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. That would just slow the whole field down.

Gene Editing 145

Gene Editing Gene Gene Therapy In-Vivo 145

Rethinking Clinical Trials

JULY 31, 2024

Adrian Hernandez In this Friday’s PCT Grand Rounds, Adrian Hernandez of Duke University will present “Precision Health to Population Health: Opportunities and Challenges for Gene Editing Therapies.” He is a co–principal investigator of the NIH Pragmatic Trials Collaboratory Coordinating Center.

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Gene Editing Gene Clinical Trials Clinical Trials 146

Worldwide Clinical Trials

NOVEMBER 12, 2024

For example, once considered incurable and terminal, patients with sickle cell disease may reach new summits in their lives with gene editing technologies such as CRISPR to repair affected DNA and, in some cases, functionally cure the condition. Operationalizing these trials requires proactive and flawless management at every stage.

Gene Therapy 182

Gene Therapy Clinical Supply Trials Gene 182

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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Gene Editing Gene Clinical Trials Clinical Trials 111

Bio Pharma Dive

OCTOBER 7, 2024

The study, of Intellia’s treatment for hereditary angioedema, is the second Phase 3 trial the biotech has begun testing its “in vivo” gene editing medicines.

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Gene Editing In-Vivo Gene Medicine 187

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

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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Gene Editing Gene Regulation Trials 187

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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Gene Editing Gene Clinical Trials Clinical Trials 98

Bio Pharma Dive

APRIL 29, 2024

The company will soon start a Phase 1/2 study of its treatment for chronic granulomatous disease, a milestone for the search-and-replace gene editing technology.

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Gene Editing Gene Trials 154

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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Gene Editing Gene Regulation Trials 154

AuroBlog - Aurous Healthcare Clinical Trials blog

SEPTEMBER 17, 2024

We are witnessing a revolution in healthcare, driven by advances in genetics, Omics, RNA and CRISPR gene-editing technology, to deliver precision and personalised medicine, said Kiran Mazumdar-Shaw, executive chairperson, Biocon and Biocon Biologics. Biology is opening up new frontiers in medicine.

Genetic Disease 189

Genetic Disease Genetics Gene Editing Medicine 189

BioSpace

APRIL 29, 2024

The FDA has cleared a clinical trial of an ex vivo prime editing candidate in patients with a rare disease, Prime Medicine announced Monday. The technique taps CRISPR technology to rewrite defective genes without breaking DNA double helix strands.

Gene Editing 88

Gene Editing Gene Medicine In-Vivo 88

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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Gene Editing Gene In-Vivo Clinical Trials 69

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.

Gene Editing 59

Gene Editing Gene In-Vivo Clinical Trials 59

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.

Gene Therapy 246

Gene Therapy Gene Editing In-Vivo Gene 246

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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Gene Editing In-Vivo Gene Trials 96

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.

Gene Editing 59

Gene Editing Gene Gene Therapy Drugs 59

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.

In-Vivo 105

In-Vivo Gene Editing Trials Drugs 105

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.

Gene Editing 111

Gene Editing In-Vivo Drugs Clinical Trials 111

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.

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Gene Editing Trials Drugs In-Vivo 98

Camargo

JULY 27, 2021

Gene therapy is a new therapeutic approach in which genes are used to treat or prevent diseases. It is a comprehensive term which encompasses a large variety of therapy products including viral and bacterial vectors, plasmid DNA, human gene editing technology, and patient-specific cellular gene therapy.

Gene Therapy 238

Gene Therapy Gene Gene Editing Genetics 238

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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Gene Editing Gene In-Vivo Genetics 52

Pharmaceutical Technology

AUGUST 24, 2022

A lead product candidate of the company, GTP-506 is a single-dose gene editing therapy. The ARCUS nuclease vector (GTP-506A) targets gene editing in the PCSK9 gene locus that is well characterised, while the therapeutic donor vector (GTP-506D) introduces the OTC gene to provide the required genetic correction.

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Gene Editing Gene Therapy Gene Genetics 130

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 97

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.,

Gene Editing 101

Gene Editing Genome Genomics Gene Therapy 101

STAT News

NOVEMBER 4, 2022

A man with muscular dystrophy who was first in line to receive an experimental gene editing therapy tailor made to treat the cause of his rare form of the disease has died. Terry had long been too old to participate in clinical trials of experimental therapies for the disease, which are often geared towards young boys.

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Gene Editing Gene Gene Therapy Scientist 105

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.

Sales 92

Sales In-Vivo Trials Gene Editing 92

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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Gene Editing Gene Trials 40

Pharmaceutical Technology

APRIL 28, 2023

EDIT-301 is made of patient-derived CD34+ haematopoietic stem and progenitor cells, which are edited using CRISPR at the gamma globin gene (HBG1 and HBG2) promoter sites using a proprietary engineered AsCas12a nuclease, per the company’s website. This increases the expression of fetal haemoglobin.

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Gene Therapy Drugs Gene Gene Editing 264

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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Gene Gene Editing Gene Therapy Clinical Trials 130

XTalks

AUGUST 29, 2022

Now a common gene editing tool, the popularity of the CRISPR-Cas9 system has increased over the past decade. CRISPR is notable for engineering living cells, allowing scientists to edit, turn off, delete, or replace genes in a cell’s genome.

Gene Editing 98

Gene Editing DNA Gene Genomics 98

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.

Gene Editing 66

Gene Editing Life Science Clinical Trials Clinical Trials 66

Worldwide Clinical Trials

MAY 30, 2024

Authors: Rich Worldwide Clinical Trials Exec. This trend is on the rise despite recent disappointments with clinical trial outcomes, which have the potential to destabilize the industry in the short term regarding drug development strategy and optimal study designs. Director, Therapeutic Area Medical Lead.

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Gene Clinical Trials Clinical Trials Gene Therapy 204

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.

In-Vivo 52

In-Vivo Gene Editing Trials Gene 52

pharmaphorum

OCTOBER 7, 2020

Drs Emmanuelle Charpentier and Jennifer Doudna have won this year’s Nobel Prize for chemistry in recognition of their work on the gene-editing technology CRISPR/Cas9. million) Nobel Prize award. “In

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Gene Editing Bacteria DNA Gene 105

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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Development Gene Editing Gene Gene Therapy 130

pharmaphorum

FEBRUARY 3, 2021

Dr Jennifer Harbottle, senior scientist in the R&D Base Editing team of PerkinElmer’s Horizon Discovery business, looks at progress made in the realms of biotechnology and next-generation diagnostics, vaccines and therapeutics, including the application of CRISPR-Cas9 gene editing in developing and refining cell therapies.

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Gene Editing Gene DNA Engineer 98