pharmaphorum

AUGUST 10, 2021

Europe market has proved so hostile to gene therapies when it comes to pricing and reimbursement for gene therapies that bluebird bio has decided to quit the market altogether, according to Andrew Obenshain, president of its severe genetic diseases unit. market and will wind down in Europe.

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Gene Therapy Gene Marketing Genetic Disease 135

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).

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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.

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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 Genomics 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.

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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

pharmaphorum

OCTOBER 25, 2022

Astellas has been having some safety-related issues with its gene therapy programmes of late, but it shows no signs of lessened enthusiasm for the category – as a new alliance with US biotech Taysha demonstrates. ” The post Astellas makes another gene therapy play, takes stake in Taysha appeared first on.

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Gene Therapy Gene Genetic Disease Genetics 104

Pharmaceutical Technology

JUNE 4, 2023

In the last three years alone, there have been over 633,000 patents filed and granted in the pharmaceutical industry, according to GlobalData’s report on Innovation in Pharmaceuticals: Transcription factors for genetically modified cells. However, not all innovations are equal and nor do they follow a constant upward trend.

Genetics 130

Genetics Gene Expression DNA Antibody 130

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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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.

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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.

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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.

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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 Genomics Genome 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.

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Gene Therapy Gene Trials FDA Approval 111

XTalks

OCTOBER 8, 2024

The 2024 Nobel Prize in Physiology or Medicine has been awarded to American scientists Victor Ambros and Gary Ruvkun for their groundbreaking discovery of microRNA (or miRNA) and its role in post-transcriptional gene regulation. A gene contains instructions within our DNA. Which means it’s not any show, it’s the show,” he said.

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Regulation Genetics Scientist RNA 52

XTalks

SEPTEMBER 30, 2020

The research shows that the activity of the immune messenger type 1 interferon (IFN) protein is diminished, either by genetic mutations or an autoimmune attack by neutralizing antibodies against it, in a subset of COVID-19 patients. Casanova’s lab is now searching for the genetic driver behind the autoantibodies.

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Antibody Genetics Research In-Vitro 135

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

XTalks

MARCH 28, 2024

Duvyzat, a histone deacetylase (HDAC) inhibitor that works to reduce inflammation and muscle loss, is the first nonsteroidal drug approved to treat patients with all genetic variants of DMD. Progressive muscle weakness in the disease is caused by genetic mutations in the dystrophin gene that lead to a lack of functional dystrophin protein.

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FDA Approval Genetics Gene Therapy Gene 105

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.

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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

APRIL 24, 2023

Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. Eagleton recently spoke on a webinar with his colleagues from Medpace about lessons learned from successful approaches from rare disease and gene therapy product approvals.

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Gene Therapy Gene Trials Clinical Trials 95

Pharmaceutical Technology

FEBRUARY 3, 2023

With scientists fervently developing mRNA vaccines, nucleic acid therapeutics, and viral vector-based gene therapies, clinicians are set to have a growing number of tools available to treat a wide range of conditions, from infectious diseases to genetic disorders and more. So, we set out to solve some of the problems,” she explains.

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Genomics Genome Medicine Gene Therapy 244

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.

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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.

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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

JUNE 28, 2023

Gene therapies for Duchenne muscular dystrophy (DMD) have been an area of intense research and Sarepta’s Elevidys is now the first one to be approved by the US Food and Drug Administration (FDA). DMD is a rare genetic disorder that leads to progressive muscle degeneration and weakness.

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Gene Therapy Gene Protein FDA Approval 98

FDA Law Blog

MARCH 13, 2023

This meeting will host a panel of rare disease patients, caregivers, and advocates to have a focused discussion on the emerging field of gene therapy (regulated at the FDA by OTP). At least some (if not all) of the panelists will have had experience participating in gene therapy trials. One family’s daughter was the first U.S.

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Gene Therapy Gene Trials Clinical Trials 98

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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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.

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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.

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Gene Genetics Regulation Cardiology 52