Camargo

JULY 27, 2021

How and When to Incorporate PK Design into Your Gene Therapy Development Plan. Gene therapy, which was in its infancy around 30 years ago, is now becoming a more prominent treatment method in many therapeutic areas, from personalized therapy to mass vaccinations against COVID-19. Gene Therapy Definition.

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Gene Therapy Gene Gene Editing Genetics 238

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. These insights were shared through several sessions, in presentation tracks specifically dedicated to “Community and Capacity Building” and “Becoming a Research Ready Organization.”

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Gene Gene Editing Gene Therapy Clinical Trials 130

pharmaphorum

OCTOBER 4, 2022

AstraZeneca’s rare disease firm Alexion is set to expand its genomic medicine portfolio with the acquisition of gene editing specialist LogicBio Therapeutics, in a deal worth approximately $68 million. The post AstraZeneca pays record 660% premium for gene editing company LogicBio appeared first on.

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Gene Editing Gene Genome Genomics 64

pharmaphorum

OCTOBER 13, 2022

Gene therapies and research into them have grown immensely in recent years, offering more novel tools in regenerative medicine to fight disease, including rare diseases and genetic disorders. Beta-thalassemia is a rare blood disorder caused by a genetic defect in hemoglobin. Novartis also recently inked an up-to $1.5

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Gene Editing Gene Gene Therapy Genetics 52

Medical Xpress

MARCH 17, 2023

Researchers in China have successfully restored the vision of mice with retinitis pigmentosa, one of the major causes of blindness in humans.

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Gene Editing Gene Genome Genomics 98

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

Medical Xpress

DECEMBER 21, 2022

Using the CRISPR-Cas9 gene editing system, UT Southwestern researchers corrected mutations responsible for a common inherited heart condition called dilated cardiomyopathy (DCM) in human cells and a mouse model of the disease.

Gene Therapy 98

Gene Therapy Gene Editing Gene Research 98

BioTech 365

MARCH 30, 2021

Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: Prime editing enables precise gene editing without collateral damage.The latest gene editing technology, prime editing, expands the ‘genetic toolbox’ for more precisely creating disease models and correcting … (..)

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Gene Editing Gene Genetics Research 40

Scienmag

NOVEMBER 19, 2020

Media contacts: Jennifer Kuzma, jkuzma@ncsu.edu Khara Grieger, kdgriege@ncsu.edu Mick Kulikowski, News Services, 919.218.5937 or mick_kulikowski@ncsu.edu Researchers at North Carolina State University call for a coalition of biotech industry, government and non-government organizations, trade organizations, and academic experts to work together to (..)

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Gene Editing Gene Research Production 44

Scienmag

JANUARY 12, 2021

Martins Scientists have used gene-editing advances to achieve a tenfold increase in the production of super-bug targeting formicamycin antibiotics. The John Innes Centre researchers used the technology to create a new strain of Streptomyces formicae bacteria which over-produces the medically promising molecules.

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Gene Editing Gene Bacteria Scientist 53

Scienmag

AUGUST 18, 2020

Credit: Rasgon Lab, Penn State Researchers who developed an improved method of gene editing for the study of arthropods will expand the technology for use in vertebrate species such as mice, fish and birds after receiving new funding from the National Science Foundation.

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Gene Editing Gene Development Research 61

BioTech 365

DECEMBER 4, 2020

Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: Advancing gene editing with new CRISPR/Cas9 variant.Using a new variant to repair DNA will improve both safety and effectiveness of the much-touted CRISPR-Cas9 tool in genetic research, … Continue reading →

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Gene Editing Gene DNA Genetics 40

STAT News

MARCH 22, 2023

DALLAS — It took just three years, after the tool’s invention, for researchers to devise ways of using CRISPR-Cas9 gene editing to treat mice with Duchenne muscular dystrophy. One company was even bought out for over $200 million. One company was even bought out for over $200 million.

