pharmaphorum

AUGUST 24, 2021

Vertex Pharma has ramped up its involvement in gene-editing medicines for the third time in a matter of months, agreeing a partnership with CRISPR specialist Arbor Biotechnologies that could be worth up to $1.2

Gene Editing 52

Gene Editing Gene In-Vivo Genetics 52

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

Gene Editing 92

Gene Editing In-Vivo Gene Sequencing Gene 92

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.

Genome 40

Genome Genomics Gene Gene Editing 40

Pharmaceutical Technology

FEBRUARY 15, 2023

The company is largely involved in genome editing using CRISPR/Cas9 and CRISPR/Cas12a systems. Developing both in vivo and ex vivo gene editing medicines, Editas Medicine is primarily involved in the haematology and oncology therapy areas.

In-Vivo 162

In-Vivo Genome Genomics Gene Editing 162

Drug Discovery World

MARCH 10, 2023

Perhaps most interestingly this week, after previous caution over in vivo gene therapy trials, the FDA has given the go ahead to Intellia Therapeutics to study NTLA-2002 in hereditary angioedema (HAE). The post This week in drug discovery (6-10 March) appeared first on Drug Discovery World (DDW).

In-Vivo 52

In-Vivo Clinical Trials Clinical Trials Drugs 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.”

Gene Therapy 40

Gene Therapy Gene Gene Editing In-Vivo 40

Drug Discovery World

APRIL 15, 2024

Gene therapy will move to ex vivo gene modification and the delivery of gene-modified cell types. DDW’s Megan Thomas speaks to experts from the drug discovery industry about their predictions on the future of stem cells in drug discovery.

Gene Editing 59

Gene Editing Clinical Trials Clinical Trials In-Vivo 59

Drug Discovery World

AUGUST 4, 2023

CRADL Seattle is opening this September as Charles River’s most recent investment in Seattle, providing flexible vivarium space and on-demand in vivo study support services. In October, CRADL Philadelphia will open in uCity Square to support Philadelphia’s growth as a life sciences biohub.

Life Science 52

Life Science Gene Therapy Gene Editing In-Vivo 52

Drug Discovery World

APRIL 10, 2024

iECURE has been given the go-ahead to begin US trials of its investigational gene editing-based therapy in newborn males with neonatal onset Ornithine Transcarbamylase (OTC) deficiency. The post FDA approves first in vivo gene insertion programme in infants appeared first on Drug Discovery World (DDW).

In-Vivo 80

In-Vivo FDA Approval Gene Gene Editing 80

Drug Discovery World

FEBRUARY 16, 2023

Challenges remain with current delivery approaches, and genetic medicines applications have been generally restricted to the liver and ex vivo settings. Aera’s technology also includes a licensed therapeutic enzyme platform based on the discovery of novel, compact, and programmable gene editing enzymes.

Genetics 52

Genetics Medicine Gene Editing In-Vivo 52

pharmaphorum

FEBRUARY 19, 2021

Snipping out this viral code with powerful CRISPR gene editing technology, which last year won Drs Emmanuelle Charpentier and Jennifer Doudna the Nobel Prize for chemistry, would in theory prevent the need for drugs to suppress the virus and the development of AIDS. The post Could CRISPR cure HIV?

Gene Editing 105

Gene Editing In-Vivo DNA In-Vitro 105

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). Lilly will provide funding for the Phase I clinical trials.

Gene Editing 130

Gene Editing Gene Gene Therapy In-Vivo 130

Drug Discovery World

JANUARY 12, 2023

The genome is the blueprint for life, consisting of genes made up of thousands of pairs of DNA bases. Altering one base pair can drastically change how the gene’s protein product functions, while mutations can lead to the development of a range of genetic disorders. . What are organoids? .

Gene Editing 98

Gene Editing Gene Drugs In-Vivo 98

The Pharma Data

DECEMBER 13, 2020

“Our company and R&D portfolio are entering into an exciting phase, as evidenced by the recent close of Series B financing and submission of the first gene editing product IND in China,” said Dong Wei, Ph.D.?CEO Dr. Zhang has around 20 years of experience in research and drug development in both industry and academia in the US.

Business Development 40

Business Development Development Gene Editing In-Vivo 40

Drug Discovery World

JULY 3, 2023

From June 20-22, 2023, Cambridge Healthtech Institute hosted the seventh annual Immuno-Oncology Summit Europe in London.

Antibody 52

Antibody In-Vitro Engineer In-Vivo 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.

Gene Editing 52

Gene Editing Gene In-Vivo Genome 52

Delveinsight

FEBRUARY 25, 2021

The gene-editing technologies, which have reached the clinic, CRISPR, zinc finger nucleases, and TALENs, make their edits by nicking DNA at the target site. Instead of snipping the genome, base editing lets for edits of individual letters in a genetic sequence.

DNA 52

DNA In-Vivo Genetics Medicine 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. . — Eric Topol (@EricTopol) June 26, 2021. — Eric Topol (@EricTopol) June 26, 2021.

In-Vivo 103

In-Vivo Gene Editing Trials Drugs 103

Drug Discovery World

NOVEMBER 16, 2022

DDW Editor Reece Armstrong learns about Molecular Devices’ Organoid Innovation Centre (OIC) and how the company is aiming to advance research into 3D biology and organoids. . 2D biology has been used by pharmaceutical researchers for decades. 2D vs 3D .

