Worldwide Clinical Trials

OCTOBER 24, 2023

Global Genes is a rare disease umbrella organization dedicated to eliminating the burdens and challenges of rare diseases for patients and families globally. Although no two rare disease patient communities are identical, there are common themes to the evolutionary arc of PAO maturation.

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Camargo

JULY 27, 2021

How and When to Incorporate PK Design into Your Gene Therapy Development Plan. Gene therapy, which was in its infancy around 30 years ago, is now becoming a more prominent treatment method in many therapeutic areas, from personalized therapy to mass vaccinations against COVID-19. Gene Therapy Definition.

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BioTech 365

JUNE 21, 2021

QIAGEN Launches Dedicated CRISPR Products for Rapid and Simplified Analysis of Gene-Editing Experiments QIAGEN Launches Dedicated CRISPR Products for Rapid and Simplified Analysis of Gene-Editing Experiments QIAprep&amp CRISPR Kit and Q-Primer Solutions expand QIAGEN portfolio for CRISPR applications Products are … Continue reading → (..)

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Scienmag

JANUARY 12, 2021

Martins Scientists have used gene-editing advances to achieve a tenfold increase in the production of super-bug targeting formicamycin antibiotics. Credit: Dr Dino J. Discovered within the last ten years, formicamycins have great […].

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BioSpace

NOVEMBER 15, 2023

s Medicines and Healthcare products Regulatory Agency on Thursday provided conditional marketing authorization for Vertex Pharmaceuticals and CRISPR Therapeutics’ gene-edited therapy exa-cel.

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Drug Discovery World

NOVEMBER 16, 2023

The Medicines and Healthcare products Regulatory Agency (MHRA) has authorised the world’s first gene therapy for sickle-cell disease (SCD) and transfusion-dependent β-thalassemia (TDT). Casgevy is designed to work by editing the faulty gene in a patient’s bone marrow stem cells so that the body produces functioning haemoglobin.

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Pharmaceutical Technology

MARCH 28, 2023

Under the terms of the deal, the company will receive non-exclusive rights to CRISPR/Cas9, a gene-editing technology of CRISPR Therapeutics, for the development of potentially curative T1D cell therapies. The gene-editing technology allows for precise, directed changes to genomic DNA.

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Scienmag

NOVEMBER 19, 2020

Media contacts: Jennifer Kuzma, jkuzma@ncsu.edu Khara Grieger, kdgriege@ncsu.edu Mick Kulikowski, News Services, 919.218.5937 or mick_kulikowski@ncsu.edu Researchers at North Carolina State University call for a coalition of biotech industry, government and non-government organizations, trade organizations, and academic experts to work together to (..)

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Fierce Pharma

NOVEMBER 27, 2023

The gene-editing therapy won a world-first approval in the U.K. earlier this month.

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BioTech 365

JANUARY 18, 2021

EdiGene Announces Approval of its IND Application for its CRISPR/Cas 9 Gene-editing Hematopoietic Stem Cell Therapy ET-01 in ?-thalassemia

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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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Pharmaceutical Technology

NOVEMBER 16, 2022

Under the deal, the LentiPeak lentiviral vector technology platform and cell therapy production expertise of ElevateBio BaseCamp will be used by Affini-T to develop its investigational oncogenic driver programmes in the clinic. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Drug Discovery World

NOVEMBER 27, 2023

A complex journey Biotherapeutics discovery and development upstream encounters challenges in cell line development (CLD), clone screening and characterisation, scale up, and eventually production. Next-generation engineering tools, such as gene editing have enabled knock-out cell lines with enhanced characteristics.

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Drug Discovery World

SEPTEMBER 25, 2023

CRISPR gene editing companies Integrated DNA Technologies (IDT) and Aldevron have inked a new global distribution agreement to expand CRISPR products for cell and gene therapy customers.

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Drug Discovery World

SEPTEMBER 6, 2022

Cell and gene therapies are not only transforming the way diseases are treated but are also aiming to cure entirely new patient populations. As researchers and developers work to transform the way we treat diseases, cell and gene therapies will continue to be the centre of innovation in the coming years. . billion raised.

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Roots Analysis

FEBRUARY 27, 2024

The global genome editing market is anticipated to grow at a CAGR of 12.6% How is the genome editing market landscape evolving: Currently, there is an evident increase in demand for complex biological therapies (including regenerative medicine products), which has created an urgent need for robust genome editing techniques.

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XTalks

DECEMBER 13, 2023

The US Food and Drug Administration (FDA) has approved the first gene therapies for the treatment of sickle cell disease, approving two on the same day. Both gene therapies are approved for individuals 12 years of age and older with sickle cell disease. It also affects Hispanic Americans, but at a lower prevalence.

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XTalks

NOVEMBER 3, 2023

After spending almost an entire day deliberating the safety of Vertex Pharmaceuticals’ and CRISPR Therapeutics’ CRISPR-based gene therapy exa-cel for sickle cell disease, a US Food and Drug Administration (FDA) advisory panel appears to be satisfied with what it saw. CRISPR works as genetic scissors to edit parts of the genome.

