STAT News

NOVEMBER 7, 2022

Verve Therapeutics said Monday that its experimental gene-editing treatment for a common form of heart disease was placed on clinical hold by the Food and Drug Administration, potentially delaying an ongoing, early-stage clinical trial. The Cambridge, Mass.-based Continue to STAT+ to read the full story…

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BioPharma Reporter

NOVEMBER 16, 2021

have received the green light from Health Canada for their Clinical Trial Application for VCTX210: an allogeneic, gene-edited, immune-evasive, stem cell-derived therapy for the treatment of type 1 diabetes (T1D). CRISPR Therapeutics and ViaCyte, Inc.

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Bio Pharma Dive

JUNE 21, 2021

The next six months could feature clinical milestones for CRISPR gene editing, the treatment of COVID-19, microbiome drugs and gene-targeted cancer therapy.

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Drug Discovery World

OCTOBER 11, 2022

A team of researchers at Northwestern University in the US has devised a new platform for gene editing that could inform the future application of CRISPR-based therapeutics. They created a system to deliver the cargo required for generating the gene editing machine known as CRISPR-Cas9. High gene editing efficiency.

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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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Bio Pharma Dive

OCTOBER 12, 2021

The gene editing company aims to expand its trial and add a second dose to improve effectiveness. However, safety worries have emerged since the FDA ordered CRISPR's rival Allogene to suspend clinical work.

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Bio Pharma Dive

JUNE 11, 2021

The latest results from the companies' trials give support to hopes that CRISPR gene editing could functionally cure sickle cell disease and beta thalassemia.

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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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Bio Pharma Dive

OCTOBER 19, 2020

The trial would be the first test of a systemically administered gene editing treatment that uses the Nobel Prize-winning technology to alter DNA.

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BioSpace

OCTOBER 18, 2023

The trial, which is the first late-stage study of an in vivo CRISPR treatment in the U.S., will start by the end of 2023. Intellia's NTLA-2001 is a treatment candidate for transthyretin amyloidosis cardiomyopathy.

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Bio Pharma Dive

DECEMBER 6, 2022

In a letter to the biotech, the regulator asked for more information on its gene editing medicine for heart disease as well as on its potential risks.

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Bio Pharma Dive

DECEMBER 7, 2022

Editas said its gene editing therapy appeared safe and was well tolerated in two patients. regulators lifted a partial hold on the trial. The data comes five months after U.S.

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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 If approved, it could mount a challenge to bluebird bio’s gene therapy Zynteglo, which is already approved in Europe.

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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. Further back in development are drugs that will be administered to edit genes within the body, but the first of these candidates are now in clinical trials.

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BioTech 365

DECEMBER 11, 2021

Graphite Bio Presents Overview of Phase 1/2 CEDAR Trial Evaluating Investigational Gene Editing Therapy GPH101 in Sickle Cell Disease at 63rd ASH Annual Meeting and Exposition Graphite Bio Presents Overview of Phase 1/2 CEDAR Trial Evaluating Investigational Gene Editing Therapy … Continue reading →

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XTalks

OCTOBER 20, 2023

Clinical-stage genome editing company Intellia Therapeutics has received clearance from the US Food and Drug Administration (FDA) for its Investigational New Drug (IND) application to start a pivotal phase III trial of NTLA-2001 for the treatment of transthyretin (ATTR) amyloidosis with cardiomyopathy.

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Drug Discovery World

OCTOBER 24, 2023

While attending Life Science Baltics 2023, Diana Spencer spoke to Monika Paule , CEO and Co-founder of Caszyme, about creating a company based on CRISPR gene editing and some of the challenges of operating in this dynamic area of research. Caszyme was founded in 2017 by the pioneers of CRISPR gene editing technologies.

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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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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. The post After disappointing trial, Editas puts lead CRISPR drug up for sale appeared first on.

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Worldwide Clinical Trials

OCTOBER 24, 2023

Global Genes is a rare disease umbrella organization dedicated to eliminating the burdens and challenges of rare diseases for patients and families globally. The Therapeutic Strategy Leads for Rare Disease at Worldwide Clinical Trials have been working alongside and championing these organizations for many years.

