pharmaphorum

SEPTEMBER 14, 2020

Here he gives us a deeper look at how genomic medicine is evolving and the barriers that are preventing it from reaching its full potential. I saw this, in particular, with the finishing of the human genome,” says Charlie. “At In reality, finishing the human genome was the first step of what is a long journey.”.

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Genome Genomics Medicine Gene 111

pharmaphorum

FEBRUARY 11, 2022

Data from the first patients enrolled into Regenxbio’s trial of its gene therapy for rare inherited disease mucopolysaccharidosis type I (MPS I) – also known as Hurler syndrome – has shown the first signs of clinical activity. The post Regenxbio expands MPS I gene therapy trial after first look at data appeared first on.

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Gene Therapy Gene Trials FDA Approval 109

pharmaphorum

JANUARY 29, 2021

Genome editing is an exciting but still nascent field, and companies in the area face as many obstacles as they do opportunities. Maybe in 50 years’ time we’ll be using gene editing to lower cholesterol, but it won’t replace statins in anyone but those with life threatening mutations for a long time”. Zinc fingers.

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Gene Editing Gene Gene Therapy In-Vivo 144

pharmaphorum

OCTOBER 12, 2022

David Del Bourgo (CEO and co-founder, Whitelab Genomics) has always been passionate about introducing disruptive, innovative technologies to markets. We founded Whitelab Genomics after realising the potential to use data, data science, and AI in a more systematic way to develop genomic therapies,” Del Bourgo says.

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Genome Genomics Medicine Development 96

Worldwide Clinical Trials

DECEMBER 12, 2022

Genetic testing provides patients with a diagnosis for their illness, helps patients and family members to understand risks of developing new diseases, and can be used to support clinical trial advancement. Can prior genetic testing results be utilized in a trial, or should a new genetic sample be taken before participating?

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Clinical Trials Clinical Trials Genetics Trials 130

Pharmaceutical Technology

APRIL 12, 2023

FoundationOne CDx is a sequencing-based in vitro diagnostic device that can identify alterations in 324 genes from tumour samples. The ROS1 gene is altered in approximately in 1-2% of lung cancer patients. Exposure to environmental factors can cause gene fusion which leads to upregulation of the ROS-1 enzyme.

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Genome Genomics In-Vitro Gene 130

Worldwide Clinical Trials

JUNE 15, 2022

At the end of May, we hosted a webinar titled “ Changing Times, Changing Therapies: Keeping Up with Advancements in Cell and Gene Therapies ” to provide a quick update on the latest advancements and ongoing in development of these advanced therapeutics. Clinical holds are becoming more common, especially in gene therapy programs.

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Gene Therapy Gene Clinical Trials Clinical Trials 130

Pharmaceutical Technology

OCTOBER 26, 2022

The biotherapeutics market is rapidly growing, with 2021 seeing the highest-ever cell and gene therapy approval number. Gene therapy uses DNA to manipulate cells and correct defective genes, whereas cell therapy is the infusion or transplantation of cells into a patient. The future of cell and gene therapies.

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Gene Therapy Gene Marketing Manufacturing 130

Drug Discovery World

JUNE 23, 2023

Sarepta Therapeutics’ Elevidys has become the first gene therapy for Duchenne muscular dystrophy (DMD) to gain marketing authorisation in the US. The Food and Drug Administration (FDA) has granted accelerated approval to Elevidys (delandistrogene moxeparvovec-rokl), an adeno-associated virus (AAV) based gene therapy for the treatment of DMD.

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Gene Therapy FDA Approval Gene Protein 97

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

Drug Discovery World

JUNE 21, 2023

The European Confederation of Pharmaceutical Entrepreneurs (EUCOPE), Exact Sciences, Guardant Health, Illumina, MSD and Novartis have come together to launch the European Coalition for Access to Comprehensive Genomic Profiling (ECGP).

Genome 52

Genome Genomics Medicine Clinical Trials 52

pharmaphorum

NOVEMBER 27, 2022

A group representing pharma companies selling precision therapies for cancer has called for a change to the way genomic testing is done in Scotland, to make sure patients get access to targeted drugs. The post Genomic testing ‘should be offered to all cancer patients in Scotland’ appeared first on. Why not lead?”

