Drug Discovery World

MAY 6, 2024

A more established Amsterdam-based enterprise is gene therapy company uniQure, developer of etranacogene dezaparvovec, the first gene therapy for haemophilia B, which was approved for use in the US in November 2022 and in the EU in February 2023. The CDD will sponsor and manage KWF-supported trials.

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Drugs Life Science Gene Editing Gene 59

The Pharma Data

DECEMBER 13, 2020

SYNB1891 is an investigational drug for the intra-tumoral treatment of solid tumors and lymphoma, composed of an engineered Synthetic Biotic designed to activate the STING pathway in the tumor microenvironment in order to upregulate the patient’s immune response. Synlogic’s President and Chief Executive Officer.

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Immune Response Engineer Clinical Trials Clinical Trials 40

Pharma Marketing Network

DECEMBER 21, 2020

those that modify the expression of an individual’s genes or repair abnormal genes) has entered clinical practice, including 11 RNA therapeutics, 2 in vivo gene therapies, and 2 gene-modified cell therapies. greater safety, transient and reversible effect on gene expression).

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Genetics Medicine Gene Therapy Marketing 52

The Pharma Data

OCTOBER 27, 2020

The Massachusetts-based biotech is investing in their proprietary “Gene Traffic Control” system, which tells the cells when to express which genes where and in what order within the chromatin regulatory system. Foghorn boomed onto the NASDAQ with a $120 million IPO , surpassing its original expectations by $20 million. AavantiBio .

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Gene Gene Therapy Medicine Genetics 52

The Pharma Data

MAY 15, 2023

The acquisition complements Gilead’s existing clinical development priorities by adding additional pipeline assets for well-validated targets in oncology and inflammation. Both programs have the potential to address multiple indications, offering broad development opportunities alone and in combination with Gilead’s portfolio.

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Drug Discovery World

FEBRUARY 28, 2024

CRISPR and brain cancer Meanwhile, elsewhere in the US, another exciting development – using CRISPR as a therapeutic approach – has possibilities for tackling brain cancer. Scientists have used CRISPR gene editing to target and rapidly destroy glioblastoma cells in an approach that could apply to other highly mutated cancers.

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The Pharma Data

MARCH 11, 2021

Clinical trial to assess safety, immune response and reactogenicity, after preclinical data showed high neutralizing antibody levels. The joint development team is working on improving the temperature stability of the mRNA vaccine candidate and targeting a -20°C storage temperature for late-stage clinical trials and at launch.

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Clinical Trials Clinical Trials Vaccination Vaccine 52

The Pharma Data

JANUARY 21, 2021

Precigen is focused on next-generation gene and cell therapy using precision technology. The compound was developed in the laboratory of Richard Childs, chief of the Laboratory of Transplantation Immunotherapy with the National Heart, Lung, and Blood Institute (NHLBI). Here’s a look. Precigen – Germantown, Md.-based

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Immune Response Trials Development Drugs 52

XTalks

MARCH 13, 2023

The US Food and Drug Administration’s (FDA) Office of Tissues and Advanced Therapies (OTAT) held a recent town hall where three experts from the regulator provided guidance on how to design and conduct gene therapy clinical trials for rare diseases. It’s a very exciting time in gene therapy.

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Gene Therapy Clinical Trials Clinical Trials Gene 52

Delveinsight

AUGUST 4, 2021

Similarly, genetics, immune response, and environmental factors also influence the occurrence of Neurological Conditions. Companies are focusing on the development of adherence-friendly drug formulations, Gene therapy, a drug for better treatment of motor symptoms.

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Drug Discovery World

FEBRUARY 5, 2024

Brian Gladsden, CEO, Mosaic Therapeutics “The industry has already made tremendous strides in targeted drug discovery in oncology, particularly as the industry moves further away from simply looking at tumour histology and relying more on cancer gene sequencing. In the past 10 years, immunotherapy trials have increased by 430%.

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Research Drug Delivery Systems Gene Sequencing Trials 116

Drug Discovery World

AUGUST 2, 2023

Palmitoyl transferases are a zinc finger Asp-His-His-Cystype (ZDHHC) family containing 23 distinct mammalian genes (excluding ZDHHC10) that catalyse this reaction 1. Those results led to further clinical development in ongoing larger studies in patients with advanced hepatocellular carcinoma and cholangiocarcinoma.

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Protein Immune Response In-Vitro Regulation 52

The Pharma Data

JULY 6, 2021

Enrolment is currently underway for a pivotal Phase 3 trial for AL001 in people at risk for or with frontotemporal dementia due to a progranulin gene mutation (FTD-GRN). billion in clinical development, regulatory and commercial launch-related milestone payments. This population represents 5% to 10% of all people with FTD.

Genetics 52

Genetics Gene Antibody Development 52

Drug Discovery World

APRIL 5, 2023

In February this year, Akamis Bio announced an expansion of its ongoing partnership with the Parker Institute for Cancer Immunotherapy (PICI) to include a clinical collaboration with the Cancer Research Institute (CRI). DDW: Why is pancreatic cancer so difficult to treat using a single therapy? DDW: You’re exploring NG-350A in the study.

