a mutation in the PKLR gene. It can correct several disease elements associated with PKD, a rare, monogenic red blood cell disorder caused by a mutation in the PKLR gene.

Gene Therapy 130

Gene Therapy Gene Licensing Licensing 130

Scienmag

MAY 5, 2021

Researchers have mapped the gene activity of osteocytes to improve their understanding of skeletal disease Credit: Garvan Institute of Medical Research Research led by the Garvan Institute of Medical Research has for the first time mapped the unique genetic profile of the skeleton’s ‘master regulator’ cells, known as osteocytes.

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Gene Genetics Regulation Research 98

Pharmaceutical Technology

MAY 4, 2023

The approval allows KALYDECO to be used in infants who have at least one mutation in their cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to the therapy, on the basis of clinical and/or in vitro assay results.

In-Vitro 246

In-Vitro Genetic Disease Medicine Genetics 246

Camargo

MAY 2, 2021

Advances in scientific knowledge and growth in the cell and gene therapy space have led to a new and exciting era of medicine for patients, as well as a new motivation for regulators to provide clear, efficient pathways for product developers. Background: The Advancement of Cell and Gene Therapies.

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Gene Therapy Gene Packaging Packaging 155

BioPharma Reporter

AUGUST 18, 2022

Food and Drug Administration (FDA) has approved bluebird bioâs Zynteglo (betibeglogene autotemcel), also known as beti-cel: a one-time gene therapy custom-designed to treat the underlying genetic cause of beta thalassemia in adult and pediatric patients who require regular red blood cell (RBC) transfusions.

Gene Therapy 105

Gene Therapy Gene FDA Approval Genetics 105

BioPharma Reporter

NOVEMBER 19, 2024

PTC Therapeutics has received FDA approval for a gene therapy to treat AADC deficiency, a rare genetic disorder that impairs the normal function of neurons.

Gene Therapy 99

Gene Therapy FDA Approval Gene Genetics 99

pharmaphorum

AUGUST 23, 2022

France’s BrainVectis, a subsidiary of Bayer’s Asklepios BioPharma (AskBio) unit, has been given the green light by regulators in France to start dosing patients with its gene therapy candidate for devastating neurodegenerative disorder Huntington’s disease.

Gene Therapy 105

Gene Therapy Gene Trials Protein 105

Medical Xpress

MAY 4, 2023

Australian researchers have identified the role that two key genes associated with age-related macular degeneration (AMD) play in the disease.

Genetics 97

Genetics Gene Regulation Research 97

BioPharma Reporter

SEPTEMBER 23, 2021

This month sees the launch of a new gene therapy player, Opus Genetics, a company backed and spun out by leading patient group Foundation Fighting Blindnessâ venture arm, the Retinal Degeneration Fund (RD Fund).

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Gene Therapy Gene Genetics Regulation 95

Scienmag

OCTOBER 27, 2021

The rapid scientific advancements that followed the mapping of the human genome have revealed just how staggeringly complex the world of genetics is. We now know that proteins are not just the products of genes, but that they also interact with genes, influencing and regulating the rhythm of their expression.

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Gene Genome Genomics Genetics 72

pharmaphorum

FEBRUARY 11, 2022

Data from the first patients enrolled into Regenxbio’s trial of its gene therapy for rare inherited disease mucopolysaccharidosis type I (MPS I) – also known as Hurler syndrome – has shown the first signs of clinical activity. The post Regenxbio expands MPS I gene therapy trial after first look at data appeared first on.

Gene Therapy 111

Gene Therapy Gene Trials FDA Approval 111

Scienmag

JULY 19, 2022

A key genetic mutation that occurs early on in cancer alters RNA “dark matter” and causes the release of previously unknown RNA biomarkers for cancer early detection, a new study by UC Santa Cruz researchers published in the journal Cell Reports shows. Kim A key genetic mutation that occurs early on in […]. Credit: Daniel H.

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RNA Regulation Gene Genetics 64

BioPharma Reporter

DECEMBER 13, 2022

Annogen has begun a research project with Pfizer to functionally test tens of thousands of disease-related non-coding sequence variants for their effect on gene regulation.

Genetics 89

Genetics Research Gene Regulation 89

Scienmag

SEPTEMBER 22, 2020

Brazilian researchers observed that in uninfected adipocytes, the hormone irisin altered the expression of genes that regulate ACE-2, which encodes a protein to which the virus binds in order to invade human cells.

Hormones 97

Hormones Gene Protein Regulation 97

pharmaphorum

SEPTEMBER 6, 2021

BioMarin Pharma has had another setback in its gene therapy development programme, announcing this morning that the FDA has placed a phase 1/2 trial of its candidate for phenylketonuria (PKU) on hold while it investigates a safety signal.

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Gene Therapy Gene Genetic Disease DNA 94

FDA Law Blog

DECEMBER 7, 2022

Valentine — On November 22, 2022, FDA approved CSL Behring’s BLA for Hemgenix (etranacogene dezaparvovec), an AAV-based gene therapy for the treatment of adults with Hemophilia B who currently use Factor IX prophylaxis therapy, have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes.

