Drug Discovery World

AUGUST 23, 2022

A Phase I/II clinical trial targeting a treatment for Huntington’s Disease (HD) using a gene therapy has been given the clearance to go ahead in France. . The disease is caused by anomalous repeating mutations in the huntingtin gene leading to abnormal protein aggregates in nerve cells.

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Drug Discovery World

SEPTEMBER 4, 2023

In the third and final part of this series of articles focused on plant-based medicines, DDW’s Megan Thomas evaluates therapeutic breakthroughs using plant-based products in drug discovery. In this study, researchers discovered how EGCG breaks apart tangles of the protein tau, a ‘hallmark’ of Alzheimer’s disease.

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Worldwide Clinical Trials

MAY 30, 2024

Authors: Rich Worldwide Clinical Trials Exec. This trend is on the rise despite recent disappointments with clinical trial outcomes, which have the potential to destabilize the industry in the short term regarding drug development strategy and optimal study designs. Director, Therapeutic Area Medical Lead. The MAA file in the E.U.

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XTalks

DECEMBER 12, 2023

The world of neuroscience trials is rapidly evolving, presenting new challenges and opportunities for researchers and clinicians. The worldwide market for neurology clinical trials stood at a valuation of $5.24 There is currently a heightened emphasis on imaging and liquid biomarkers in neuroscience clinical trials.

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Drug Discovery World

AUGUST 16, 2023

A new study has shed light on the genetic messages encoded by genes within ‘triple negative’ breast cancers (TNBC), and shows they could predict response to chemotherapy. The presence of these biomarkers could be used to accelerate the development of more personalised treatments for triple negative breast cancers.

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Pharmaceutical Technology

OCTOBER 18, 2022

Last week, Nature magazine published a paper detailing an in-situ study of Parkinson’s disease (PD), showing protein structural changes in cerebrospinal fluid (CSF) between healthy individuals and Parkinson’s patients. Genetic biomarkers are being investigated to aid in the development of precision medicine for PD.

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pharmaphorum

APRIL 23, 2021

Vertex Pharma has ramped up its involvement in gene-editing medicines for the second time in a week, paying Obsidian Therapeutics $75 million upfront to access its technology platform. . For example, adding a small-molecule might stabilise the medicine, allowing levels of the target protein to increase.

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FDA Law Blog

DECEMBER 13, 2023

Sasinowski — On December 12, 2023, FDA announced the creation of a new advisory committee specifically for treatments for genetic metabolic diseases, the Genetic Metabolic Diseases Advisory Committee, or “GeMDAC.” There are hundreds of known genetic metabolic diseases, most of which are rare and carry significant morbidity.

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XTalks

FEBRUARY 19, 2024

In a recent study published by Weill Cornell Medicine in the Journal of Infectious Diseases , researchers have reported the potential of Moderna’s experimental mRNA vaccine in protecting adults against CMV, which could potentially also stop the transmission of the virus from mothers to their infants during pregnancy.

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The Pharma Data

JULY 8, 2021

Today, access to the world’s largest browsable resource linking rare protein-coding genetic variants to human health and disease was launched through a genetic exome sequence analysis collaboration between AbbVie (NYSE: ABBV), Biogen Inc. Nasdaq: BIIB) and Pfizer (NYSE: PFE). The browser can be accessed via [link]. About AbbVie.

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XTalks

OCTOBER 20, 2023

Clinical-stage genome editing company Intellia Therapeutics has received clearance from the US Food and Drug Administration (FDA) for its Investigational New Drug (IND) application to start a pivotal phase III trial of NTLA-2001 for the treatment of transthyretin (ATTR) amyloidosis with cardiomyopathy.

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pharmaphorum

SEPTEMBER 14, 2020

Last week geneticist Dr Charles Steward shared with us his experiences of searching for a genetic cause for his children’s rare neurological diseases. Here he gives us a deeper look at how genomic medicine is evolving and the barriers that are preventing it from reaching its full potential.

