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

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. Roughly 80% of rare diseases are due to a known genetic driver. The post 3 Key Takeaways from Global Genes Summit 2023 appeared first on Worldwide Clinical Trials.

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Gene Gene Editing Gene Therapy Clinical Trials 130

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. . Alternatively, a drug can be administered that switches off the genetic medicine and brings protein expression to a halt.

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Gene Editing Genetics Drugs Gene 52

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.

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Gene Editing DNA Gene Genome 98

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. There is enormous power in this genetic tool, which affects us all. What started as a curiosity?driven, million) Nobel Prize award. “In

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Gene Editing Bacteria DNA Gene 105

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 therapeutic candidate comprises allogeneic NK cells genetically engineered to express chimeric antigen receptors (CAR-NK) targeting cells CD33.

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Gene Editing Genetic Engineering Engineer Clinical Trials 100

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 therapeutic candidate (CAR-NK) comprises allogeneic genetically modified NK cells expressing chimeric antigen receptors (CAR) targeting cells expressing B lymphocyte antigen CD19.

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Gene Editing Clinical Trials Clinical Trials Genetics 100

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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In-Vivo Gene Editing Trials Gene 52

pharmaphorum

MARCH 11, 2021

Vertex Pharma has made its name with treatment for cystic fibrosis, but has big plans in cell and genetic therapies – and has just chalked up fast-track status from the FDA for a cell-based therapy for diabetes. . That implantable technology was at the heart of the Semma acquisition. ViaCyte also has an unencapsulated version in phase 1/2.

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Gene Editing Insulin Immune Response Gene 98

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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Gene Editing Gene DNA Bacteria 64

XTalks

JANUARY 26, 2024

Innovations in Cancer Therapy CRISPR/Cas9, a groundbreaking gene-editing technology, has demonstrated significant potential in oncology, offering new avenues for cancer treatment. By tailoring treatments based on an individual’s genetic makeup, it may allow for more effective and targeted therapies.

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Research Gene Editing Gene Genetics 118

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.

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Gene Editing Gene In-Vivo Genetics 52

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.

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Genetics Medicine Gene Therapy Marketing 52

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. These companies are at various stages of research and clinical trials.

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Life Science Gene Editing Development Clinical Trials 119

pharmaphorum

OCTOBER 8, 2021

A phase 1 trial of Allogene’s of-the-shelf CAR-T for lymphoma has been placed on hold by the FDA, after a “chromosomal abnormality” was seen in a patient receiving the cell therapy. The post FDA halts Allogene CAR-T trial after safety scare appeared first on.

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Trials Gene Editing Genetics Gene 52

XTalks

MAY 22, 2023

Related: 5 Food Companies Working With Precision Fermentation Technology How Pairwise Leverages CRISPR Technology CRISPR, or Clustered Regularly Interspaced Short Palindromic Repeats, is a gene-editing technique that can be used to alter the DNA of cells to enhance certain characteristics or reduce less desirable ones.

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Branding Gene Editing DNA Production 98

pharmaphorum

FEBRUARY 23, 2022

Leverage their knowledge and experiences to ultimately ensure effective creation and implementation of patient-centric gene therapy programs.

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Gene Therapy Gene Gene Editing Clinical Trials 52

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.

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In-Vivo Gene Editing Gene Therapy Gene 52

WCG Clinical

NOVEMBER 17, 2023

Since then, however, certain genetic engineering technologies (e.g., CRISPR-based gene editing) have advanced to the point where cellular genomes can be edited without using viral vectors or any other materials subject to the NIH Guidelines and without integration of rsNA into the chromosome.

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Genome Genomics Gene Editing Genetic Engineering 52

pharmaphorum

APRIL 20, 2022

The potential of CRISPR technology has been a hot topic in the industry ever since it was first developed, but as trials progress further into the clinic, what therapeutic areas could be set to benefit? Cancer is a genetic disease, caused by certain changes in the way that genes control cell function, such as how they grow and divide.

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Gene Editing DNA Gene Genetics 52

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.

