XTalks

JANUARY 3, 2024

In individuals affected by ATTR, which includes both hereditary and wild-type (non-hereditary) variants, the TTR protein forms fibrils that accumulate in various tissues. Safety and Efficacy of Wainua The FDA’s approval of Wainua is underpinned by compelling clinical data. This conjugation facilitates ASO delivery to hepatocytes.

FDA Approval 105

FDA Approval Protein RNA Clinical Trials 105

Drug Discovery World

SEPTEMBER 4, 2023

In August 2023, a study was conducted by Dr Soriful Islam, and Dr James Segars of the Johns Hopkins University School of Medicine and colleagues which found that in laboratory tests of human fibroid cells, EGCG reduced the amount of a compound that promotes cell division and the quantity of proteins responsible for the tumours’ fibrous contents 1.

Medicine 59

Medicine Protein Containment Drugs 59

pharmaphorum

OCTOBER 2, 2020

Confirming press reports, the EMA said that large scale clinical trials from the vaccine codenamed AZD1222 involving several thousands of people are ongoing and results will become available over “the coming weeks and months”. The post European regulators begin rolling review of AZ’s COVID-19 vaccine appeared first on.

Regulation 100

Regulation Vaccination Vaccine Protein 100

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). The move follows a green light by the UK's MHRA. Source link.

Vaccination 52

Vaccination Vaccine Trials Immune Response 52

XTalks

JUNE 28, 2023

The approval is restricted to ambulatory patients within this narrow age range due to uncertainty around its effectiveness in older children, which Sarepta hopes to clarify in a confirmatory trial. DMD is a rare genetic disorder that leads to progressive muscle degeneration and weakness. It is injected into a patient’s muscle tissue.

Gene Therapy 98

Gene Therapy Gene Protein FDA Approval 98

XTalks

MAY 1, 2023

This makes Qalsody the first approved treatment to target a genetic cause of ALS. Since SOD1 mutations were first identified as a cause of ALS 30 years ago, the familial ALS community has been searching for genetically targeted treatments. To date, over 200 mutations in the SOD1 gene have been associated with the development of ALS.

FDA Approval 89

FDA Approval Gene Genetics Protein 89

The Pharma Data

NOVEMBER 9, 2020

Currently in late-phase clinical development, NVXCoV2373 is a stable, prefusion protein made using Novavax’ nanoparticle technology and includes its proprietary MatrixM adjuvant. Novavax expects to begin its pivotal Phase 3 clinical trial in the United States and Mexico by the end of November. and globally.”.

Vaccination 52

Vaccination Vaccine Clinical Trials Clinical Trials 52

WCG Clinical

NOVEMBER 17, 2023

Although the NIH Guidelines were originally written with non-clinical laboratory research in mind, they also apply to human gene transfer (HGT) research, wherein rsNA or rsNA-containing products are administered to research participants. Since then, however, certain genetic engineering technologies (e.g.,

Genome 52

Genome Genomics Gene Editing Genetic Engineering 52

pharmaphorum

DECEMBER 2, 2021

In September, it reported a fourth patient death in a clinical trial of its AT132 (resamirigene bilparvovec) gene therapy for the rare disease X-linked myotubular myopathy (XLMTM), possibly linked to liver side effects. Those safety issues have hit Astellas’ aspirations in gene therapy directly.

Gene Therapy 79

Gene Therapy Gene Manufacturing Containment 79

The Pharma Data

NOVEMBER 30, 2020

CoV2373 is a stable, prefusion protein antigen derived from the genetic sequence of the SARS-CoV-2 coronavirus spike (S) protein and adjuvanted with Novavax’ proprietary Matrix?M. pivotal Phase 3 trial update. Mexico pivotal Phase 3 trial update. and Mexico pivotal Phase 3 trial and manufacturing scale-up. .

Clinical Development 52

Clinical Development Vaccination Vaccine Development 52

XTalks

NOVEMBER 23, 2020

The global trials are assessing the safety and efficacy of the vaccine in individuals aged 18 years or over from diverse racial and geographic groups who are healthy or have stable underlying medical conditions. Oxford-AstraZeneca COVID-19 Vaccine Induces Immune Responses in Early Clinical Trials.

Vaccination 97

Vaccination Vaccine Clinical Trials Clinical Trials 97

XTalks

SEPTEMBER 8, 2020

AZD1222 is now in Phase III clinical trials which will enroll approximately 30,000 adult volunteers in 80 states in the US, to determine whether the vaccine can help prevent symptomatic coronavirus disease from spreading. The genetic material contains SARS-CoV-2 spike protein.