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pharmaphorum

JUNE 28, 2021

Preliminary results from a phase 1 trial run by Regeneron and CRISPR specialist Intellia Therapeutics – co-founded by Nobel Prize winner Jennifer Doudna – showed steep reductions in a biomarker of ATTR amyloidosis disease activity with a single dose of the NTLA-2001 drug. — Eric Topol (@EricTopol) June 26, 2021.

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pharmaphorum

SEPTEMBER 14, 2020

AstraZeneca has resumed UK trials for its coronavirus vaccine, after the country’s medicines regulator gave the all-clear following a safety scare. A UK safety committee has concluded its investigations and recommended to the country’s Medicines and Healthcare products Regulatory Agency (MHRA) that trials are safe to resume.

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Advarra

JANUARY 11, 2024

Adoptive T Cell therapies, therapeutic antibodies, and immunomodulatory proteins represent just some of the potentially beneficial treatment strategies for successful mRNA cancer trials. One strategy proposed the use of synthetic mRNA or modified mRNA analogs, which can enhance the stability and protein expression of mRNA.

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Drug Discovery World

OCTOBER 6, 2022

Precision medicine promises a paradigm shift to confidently match the right patients to the right treatment at the right time. In the cancer space alone over the last five years, clinical trials have increased 500% for new drugs and new drug combinations. In pursuit of this, the number of new therapeutics in development has boomed.

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Pharmaceutical Technology

JULY 5, 2022

Researchers at the UNC School of Medicine have discovered that hepatitis A virus (HAV) replication needs particular interactions between the human protein ZCCHC14 and TENT4 poly(A) polymerases, a group of enzymes. In addition, the scientists later found that the HAV needs TENT4A/B for its replication.

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Drug Discovery World

DECEMBER 9, 2022

A new type of targeted medicine has shown ‘remarkable’ benefits for patients with advanced breast cancer in a major Phase III clinical trial. . Capivasertib is a potential first-in-class drug that blocks activity of the cancer-driving protein molecule AKT. The CAPItello-291 trial. Genetic alterations of the AKT pathway.

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The Pharma Data

SEPTEMBER 14, 2020

. AstraZeneca has confirmed that clinical trials assessing its Oxford University partnered coronavirus vaccine AZD1222 have resumed in the UK, following a green light by the Medicines Health Regulatory Authority (MHRA). Source link.

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Pharmaceutical Technology

AUGUST 5, 2022

As per the prevailing prion theory, a misfolded version of a normal cell-surface protein acts as the chief infectious agent. It is ultra-rare,” says Jesús Rodriguez Requena, PhD, associate professor in the Department of Medicine at the University of Santiago de Compostela. And then comes the question of the right target.

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Drug Discovery World

NOVEMBER 14, 2023

A Phase Ib trial has shown low-density lipoprotein cholesterol (LDL-C) reductions up to 55% and blood PCSK9 protein reductions up to 84% after a single infusion of Verve Therapeutics’ Verve-101. FH is one of the most common genetic conditions, affecting around one in 300 people globally.

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Drug Discovery World

JANUARY 9, 2024

On Monday 5 February at 3.30pm, he will speak on ‘ Proximity-inducing drugs: using technological innovation to build the next generation of medicine. Simon Bushell DDW is delighted to announce that the programme will feature Simon Bushell, SVP of Molecular Discovery of Entact Bio.

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Drug Discovery World

FEBRUARY 21, 2024

In October 2023, pharmaceutical company NKGen Biotech released data from its Phase I clinical trial on the use of its investigational natural killer therapy, SNK01, to treat patients with Alzheimer’s disease. This is done without any genetic modification. RA: What are some of the unmet needs in current treatments for Alzheimer’s?

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Drug Discovery World

APRIL 3, 2024

In the last few years, immunotherapies like immune checkpoint inhibitors, vaccines and cell and gene therapies have revolutionised the treatment of cancer, and targeted drugs like monoclonal antibodies (mAbs), poly-ADP ribose polymerase (PARP) inhibitors and targeted protein degraders are also playing an important role.

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Drug Discovery World

JUNE 23, 2023

Elevidys addresses the root genetic cause of Duchenne – mutations in the dystrophin gene that result in the lack of dystrophin protein – by delivering a gene that codes for a shortened form of dystrophin to muscle cells known as Elevidys micro-dystrophin.