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BioSpace

OCTOBER 14, 2021

Read about TFF Pharmaceuticals and Augmenta Bioworks' dry powder COVID-19 antibody formula, the world's first gene editing clinical trial for PKU, Bayer's COVID-19 vaccine and other key developments in life sciences research.

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BioTech 365

OCTOBER 8, 2021

Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: FDA stops all Allogene’s CAR-T trials over safety scare, raising questions about future of gene editing.FDA stops all Allogene’s CAR-T trials over safety scare, raising questions about … Continue reading →

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Scienmag

JANUARY 19, 2021

publishers New Rochelle, NY, January 19, 2021–Gene editing therapies, including CRISPR-Cas systems, offer the potential to correct mutations causing inherited retinal degenerations, a leading cause of blindness. Credit: Mary Ann Liebert, Inc.,

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BioPharma Reporter

FEBRUARY 23, 2023

After voluntarily pausing a phase 1/2 trial due to safety concerns in January 2023, the gene edited cell therapy firm Graphite Bio has discontinued the development of nulabeglogene autogedtemcel (nula-cel), its lead candidate cell therapy for sickle cell disease, and has halved its workforce.

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Drug Discovery World

NOVEMBER 16, 2023

The Medicines and Healthcare products Regulatory Agency (MHRA) has authorised the world’s first gene therapy for sickle-cell disease (SCD) and transfusion-dependent β-thalassemia (TDT). Trial results for Casgevy The authorisation is based on positive clinical trial data.

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Drug Discovery World

OCTOBER 10, 2023

The National Institutes of Health has awarded the first phase of a roughly $40m grant to Yale University to advance a novel CRISPR-based gene-editing delivery platform for the targeted treatment of neurogenetic diseases. The two diseases being targeted in the project are Angelman syndrome and H1-4 (HIST1H1E) syndrome.

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Drug Discovery World

NOVEMBER 14, 2023

A Phase Ib trial has shown low-density lipoprotein cholesterol (LDL-C) reductions up to 55% and blood PCSK9 protein reductions up to 84% after a single infusion of Verve Therapeutics’ Verve-101. The post Single-course in vivo base editing therapy proven to lower cholesterol appeared first on Drug Discovery World (DDW).

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Drug Discovery World

DECEMBER 8, 2023

“The future development of this combination therapy alongside our monotherapy clinical trials will help expand patient options and narrow the gap towards improving the long-term survival of patients with hepatocellular carcinoma.” Both companies will share the costs of manufacturing and preclinical assessments.

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BioTech 365

DECEMBER 17, 2020

Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: Scientists set a path for field trials of gene drive organisms.The modern rise of gene drive research, accelerated by CRISPR-Cas9 gene editing technology, has led to transformational … Continue reading →

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pharmaphorum

APRIL 23, 2021

CRISPR Therapeutics is to receive a hefty $900m payment from Vertex after the companies amended a collaboration to develop, manufacture and market a gene editing therapy for sickle cell disease and beta thalassemia.

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Pharmaceutical Technology

MARCH 2, 2023

Attributes of the drug, company and its clinical trials play a fundamental role in drug-specific PTSR and likelihood of approval. The company develops gene-editing organ transplantation technology. QN-023a overview QN-023a is under development for the treatment of relapsed or refractory acute myeloid leukemia (AML).

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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

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Pharmaceutical Technology

MARCH 2, 2023

Attributes of the drug, company and its clinical trials play a fundamental role in drug-specific PTSR and likelihood of approval. The company develops gene-editing organ transplantation technology. Hangzhou Qihan Biotechnology overview Hangzhou Qihan Biotechnology is a healthcare company.

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pharmaphorum

FEBRUARY 16, 2022

Having a third functional cure of HIV nevertheless does give pointers to how a more widely applicable cure might be developed in the future, for example using gene-editing drugs to introduce CCR5?32 Sangamo was working on a treatment based on autologous (patient-derived) stem cells and T cells that were gene-edited to carry the CCR5?32

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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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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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Pharmaceutical Technology

OCTOBER 13, 2022

Vita Therapeutics intends to use the funding to advance its lead pre-clinical programme VTA-100 for limb-girdle muscular dystrophy (LGMD2A) to clinical trials. VTA-100 has been designed to be an autologous therapy which combines gene correction as well as induced iPSC technology for repairing and replacing muscle cells for LGMD2A patients.

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