Bio Pharma Dive

DECEMBER 7, 2022

Editas said its gene editing therapy appeared safe and was well tolerated in two patients. regulators lifted a partial hold on the trial. The data comes five months after U.S.

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Gene Editing Regulation Gene Trials 177

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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In-Vivo Gene Editing Engineer Gene 52

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 Regulation Gene Trials 148

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 Genome Genomics 52

Drug Discovery World

OCTOBER 24, 2023

While attending Life Science Baltics 2023, Diana Spencer spoke to Monika Paule , CEO and Co-founder of Caszyme, about creating a company based on CRISPR gene editing and some of the challenges of operating in this dynamic area of research. Caszyme was founded in 2017 by the pioneers of CRISPR gene editing technologies.

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Gene Editing Gene Life Science Regulation 105

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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Gene Editing Gene Development Regulation 98

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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Genome Genomics Gene Editing Genetics 98

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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Gene Silencing Gene Gene Editing Regulation 59

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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Gene Editing Gene Regulation Marketing 52

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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Gene Editing DNA Genetics Scientist 105

Drug Discovery World

MARCH 22, 2023

The findings showed that targeting two regulators that control gene functions related to inflammation led to at least 10 times greater T cell expansion in models, resulting in increased antitumour immune activity and durability. “We

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Gene Editing Gene Engineer Regulation 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.

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Gene Editing Genetics Gene Development 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.

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Gene Editing Medicine Genome Genomics 52

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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In-Vitro Gene Editing Medicine Licensing 130

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

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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Gene Editing Bacteria DNA Gene 102

Drug Discovery World

MAY 6, 2024

Mazzetti and Schneider give the example of T3 Pharmaceuticals, a University of Basel spin-off that developed a proprietary cancer therapy platform based on regulating and harnessing the natural behaviours of live bacteria. One such company, drawing on Vilnius’ gene editing expertise, is Caszyme, which was founded by Professor Šikšnys.

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Drugs Life Science Gene Editing Gene 59

Drug Discovery World

MARCH 21, 2023

Published in Molecular Cell, the study explains the rules determining to what extent genes respond to CRISPR activation, ensuring that future research can be designed as efficiently as possible. CRISPR activation (CRISPRa) is a type of CRISPR gene editing that is used to overexpress certain genes.

Research 52

Research Gene Gene Editing Genome 52

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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Gene Editing Gene Vaccination Vaccine 102

Drug Discovery World

NOVEMBER 27, 2023

In addition, it has stimulated increased collaboration among drug developers, technology providers, supply chain organisations, and regulators. Next-generation engineering tools, such as gene editing have enabled knock-out cell lines with enhanced characteristics.

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Production Engineer Gene Editing Gene 59

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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Marketing Sales Gene Editing Antibody 111

Drug Discovery World

DECEMBER 15, 2022

As gene editing technologies like CRISPR progress toward clinical study, researchers must continue to advance new approaches and address inherent challenges, explains Jon Chesnut, PhD, Senior Director, Cell Biology R&D, Thermo Fisher Scientific. Early phase clinical trials for gene editing therapies.

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Gene Editing Gene In-Vivo Genome 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.

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Branding Gene Editing DNA Production 98

Drug Discovery World

JULY 18, 2023

The centre also features a newly built demo lab which will bring the Revvity portfolio closer to scientists.

Genome 52

Genome Genomics Life Science Gene Editing 52

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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Gene Editing Gene Genome Genomics 52

Drug Discovery World

APRIL 4, 2023

The decision will enable the company to include the US in the global Phase II portion of its ongoing Phase I/II study of NTLA-2002, an in vivo genome editing candidate designed to inactivate the target gene, kallikrein B1 (KLKB1).

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FDA Approval In-Vivo Gene Editing Drugs 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.

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Gene Editing Engineer Development Gene 78

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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Gene Therapy Gene Production Clinical Trials 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.

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Protein Gene Genome Genomics 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.

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Containment Genetics Gene Editing Production 52

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.

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Life Science Gene Therapy Gene Manufacturing 52

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

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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Gene Editing Gene DNA Development 52

Drug Discovery World

APRIL 14, 2023

He is being recognised for his revolutionary contributions to developing the first gene-edited cell-based therapy for cancer that involves the genetic re-engineering of a patient’s own T cells to combat their disease, and for demonstrating that adoptive T-cell therapy can induce remission and in some cases cure patients with advanced cancer.

Genetics 52

Genetics Research Medicine Clinical Research 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

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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Research Drugs Gene Therapy Clinical Trials 59

Drug Discovery World

JULY 18, 2023

Current research is focused on designing CRISPR-enhanced CAR T cells that are resistant to immunosuppressive cytokines such as transforming growth factor-β (TGF-β) 2 , programmed cell death protein (PD-1) 3 , or other negative T cell regulators 4 (CTLA-4, LAG-3, and TIM-3), thus improving anti-tumour functions.

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DNA Genome Genomics RNA 98