Manufacturing 83

Manufacturing Vaccination Vaccine Allergies 83

pharmaphorum

SEPTEMBER 1, 2020

AstraZeneca has expanded development of COVID-19 vaccine AZD1222 into the US, beginning a phase 3 clinical trial across all adult age groups. The UK pharma said that the trial will recruit up to 30,000 adults aged 18 years or over to assess the safety, efficacy and immune response.

Vaccination 53

Vaccination Vaccine Trials Immune Response 53

XTalks

JUNE 16, 2021

Novavax shared the latest data from its ongoing late-stage COVID-19 vaccine trial, which shows that its yet-to-be authorized shot provides 100 percent protection against moderate and severe disease and has an overall efficacy of 90.4 Related: Latest Data from Novavax COVID-19 Vaccine Trial Involving B.1.351

Vaccination 98

Vaccination Vaccine Protein Trials 98

The Pharma Data

SEPTEMBER 5, 2021

In clinical trials, Vaxzevria demonstrated 100% efficacy against severe disease and hospitalisation after two doses. After vaccination, the surface spike protein is produced, priming the immune system to attack the SARS-CoV-2 virus if it later infects the body. To date, AstraZeneca and its partners have supplied more than 1.1

Vaccination 52

Vaccination Vaccine Protein Containment 52

pharmaphorum

AUGUST 16, 2020

The order comes after the Commission put agreements in place for the supply of two other vaccines – from Johnson & Johnson and Sanofi/GlaxoSmithKline – that will be activated if the shots prove their worth in clinical trials. Those deals cover 200 million doses of J&J’s Ad26.COV2.S

Vaccination 110

Vaccination Vaccine Immune Response Trials 110

XTalks

MARCH 10, 2023

It is caused by a mutation in the FXN gene , which is responsible for the production of the protein frataxin. Omaveloxolone Clinical Trials Skyclarys is an oral medication that contains omaveloxolone, an activator of the nuclear factor-erythroid 2 related factor 2 (Nrf2) pathway.

FDA Approval 52

FDA Approval Engineer Clinical Trials Clinical Trials 52

The Pharma Data

JANUARY 29, 2021

Additional safety and efficacy data for the vaccine will continue to accumulate from ongoing clinical trials and is expected to be published in the coming weeks. After vaccination, the surface spike protein is produced, priming the immune system to attack the SARS-CoV-2 virus if it later infects the body.

Vaccination 52

Vaccination Vaccine Clinical Trials Clinical Trials 52

The Pharma Data

JANUARY 29, 2021

Additional safety and efficacy data for the vaccine will continue to accumulate from ongoing clinical trials and is expected to be published in the coming weeks. After vaccination, the surface spike protein is produced, priming the immune system to attack the SARS-CoV-2 virus if it later infects the body.

Vaccination 52

Vaccination Vaccine Clinical Trials Clinical Trials 52

XTalks

SEPTEMBER 15, 2020

A vaccine developed by Swedish company Diamyd Medical has demonstrated significant treatment efficacy in a predefined genetic subgroup of individuals with type 1 diabetes in a Phase IIb clinical trial. So, this really validates what we already knew and now we have prospective results supporting that [from this trial].”.

Vaccination 103

Vaccination Vaccine Insulin Trials 103

The Pharma Data

APRIL 18, 2022

Takeda today announced that it has received manufacturing and marketing approval from the Japan Ministry of Health, Labour and Welfare (MHLW) for Nuvaxovid ® Intramuscular Injection (Nuvaxovid), a novel recombinant protein-based COVID-19 vaccine, for primary and booster immunization in individuals aged 18 and older. TAK-019 Clinical Trial.

Vaccination 52

Vaccination Vaccine Manufacturing Clinical Trials 52

The Pharma Data

NOVEMBER 17, 2021

2) From the body of evidence in clinical trials and real-world data, the vaccine has been shown to have an acceptable safety profile.(3,4,5,6,7). After vaccination, the surface spike protein is produced, priming the immune system to attack the SARS-CoV-2 virus if it later infects the body. 3,4,5,6,7).

Vaccination 52

Vaccination Vaccine Clinical Trials Clinical Trials 52

XTalks

AUGUST 17, 2020

Cue Biopharma is a Cambridge, Massachusetts-based clinical-stage biotechnology company that is focused on developing injectable immune biologics using its proprietary Immuno-STAT (Selective Targeting and Alteration of T cells) platform. protein-based biologics) and vaccine treatments. Cell-Based Immunotherapy vs. Immune Biologics.

Protein 98

Protein Engineer Immune Response In-Vivo 98

Drug Discovery World

OCTOBER 27, 2023

Organ-chips combine cell culture with microfluidics to emulate the biological forces of different organ tissues and/or disease states, allowing pharmaceutical researchers to determine a drug candidates’ efficacy and toxicity ahead of clinical trials. Could organ-chips replace other research methods?

