pharmaphorum

JANUARY 29, 2021

Cutting edge’ is, for once, a truly apt description when it comes to gene editing – both because the field is pushing medicine into areas we might never have dreamed possible, and because these technologies involve literally cutting DNA at a specific point in the genome. Zinc fingers. billion in funding.

Gene Editing 142

Gene Editing Gene Gene Therapy In-Vivo 142

Drug Discovery World

APRIL 3, 2023

As the industry’s awareness of alternatives to traditional antibodies continues to grow, there is increasing demand for these alternatives, which we are seeing particularly within the therapeutic sector, to enable targeted therapeutics and enable the emerging gene therapy market. DS: The company now has clients across big pharma.

Antibody 52

Antibody Gene Therapy Gene In-Vitro 52

Drug Discovery World

OCTOBER 27, 2023

With cues from a diverse population of tissue-specific cell types, extracellular matrix proteins, and biomechanical forces, cells grown in organ-chips closely emulate their in vivo counterparts and replicate tissue-level functions. But can organ-chips entirely replace other models? Not yet, according to Ewart.

Research 72

Research Gene Therapy In-Vivo In-Vitro 72

Roots Analysis

JANUARY 14, 2024

Moreover, the exuberant development of biologics has revolutionized the treatment of a range of therapeutic conditions, which has further contributed to the exponential growth in the current demand for biologic therapies developed by biologics manufacturing companies.

Contract Manufacturing 40

Contract Manufacturing Manufacturing Protein Bacteria 40

Delveinsight

JANUARY 12, 2021

Birinapant is a clinical-stage SMAC mimetic that degrades Inhibitors of Apoptosis Proteins (IAPs) by binding to them, ultimately leading to cell death in tumor cells. The companies believe that their candidate in its original IgG format has shown potent neutralization activity in in vitro assays and in an in vivo animal model.

Licensing 52

Licensing Licensing Antibody Gene Therapy 52

XTalks

AUGUST 19, 2022

Bluebird bio’s gene therapy Zynteglo (betibeglogene autotemcel, beti-cel) has been awarded a much anticipated approval from the US Food and Drug Administration (FDA) for the treatment of adult and pediatric patients with beta thalassemia who need regular blood transfusions. Bluebird has a total of three gene therapies in its pipeline.

Gene Therapy 95

Gene Therapy FDA Approval Gene Clinical Trials 95

XTalks

FEBRUARY 27, 2024

Since the activity of TILs can be suppressed by tumor cells, TIL therapies are designed to bolster the number of TILs. Related: FDA Issues Letters to CAR T-Cell Therapy Makers to Include Boxed Warning Amtagvi was approved through the FDA’s Accelerated Approval pathway. TILs are immune cells that attack and penetrate tumors.

FDA Approval 52

FDA Approval Manufacturing In-Vivo Trials 52

Drug Discovery World

MARCH 9, 2023

This includes the measurement of plasma kallikrein protein levels and clinical activity as determined by HAE attack rate measures. The post FDA clears in vivo gene editing therapy for trials in US appeared first on Drug Discovery World (DDW).

In-Vivo 52

In-Vivo Gene Editing Gene Trials 52

Drug Discovery World

OCTOBER 13, 2022

Over the two days at ExCel, 1554 delegates attended the event. Of those delegates, 75% work in drug discovery. There were 127 exhibitors, 180 posters, 40 technology speakers, 52 scientific speakers as well as a series of eight seminars focused on automation and technology which DDW organised and moderated in the dedicated Tech Theatre. .

Drugs 52

Drugs In-Vivo In-Vitro Biobanking 52

Roots Analysis

AUGUST 31, 2023

Overview of Gene Switch The notion that genes might be turned on and off was discovered several decades ago when studies revealed that E. Gene switches are sites on genes where regulatory molecules can bind to trigger transcription process, leading to expression of a particular gene.

Gene 40

Gene Gene Therapy Gene Expression Regulation 40

Drug Discovery World

NOVEMBER 17, 2023

The breaking news this week was certainly the first regulatory approval for Casgevy, not only the first gene therapy approved for sickle cell disease, but also the first therapy to use CRISPR gene editing. However, there were a number of other breakthroughs announced in the cell and gene therapy field.

Gene Therapy 52

Gene Therapy Gene Drugs Gene Editing 52

Drug Discovery World

SEPTEMBER 28, 2022

Yet despite this, the biggest players in the global pharma sector are increasingly opening their doors to partnerships with AI and machine learning companies, cognisant of the potential benefits these could provide across broad therapy areas such as oncology, autoimmune and rare diseases. . The simple answer is no.

Pharma Companies 52

Pharma Companies Drugs In-Vivo Genetics 52

Drug Discovery World

NOVEMBER 9, 2023

Ahead of protein and antibody engineering conference PEGS Europe 2024 in Lisbon, DDW’s Megan Thomas looks at what to expect from each track of the annual biologics technology meeting. Croasdale-Wood will then participate in a keynote chat, interviewed by Miho.

