Worldwide Clinical Trials

NOVEMBER 12, 2024

By Amy Raymond, PhD, PMP, Executive Director, Therapeutic Strategy Lead, Rare Disease Cell and gene therapies (CGTs) include cutting-edge approaches that offer the hope of a healthier, happier, and better tomorrow for a wide range of patient populations. The global nature of cell and gene therapy trials adds another layer of complexity.

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Bio Pharma Dive

DECEMBER 6, 2022

In a letter to the biotech, the regulator asked for more information on its gene editing medicine for heart disease as well as on its potential risks.

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Gene Editing Gene Regulation Trials 154

Fierce Pharma

AUGUST 7, 2024

On the rocky road to ensuring access to gene therapy Casgevy in the U.K.,

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Gene Editing Gene Therapy Gene Regulation 97

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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The Pharma Data

SEPTEMBER 3, 2020

Current scientific techniques are not yet safe or effective enough to be used to create gene-edited babies, an international committee says. The world’s first gene-edited babies were born in China in November 2018. Why is gene-editing babies controversial? The committee was set up in response.

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BioPharma Reporter

JANUARY 9, 2023

The collaboration will combine ReCodeâs delivery technology with AskBioâs gene editing and DNA cargoes to develop gene correction therapies for liver and lung diseases.

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BioPharma Reporter

MARCH 28, 2023

Vertex will pay CRISPR Therapeutics $100m up-front for non-exclusive rights to its technology with the goal of developing a certain type of therapy for diabetes.

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BioPharma Reporter

OCTOBER 22, 2020

The use of CRISPR, the genetic scissors that allow scientists to edit the instruction manual of life, DNA, has drawn massive global attention over the last several years.

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STAT News

MARCH 6, 2023

LONDON — The first gene-edited children were born in China five years ago , but it’s unlikely to happen again there anytime soon. That was the message Chinese scientists delivered Monday on the opening day of the Third International Summit on Human Genome Editing in London.

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BioPharma Reporter

OCTOBER 2, 2023

Laverock Therapeutics, the gene editing-induced gene silencing platform for human therapeutic applications, has expanded its seed funding round to Â13.5 million.

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XTalks

MARCH 7, 2024

In a pivotal move for the food industry, the US Food and Drug Administration (FDA) has unveiled industry guidance for genome edited foods derived from plants. Genome editing stands as a cutting-edge technique that ingeniously introduces new traits into plants.

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Gene Editing Gene Genomics Genome 52

pharmaphorum

MARCH 19, 2024

Achieve Safe & Targeted In Vivo Delivery of Payloads with Clarity in Regulations & Appropriate Models to Advance the Next Frontier of In Vivo Editing from Research to Clinic

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pharmaphorum

OCTOBER 7, 2020

Drs Emmanuelle Charpentier and Jennifer Doudna have won this year’s Nobel Prize for chemistry in recognition of their work on the gene-editing technology CRISPR/Cas9. I encourage continued support of fundamental science as well as public discourse about the ethical uses and responsible regulation of CRISPR technology.”.

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pharmaphorum

SEPTEMBER 18, 2020

Vertex is already the top player in CF with a quartet of exon-skipping medicines that tackle the mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that underlies the disease, but there are still a sizeable group of patients with no treatment options.

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Pharmaceutical Technology

MARCH 9, 2023

T-cell activation can be measured by assessing proliferation, up-regulation of activation markers and production of effector cytokines (IFN-gamma and TNF-alpha). Activation of T-cell proliferation specifically in a tumour is crucial for reducing the autoimmune side effects of antitumour immunotherapy.

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pharmaphorum

DECEMBER 6, 2020

A gene-editing drug developed by CRISPR Therapeutics and Vertex Pharma has achieved “remarkable” improvements in patients with beta thalassaemia and sickle cell disease in an early-stage trial reported at the American Society of Haematology (ASH) annual meeting.

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Gene Editing Gene In-Vivo Genetics 52

BioPharma Reporter

DECEMBER 18, 2023

Eligo Bioscience, a gene-editing company focused on addressing diseases driven by the expression of bacterial genes from the microbiome, has announced a successful $30 million Series B funding round, led by Sanofi Ventures.

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BioPharma Reporter

MARCH 2, 2021

The gene-editing and genome-engineering company, eGenesis, today announced the completion of a US$125m Series C financing round.

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BioPharma Reporter

AUGUST 30, 2021

Boston-based company, Vertex, has signed a deal to use Arbor Biotechnologiesâ CRISPR gene-editing technology to develop novel cell therapies for the treatment of serious diseases.

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XTalks

MAY 4, 2021

Researchers at the University of California San Francisco (UCSF) and the Whitehead Institute have developed a novel CRISPR-based tool called “CRISPRoff” that can switch off genes in human cells through epigenetic editing without altering the genetic sequence itself. It’s a great tool for controlling gene expression.”.