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Genome Genomics Genetics Medicine 89

Drug Discovery World

MARCH 9, 2023

The post FDA clears in vivo gene editing therapy for trials in US appeared first on Drug Discovery World (DDW).

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In-Vivo Gene Editing Gene Trials 52

XTalks

SEPTEMBER 9, 2021

The US Food and Drug Administration (FDA) has placed a clinical hold on BioMarin Pharmaceutical’s investigational gene therapy BMN 307 for the rare inherited disease phenylketonuria (PKU) over safety concerns found during preclinical testing. The mice developed the tumors one year after being given BMN 307.

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Gene Therapy Gene Genome Genomics 105

Pharmaceutical Technology

SEPTEMBER 22, 2022

You have just received the results from your whole genome sequencing test, offered through your public health provider, and discovered that you have a 75% chance of developing a rare form of cancer. Alternatively, there are vaccines ready to enter decentralised third-phase clinical trials, so you can enrol quickly. It is the year 2030.

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Genome Genomics Clinical Trials Clinical Trials 342

Scienmag

OCTOBER 13, 2020

NCI-MATCH precision medicine trial makes major contributions to the use of tumor gene testing to select treatment for patients; new data has broad relevance in medicine and provides a roadmap for future precision medicine trials Credit: ECOG-ACRIN Cancer Research Group Five years ago, the ECOG-ACRIN Cancer Research Group (ECOG-ACRIN) and National Cancer (..)

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Clinical Trials Clinical Trials Trials Genome 88

pharmaphorum

AUGUST 31, 2022

After a recent approval, there are now three gene therapies available on the US market. In recent years, gene therapy has transitioned from a promising idea to a reality for patients, with many of the severe safety issues that emerged in early iterations of the technology being overcome. from 2021 to 2029.

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Gene Therapy Gene Gene Editing DNA 88

pharmaphorum

MARCH 10, 2021

bluebird bio is to ask regulators to restart clinical studies of its LentiGlobin for sickle cell disease, after an investigation concluded that a case of acute myeloid leukaemia (AML) was “very unlikely” to be caused by the gene therapy. He added: “We have seen no significant gene misregulation attributable to the insertion event.

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Gene Therapy Gene Trials Gene Expression 52

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

Drug Discovery World

DECEMBER 8, 2023

Andrew Busey , Co-Founder, Form Bio discusses why vertical AI is set to reshape the cell and gene therapy sector. The cell and gene therapy industry is no exception to this digital transformation. General AI tools such as ChatGPT have acted as a catalyst, igniting a wave of AI innovation akin to the early days of the internet era.

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Gene Therapy Gene In-Vitro Development 64

BioSpace

OCTOBER 18, 2021

On Monday, LogicBio Therapeutics unveiled clinical trial results demonstrating the first-ever in vivo, nuclease-free genome editing in little humans.

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In-Vivo Gene Genome Genomics 93

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.

In-Vivo 52

In-Vivo Gene Editing Trials Gene 52

Scienmag

JANUARY 19, 2021

publishers New Rochelle, NY, January 19, 2021–Gene editing therapies, including CRISPR-Cas systems, offer the potential to correct mutations causing inherited retinal degenerations, a leading cause of blindness. Credit: Mary Ann Liebert, Inc.,

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Gene Editing Genome Genomics Gene Therapy 101

Drug Discovery World

DECEMBER 6, 2023

Gene editing company Eligo Bioscience has announced a successful $30 million Series B funding round, led by Sanofi Ventures. “We The investment will fuel pre-IND and IND activities to achieve early human data readouts in a PhIb/IIa clinical trial.

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Gene Editing Gene In-Vivo Genetics 52

Drug Discovery World

MARCH 27, 2023

Genomics is driving a revolution in drug development and, in the long run, the state of public healthcare. The first draft human genome took a decade to create, and 13 years to complete. Advances in sequencing are leading to scientific breakthroughs in preventative care, faster diagnoses, and proactive medical interventions.

Genome 52

Genome Genomics Research Scientist 52

pharmaphorum

MARCH 15, 2022

Gene sequencing specialist Illumina has started rolling out a new test that looks for a battery of tumour-associated genes from a single tissue sample, and could be used to help guide treatment for a broad range of cancers. “Comprehensive genomic profiling has proven to be this platform.”