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Gene Therapy Gene Gene Editing In-Vivo 59

Delveinsight

JANUARY 14, 2021

The companies schedule to initiate a Phase 2 trial assessing combination therapy for both treatment-experienced and treatment-naïve people living with HBV. People in the trial with HBV treatment experience may also receive Gilead’s Vemlidy (tenofovir alafenamide fumarate, TAF). Gilead Sciences, Inc. and Vir Biotechnology, Inc.

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Gene Editing Gene Genetics DNA 40

pharmaphorum

FEBRUARY 17, 2022

SCD is one of the most prevalent genetic diseases in Europe, affecting approximately 52,000 people, and is more common in those whose ancestors are from sub-Saharan Africa, although it is also seen in people of Hispanic, South Asian, Southern European and Middle Eastern ancestry.

Drugs 59

Drugs Gene Therapy Gene Editing Gene 59

pharmaphorum

SEPTEMBER 14, 2022

SparingVision has raised €75 million in second-round funding that will be used to fund clinical trials of gene therapies for ocular diseases retinitis pigmentosa (RP) and dry age-related macular degeneration (AMD). The current schedule is for trials to start in 2024, with first safety and efficacy data coming a year later.

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Gene Therapy Gene Gene Sequencing Gene Editing 52

XTalks

FEBRUARY 27, 2024

There have been significant advancements in new rare disease drugs, particularly for genetic disorders that can be treated by correcting, replacing or silencing defective genes. Sarepta hopes to clarify Elvidys’ effectiveness in older children in a confirmatory trial. The FDA introduced its Orphan Drug Act in 1983.

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Research Drugs Gene Therapy Clinical Trials 59

BioSpace

NOVEMBER 17, 2022

Editas Medicine is pausing its ocular gene therapy program after demonstrating a favorable safety profile and seeking a potential partner to develop EDIT-101, the company announced Thursday.

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Gene Therapy Gene Gene Editing Medicine 42

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.

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Gene Editing Gene Gene Therapy In-Vivo 145

Bioengineer

JULY 23, 2021

“By applying high-resolution imaging, advanced cell and molecular readouts and RNA sequencing methods leveraging the OTME-Chip, we discovered the actual genetic signaling pathways behind the blood cell triggered metastasis of ovarian cancer and a new drug strategy to stop this process.” ” ###.

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Engineer Gene Editing Clinical Trials Clinical Trials 52

Trialfacts

SEPTEMBER 12, 2024

Their work is driven by a profound understanding of disease biology and supported by advanced discovery platforms such as genetics and genomics, gene editing, bioinformatics, proteomics, bioengineering, image analysis, biobanks, disease-specific stem cell lines, and various animal models.

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Research Gene Editing Biobanking Bioinformatics 52

The Pharma Data

NOVEMBER 3, 2020

Nasdaq:LOGC) (LogicBio), a company dedicated to extending the reach of genetic medicine with pioneering targeted delivery platforms, announced today the U.S. LogicBio Therapeutics is dedicated to extending the reach of genetic medicine with pioneering targeted delivery platforms. LEXINGTON, Mass., About LogicBio Therapeutics.

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Clinical Trials Clinical Trials Gene Editing Genome 40

Advarra

NOVEMBER 29, 2022

While each ultra-rare genetic disorder may have a very small patient population, when these conditions are grouped together, they make up a significant number. A major topic of discussion is the significant problems in multi-country clinical trials due to the different requirements of the parties involved.

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Gene Therapy Gene Production Clinical Trials 52

pharmaphorum

NOVEMBER 2, 2021

In 2019, there were 57 bsAb candidates in clinical trials, against both haematological and solid tumours. In the pipeline , Amgen has BiTE molecules targeting seven types of cancer in clinical trials, as well as further structures in the design stage. Simplified schematic overview of the proposed mechanisms of action for bsAbs.

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Antibody Immune Response Production Engineer 52

Camargo

JULY 27, 2021

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. Additionally, gene editing allows us either to remove or to modify harmful genes.

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Gene Therapy Gene Gene Editing Genetics 238

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

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

FEBRUARY 16, 2022

She received a cord blood transplant to treat the AML, receiving stem cells carrying a genetic mutation (CCR?5/?32) 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

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Gene Editing Research Gene Genetics 69

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