Worldwide Clinical Trials

NOVEMBER 12, 2024

For example, once considered incurable and terminal, patients with sickle cell disease may reach new summits in their lives with gene editing technologies such as CRISPR to repair affected DNA and, in some cases, functionally cure the condition. Cell therapy, specifically, is changing our narrative around chronic and lethal conditions.

Gene Therapy 182

Gene Therapy Clinical Supply Trials Gene 182

Fierce Pharma

AUGUST 7, 2024

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

Gene Editing 97

Gene Editing Gene Therapy Gene Regulation 97

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.

Gene Editing 52

Gene Editing Gene Genome Genomics 52

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.

Gene Editing 52

Gene Editing Gene DNA Development 52

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.

Gene Editing 96

Gene Editing Gene Development Regulation 96

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.

Genome 98

Genome Genomics Gene Editing Genetics 98

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.

Gene Editing 105

Gene Editing DNA Genetics Scientist 105

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

In-Vivo 52

In-Vivo Gene Editing Engineer Gene 52

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.

Gene Editing 52

Gene Editing Gene Genome Genomics 52

Pharmaceutical Technology

FEBRUARY 23, 2023

It covers a broad range of techniques from the less invasive practice of intermittent fasting to drastic and invasive procedures, such as the implanting of microchips or injecting of gene-editing enzymes. As a broader concept, biohacking encourages experimental biotechnology outside the confines of traditional research environments.

Gene Editing 278

Gene Editing Regulation Production Nurses 278

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.”.

Gene Editing 105

Gene Editing Bacteria DNA Gene 105

XTalks

JULY 14, 2022

However, there is limited knowledge about the upstream regulators of most genes. A downstream approach has traditionally been used by scientists to map networks, wherein a gene for a protein is removed one at a time and the impact on the immune cell’s function is observed. All 24 of the regulators affected the levels of IL2RA.

Gene 98

Gene Regulation Protein Gene Editing 98

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.

In-Vitro 130

In-Vitro Gene Editing Medicine Licensing 130

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.

Gene Silencing 59

Gene Silencing Gene Gene Editing Regulation 59

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.

Gene Editing 52

Gene Editing Gene In-Vivo Genetics 52

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.

Gene Editing 52

Gene Editing Genome Genomics Medicine 52

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.

Gene Editing 52

Gene Editing Genetics Gene Development 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.

Gene Editing 52

Gene Editing Gene Regulation Marketing 52

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. The authors suggest natural tracr-L regulates autoimmunity. And removing tracr-L altogether greatly increased CRISPR-Cas9 expression.

Bacteria 52

Bacteria Gene Editing Research RNA 52

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.

Gene Editing 52

Gene Editing Manufacturing Gene In-Vivo 52

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.

Branding 98

Branding Gene Editing DNA Production 98

pharmaphorum

JANUARY 29, 2021

Cutting edge’ is, for once, a truly apt description when it comes to gene editing – both because the field is pushing medicine into areas we might never have dreamed possible, and because these technologies involve literally cutting DNA at a specific point in the genome. Zinc fingers. The genomic medicine journey.

Gene Editing 145

Gene Editing Gene Gene Therapy In-Vivo 145

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.

Research 52

Research Gene Editing Biobanking Bioinformatics 52

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.

Gene Therapy 52

Gene Therapy Gene Production Clinical Trials 52

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.

Research 59

Research Drugs Gene Therapy Clinical Trials 59

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. If so, why?

Clinical Trials 52

Clinical Trials Clinical Trials Trials Gene Therapy 52

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.

Gene Editing 88

Gene Editing Engineer Development Gene 88

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.

Marketing 111

Marketing Sales Gene Editing Antibody 111

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.

Gene Editing 105

Gene Editing Gene Vaccination Vaccine 105

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.

DNA 98

DNA Gene Gene Silencing Genome 98

XTalks

DECEMBER 29, 2022

As of December 16, 2022, there are seven US Food and Drug Administration (FDA) approved gene therapies. In 2022 alone, the US regulator approved four new gene therapies, showing the high interest in getting these therapies to market. In 2023, a number of gene therapies are expected to get the FDA green light.

Life Science 52

Life Science Gene Therapy Gene Manufacturing 52

The Pharma Data

MARCH 7, 2022

The new technique controls gene activity without altering the DNA sequence of the genome by targeting chemical modifications that help package genes in our chromosomes and regulate their activity. The chemical modifications that regulate gene activity are called epigenetic markers.

Protein 52

Protein Gene Genome Genomics 52

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.

DNA 52

DNA Gene Gene Silencing Genome 52

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.

Containment 52

Containment Genetics Gene Editing Production 52

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.

Genetics 40

Genetics Containment Sales Production 40

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.

Vaccination 52

Vaccination Vaccine In-Vivo Genetics 52

XTalks

AUGUST 2, 2023

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. The TPO receptor agonist regulates blood platelet production by binding to and activating TPO receptors on megakaryocyte cells and inducing signalling cascades that increase platelet production.

Sales 98

Sales Manufacturing FDA Approval Life Science 98