Drug Discovery World

AUGUST 15, 2023

Reliable analytical methods such as digital PCR to characterise lentiviral vectors and the cells transduced with them are critical to helping ensure the safety and efficacy of these biologics.

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Gene Therapy In-Vivo Gene DNA 52

Drug Discovery World

AUGUST 14, 2023

Lung-I Cheng, Vice President and Head of Cell & Gene Therapy Service Line at AmerisourceBergen, and Cori Gorman, Senior Director of CMC and Regulatory Affairs at Biopharma Excellence, offer advice on navigating the different and sometimes contradictory regulatory requirements in the US and EU. billion in 2020 to $15.5

Gene Therapy 52

Gene Therapy Marketing Gene DNA 52

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.

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Gene Editing Gene Gene Therapy In-Vivo 145

Drug Discovery World

AUGUST 15, 2023

Recombinant lentiviral vectors are used for ex vivo transgene delivery in several gene-modified cell therapies, such as chimeric antigen receptor (CAR) T cell therapy.

In-Vivo 52

In-Vivo DNA Genome Genomics 52

WCG Clinical

MAY 28, 2024

Recent headlines have highlighted the potential for Chimeric Antigen Receptor (CAR)-based therapies to provide clinical benefits to persons affected by lupus. An expected and well-known side effect of these B cell-targeted therapies is “B cell aplasia”— i.e. partial or complete depletion of B cells from circulation and immune organs.

In-Vivo 52

In-Vivo Clinical Trials Clinical Trials FDA Approval 52

XTalks

DECEMBER 20, 2023

In 2024, pharma companies and emerging biotechs will improve candidate safety profiles and reduce off-target effects by exploiting predictive intelligence unlocked by AI,” she predicts. This year, the very first CRISPR-based therapy received FDA approval for sickle cell disease.

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Life Science Gene Editing Development Clinical Trials 119

Drug Discovery World

JULY 5, 2024

The last few days have seen some interesting developments related to gene editing, including the discovery of a new mechanism for genetic programming and evidence in favour of redosing CRISPR-based therapies, as well as significant investment and new indications for gene therapies.

Gene Therapy 59

Gene Therapy Gene Editing In-Vivo Gene 59

Drug Discovery World

MAY 15, 2024

In vivo proof-of-concept for Circio Holding’s differentiated circVec platform approach to gene therapy has been demonstrated in two posters at the American Society of Gene & Cell Therapy (ASGCT) 2024 annual meeting. DNA vectors compared to standard linear mRNA-based expression. “We

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RNA In-Vivo Gene Therapy Genetic Disease 52

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

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

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.

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Gene Editing Gene In-Vivo Genome 52

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

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

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. CRISPR also addresses another limitation in CAR T therapies related to the insufficient quantity and poor quality of autologous T cells.

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DNA Genome Genomics RNA 98

Drug Discovery World

NOVEMBER 9, 2023

Klaus Wagenbauer, PhD, Founder & CEO, Plectonic, on: ‘Programmable DNA-origami-based T cell engagers: PTE’. 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.

Engineer 52

Engineer Antibody Protein In-Vivo 52

Roots Analysis

SEPTEMBER 18, 2023

Owing to the ability to preserve a wide variety of elements for such a long period of time, including cells, tissues, blood and DNA, cryopreservation has emerged as a key focus area for stakeholders in the biopharmaceutical industry.

Engineer 52

Engineer Containment In-Vivo Production 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.

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Gene Editing Gene Engineer DNA 52

Drug Discovery World

NOVEMBER 13, 2023

Noel Pauli, PhD, Group Leader, Antibody Engineering, Adimab, on: ‘Harnessing in vivo diversities with a yeast-based platform for the discovery of antibodies against multi-pass membrane proteins’. Ali Madani, PhD, Founder and CEO, Profluent Bio, on: ‘Protein engineering with large language models’.

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Protein Antibody Engineer Scientist 59