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XTalks

MAY 26, 2023

DEB is a genetic disorder characterized by very fragile skin that rips and blisters easily even from minor friction (like rubbing or scratching) or injury, resulting in open wounds that are prone to skin infections and fibrosis. Results of the trials are published in the journals Nature Medicine and The New England Journal of Medicine.

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Pharmaceutical Technology

JUNE 29, 2022

The next wave of medicine is well on course to be cell and gene-based. In September 2021, GlobalData figures revealed there to be 1,320 industry-sponsored regenerative medicine and advanced therapy trials ongoing worldwide. Meanwhile, the pharma industry is undergoing somewhat of a transformation itself.

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Drug Discovery World

DECEMBER 19, 2022

A double-blind, placebo-controlled clinical trial of a gene therapy gel developed at Stanford Medicine shows improved wound healing in 31 people with the disease, including 19 who were 18 years old or younger. . One trial participant was Vincenzo Mascoli, aged 22.

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The Pharma Data

JANUARY 10, 2021

11, 2021 (GLOBE NEWSWIRE) — Editas Medicine, Inc. Food and Drug Administration (FDA) has cleared the initiation of the safety phase of the Company’s EDIT-301 clinical trial, and the Company can begin dosing patients. EDIT-301 is the first experimental medicine in development generated using CRISPR/Cas12a gene editing. “The

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The Pharma Data

DECEMBER 3, 2020

New Tau Protein is Abnormal in Very Early Alzheimer’s Disease. Investigators with the University of Gothenburg identified new forms of tau protein that become abnormal in the very early stages of Alzheimer’s disease, even before the development of cognitive problems. “The Every week there are numerous scientific studies published.

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Drug Discovery World

NOVEMBER 27, 2023

In the UK, following Medicines and Healthcare products Regulatory Agency (MHRA) approval on 15 November 2023, and as a result of the existing reimbursement agreement between Vertex and the NHS, children aged two years and above in the UK have access to this expanded indication for Kaftrio in a combination regimen with ivacaftor.

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The Pharma Data

DECEMBER 16, 2020

Currently in Phase 3 clinical testing in the United Kingdom for the prevention of COVID-19, NVX-CoV2373 is a recombinant protein vaccine adjuvanted with Novavax’ proprietary Matrix-M to enhance the immune response. NVX-CoV2373 contains purified protein antigen and can neither replicate, nor can it cause COVID-19. About NVX-CoV2373.

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Drug Discovery World

SEPTEMBER 13, 2023

A Translational Psychiatry publication indicates that SARS-CoV-2 infection, even when unnoticed or asymptomatic, can have an influence on the activation of the Human Endogenous Retrovirus W (W-ENV) protein in psychotic patients, in a context of innate immune inflammation.

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XTalks

APRIL 3, 2024

The RBCs in circulation are marked by proteins in the complement system for removal by the spleen and liver. Voydeya’s FDA approval was based on positive results from the pivotal ALPHA Phase III trial. In the trial, the most common adverse events were headache, nausea, arthralgia (joint stiffness) and diarrhea.

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The Pharma Data

DECEMBER 3, 2020

Dravet syndrome is a severe and progressive genetic epilepsy characterized by frequent, prolonged and refractory seizures that usually begin within the first year of life. Our goal is to develop a medicine that goes beyond seizure control to address many of the other comorbidities associated with Dravet syndrome. Clinical Trials.

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pharmaphorum

JULY 20, 2022

Armed with a $100 million second-round financing, CAMP4 Therapeutics is preparing to start the first clinical trial of a drug targeting regulatory RNA (regRNA) molecules that can be used to fine-tune the expression of genes. ” The post CAMP4 raises $100m to take lead RNA drugs into clinic appeared first on. .

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pharmaphorum

JANUARY 10, 2022

The opt-in has been prompted by the results of a phase 2 trial of ensovibep (MPO420), which showed that a single intravenous dose of the drug was able to not only reduce viral load in non-hospitalised COVID-19 patients over eight days, but also cut the risk of hospitalisation or death by 78% versus placebo.

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