Pharmaceutical Technology

FEBRUARY 13, 2023

While several therapeutics are available for treating symptoms associated with epilepsy, researchers and patients have strongly called out the need for more holistic treatments that would address the condition as a whole. Meanwhile, others are researching the link between gene variation and different responses to treatments.

Research 264

Research Gene Gene Therapy Genomics 264

XTalks

DECEMBER 14, 2022

Expanding upon the CRISPR-Cas9 gene editing system, researchers at MIT have designed a new technique called PASTE gene editing that can cut out defective genes and replace them with new genes in a safer and more efficient way. How does the Gene Editing Tool PASTE Work?

Gene Editing 98

Gene Editing DNA Gene Gene Sequencing 98

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.

Gene Therapy 238

Gene Therapy Gene Gene Editing Genetics 238

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.

Gene Editing 235

Gene Editing DNA Gene Gene Therapy 235

Pharmaceutical Technology

MAY 25, 2023

ElevateBio has raised $401m in a Series D financing round for advancing its technology platforms to expedite the design, production and development of cell and gene therapies. The technology platforms include the Life Edit gene editing platform, an RNA, cell, protein, vector engineering and induced pluripotent stem cells (iPSCs) platform.

Gene Therapy 243

Gene Therapy Gene Gene Editing Development 243

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.

Gene Editing 98

Gene Editing Gene Genome Genomics 98

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. Continue to STAT+ to read the full story…

Gene Editing 98

Gene Editing Gene Therapy Research Gene 98

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.

Gene Editing 64

Gene Editing Gene Genomics Genome 64

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 97

Gene Therapy Gene Editing Gene Research 97

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.

Gene Editing 144

Gene Editing Scientist Gene Research 144

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.

Genomics 98

Genomics Genome Gene Editing Genetics 98

STAT News

MARCH 3, 2023

Continue to STAT+ to read the full story…

Genomics 111

Genomics Genome Gene Editing Genetic Disease 111

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.

Gene Editing 61

Gene Editing Gene Development Research 61

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.

Gene Editing 52

Gene Editing Gene In-Vivo Genetics 52

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.

Research 118

Research Gene Editing Gene Genetics 118

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.

Gene Editing 53

Gene Editing Gene Bacteria Scientist 53

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

Gene Editing 52

Gene Editing Gene Gene Therapy Genetics 52

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 (..)

Gene Editing 44

Gene Editing Gene Research Production 44

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 (..)

Gene Editing 72

Gene Editing Genetics Hormones Medicine 72

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.”

Gene 130

Gene Gene Editing Gene Therapy Clinical Trials 130

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.

Genomics 130

Genomics Genome Medicine Development 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

Gene Editing 97

Gene Editing In-Vivo Gene Sequencing Gene 97

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 … (..)

Gene Editing 40

Gene Editing Gene Genetics Research 40

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

Gene Editing 105

Gene Editing Bacteria DNA Gene 105

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 →

Gene Editing 40

Gene Editing Gene DNA Genetics 40

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 →

Gene Editing 40

Gene Editing In-Vivo Genetics Gene 40

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. However, researchers are determined to overcome this hurdle.

Gene Editing 98

Gene Editing DNA Gene Genomics 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

Gene Editing 69

Gene Editing Research Gene Genetics 69

Pharmaceutical Technology

FEBRUARY 20, 2023

Hans Bunschoten, chief strategy officer, Cerba Research: “Cerba Research and Viroclinics-DDL have a lot of synergies” The discrepancy is magnified in pivotal studies with a 76.1% Why did you accept the offer from Cerba Research? Cerba Research and Viroclinics-DDL have a lot of synergies.

Research 130

Research Clinical Trials Clinical Trials Trials 130

pharmaphorum

JUNE 28, 2021

The reductions matched the efficacy of current therapies for ATTR amyloidosis that require chronic dosing such as Alnylam’s Onpattro (patisiran) and Ionis/Akcea’s Tegsedi (inotersen) – both gene-silencing agents which can cost around $450,000 a year. — Eric Topol (@EricTopol) June 26, 2021.

In-Vivo 105

In-Vivo Gene Editing Trials Drugs 105

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

Worldwide Clinical Trials

MAY 30, 2024

These advancements have fostered increased focus from politicians, policymakers, and the investment communities backing academic and industry-sponsored research projects. Researchers established efficacy of edaravone in a 24 week, randomized, placebo-controlled double-blind study in 137 Japanese patients.

Gene 204

Gene Clinical Trials Clinical Trials Gene Therapy 204

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.

DNA 56

DNA Gene Gene Editing Scientist 56

Scienmag

JULY 2, 2021

The ongoing effort to explore […].

Gene Silencing 75

Gene Silencing Gene Editing Gene DNA 75

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.

Gene Editing 64

Gene Editing Gene DNA Bacteria 64

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., […].

Gene Editing 41

Gene Editing Gene DNA Genetics 41

Pharmaceutical Technology

MARCH 2, 2023

ImmunoGen has announced an agreement granting rights to Vertex Pharmaceuticals to carry out research using antibody-drug conjugate (ADC) technology for the discovery of new targeted conditioning agents. The research using ImmunoGen’s technology will enable the discovery of the agents for use with gene editing.

Gene Editing 130

Gene Editing Gene Development Research 130