Research 72

Research Gene Therapy In-Vivo In-Vitro 72

The Pharma Data

MARCH 14, 2021

Furthermore, in clinical trials, even though the number of thrombotic events was small, these were lower in the vaccinated group. After vaccination, the surface spike protein is produced, priming the immune system to attack the SARS-CoV-2 virus if it later infects the body.

Vaccination 52

Vaccination Vaccine Licensing Licensing 52

XTalks

NOVEMBER 10, 2023

The US Food and Drug Administration (FDA) has approved Takeda Pharmaceuticals’ Adzynma, the first recombinant protein product for prophylactic (preventive) or on‑demand enzyme replacement therapy (ERT) in adult and pediatric patients with congenital thrombotic thrombocytopenic purpura (cTTP), an ultra-rare blood clotting disorder.

FDA Approval 59

FDA Approval Gene Therapy Gene Clinical Trials 59

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. PFIZER DISCLOSURE NOTICE: The information contained in this release is as of July 8, 2021.

Genetics 52

Genetics Biobanking Protein Containment 52

The Pharma Data

JANUARY 10, 2021

The new year began with a fairly low level of clinical trial news. Arcturus Therapeutics got the FDA go-ahead for its Phase II trial of its COVID-19 vaccine candidate ARCT-021. The trial will enroll 600 participants, with 450 receiving ARCT-021 and 150 receiving placebo. Here’s a look. COVID-19-Related.

Gene Therapy 52

Gene Therapy Trials Gene Clinical Trials 52

The Pharma Data

JANUARY 6, 2021

The vaccine was shown in clinical trials to be safe and effective at preventing symptomatic COVID-19, with no severe cases and no hospitalisations more than 14 days after the second dose. Additional safety and efficacy data will continue to accumulate from ongoing clinical trials.

Vaccination 52

Vaccination Vaccine Clinical Trials Clinical Trials 52

The Pharma Data

NOVEMBER 20, 2020

Progeria and Progeroid Laminopathies are separate and distinct ultra-rare, genetic, premature aging diseases that accelerate mortality in young patients. The increase in survival observed with Zokinvy was derived from two open-label clinical trials (N=62) conducted at Boston Children’s Hospital. ABOUT ZOKINVY (LONAFARNIB).

FDA Approval 52

FDA Approval Genetics Clinical Trials Clinical Trials 52

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. In addition, the lethality of prion diseases effectively means that any effective treatment could be fast-tracked into the clinic, streamlining the regulatory journey for companies, says Wille.

Drugs 299

Drugs Protein Vaccination Vaccine 299

The Pharma Data

SEPTEMBER 8, 2020

More specifically, the agency requires that scientific evidence for regulatory approval must come from large, high quality clinical trials that are randomized and observer-blinded, with an expectation of appropriately designed studies with significant numbers of participants across diverse populations. Sanofi, Empowering Life.

Vaccination 52

Vaccination Vaccine Containment Clinical Trials 52

The Pharma Data

DECEMBER 18, 2020

The Moderna COVID-19 Vaccine contains messenger RNA (mRNA), which is genetic material. The vaccine contains a small piece of the SARS-CoV-2 virus’s mRNA that instructs cells in the body to make the virus’s distinctive “spike” protein. The vaccine was 94.1% ModernaTX, Inc.

Vaccination 52

Vaccination Vaccine Production Manufacturing 52

The Pharma Data

SEPTEMBER 8, 2020

More specifically, the agency requires that scientific evidence for regulatory approval must come from large, high quality clinical trials that are randomized and observer-blinded, with an expectation of appropriately designed studies with significant numbers of participants across diverse populations. For more information, visit.

Vaccination 52

Vaccination Vaccine Containment Clinical Trials 52

Drug Discovery World

DECEMBER 14, 2023

As well as the Cancer Research UK Scotland Institute, Cancer Research UK awards grants to research groups at universities and funds Experimental Cancer Medicine Centres (ECMCs) in Glasgow and Edinburgh, which deliver clinical trials of new experimental treatments.

Drugs 75

Drugs Life Science Research Scientist 75

The Pharma Data

OCTOBER 30, 2020

To develop REGN-COV2, Regeneron scientists evaluated thousands of fully-human antibodies produced by the company’s VelocImmune ® mice, which have been genetically modified to have a human immune system, as well as antibodies identified from humans who have recovered from COVID-19.

Production 52

Production Antibody Manufacturing Trials 52

The Pharma Data

JULY 27, 2021

This approach begins by using tools like advanced human genetics to unravel the complexities of disease and understand the fundamentals of human biology. is a clinical stage biotechnology company developing a new class of biologics, Human Heavy-Chain Antibodies (UniAb®), for the treatments of cancer, autoimmunity, and infectious diseases.

Production 52

Production Antibody Sales Manufacturing 52