Engineer 52

Engineer Antibody Protein In-Vivo 52

Drug Discovery World

NOVEMBER 13, 2023

Ahead of protein and antibody engineering conference PEGS Europe 2024 in Lisbon, DDW’s Megan Thomas looks at what to expect from each track of the annual biologics technology meeting. Ali Madani, PhD, Founder and CEO, Profluent Bio, on: ‘Protein engineering with large language models’.

Protein 59

Protein Antibody Engineer Scientist 59

The Pharma Data

AUGUST 3, 2021

SVP, Chief Medical Officer, Novartis Gene Therapies. “We SVP, Chief Medical Officer, Novartis Gene Therapies. “We Additionally, STEER will add to the clinical data and emerging real-world evidence for the use of gene therapy to treat SMA. 2 years and <5 years old with SMA Type 2.

Clinical Trials 52

Clinical Trials Clinical Trials Gene Therapy Trials 52

Drug Discovery World

APRIL 12, 2023

Existing in vivo and in vitro models aren’t up to the challenge Preclinical tools exist for the evaluation and screening of potential drug candidates. Existing in vivo and in vitro models aren’t up to the challenge Preclinical tools exist for the evaluation and screening of potential drug candidates.

Development 52

Development In-Vivo Drugs In-Vitro 52

Drug Discovery World

JANUARY 20, 2023

Existing in vivo and in vitro models aren’t up to the challenge Preclinical tools exist for the evaluation and screening of potential drug candidates. Existing in vivo and in vitro models aren’t up to the challenge Preclinical tools exist for the evaluation and screening of potential drug candidates.

Development 52

Development In-Vivo Drugs In-Vitro 52

Drug Discovery World

DECEMBER 15, 2022

These platforms all induce double strand cuts in the chromosomal DNA that can be sealed by the cell, leading to a specific gene disruption, or resulting in a new target site for inserting DNA segments. Gene editing tools can sometimes bind to unintended sites, typically because of sequence homologies and/or mismatch tolerance.

Gene Editing 52

Gene Editing Gene In-Vivo Genome 52

The Pharma Data

MARCH 20, 2023

Children in LT-001 treated after SMA symptom onset maintained or achieved additional milestones up to 7.5 Children in LT-001 treated after SMA symptom onset maintained or achieved additional milestones up to 7.5 years post-dosing, children who were treated after presenting symptoms of SMA maintained all previously achieved motor milestones.

Gene Therapy 40

Gene Therapy Gene In-Vivo Licensing 40

Delveinsight

JANUARY 18, 2021

Usually, only a single copy of the gene is required that too from the mother, however, in cases of AS, either the child does not get any copy, or the child receives two copies – each from mother and father. GeneTx and Ultragenyx are co-developing the therapy.

Marketing 52

Marketing Gene Drugs Protein 52

Drug Discovery World

JULY 18, 2023

Rolf Turk , Senior Manager, Genomics Medicine at Integrated DNA Technologies, examines how CRISPR is being used to enhance cancer therapies. Better, faster, stronger: targeting cancer with precision by CRISPR-enhanced CAR T therapies Cancer is a global public health crisis that demands powerful, innovative solutions in the battle against it.

DNA 98

DNA Genome Genomics RNA 98

Drug Discovery World

OCTOBER 13, 2022

One aspect that makes mRNA technology popular with drug developers is that its proteins can be easily edited. One aspect that makes mRNA technology popular with drug developers is that its proteins can be easily edited. Why mRNA has potential . mRNA presents versatility plus flexibility to target a broader range of diseases. .

Drugs 52

Drugs Manufacturing Vaccination Vaccine 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. Adult fibroblasts were cultured in a cocktail of transcription factors to reactivate embryonic gene expression patterns 2.

Gene Editing 52

Gene Editing Gene Engineer DNA 52

The Pharma Data

APRIL 27, 2021

Strong increase in Q1 2021 business EPS ( 1) at CER. Q1 2021 sales increase of 2.4% at CER driven by growth drivers Dupixent ® and Vaccines. Specialty Care sales grew 15.3%, due to strong Dupixent ® performance (+45.6% to €1,047 million) and oncology launches. General Medicines core assets grew 4.4%, while GBU sales were down 3.8%.

Sales 52

Sales Vaccination Vaccine Medicine 52

Drug Discovery World

FEBRUARY 22, 2024

According to Medicine Discovery Catapult, these include: “Navigating the complexity of the CNS; understanding the disease pathology, and accessing predictable in vitro and in vivo cell models 2 ”. Dementia is the only major cause of death without a treatment to prevent, slow or stop disease progression. “In In 2019, dementia accounted for 1.8

Drugs 59

Drugs Gene Clinical Trials Clinical Trials 59