Pharmaceutical Technology

MAY 24, 2023

The regulator granted RMAT designation based on the data obtained from the ongoing Phase I RP-L301 clinical trial. 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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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). Biogen Safe Harbor.

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Roots Analysis

AUGUST 31, 2023

Further, the transcription factors contain two molecular domains, the DNA binding domain and the activation domain. Moreover, some transcription factors contain additional domains, such as ligand binding domain in order to interact with chemical signals.

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Advarra

OCTOBER 26, 2023

Controlled Environment and IP Containment The potential for a release, and the risk associated with a genetically engineered IP, are part of the IBC’s assessment purview under National Institutes of Health (NIH) Guidelines. The containment must then prevent spills or leaks if the IP is dropped. Size matters.

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Drug Discovery World

JANUARY 11, 2024

The team, led by University College London (UCL), King’s College London and Moderna scientists, found that mRNA could be used to correct a rare genetic liver disease known as argininosuccinic aciduria in a mouse model. mRNA contains instructions that direct the cells to make proteins.

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XTalks

NOVEMBER 7, 2022

Gleich, MD, FACS, Senior Vice President, Medical Department, and Dr. Christopher Huth, PhD, Senior Clinical Trial Manager, Clinical Trial Management. Liquid Biopsy Use in Oncology Clinical Trials. Regulators may ask at some point to provide that documentation, so it’s important to track that across the study.

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The Pharma Data

OCTOBER 25, 2020

NASDAQ: PTCT), today announced the initiation of a registration-directed Phase 2/3 clinical trial to evaluate vatiquinone (PTC743) in patients with mitochondrial epilepsy, the highly morbid condition of refractory seizures in children with inherited mitochondrial disease. . SOUTH PLAINFIELD, N.J. , Peltz , Ph.D.,

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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.

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Drug Discovery World

DECEMBER 15, 2022

Gene editing tools such as zinc finger nucleases, transcription activator-like effector nucleases (TALEN) and clustered regularly interspaced short palindromic repeat (CRISPR) nucleases have been heralded for their enormous potential to treat diseases and genetic disorders. Early phase clinical trials for gene editing therapies.

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The Pharma Data

NOVEMBER 22, 2020

As the Pfizer and BioNTech COVID-19 vaccine goes to regulators and the Moderna vaccine approaches the end of its Phase III trials, AstraZeneca and the University of Oxford announced high-level results from an interim analysis of their COVID-19 vaccine, AZD1222. Trials are also being run in the U.S., Featured Jobs on BioSpace.

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The Pharma Data

NOVEMBER 27, 2020

Nasdaq:RYTM), a biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic diseases of obesity, announced today that the U.S. With this approval, Imcivree becomes the first-ever FDA approved therapy for these rare genetic diseases of obesity. BOSTON, Nov.

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The Pharma Data

AUGUST 3, 2021

Food and Drug Administration (FDA) has determined that OAV-101 intrathecal (IT) clinical trials for spinal muscular atrophy (SMA) patients may proceed, thereby lifting the partial clinical trial hold initiated in October 2019. Novartis today announced that the U.S. SVP, Chief Medical Officer, Novartis Gene Therapies. “We

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XTalks

MARCH 10, 2023

Omaveloxolone Clinical Trials Skyclarys is an oral medication that contains omaveloxolone, an activator of the nuclear factor-erythroid 2 related factor 2 (Nrf2) pathway. Skyclarys was approved based on the results of the MOXIe Part 2 clinical trial.

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Drug Discovery World

AUGUST 14, 2023

For example, the FDA’s Center for Biologics Evaluation and Research (CBER) 3 , which regulates biological products for human use, differentiates between in vivo gene therapy (new genes are inserted into the body to target cells) and ex vivo gene therapy (cells are modified outside the body and then administered back into the patient).

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Drug Discovery World

APRIL 4, 2023

Read more: FDA Fast Track Designation for retinitis pigmentosa treatment Drug candidate for TUBB4a leukodystrophy, SynaptixBio The FDA has granted Rare Paediatric Disease (RPD) Designation to SynaptixBio’s drug candidate for TUBB4a leukodystrophy – a genetic and debilitating condition which mainly affects babies and young children.

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Advarra

SEPTEMBER 29, 2022

Informed consent is one of the central protections the regulations provide to research subjects. The regulatory requirements for informed consent will vary depending upon which regulations apply to the conduct of a particular study. The regulations are very specific regarding what to include in the ICF and what to exclude.

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Drug Discovery World

AUGUST 3, 2023

Medical cannabis is often a first-thought when regarding plant-based medicines, but there remains public scepticism, arguably due to the regulations relating to the substance. What is the regulatory landscape of medical cannabis? There is, however, one cannabis-derived drug product that the FDA has approved, which is Epidiolex (cannabidiol).

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The Pharma Data

SEPTEMBER 22, 2020

Trial design and other details are being evaluated and a comprehensive update on the overall Novartis SMA clinical development program will be provided at a future time following further discussions with health authorities. All patients deserve a gene therapy designed to address the genetic root cause of their disease with a single dose.

