Pharmaceutical Technology

FEBRUARY 13, 2023

Several biotech companies and researchers are now exploring medical devices and gene therapies to address not just common forms of epilepsy, but also rare conditions such as Dravet Syndrome. Common gene therapies use vehicles such as adeno-associated vectors (AAVs), which cannot carry the required payload in this case, says Carney.

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Research Gene Gene Therapy Genome 264

pharmaphorum

OCTOBER 4, 2022

AstraZeneca’s rare disease firm Alexion is set to expand its genomic medicine portfolio with the acquisition of gene editing specialist LogicBio Therapeutics, in a deal worth approximately $68 million. The post AstraZeneca pays record 660% premium for gene editing company LogicBio appeared first on.

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Gene Editing Gene Genome Genomics 64

BioSpace

OCTOBER 26, 2021

With the complete acquisition of Life Edit, ElevateBio will be able to integrate that company’s genome editing capabilities with its cell and gene therapies.

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Gene Editing Gene Gene Therapy Genomics 76

Scienmag

OCTOBER 21, 2020

–Genome editing of human embryos represents one of the most contentious potential scientific applications today. But what if geneticists could sidestep the controversy by editing sperm and eggs instead? Credit: Photo by L. Brian Stauffer CHAMPAIGN, Ill.

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Gene Editing Gene Genomics Genome 88

pharmaphorum

JUNE 23, 2022

Novartis has shouldered its way into the in vivo gene editing category via a deal with US biotech Precision BioSciences, focused on a therapy for sickle cell disease (SCD). It is also working with Eli Lilly on in vivo gene-editing drugs against three targets, including one in Duchenne muscular dystrophy, under the terms of a $1.4

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Gene Editing In-Vivo Gene Sequencing Gene 97

XTalks

MAY 22, 2024

In this episode, Ayesha spoke with John Finn, PhD, Chief Scientific Officer at Tome Biosciences , a company developing programmable gene insertion (PGI) technology. PGI is a cutting-edge gene editing technology that allows for the insertion of large sequences of DNA with site-specific precision.

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Gene Editing Life Science Gene Gene Therapy 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.

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Gene Therapy Gene Production Clinical Trials 52

The Pharma Data

JANUARY 12, 2021

In December, life sciences giant Bayer launched a cell and gene therapy platform within its pharmaceutical division in order to become a leading company within a rapidly emerging and evolving field that offers the potential of life-saving therapies. However, she said the company wants to strengthen its position in gene editing.

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Gene Therapy Gene Gene Editing Business Development 52

The Pharma Data

JUNE 7, 2023

Bayer strengthens gene therapy portfolio with lipid nanoparticle technology from Acuitas Therapeutics Bayer AG is joining forces with Acuitas Therapeutics, Inc., “Accessing state-of-the-art LNP technology through this collaboration will add momentum to our gene editing efforts for the benefit of patients.”

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Gene Therapy Gene Gene Editing In-Vivo 40

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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Genomics Genome Gene In-Vivo 40

XTalks

MAY 4, 2021

The epigenome plays a key role in many diseases such as heart disease, viral infections and cancer, and the new CRISPRoff technology could lead to powerful epigenetic therapies. Epigenetic Editing with CRISPR. Nearly one third of human genes lack CGIs, which would limit the use of the tool.

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DNA Gene Gene Silencing Genome 98

XTalks

DECEMBER 20, 2023

CRISPR Gene Editing Therapeutics CRISPR/Cas-9 technology makes lists like this almost every year, and for good reason. Given its ability to precisely edit genes, there is tremendous promise for the revolutionary technology to offer cures for genetic disorders and to treat various diseases.

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Life Science Gene Editing Development Clinical Trials 119

XTalks

NOVEMBER 30, 2023

More Investigational Therapies for HIV Are on the Way American Gene Technologies, a pioneering biotech company headquartered in Rockville, Maryland, has released promising results from its Phase I trial of AGT103-T, a gene therapy designed for individuals with chronic HIV disease.

