pharmaphorum

SEPTEMBER 28, 2022

Vertex Pharma and partner CRISPR Therapeutics will start a rolling marketing application in the US for their gene-editing drug for sickle cell disease (SCD) and beta thalassaemia later this year. The time places exa-cel in pole position to become the first drug developed based on CRISPR/Cas9 gene-editing technology to reach the market.

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Gene Editing Gene In-Vivo Clinical Trials 59

pharmaphorum

JUNE 12, 2022

A gene-editing drug developed by CRISPR Therapeutics and Vertex Pharma has continued to show impressive results in clinical trial, with an update at the EHA congress showing long-term effects on the symptoms of both beta thalassaemia and sickle cell disease. billion product if it gets approved for both indications.

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Gene Editing Gene Gene Therapy Drugs 59

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

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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

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Gene Editing In-Vivo Gene Sequencing Gene 92

pharmaphorum

OCTOBER 8, 2020

Scribe Therapeutics, a start-up focusing on gene-editing using CRISPR/Cas9, has burst onto the biotech scene with a $415 million deal with Biogen. Its main platform – called X-editing – can generate CRISPR drugs with improved activity, specificity and deliverability compared to rivals, according to the start-up’s website.

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Gene Editing Gene In-Vivo Clinical Trials 56

pharmaphorum

NOVEMBER 18, 2022

Gene-testing specialist Editas Medicine has halted development of its lead clinical programme for congenital eye disorders after it generated lacklustre results in a phase 1/2 trial. LCA10 is caused by mutations in the CEP290 gene and accounts for approximately 20?30% 30% of all LCA cases.

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Sales Trials In-Vivo Gene Editing 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.

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Genome Genomics Gene Gene Editing 40

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).

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In-Vivo Clinical Trials Clinical Trials Drugs 52

Pharmaceutical Technology

APRIL 28, 2023

The US Food and Drug Administration (FDA) has granted an Orphan Drug Designation to Editas Medicine’s gene therapy EDIT-301 in sickle cell disease, based on an April 27 announcement. The US agency previously granted the Orphan Drug Designation to EDIT-301 for its study in beta thalassemia, in May 2022. g/dL and 45.4%

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Gene Therapy Drugs Gene Gene Editing 264

pharmaphorum

JANUARY 10, 2022

Bayer has bolstered its cell and gene therapy platform by securing access to a CRISPR-based gene-editing platform developed by US biotech Mammoth Biosciences. CRISPR drugs can be used to modify the expression of disease-associated proteins in the body, for example, by correcting a mutation in a specific gene.

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In-Vivo Gene Editing Gene Therapy Gene 52

Drug Discovery World

APRIL 15, 2024

Melissa Little, CEO of the Novo Nordisk Foundation Centre for Stem Cell Medicine “In the stem cell space, 2023 has seen significant increases in the delivery of pluripotent stem cell-derived cell types into first-in-human clinical trials. Gene therapy will move to ex vivo gene modification and the delivery of gene-modified cell types.

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Gene Editing Clinical Trials Clinical Trials In-Vivo 59

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 also offering up to $1.3

Gene Editing 52

Gene Editing Genetics Drugs Gene 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).

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In-Vivo FDA Approval Gene Gene Editing 80

XTalks

NOVEMBER 3, 2023

CRISPR works as genetic scissors to edit parts of the genome. The CRISPR-Cas9 gene editing system was first discovered to be endogenous in bacteria. Given the system’s powerful ability to make cuts in genes in a precise manner, the system is being leveraged in gene therapies.

Gene Therapy 59

Gene Therapy Gene Gene Editing In-Vivo 59

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.

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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.

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Gene Editing Gene Gene Therapy In-Vivo 130

Drug Discovery World

APRIL 4, 2023

The Food and Drug Administration (FDA) in the US has made several key drug decisions over the last few weeks. Here’s a summary. Orserdu (elacestrant), Stemline Therapeutics The first and only treatment specifically indicated for patients with ESR1 mutations in ER+, HER2- advanced or metastatic breast cancer was approved in the US in February.

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FDA Approval In-Vivo Gene Editing Drugs 52

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). Intellia’s multi-national Phase I/II study is evaluating the safety, tolerability, pharmacokinetics and pharmacodynamics of NTLA-2002 in adults with Type I or Type II HAE.

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In-Vivo Gene Editing Gene Trials 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?

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Gene Therapy FDA Approval Gene In-Vivo 98

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. . Intellia and Regeneron’s phase 1 trial looked at patients who had nerve damage (polyneuropathy) as a result of the disease.

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In-Vivo Gene Editing Trials Drugs 103

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

Drug Discovery World

JULY 3, 2023

This was centred around pre-clinical models (in vitro and in vivo) to support discovery and development, re-entering the fray with costimulatory agonists, and clinical development strategies for combinations with immune cell engagers​.

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Antibody In-Vitro Engineer In-Vivo 52

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.,

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Genetics Medicine Gene Therapy Marketing 52

pharmaphorum

FEBRUARY 1, 2024

A single dose of a gene-editing drug being developed by Intellia Therapeutics almost eliminated attacks in patients with hereditary angioedema (HAE) in a clinical trial, pointing to the potential of CRISPR-based drugs delivered in vivo to treat diseases.

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Gene Editing In-Vivo Drugs Clinical Trials 107

Pharmaceutical Technology

APRIL 4, 2023

Formerly known as CTX001, exa-cel is an investigational, autologous, ex vivo CRISPR/Cas9 gene-edited therapy. The BLAs are supported by data obtained from the ongoing Phase III CLIMB-111 and CLIMB-121 trials, along with an ongoing long-term follow-up CLIMB-131 trial.

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Gene Therapy Gene Editing In-Vivo Gene 246

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

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Clinical Trials Clinical Trials Trials Gene Therapy 52

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 .

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Drugs In-Vivo In-Vitro Scientist 97

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.

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Gene Editing Trials Drugs Gene 98

XTalks

DECEMBER 20, 2023

This includes analyzing how drug combinations may impact individual patients or groups of patients before even entering a clinical trial. With a staggering 90 percent of drugs failing in clinical trials, AI has the potential to help improve these statistics.

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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

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

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

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.

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DNA Genome Genomics RNA 98

The Pharma Data

JANUARY 10, 2021

Food and Drug Administration (FDA) has cleared the initiation of the safety phase of the Company’s EDIT-301 clinical trial, and the Company can begin dosing patients. The Company is required to develop and submit to the FDA an improved potency assay prior to enrolling the efficacy phase of the RUBY trial.

Medicine 52

Medicine Development Clinical Trials Clinical Trials 52

Drug Discovery World

FEBRUARY 26, 2024

These therapies, which utilise human cells to replace diseased or damaged cells or use engineered cells to act as therapies for a specific disease, have become more common in recent years, with approvals and clinical trials increasing. It’s safe to say there is a lot of potential for cell therapies.

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Manufacturing In-Vivo Development Production 52

Pharma Marketing Network

JANUARY 20, 2021

Assuming these findings are replicated in clinical trials, this vaccine, with its more-appealing route of administration and convenient (refrigerator) storage requirements could make an important contribution to containing the disease.

Vaccination 52

Vaccination Vaccine In-Vivo Genetics 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.

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Antibody Protein Engineer In-Vivo 59