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Gene Therapy Gene Immune Response Development 52

Roots Analysis

OCTOBER 30, 2023

These RNA based therapeutics play a crucial role in protein production and regulation of gene functions. Self-amplifying RNA (saRNA): Self amplifying RNA is an engineered form of RNA, combining the amplifying genome (native to certain viruses) to the therapeutic gene of interest.

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RNA Vaccination Vaccine Development 40

The Pharma Data

DECEMBER 27, 2020

Notably, we never modify the DNA of these cells, which avoids some of the safety concerns that have been reported with certain gene-editing technologies. From these cells we apply proprietary methods to manufacture pure populations of only the cell types which we wish to use in patients. Forward-Looking Statements.

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Manufacturing Production Development Clinical Trials 52

XTalks

JUNE 9, 2022

When a child born from parents with FH genes, inherits one FH gene from each parent, they might develop homozygous familial hypercholesterolemia (HoFH). Children suffering from HoFH develop xanthanomas within the first few years of their life. Familial hypercholesterolemia (FH) is a hereditary disorder.

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Trials Antibody Genetics Gene 52

Drug Discovery World

OCTOBER 17, 2023

Analysing spatial changes during disease progression or treatment response is key to fully understand pathologies. RA: With spatial biology being cross-disciplinary – in that it uses techniques in both gene expression analysis and immunofluorescence – is expertise in the field limited? Let’s take CAR T cells as an example.

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Gene Expression Drugs Protein Gene 52

Drug Discovery World

MAY 30, 2023

FAPi PET is being developed as a highly sensitive imaging agent and can be used as a predictive biomarker for FAP targeted therapeutics, several of which are currently in clinical development. We expect this field to grow significantly in the coming years as these and other earlier stage assets are developed.

Research 52

Research Drugs Protein RNA 52

Drug Discovery World

FEBRUARY 15, 2023

Cancer is a complex heterogenous multistep disease characterised by uncontrolled cell proliferation combined with a dysregulated immune response. A healthy immune system is naturally primed to eliminate malignant and abnormal cells through a synchronised and dynamic interplay between adaptive and innate immunity.

Research 52

Research In-Vitro In-Vivo Development 52

The Pharma Data

OCTOBER 14, 2020

(Nasdaq: TNXP) (Tonix or the Company), a clinical-stage biopharmaceutical company, today announced that the first participant was enrolled in the observational PRECISION study (TNX-C002), to examine the immune responses to COVID-19 in healthy volunteers who have recovered from COVID-19 or were asymptomatic.

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Vaccination Vaccine Development Medicine 40

The Pharma Data

SEPTEMBER 15, 2020

In addition to the COVID-19 vaccine program, Pfizer aims to deliver five innovative vaccines by 2025, subject to clinical success and regulatory approval. Based on the acceptable safety profile and the favorable immune response data, including the 4th dose response data, Pfizer received Breakthrough Therapy Designation.

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Gene Therapy Vaccination Vaccine Trials 52

The Pharma Data

MARCH 31, 2023

Exploratory analyses included EFS by surgical approach, EFS by extent or completeness of resection, and EFS and pathologic complete response (pCR) by a four-gene ( CD8A, CD274, STAT-1, LAG-3 ) inflammatory signature score derived from RNA sequencing of baseline tumor samples.

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Trials HR Medicine Development 40

Drug Discovery World

NOVEMBER 13, 2023

Enkelejda Miho, PhD, Professor, University of Applied Sciences and Arts Northwestern Switzerland, and Managing Director, aiNET, on: ‘The singular immune response to dengue and machine learning identification of antibodies in high-throughput sequences’.

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Protein Antibody Engineer Scientist 59

The Pharma Data

OCTOBER 26, 2020

The treatment induced HPV-specific T-cell responses and was associated with increased levels of immune cell infiltration in the tumors and expression of genes associated with activation of the immune system. An overall response rate of 23.5% . – .

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The Pharma Data

AUGUST 11, 2020

In the field of immune cell therapy, this includes registrational CAR T cell agents for numerous diseases, and a growing early-stage pipeline that expands cell and gene therapy targets, and technologies. By harnessing the body’s own immune system to fight cancer, Opdivo has become an important treatment option across multiple cancers.

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The Pharma Data

AUGUST 11, 2020

In the field of immune cell therapy, this includes registrational CAR T cell agents for numerous diseases, and a growing early-stage pipeline that expands cell and gene therapy targets, and technologies. By harnessing the body’s own immune system to fight cancer, Opdivo has become an important treatment option across multiple cancers.

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Trials Hormones Clinical Trials Clinical Trials 52

XTalks

SEPTEMBER 14, 2020

It was recently reported that the addition of an adjuvant to their vaccine led to enhanced immune responses that exceeded responses in convalescent serum from symptomatic COVID-19 patients. Novavax has also tested its recombinant SARS-CoV-2 nanoparticle adjuvanted vaccine in a Phase I/II trial.

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