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FDA Approval Gene Therapy Clinical Trials Clinical Trials 111

The Pharma Data

DECEMBER 13, 2020

which develops genome editing technologies to accelerate drug discovery and develop novel therapeutics for a broad range of diseases, today announced the appointment of Bo Zhang, Ph.D., Prior to joining EdiGene, he was Vice President of KLUS Pharma and focused on cell therapy and new technologies. 14, 2020 10:00 UTC.

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Business Development Development Gene Editing In-Vivo 40

Pharma Marketing Network

DECEMBER 21, 2020

Almost two decades after the human genome was sequenced, a trickle of new genetic medicines (i.e., 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.

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Genetics Medicine Gene Therapy Marketing 52

XTalks

FEBRUARY 27, 2024

Technologies like CRISPR-Cas9 have revolutionized the potential for gene editing, offering hope for curative treatments for conditions like Duchenne muscular dystrophy (DMD) and certain types of inherited blindness. Both therapies were awarded their approvals on the same day in December 2023.

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Research Drugs Gene Therapy Clinical Trials 59

Cloudbyz

MARCH 6, 2024

Additionally, there is a growing need for regulatory frameworks to ensure the safety, efficacy, and affordability of biologic therapies, particularly as they become more prevalent in clinical practice. Looking ahead, the future of pharmaceutical therapies is undeniably intertwined with the continued advancement of biologics.

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Delveinsight

SEPTEMBER 14, 2021

Vor Biopharma has declared that the FDA has awarded their acute myeloid leukemia (AML) engineered hematopoietic stem cell therapy (eHSC), VOR33, a Fast Track Designation. VOR33 comprises CRISPR genome-edited hematopoietic stem and progenitor cells , which have been engineered to exclude CD33.

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RNA Clinical Trials Clinical Trials Drugs 67

XTalks

MAY 4, 2021

The epigenome plays a key role in many diseases such as heart disease, viral infections and cancer, and the new CRISPRoff technology could lead to powerful epigenetic therapies. Epigenome Editing with CRISPR. Nearly one third of human genes lack CGIs, which would limit the use of the tool.

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DNA Gene Gene Silencing Genome 52

The Pharma Data

NOVEMBER 3, 2020

LogicBio’s proprietary genome editing technology platform, GeneRide, enables the site-specific integration of a therapeutic transgene without nucleases or exogenous promoters by harnessing the native process of homologous recombination.

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Clinical Trials Clinical Trials Gene Editing Genome 40

Pharma Marketing Network

JANUARY 20, 2021

Regarding the use of direct-acting anti-SARS-CoV-2 therapeutics (such as nucleoside analogs, protease inhibitors, and monoclonal antibodies), the panel stressed the need for constant surveillance to track changes in the viral genome that might result from their use.

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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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Gene Therapy Gene Gene Editing In-Vivo 59

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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In-Vivo Gene Editing Trials Gene 52

XTalks

DECEMBER 24, 2020

From isolating SARS-CoV-2 in early January to sequencing its genome shortly thereafter and having a prototype vaccine against it within days, scientific process and progress have held steadfast throughout the pandemic. CRISPR Gene Editing Inventors Win Nobel Prize. The illness was later named coronavirus disease 2019 (COVID-19).

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Life Science Vaccine Vaccination Gene 111

XTalks

JANUARY 6, 2025

While these therapies span all stages of clinical development, they are particularly concentrated in earlier phases, indicating strong future growth. RNA-based therapies hold the potential to offer new treatment options for diseases with limited or inadequate therapeutic alternatives. billion in 2021, is projected to reach $42.56

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Pharmaceutical Technology

FEBRUARY 20, 2023

This may lead to therapies and dosing regimes being less suitable for minority populations – and according to Hans Bunschoten, chief strategy officer at clinical research company Cerba Research, part of Cerba Healthcare, this is a major problem. The technology platforms are almost the same.

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