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XTalks

OCTOBER 20, 2023

Clinical-stage genome editing company Intellia Therapeutics has received clearance from the US Food and Drug Administration (FDA) for its Investigational New Drug (IND) application to start a pivotal phase III trial of NTLA-2001 for the treatment of transthyretin (ATTR) amyloidosis with cardiomyopathy.

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pharmaphorum

OCTOBER 21, 2020

million) to develop its gene therapy for a rare ocular disease. SPVN06 is a proprietary, mutation-agnostic, AAV gene therapy consisting of one neurotrophic factor and one oxidative stress reducing enzyme which, acting together, aim to slow or stop the degeneration of photoreceptors. French biotech SparingVision has raised 44.5

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Drug Discovery World

DECEMBER 11, 2023

The US Food and Drug Administration (FDA) has approved the first cell-based gene therapies for the treatment of sickle cell disease (SCD), Casgevy and Lyfgenia. Casgevy, from Vertex Pharmaceuticals and CRISPR Therapeutics, is also the first FDA-approved treatment to use CRISPR gene editing technology.

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Drug Discovery World

MAY 23, 2023

DDW’s Megan Thomas caught up with George White , General Manager, Product Management, Cell & Gene Therapy at Cytiva, to learn more about the company’s insight into the CGT market. Key challenges for cell and gene therapies In order for cell and gene (CGT) therapies to succeed, George White identifies several challenges to overcome.

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pharmaphorum

SEPTEMBER 14, 2022

SparingVision has raised €75 million in second-round funding that will be used to fund clinical trials of gene therapies for ocular diseases retinitis pigmentosa (RP) and dry age-related macular degeneration (AMD). The post SparingVision raises €75m for eye disease gene therapies appeared first on.

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Pharmaceutical Technology

AUGUST 26, 2022

ElevateBio has signed a long-term strategic collaboration with the University of Pittsburgh, US, to establish a biomanufacturing centre for expediting cell and gene therapy development. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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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. Recent Problems in Cell and Gene Therapy Development. Dealing with confusing regulatory guidelines is one of the top obstacles researchers face as they develop CGT products 1.

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pharmaphorum

MAY 7, 2021

Gene editing firm CRISPR Therapeutics has announced a collaboration with US biotech Nkarta to develop natural killer (NK) cell therapies for cancer. It’s at the forefront of gene editing although the technology has spawned rivals including Intellia Therapeutics, Caribou Biosciences, Sangamo Therapeutics and Mammoth Biosciences.

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Drug Discovery World

MAY 5, 2023

The acquisition will provide Eterna with a pipeline of allogeneic immuno-oncology products under development for the treatment of haematologic and solid tumours. In contrast to DNA-based reprogramming and gene editing, the mRNA-based approach does not expose cells to expensive and potentially harmful viruses or DNA vectors.

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Pharmaceutical Technology

JANUARY 19, 2023

GlobalData’s Pharma Intelligence Center shows 19 autologous CAR-T cell products in Phase I, and nine products in Phase II, globally. None of these products are in a registrational trial, and as such, GlobalData does not anticipate their entry into the AML market earlier than 2028.

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WCG Clinical

NOVEMBER 17, 2023

Although the NIH Guidelines were originally written with non-clinical laboratory research in mind, they also apply to human gene transfer (HGT) research, wherein rsNA or rsNA-containing products are administered to research participants. Since then, however, certain genetic engineering technologies (e.g.,

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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. After CRISPR editing, the T cells were expanded and infused in solid tumour mice models.

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BioTech 365

OCTOBER 26, 2020

.–(BUSINESS WIRE)–EdiGene, Inc. ( “Company”, or “EdiGene”), today announced the Center for Drug Evaluation (CDE) of China National Medical Products Administration (NMPA) has accepted for review the Company’s Investigational New Drug (IND) application for ET-01, autologous CD34+ … Continue reading → (..)

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Pharmaceutical Technology

JANUARY 10, 2023

Asklepios BioPharmaceutical has entered a research partnership and option agreement with ReCode Therapeutics for exploring its single-vector gene-editing platform. The new solution will enable complete gene insertion by delivering the gene-editing tool and DNA as mixed cargo to desired targets in one LNP.

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Pharmaceutical Technology

JUNE 15, 2023

Verve Therapeutics and Eli Lilly and Company have entered an exclusive research partnership to advance the former’s preclinical stage in vivo gene editing programme targeting lipoprotein(a) (Lp(a)) to treat atherosclerotic cardiovascular disease (ASCVD). The company will receive a combined upfront payment and equity investment of $60m.

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Drug Discovery World

AUGUST 11, 2023

GenScript will provide various CRISPR reagents to support the development of the universal CAR-T products from discovery to commercialisation. T-MAXIMUM Biotech hopes to progress multiple products to the Phase II clinical research stage and deliver products to market within the next five years.

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Scienmag

NOVEMBER 9, 2020

Their approach […].

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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 rights for etranacogene dezaparvovec were subsequently sold to CSL Pharmaceuticals.

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The Pharma Data

DECEMBER 5, 2020

5, 2020 — A pair of new gene therapies promise a potentially lasting cure for sickle cell disease by subtly altering the genetic information in patients’ bone marrow cells, researchers report. Selim Corbacioglu from the University of Regensburg, relies on CRISPR-Cas9 gene editing to switch off BCL11A. SATURDAY, Dec.

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