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

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

pharmaphorum

APRIL 23, 2021

Vertex Pharma has ramped up its involvement in gene-editing medicines for the second time in a week, paying Obsidian Therapeutics $75 million upfront to access its technology platform. . Vertex is taking over 60% of the costs of the programme in return for getting the same proportion of profits on sales if CTX001 reaches the market.

Gene Editing 52

Gene Editing Genetics Drugs Gene 52

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

XTalks

DECEMBER 20, 2023

CRISPR Gene Editing Therapeutics CRISPR/Cas-9 technology makes lists like this almost every year, and for good reason. Given its ability to precisely edit genes, there is tremendous promise for the revolutionary technology to offer cures for genetic disorders and to treat various diseases.

Life Science 119

Life Science Gene Editing Development Clinical Trials 119

Pharma Marketing Network

DECEMBER 21, 2020

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. Almost two decades after the human genome was sequenced, a trickle of new genetic medicines (i.e.,

Genetics 52

Genetics Medicine Gene Therapy Marketing 52

Drug Discovery World

APRIL 4, 2023

The designation is the first step to gaining a priority review voucher (PRV), which can accelerate market access for therapeutics. The Food and Drug Administration (FDA) in the US has made several key drug decisions over the last few weeks. Here’s a summary. The drug was granted orphan drug designation by the FDA in May 2021.

FDA Approval 52

FDA Approval In-Vivo Gene Editing Drugs 52

XTalks

DECEMBER 13, 2023

Both gene therapies are approved for individuals 12 years of age and older with sickle cell disease. The disease occurs due to a point mutation in the hemoglobin beta globin ( HBB ) gene that codes for one of the proteins that make up hemoglobin, the oxygen carrier in red blood cells. How do Casgevy and Lyfgenia Work?

Gene Therapy 98

Gene Therapy FDA Approval Gene In-Vivo 98

Drug Discovery World

NOVEMBER 16, 2022

Therefore, while researchers can understand just how a drug molecule impacts a cell, they can’t be sure that it will perform the same when used in-vivo. . One of these benefits is how cells grown in this type of culture are able to interact with their surroundings, similar to how they would perform in-vivo. 2D vs 3D .

Drugs 97

Drugs In-Vivo In-Vitro Scientist 97

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?    I quickly recognised the commercial value of aptamers as being able to address the gap in the market where antibodies fail to perform, and together we established the business to begin aptamer development.

Antibody 52

Antibody Gene Therapy Gene In-Vitro 52

pharmaphorum

JULY 13, 2022

Verve Therapeutics has started dosing patients in a phase 1b trial of its in vivo gene-editing drug for high cholesterol, designed to permanently switch off the PCSK9 gene with a one-shot treatment. dosing of *first patient* with VERVE-101, an in vivo CRISPR base editing medicine. Today: we are announcing.

Gene Editing 98

Gene Editing Trials Drugs Gene 98

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. Enrolled patients will receive a single administration of EDIT-301.

Medicine 52

Medicine Development Clinical Trials Clinical Trials 52

Drug Discovery World

FEBRUARY 26, 2024

For example, Vertex Pharmaceuticals’ stem-cell treatment for sickle-cell disease and transfusion-dependent β-thalassemia was authorised by the Medicines and Healthcare products Regulatory Agency (MHRA) in November 2023, making it the first licensed therapy that uses the gene-editing tool CRISPR to help treat patients.

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

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.

Vaccination 52

Vaccination Vaccine In-Vivo Genetics 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 ”. But psychopharmacology developments petered out, and few new drugs reached the market during the last four decades. to £104.7m

Drugs 59

Drugs Gene Clinical Trials Clinical Trials 59