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Gene Genome Genomics Gene Sequencing 52

pharmaphorum

JUNE 28, 2021

The first ever clinical data with a CRISPR/Cas9 drug used to edit the genomes of cells within the body has yielded impressive results in patients with ATTR amyloidosis, a life-threatening rare disease. . Intellia and Regeneron’s phase 1 trial looked at patients who had nerve damage (polyneuropathy) as a result of the disease.

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In-Vivo Gene Editing Trials Drugs 96

Drug Discovery World

MARCH 13, 2023

GenSight Biologics has revealed pooled safety data from five clinical studies of lenadogene nolparvovec (Lumevoq), the largest cohort of ND4-LHON (Leber hereditary optic neuropathy) patients studied after gene therapy treatment. The post Five-year data show gene therapy safe for ND4-LHON appeared first on Drug Discovery World (DDW).

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Gene Therapy Gene Immune Response Genome 52

The Pharma Data

OCTOBER 26, 2021

The AVENIO Tumor Tissue Comprehensive Genomic Profiling (CGP) Kit is the first jointly-developed product that brings together the expertise and reach of Roche with Foundation Medicine’s pioneering leadership in genomic science. Cancer is a disease of the genome and treatment no longer depends solely on the tissue of origin.

Genome 52

Genome Genomics Research Medicine 52

Drug Discovery World

FEBRUARY 27, 2023

An investigational gene therapy for Sanfilippo syndrome – which leads to a form of childhood dementia – has shown promising early results in a proof-of-concept study. These molecules then accumulate in the cells of the body causing irreparable damage to many organs including the brain, leading to inflammation and damage to brain tissue.

Gene Therapy 52

Gene Therapy Gene Genetics Genetic Disease 52

pharmaphorum

JULY 13, 2022

Verve Therapeutics has started dosing patients in a phase 1b trial of its in vivo gene-editing drug for high cholesterol, designed to permanently switch off the PCSK9 gene with a one-shot treatment. Early trials suggest it as efficacy that matches the injectables, but the winner in the PCSK9 category remains hard to pick.

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Gene Editing Trials Drugs Gene 98

Drug Discovery World

MAY 17, 2024

Deepika Khedekar , Associate Centralized Clinical Trial Lead at IQVIA Inc, looks at the challenges of utilising artificial intelligence (AI) in oncology clinical research. MIT research 4 tells us a hard truth: about 95% of these trials fall short, leaving behind a trail of broken hopes for cancer patients each year.

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Trials Clinical Trials Clinical Trials Protein 59

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. Current Regulatory Trends in Cell & Gene Therapy. Trend One: Expected Growth in Cell and Gene Therapy IND filings.

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Gene Therapy Gene Production Clinical Trials 52

STAT News

JANUARY 30, 2023

The human genome was sequenced in 2003. It is now possible to treat diseases with gene therapy, antisense oligonucleotides, messenger RNA (mRNA), noncoding RNA (known as small interfering RNA, or siRNA), and other gene-based modalities. New ways of conducting clinical trials have also emerged. Read the rest…

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RNA Gene Therapy Drugs Gene 111

Pharmaceutical Technology

JULY 29, 2022

CAMP4’s CSO David Bumcrot PhD tells Pharmaceutical Technology that the company plans to see clinical trials go forward for their urea cycle disorder programs late next year. The most common marketed drugs in this space aim to address the hyperammonaemia caused by the defective genes in this disorder.

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RNA Gene Expression Gene Gene Therapy 245

pharmaphorum

APRIL 27, 2021

Dosing can start once again in uniQure’s phase 3 trial of its haemophilia B gene therapy, after the FDA concluded that a case of liver cancer seen in the study was unlikely to be caused by the treatment. . The post FDA lifts hold on uniQure gene therapy after cancer case review appeared first on.

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Gene Therapy Gene Genome Genomics 52

XTalks

AUGUST 29, 2022

Now a common gene editing tool, the popularity of the CRISPR-Cas9 system has increased over the past decade. CRISPR is notable for engineering living cells, allowing scientists to edit, turn off, delete, or replace genes in a cell’s genome. Harnessing the Cellular Engineering Potential of CRISPR.

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