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. billion in funding.

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Gene Editing Gene Gene Therapy In-Vivo 144

Pharmaceutical Technology

SEPTEMBER 29, 2022

Scribe Therapeutics and Sanofi have signed a strategic partnership to expedite the development of breakthrough clustered regularly interspaced short palindromic repeats (CRISPR)-based cell therapies for cancer. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Development In-Vivo Genome Genomics 130

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. The post New whitepaper explores digital PCR for lentiviral vector characterisation appeared first on Drug Discovery World (DDW).

Gene Therapy 52

Gene Therapy In-Vivo Gene DNA 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

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Gene Editing In-Vivo Gene Sequencing Gene 88

Drug Discovery World

DECEMBER 6, 2023

“We are impressed by the unique delivery and editing technologies that Eligo has developed, and we believe this will set the stage for Eligo to forge new paths in the application of in vivo gene-editing technologies.” The investment will fuel pre-IND and IND activities to achieve early human data readouts in a PhIb/IIa clinical trial.

Gene Editing 52

Gene Editing Gene In-Vivo Genetics 52

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

The Pharma Data

DECEMBER 13, 2020

which develops genome editing technologies to accelerate drug discovery and develop novel therapeutics for a broad range of diseases, today announced the appointment of Bo Zhang, Ph.D., Prior to joining EdiGene, he was Vice President of KLUS Pharma and focused on cell therapy and new technologies. 14, 2020 10:00 UTC.

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Business Development Development Gene Editing In-Vivo 40

Pharma Marketing Network

DECEMBER 21, 2020

Almost two decades after the human genome was sequenced, a trickle of new genetic medicines (i.e., those that modify the expression of an individual’s genes or repair abnormal genes) has entered clinical practice, including 11 RNA therapeutics, 2 in vivo gene therapies, and 2 gene-modified cell therapies.

Genetics 52

Genetics Medicine Gene Therapy Marketing 52

Drug Discovery World

SEPTEMBER 14, 2023

Also in July 2023, Alexion, AstraZeneca Rare Disease (Alexion) and Pfizer entered into an agreement for Alexion to purchase and license the assets of Pfizer’s early-stage rare disease gene therapy portfolio for a total consideration of up to $1bn, plus tiered royalties on sales. billion, a decrease of $15.0 Vaccines were up 9.1%

Drugs 59

Drugs Vaccination Vaccine Medicine 59

Pharmaceutical Technology

MAY 17, 2023

Scribe Therapeutics has entered a strategic collaboration with Eli Lilly and Company subsidiary Prevail Therapeutics for accelerating in vivo CRISPR-based therapies to target the causes of serious neurological and neuromuscular diseases. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

Genetics 130

Genetics Medicine In-Vivo Gene Therapy 130

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). The US Food and Drug Administration (FDA) has cleared Intellia Therapeutics’ Investigational New Drug (IND) application for NTLA-2002 for the treatment of hereditary angioedema (HAE).

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In-Vivo Gene Editing Gene Trials 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. In vivo approaches involve delivering the gene editing components directly into the patient. In one example, Choi et al.

Gene Editing 52

Gene Editing Gene In-Vivo Genome 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 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

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

Pharma Marketing Network

JANUARY 20, 2021

In other areas of the industry (such as cell and genetic medicine development) progress continued at an impressively brisk pace despite the pandemic. Regarding OWS’s support for vaccine development, Dr Slaoui noted that 5 of the 6 vaccines selected (from a total of 94 programs) are currently in phase 3 development or approved.

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Vaccination Vaccine In-Vivo Genetics 52

Drug Discovery World

OCTOBER 17, 2022

For pharma and biotech companies – whether working on small or large molecule drugs, or cell and gene therapies – this means empowering scientists with better and faster data management approaches, best practices, and leading technologies so they can pinpoint new molecules and formulations and modify existing ones more easily and quickly.

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Marketing Scientist Gene Expression Drugs 52