Drugs 97

Drugs In-Vivo In-Vitro Scientist 97

Drug Discovery World

JUNE 7, 2023

DDW’s Diana Spencer chats to Macomics’ co-founder and VP Immunology Dr Luca Cassetta about the company’s approach to tumour suppression and the challenges of launching a new business in early 2020. DS: Congratulations on the publication of your discoveries in Nature Reviews Cancer. Your studies focus on tumour associated macrophages (TAMs).

In-Vivo 52

In-Vivo Gene Editing Engineer Bioinformatics 52

Drug Discovery World

APRIL 3, 2023

DS: What motivated you and Dr David Bunka, CTO, to create Aptamer and did you imagine you would one day be operating on such a global scale?    AT : We initially met at the University of Leeds, where David was a post-doctoral researcher studying aptamers, and I was completing my PhD. Optimer binders are engineered to overcome these problems.

Antibody 52

Antibody Gene Therapy Gene In-Vitro 52

XTalks

DECEMBER 20, 2023

AI and Machine Learning: Transforming Research Artificial intelligence (AI) and machine learning (ML) are revolutionizing research approaches in the life sciences. CRISPR Gene Editing Therapeutics CRISPR/Cas-9 technology makes lists like this almost every year, and for good reason.

Life Science 119

Life Science Gene Editing Development Clinical Trials 119

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.

Gene Editing 52

Gene Editing Gene In-Vivo Genetics 52

Drug Discovery World

JANUARY 23, 2023

Kevin Hemphill, R&D Manager at PerkinElmer’s Horizon Discovery explores how base editing has emerged as an attractive gene editing option for researchers wanting to develop stem cell-based therapies. Pioneering work in the mid-2000s, led by the Yamanaka group at Kyoto University, further advanced the field of PSCs.

Gene Editing 52

Gene Editing Gene Engineer DNA 52

Drug Discovery World

DECEMBER 12, 2022

The meeting will focus on the application of genome engineering in identification of novel drug targets through large-scale CRISPR based functional genomics studies, developing advanced cellular and in vivo disease models for target validation, and its applications in therapeutic genome editing. Register here.

Genome 52

Genome Genomics Engineer In-Vivo 52

The Pharma Data

DECEMBER 11, 2020

of Children’s Hospital at TriStar Centennial in Nashville, Tennessee, and colleagues found that gene editing using CRISPR-Cas9 is safe and feasible and can provide clinical benefit to patients with sickle cell disease and transfusion-dependent ß-thalassemia. In one study, Haydar Frangoul, M.D., Abstract No.

Gene Editing 52

Gene Editing Gene Gene Therapy In-Vivo 52

Drug Discovery World

JULY 18, 2023

Since its discovery in 2012, CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) technology has revolutionised the biomedicine and cell and gene therapy fields, providing a versatile tool for precise and efficient genome editing.

DNA 98

DNA Genome Genomics RNA 98

The Pharma Data

NOVEMBER 22, 2020

Scientists in Israel have used the CRISPR Cas-9 gene editing system to destroy cancerous cells in mice without damaging other cells. To conduct the research, the scientists used hundreds of mice with two of the most aggressive forms of cancer: glioblastoma and metastatic ovarian cancer. Photo courtesy of Science Advances.

Gene Editing 52

Gene Editing DNA Genome Genomics 52

The Pharma Data

JANUARY 10, 2021

EDIT-301 is an experimental, ex vivo gene editing cell medicine in development for the treatment of sickle cell disease. Previously, the Company received Rare Pediatric Disease designation from the FDA for EDIT-301. Clinical trial materials are being manufactured by Editas Medicine.

Medicine 52

Medicine Development Clinical Trials Clinical Trials 52

XTalks

FEBRUARY 10, 2023

According to a recent review published in Nature , as of April 2022 almost 1,800 active cell therapy clinical trials were listed in ClinicalTrials.gov, a 33 percent increase from 2021. The rapidly expanding landscape of cell therapy trials has put an operational strain on many clinical sites due to limited resources and infrastructure. “In

Clinical Trials 52

Clinical Trials Clinical Trials Trials Gene Therapy 52

Drug Discovery World

FEBRUARY 26, 2024

And it’s not just bit.bio – there are many other companies exploring different cell types within clinical research. Moreso, research using natural killer cells blossomed in 2023 with a number of companies targeting use-cases in Alzheimer’s oncology, and even diabetes. It’s safe to say there is a lot of potential for cell therapies.

Manufacturing 52

Manufacturing In-Vivo Development Production 52

Drug Discovery World

NOVEMBER 30, 2023

DDW’s Megan Thomas attended PEGS Europe 2023 in Lisbon, Portugal, from Tuesday 14 November to Thursday 16 November 2023. The company’s goal is to accelerate functional characterisation and shortenoptimisation time in antibody discovery and t-cell workflows. GSK has developed high-throughput mammalian and E.

Antibody 59

Antibody Protein Engineer In-Vivo 59

Drug Discovery World

FEBRUARY 22, 2024

Recent advances in biomarkers, research into the central nervous system and breakthroughs in human iPSC cell models, have bolstered neuroscience drug discovery research. ARUK 3 adds that while the UK government increased funding in dementia research from £28.2m In 2019, dementia accounted for 1.8 million deaths worldwide” 1.

Drugs 59

Drugs Gene Clinical Trials Clinical Trials 59