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 108

Drug Discovery World

MAY 5, 2023

Anthony Schmitt , PhD, Senior Vice President, Science at Arima Genomics, discusses the applications of 3D genomics in cancer research. But what about the patients whose genomic tests do not identify a cancer driver matched to an existing targeted therapy? with regards to detecting cancer driver mutations.

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Genome Genomics Gene Genetics 52

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 142

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

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 92

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.

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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).

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Genome Genomics Medicine Clinical Trials 52

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

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?”

Genome 85

Genome Genomics Genetics Medicine 85

XTalks

APRIL 24, 2023

Awareness of rare diseases is growing, and with a better understanding of the pathophysiology of many rare diseases, innovative treatment options are emerging, like gene therapies that can treat the root cause of rare genetic diseases and potentially provide long-term symptom relief, or even a definitive cure.

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Gene Therapy Gene Trials Clinical Trials 95

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

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

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

NOVEMBER 7, 2022

These tumor-derived entities are used to derive genomic and proteomic data. Such information can reveal details about tumor staging, tumor progression, heterogeneity, gene mutations and clonal evolution. Liquid Biopsy Use in Oncology Clinical Trials. Guardant360 ™ (Guardant Health; end-to-end commercial provider).

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Clinical Trials Clinical Trials Trials FDA Approval 98

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

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 We are excited to lead this financing for Eligo and support the company as they move to the clinic,” said Laia Crespo, Partner at Sanofi Ventures. “We

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Gene Editing Gene In-Vivo Genetics 52

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.

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Gene Therapy Gene Trials FDA Approval 109

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.

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RNA Gene Therapy Drugs Gene 111

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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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

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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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.

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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.

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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

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

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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Gene Editing DNA Gene Genome 98

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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Drug Discovery World

JANUARY 26, 2023

The modernisation of R&D models could be key to reducing spend on new drugs, a point that Deloitte makes about the need to transform the clinical trials process. At the height of the pandemic, DCTs were seen as a viable alternative to keeping the engines running on clinical trials when non-critical in-person care was paused.

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Drug Discovery World

FEBRUARY 6, 2024

Her current work focuses on advancing the fields of genome sequencing and analysis, with an emphasis on identifying complex structural variation. She speaks to Megan Thomas on how genomics can advance drug discovery and development. MT: Where is technology being implemented to progress the drug discovery process?

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Genome Genomics Genetics In-Vitro 59

Drug Discovery World

FEBRUARY 12, 2024

We’re already seeing biotechs making breakthroughs with AI in this area, with more than 150 small molecule drugs discovered and 15 already in clinical trials. Matt Todd, Head of Architecture, Ori Biotech “We expect AI and data analytics to support pivotal advances in cell and gene therapy (CGT) manufacturing.

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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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XTalks

MAY 23, 2024

Eterovic brings much knowledge and experience to the table, having led the development of a new Oncology Diagnostic program at Eurofins Viracor that integrates the latest genomic profiling techniques. Eterovic highlights that the financial and logistical barriers to comprehensive genomic profiling are high but not impossible to overcome.

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Drug Discovery World

SEPTEMBER 6, 2022

DDW Editor Reece Armstrong looks at the cell and gene therapy landscape, examining the challenges facing developers and the trends we can expect to see throughout the year. . There’s no doubt that cell and gene therapies present some of the most exciting opportunities for emerging drugs. billion, compared to $19.9 Cancer is king .

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Gene Therapy Gene Manufacturing Medicine 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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Drug Discovery World

NOVEMBER 14, 2023

It is caused by a single gene mutation that impairs the body’s ability to mediate LDL cholesterol. Since the mutation only affects a single gene, it makes it a prime candidate for genome editing treatments. The data showed that Verve-101 could meaningfully and durably lower LDL-C in these patients.

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In-Vivo Gene Editing Gene Genome 52

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.,

Genome 52

Genome Genomics Gene Editing Genetic Engineering 52

Drug Discovery World

MARCH 13, 2023

The cell and gene therapy (CGT) landscape has grown significantly in the past year. Between the 24 therapies already approved by the FDA 1 and a marked increase in clinical trials, widespread accessibility to precision medicine feels within reach.

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