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Gene Therapy Gene FDA Approval Production 119

XTalks

FEBRUARY 21, 2025

Current Share Price: PWS is a complex, genetically determined neurodevelopmental disorder that affects multiple organ systems. And hyperphagia is common not only to PWS but also to hypothalamic obesity, binge-eating disorder and certain rare genetic disorders like Bardet-Biedl syndrome.

Genetics 59

Genetics Hormones Gene Expression Gene 59

BioPharma Reporter

SEPTEMBER 3, 2020

AskBio, which develops adeno-associated virus (AAV) gene therapies for genetic disorders, has won an R&D grant valued at Â2m (around US$2.7m) from Scottish Enterprise.

Gene Therapy 74

Gene Therapy Gene Genetics Development 74

Medical Xpress

APRIL 7, 2023

A team of scientists at the Medical University of South Carolina (MUSC) has identified a stress-regulated gene that plays a role in the link between long-term stress and a common type of depressive behavior in mice.

Genetics 75

Genetics Gene Scientist Regulation 75

XTalks

APRIL 8, 2025

There are over 10,000 rare diseases affecting an estimated 300 million people worldwide where 80% are genetic , 95% lack approved treatments and nearly half begin in childhood. Cross-border enrollment captures a wider range of genetic backgrounds, environmental influences, and cultural perspectives.

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Clinical Trials Clinical Trials Trials Gene Therapy 88

Medical Xpress

JANUARY 3, 2023

Alcohol exposure in early pregnancy can change gene function during the tightly regulated embryonic development, and consequently cause developmental disorders—especially neurodevelopmental disorders.

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Gene Development Regulation Genetics 81

BioPharma Reporter

JANUARY 2, 2023

Last week saw Opus Genetics announce it had acquired the rights to two preclinical-stage adeno-associated virus (AAV)-based gene therapy product candidates for inherited retinal diseases (IRDs) from Iveric Bio.

Gene Therapy 118

Gene Therapy Genetics Gene Production 118

Scienmag

FEBRUARY 24, 2021

According to a new study published in The American Journal of Human Genetics, more than one third of genetic variants that increase the risk of coronary artery disease regulate the expression of genes in the liver. Credit: UEF/Raija Törrönen.

Gene 52

Gene Genetics Regulation Cardiology 52

Pharmaceutical Technology

MAY 22, 2023

Vyjuvek is a non-invasive, topical, re-dosable gene therapy that delivers functional human COL7A1 gene copies to offer wound healing. We offer our sincere gratitude to DEB patients, caregivers, investigators, US regulators and our employees who made this approval possible.

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FDA Approval Gene Therapy Genetic Disease Gene 130

BioPharma Reporter

FEBRUARY 27, 2023

AbbVie and Capsida Biotherapeutics have expanded their strategic collaboration to develop genetic medicines for eye diseases with high unmet need.

Gene Therapy 97

Gene Therapy Gene Genetics Medicine 97

Advarra

JUNE 12, 2023

Gene therapy research is exciting and full of promise, but because of the risks involved, it’s also highly regulated, requiring an institutional biosafety committee (IBC) to provide additional oversight and risk assessment. What do the Regulations Say about IBC Review? What Does an IBC Review? How is IBC Membership Composed?

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Genetic Engineering Gene Therapy Genetics Engineer 98

STAT News

MARCH 6, 2023

LONDON — The first gene-edited children were born in China five years ago , but it’s unlikely to happen again there anytime soon. That was the message Chinese scientists delivered Monday on the opening day of the Third International Summit on Human Genome Editing in London. Continue to STAT+ to read the full story…

Genomics 98

Genomics Genome Gene Editing Genetics 98

Pharmaceutical Technology

JANUARY 22, 2023

One of these is MCT8 deficiency, a condition caused by a mutation in the MCT8 gene, which affects one in 700,000 males. T3 is a major hormone in the blood that regulates thyroid levels. In 2023, Sarepta Therapeutics has a PDUFA date for the first gene transfer DMD therapy, delandistrogene moxeparvovec (SRP 9001), on May 29.

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Drugs Gene Hormones Clinical Trials 317

Scienmag

MAY 7, 2021

The two meters of -stretched- DNA contained in human cells are continuously twisting and untwisting to give access to genetic information: when a gene is expressed to generate a protein, the two strands of DNA are separated to give access to all the machinery necessary for this expression, resulting in an excessive accumulation of coiling […]. (..)

DNA 62

DNA Regulation Genome Genomics 62

BioPharma Reporter

FEBRUARY 27, 2023

AbbVie and Capsida Biotherapeutics have expanded their strategic collaboration to develop genetic medicines for eye diseases with high unmet need.

Gene Therapy 95

Gene Therapy Gene Genetics Medicine 95

Medical Xpress

MARCH 1, 2023

Epigenetic changes in the regulation of a key gene in the body's stress response system were detected in babies and young children with abusive injuries, as opposed to accidental, according to a pilot study published in the journal Pediatric Research.

Gene 75

Gene Regulation Research Genetics 75

pharmaphorum

MARCH 10, 2021

bluebird bio is to ask regulators to restart clinical studies of its LentiGlobin for sickle cell disease, after an investigation concluded that a case of acute myeloid leukaemia (AML) was “very unlikely” to be caused by the gene therapy. bluebird bio’s Philip Gregory.

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Gene Therapy Gene Trials Gene Expression 59