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Gene Editing Gene Therapy Research Gene 98

BioTech 365

MAY 1, 2021

Editas Medicine to Present Preclinical Data Demonstrating Advancements in In Vivo Gene Editing Approach for the Treatment of Genetic Ocular Diseases at the Association for Research in Vision and Ophthalmology Annual Meeting Editas Medicine to Present Preclinical Data Demonstrating Advancements … Continue reading →

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Gene Editing In-Vivo Genetics Gene 40

Pharmaceutical Technology

MARCH 28, 2023

Under the terms of the deal, the company will receive non-exclusive rights to CRISPR/Cas9, a gene-editing technology of CRISPR Therapeutics, for the development of potentially curative T1D cell therapies. The gene-editing technology allows for precise, directed changes to genomic DNA.

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Gene Editing DNA Gene Gene Therapy 235

XTalks

JANUARY 26, 2024

Technological advancements are revolutionizing cancer research and therapies. Innovations in Cancer Therapy CRISPR/Cas9, a groundbreaking gene-editing technology, has demonstrated significant potential in oncology, offering new avenues for cancer treatment.

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Research Gene Editing Gene Genetics 118

Medical Xpress

DECEMBER 12, 2022

An international research team led by Dr. Ana Guadaño at the Alberto Sols Biomedical Research Institute (IIBM, a combined CSIC-UAM center) and involving the Complutense University of Madrid (UCM), used CRISPR gene editing techniques to incorporate into mice a mutation of the MCT8 protein responsible for transporting thyroid hormones to the interior (..)

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Gene Editing Genetics Hormones Medicine 75

Scienmag

FEBRUARY 23, 2021

CRISPR gene editing has transformed research, but it is not perfect, and can sometimes target unintended genes; to watch CRISPR enzymes respond to different genes, Leipzig University researchers developed a new method using DNA origami and were able to me Credit: Image courtesy of Julene Madariaga Marcos.

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DNA Gene Gene Editing Scientist 56

STAT News

MARCH 6, 2023

LONDON — The first gene-edited children were born in China five years ago , but it’s unlikely to happen again there anytime soon. That was the message Chinese scientists delivered Monday on the opening day of the Third International Summit on Human Genome Editing in London.

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Genome Genomics Gene Editing Genetics 98

Drug Discovery World

FEBRUARY 16, 2023

Challenges remain with current delivery approaches, and genetic medicines applications have been generally restricted to the liver and ex vivo settings. Aera’s technology also includes a licensed therapeutic enzyme platform based on the discovery of novel, compact, and programmable gene editing enzymes.

Genetics 52

Genetics Medicine Gene Editing In-Vivo 52

Pharmaceutical Technology

JUNE 8, 2023

The ttLNP platform can deliver a range of high-impact genomic payloads, including gene editing, siRNA, mRNA, DNA and gene-writing apparatus, to tissues, cells and organs. The development will be advanced through both internal pipeline programmes and external collaborations.

Genome 130

Genome Genomics Medicine Development 130

Scienmag

DECEMBER 3, 2020

Using a new variant to repair DNA will improve both safety and effectiveness of the much-touted CRISPR-Cas9 tool in genetic research, Michigan Medicine researchers say. Eugene Chen, M.D., […].

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Gene Editing Gene DNA Genetics 41

Drug Discovery World

JANUARY 17, 2023

Oregon State University College of Pharmacy scientists have shown that lipid nanoparticles and messenger RNA could treat blindness associated with a rare genetic condition. . Researchers developed nanoparticles able to penetrate the neural retina and deliver mRNA to the photoreceptor cells whose proper function makes vision possible. .

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Gene Editing Gene Gene Therapy Genetics 98

XTalks

JULY 14, 2022

Every gene in a cell is expressed at exact levels due to complex gene regulatory networks. The connections between regulatory genes and their downstream targets have been mapped to some extent — they can be thought of as a subway or metro map, with connections existing between major “hubs.”