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Advarra

NOVEMBER 29, 2022

The field of cell and gene therapies (CGT) is constantly evolving, and there has been significant progress in this area of research. However, despite the promise of these therapies, the regulations governing them lag the science, which in turn hinders the clinical translation of these novel medicines.

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The Pharma Data

MARCH 7, 2022

By combining CRISPR technology with a protein designed with artificial intelligence (AI), it is possible to awaken individual dormant genes by disabling the chemical “off switches” that silence them. The chemical modifications that regulate gene activity are called epigenetic markers. it can be reawakened.

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pharmaphorum

AUGUST 8, 2022

The boards of both companies have recommended the deal to shareholders, and the two companies suggested it should close before the end of the year – assuming of course it doesn’t fall foul of any antitrust issues raised by financial regulators.

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The Pharma Data

JANUARY 19, 2021

A naturally occurring system for tuning CRISPR-Cas9 expressing in bacteria, identified in a study published in Cell , could have implications for gene editing therapies as well. A CRISPR-Cas9 system has two components: the Cas9 guide RNA that directs the system to edit a particular gene, and the CRISPR “scissors” that make the cut.

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XTalks

MAY 22, 2023

Related: 5 Food Companies Working With Precision Fermentation Technology How Pairwise Leverages CRISPR Technology CRISPR, or Clustered Regularly Interspaced Short Palindromic Repeats, is a gene-editing technique that can be used to alter the DNA of cells to enhance certain characteristics or reduce less desirable ones.

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XTalks

SEPTEMBER 20, 2024

Vittoria Biotherapeutics is a clinical-stage cell therapy company that incorporates the latest advancements in cell therapy and gene editing with a unique approach that potentially overcomes some of the issues with this therapeutic modality. Navigating the Regulatory Landscape Regulations for novel therapies like CAR T are complex.

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pharmaphorum

JULY 1, 2021

Synthetic biology has grown exponentially in less than a decade, with CRISPR gene-editing as the key technology driving this through its use in areas like cancer, infectious diseases, and rare, undertreated indications.

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Gene Editing Engineer Development Gene 88

XTalks

MAY 4, 2021

Researchers at the University of California San Francisco (UCSF) and the Whitehead Institute have developed a novel CRISPR-based tool called “CRISPRoff” that can switch off genes in human cells without editing the genetic sequence itself. These modifications regulate gene expression without altering the sequence or structure of DNA.

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XTalks

FEBRUARY 27, 2024

There have been significant advancements in new rare disease drugs, particularly for genetic disorders that can be treated by correcting, replacing or silencing defective genes. The US Food and Drug Administration (FDA) has approved a number of new rare disease drugs in recent years, including gene therapies.

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XTalks

DECEMBER 29, 2022

For example, on the pharma and biotech side, there has been growing demand for new drugs and biologics, from new classes of dual-action diabetes and weight loss drugs like Ozempic and Mounjaro to life-saving gene therapies. 1. Cell and Gene Therapies. In 2023, a number of gene therapies are expected to get the FDA green light.

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Trialfacts

SEPTEMBER 12, 2024

Their work is driven by a profound understanding of disease biology and supported by advanced discovery platforms such as genetics and genomics, gene editing, bioinformatics, proteomics, bioengineering, image analysis, biobanks, disease-specific stem cell lines, and various animal models.

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Research Gene Editing Biobanking Bioinformatics 52

XTalks

FEBRUARY 10, 2023

It is important to have a comprehensive Product Development Plan (PDP) that is developed early and in collaboration with regulators and the non-clinical, manufacturing, clinical operations and logistics teams. An overview of Medpace’s experience in conducting cell and gene therapy trials.

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The Pharma Data

JANUARY 17, 2021

As a result, the actual future financial condition, performance and results of the Company may differ materially from the plans, goals and expectations set forth in any forward-looking statements.

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Pharma Marketing Network

JANUARY 20, 2021

According to Bill Evans of Rock Health, the ongoing adoption of digital health products, such as telehealth, will depend on the degree to which CMS and state regulators retain relaxed rules that allow physicians to cross state lines and enable reimbursement for digital services.

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Vaccination Vaccine In-Vivo Genetics 52

XTalks

AUGUST 2, 2023

Meanwhile, BioMarin’s gene therapy Roctavian is approved by the FDA for the treatment of hemophilia A. In September 2022, Roche acquired Good Therapeutics for an upfront payment of $250 million, and has access to their PD-1-regulated IL-2 program. But all three drugs have new competitors. Both are anti-CD20 agents.

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XTalks

DECEMBER 24, 2020

From rare disease drug approvals to treatments involving immunotherapies and gene therapies and awarding of a Nobel Prize to the inventors of the gene-editing tool CRISPR, 2020 was a year of great activity and productivity despite the backdrop of the pandemic. CRISPR Gene Editing Inventors Win Nobel Prize.

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The Pharma Data

NOVEMBER 24, 2020

As a result, the actual future financial condition, performance and results of the Company may differ materially from the plans, goals and expectations set forth in any forward-looking statements.

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