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The Pharma Data

DECEMBER 30, 2020

AstraZeneca’s COVID-19 vaccine candidate AZD1222 is a viral vector-based, weakened version of adenovirus containing the genetic material of SARS-CoV-2 spike protein. During Phase III trials, one of the cohorts was administered one and a half doses, as opposed to two full doses. AstraZeneca’s efficacy is at 70.4%.

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The Pharma Data

DECEMBER 21, 2020

21, 2020 /PRNewswire/ — Amgen (NASDAQ:AMGN) and AstraZeneca today announced the SOURCE trial did not meet the primary endpoint of a statistically significant reduction in the daily oral corticosteroid (OCS) dose, without loss of asthma control, with tezepelumab compared to placebo. THOUSAND OAKS, Calif. ,

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The Pharma Data

JANUARY 29, 2021

Following review of the application, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency based its positive opinion on data from a rolling review of trial data from the primary analysis of the Phase III programme led by the University of Oxford.

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The Pharma Data

JANUARY 29, 2021

Following review of the application, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency based its positive opinion on data from a rolling review of trial data from the primary analysis of the Phase III programme led by the University of Oxford.

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The Pharma Data

MARCH 23, 2022

Unfortunately, in a Phase 3 clinical trial, treatment with remdesivir did not make a difference in mortality for Ebola virus disease patients. Early in infection, it appears that host cellular proteins are in defense mode against Ebola virus polymerase and the viral genetic material.

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The Pharma Data

NOVEMBER 9, 2020

Trial Also Met the Primary Endpoint in Patients With Low Levels of Eosinophils. In the subgroup of patients with baseline eosinophil counts less than 300 cells per microliter, the trial met the primary endpoint with tezepelumab demonstrating a statistically significant and clinically meaningful reduction in AAER.

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XTalks

AUGUST 6, 2020

Epidiolex is the only FDA-approved formulation that contains CBD derived from the cannabis plant. CBD is the most abundant non-psychoactive cannabinoid compound found in the cannabis plant, making up about 40 percent of the plant’s extract, which contains over 100 different cannabinoid compounds.

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XTalks

NOVEMBER 10, 2023

cTTP is a very rare, inherited and life-threatening blood clotting disorder caused by a disease-causing mutation in the ADAMTS13 (A disintegrin and metalloproteinase with thrombospondin motifs 13) gene, which encodes the ADAMTS13 enzyme that regulates blood clotting by cleaving the von Willebrand factor (VWF) protease.

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The Pharma Data

NOVEMBER 30, 2020

The rolling review process began on October 6, which allowed European regulators to evaluate the data as it was submitted. The AstraZeneca-Oxford product had demonstrated an efficacy rate between about 62% and 90%, but there were issues with the Phase III trial. The combined analysis averaged 70%.

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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. While there may be just under 20,000 confirmed protein coding genes, it turns out that much of the genome outside of these genes is also important in regulating how the genome is controlled.

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The Pharma Data

JANUARY 5, 2021

RAD011 is a pivotal-trial ready synthetic cannabidiol oral solution with potential utilization in multiple endocrine and metabolic orphan diseases. Acquisition and associated PWS trial costs to be funded from existing operations. Management to host conference call and webcast this morning at 8:15 a.m.

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The Pharma Data

DECEMBER 30, 2020

This regimen 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. We would like to thank our many colleagues at AstraZeneca, Oxford University, the UK government and the tens of thousands of clinical trial participants.”.

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Drug Discovery World

AUGUST 10, 2022

And now, with more than 600 CAR T trials recruiting or ongoing, these cells represent a majority of the cellular immunotherapies in development today. Cells from solid tumours are genetically heterogeneous both between and within tumours, making it challenging to identify a single, selective antigen target for treatment.

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The Pharma Data

MARCH 4, 2021

Together we generated positive Phase 2 ELEKTRA study data, and as a result, soticlestat is poised to enter two pivotal trials,” said Andy Plump, M.D., Dravet syndrome is most commonly caused by a genetic mutation in the SCN1A gene and affects approximately 1 in 15,000 to 1 in 21,000 people in the United States.

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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. All tests need to meet stringent criteria for quality control and this data is submitted to regulators within each country or region for independent review before any batch can be released to countries.

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The Pharma Data

DECEMBER 8, 2020

Its development program is the first and largest EoE Phase 3 clinical trial program in the U.S. to report results, and includes the pivotal Phase 3 trials ORBIT1 and ORBIT2 which investigated the safety and efficacy of TAK-721 in adolescent and adult patients (11 to 55 years of age) with EoE. For more information, visit [link].

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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. In addition to the University of Oxford-led trials, the Company is conducting a large trial in the US as part of a global programme.

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XTalks

FEBRUARY 4, 2021

Moreover, when the foundation initiated their flagship observational clinical trial —the Parkinson’s Progression Markers Initiative or PPMI in 2010 — there weren’t really clear examples in the field of standardized longitudinal sample collection across multi-international sites. A Biorepository versus a Bioresource.

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Pharma Marketing Network

JANUARY 20, 2021

In other areas of the industry (such as cell and genetic medicine development) progress continued at an impressively brisk pace despite the pandemic. The first of these is the large number of clinical trials, many with a small enrollment and observational design.

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