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Gene Regulation Protein Gene Editing 98

pharmaphorum

FEBRUARY 16, 2022

A woman with HIV who received a stem cell transplant to treat acute myeloid leukaemia (AML) appears to have been cured of the infection according to US researchers. She received a cord blood transplant to treat the AML, receiving stem cells carrying a genetic mutation (CCR? “This third case of HIV remission suggests that CCR?5/?32

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Gene Editing Research Gene Genetics 69

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 96

STAT News

NOVEMBER 29, 2022

He Jiankui, the Chinese biophysicist who created the first gene-edited children , had been quiet since completing a three-year prison sentence in April, leaving many to wonder whether he had plans to return to scientific research. Earlier this month, we got his answer.

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Gene Editing Scientist Gene Research 144

BioTech 365

DECEMBER 23, 2020

Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: Genetic engineering without unwanted side effects helps fight parasites.Modified CRISPR-Cas9 gene editing scissors are enabling researchers at the University of Zurich to make alterations to the genetic … Continue reading (..)

Genetic Engineering 40

Genetic Engineering Engineer Genetics Gene Editing 40

Drug Discovery World

MARCH 21, 2023

UK researchers, from the Wellcome Sanger Institute and collaborators, have used human stem cells and neurons to investigate what features influence how well CRISPR activation works for different sets of genes. CRISPR activation (CRISPRa) is a type of CRISPR gene editing that is used to overexpress certain genes.

Research 52

Research Gene Gene Editing Genome 52

Drug Discovery World

DECEMBER 11, 2023

The US Food and Drug Administration (FDA) has approved the first cell-based gene therapies for the treatment of sickle cell disease (SCD), Casgevy and Lyfgenia. Casgevy, from Vertex Pharmaceuticals and CRISPR Therapeutics, is also the first FDA-approved treatment to use CRISPR gene editing technology.

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Gene Therapy FDA Approval Gene Gene Editing 52

Drug Discovery World

OCTOBER 6, 2023

In celebration of the Nobel Prize for Medicine going to two of the early proponents of mRNA technology for creating therapeutics, Katalin Karikó and Drew Weissman, this week our round-up highlights the importance of genetics, genomics and gene editing in drug discovery.

Drugs 52

Drugs Gene Editing Genome Genomics 52

BioTech 365

APRIL 9, 2021

Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: New, reversible CRISPR method can control gene expression while leaving underlying DNA sequence unchanged.Over the past decade, the CRISPR-Cas9 gene editing system has revolutionized genetic engineering, allowing … Continue (..)

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Gene Expression DNA Gene Editing Gene 40

STAT News

MARCH 3, 2023

Continue to STAT+ to read the full story…

Genome 111

Genome Genomics Gene Editing Genetic Disease 111

Drug Discovery World

MARCH 25, 2024

Allogene Therapeutics and Arbor Biotechnologies Biotechnology companies Allogene Therapeutics and Arbor Biotechnologies entered a non-exclusive, global gene editing licensing agreement for use of Arbor’s proprietary CRISPR gene-editing technology in Allogene’s AlloCAR T platform for the treatment of autoimmune disease (AID).

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Gene Editing Drugs Genome Genomics 52

Drug Discovery World

OCTOBER 25, 2022

Scientists have shown how stomach cancers can develop resistance to a new class of drugs called ATR inhibitors by switching off the activity of two key genes – raising the possibility of outsmarting cancer by predicting drug resistance in advance. . It was funded by Cancer Research UK. . CRISPR used to identify genes involved.

Development 52

Development Drugs Gene Editing Gene 52

Drug Discovery World

FEBRUARY 27, 2023

An investigational gene therapy for Sanfilippo syndrome – which leads to a form of childhood dementia – has shown promising early results in a proof-of-concept study. Patients with MPS-IIIA have a mutation in the SGSH gene, causing them to lack an enzyme which normally breaks down large sugar molecules.

Gene Therapy 52

Gene Therapy Gene Genetics Genetic Disease 52

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. The post Nobel winner Charpentier’s CRISPR Therapeutics gets $900m in reworked Vertex deal appeared first on.

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Gene Editing Gene DNA Bacteria 64