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. The genomic medicine journey. Zinc fingers.

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Gene Editing Gene Gene Therapy In-Vivo 145

Pharmaceutical Technology

JUNE 8, 2023

Biotechnology company Hopewell Therapeutics has raised $25m in seed financing to accelerate the development of next-generation lipid nanoparticles for targeted delivery of genomic medicines. Hopewell Therapeutics is engaged in discovering, synthesising and developing advanced ttLNPs to provide next-generation genomic medicines.

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Genome Genomics Medicine Development 130

Pharmaceutical Technology

MAY 12, 2023

The research team has designed four bacterial viruses which use CRISPR technology to kill the unwanted bacteria precisely. The division of infectious diseases at Weill Cornell Medicine carried out the work in collaboration with JAFRAL and JMI Laboratories.

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Development Bacteria Research Gene Editing 246

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

Drug Discovery World

SEPTEMBER 25, 2023

CRISPR gene editing companies Integrated DNA Technologies (IDT) and Aldevron have inked a new global distribution agreement to expand CRISPR products for cell and gene therapy customers. The post Partners working to realise the future of genomic medicine appeared first on Drug Discovery World (DDW).

Genome 52

Genome Genomics Gene Editing Medicine 52

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. The move marks a continuation in its ongoing commitment to exploring gene-editing technology.

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Gene Editing Gene Gene Therapy Genetics 52

BioTech 365

JULY 29, 2021

Editas Medicine and IDT Announce Publication in Nature Communications of Research Data Supporting the Use of Optimized AsCas12a Nuclease Variant, Alt-R A.s. Cas12a (Cpf1) Ultra, in Researching the Potential of Gene-Edited Cell Medicines Editas Medicine and IDT Announce Publication in … Continue reading →

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Gene Editing Medicine Gene Research 40

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

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.

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Gene Therapy Gene Editing Gene Research 98

BioTech 365

NOVEMBER 25, 2021

Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: Scientists produce new antibiotics by gene editing.Scientists have discovered a new route to produce complex antibiotics exploiting gene editing to re-program pathways to future medicines urgently required … Continue reading (..)

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Gene Editing Scientist Gene Medicine 40

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 The post Vertex builds in gene editing yet again, with $1.2bn Arbor deal appeared first on.

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Gene Editing Gene In-Vivo Genetics 52

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

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

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

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

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.

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Gene Therapy Gene Gene Editing Manufacturing 243

Scienmag

JULY 18, 2022

HOUSTON – (July 18, 2022) – Rice University researchers have demonstrated that CRISPR-Cas9, increasingly famous as a gene-editing tool, can be employed in powerful additional ways in human cells.

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Gene Editing DNA Gene Research 84

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. 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 32 mutation into patients.

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Gene Editing Research Gene Genetics 69

Scienmag

OCTOBER 20, 2021

The ability to edit the genome by altering the DNA sequence inside a living cell is powerful for research and holds enormous promise for the treatment of diseases. However, existing genome editing technologies frequently result in unwanted mutations or can fail to introduce any changes at all.

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DNA Gene Editing Genome Genomics 75

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

The Pharma Data

JULY 28, 2021

AstraZeneca has entered into a collaboration with Regeneron to research, develop and commercialise small molecule compounds directed against the GPR75 target with the potential to treat obesity and related co-morbidities. The companies will evenly split research and development costs and share equally in any future potential profits.

Medicine 52

Medicine Development Research Insulin 52

Drug Discovery World

OCTOBER 14, 2022

Meanwhile, Ochre Bio successfully raised $30 million to advance its pipeline of RNA therapies for liver disease, and researchers found that combining CRISPR gene editing with Spherical Nucleic Acids could expand the potential of the technology. . Partnership seeks to transform Cystic Fibrosis research .

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Gene Editing RNA Drugs Gene 52

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.

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Research Gene Gene Editing Genome 52

Drug Discovery World

JANUARY 9, 2023

In this early research, Khalid Shah, Professor of Neurosurgery at Harvard Medical School and Brigham and Women’s Hospital, and colleagues tested the vaccine in an advanced mouse model of brain cancer glioblastoma, with promising results. The findings are published in Science Translational Medicine. . CRISPR-Cas9 gene editing .

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Scientist Gene Editing Gene Engineer 52

pharmaphorum

SEPTEMBER 29, 2020

Cell therapy research has been built on collaborations amongst scientists and entrepreneurs, providing early proof of concept for modalities thought to be too difficult to commercialise but with a strong potential for patient benefit”.

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Research Gene Editing Gene Development 128

Drug Discovery World

OCTOBER 31, 2022

The last month has seen huge strides forward in our understanding of cancers, particularly in how they develop resistance to therapies and how we can ‘outsmart’ them using gene editing or different therapeutic pathways, but also how we can better target drugs to individuals and accurately predict treatment outcomes.

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Drugs Research Gene Editing Protein 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

Pharmaceutical Technology

MARCH 9, 2023

In 2022, Immatics and Editas Medicine entered a research collaboration and licensing agreement to combine gamma-delta T-cell adoptive cell therapies and gene editing to develop medicines for the treatment of cancer. Bristol-Myers Squibb and Cellectis are the other key patent filers in in-vitro T-cell activation.

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In-Vitro Gene Editing Medicine Licensing 130

Drug Discovery World

MAY 5, 2023

With Exacis’ pipeline of engineered iPSC-derived cell therapy candidates, we believe Eterna is well-positioned to develop more effective, targeted and highly differentiated medicines to treat cancer,” said Matt Angel, Chief Executive Officer and President of Eterna.

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Gene Editing Engineer DNA Life Science 52

Drug Discovery World

NOVEMBER 24, 2023

 Traditional approaches to research won’t cut it The pursuit of scientific knowledge is not merely an academic endeavour, but a necessary pathway to improving the longevity and quality of human lives.  Replicating success beyond the pandemic Historically (pre 1940s) medical innovation was often achieved through chance.

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Research Life Science Gene Editing Vaccination 59

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

Drug Discovery World

MARCH 22, 2023

A new approach that delivers a ‘one-two punch’ to help T cells attack solid tumours is the focus of a preclinical study by researchers from the Perelman School of Medicine at the University of Pennsylvania. After CRISPR editing, the T cells were expanded and infused in solid tumour mice models.

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Gene Editing Gene Engineer Regulation 52

Roots Analysis

FEBRUARY 27, 2024

How is the genome editing market landscape evolving: Currently, there is an evident increase in demand for complex biological therapies (including regenerative medicine products), which has created an urgent need for robust genome editing techniques. Get a complete list of the presentations, here.

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Genome Genomics Gene Gene Editing 40

The Pharma Data

JUNE 7, 2023

Bayer strengthens gene therapy portfolio with lipid nanoparticle technology from Acuitas Therapeutics Bayer AG is joining forces with Acuitas Therapeutics, Inc., “Accessing state-of-the-art LNP technology through this collaboration will add momentum to our gene editing efforts for the benefit of patients.”

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Gene Therapy Gene Gene Editing In-Vivo 40

XTalks

OCTOBER 12, 2021

The experimental arthritis vaccine was developed by researchers at the University of Toledo (UToledo) and is a major breakthrough for rheumatoid arthritis and other autoimmune diseases as well. Ritu Chakravarti, assistant professor in the UToledo College of Medicine and Life Sciences, and the paper’s lead author, was investigating 14-3-3?

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Vaccination Vaccine Research Immune Response 105

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. driven, fundamental discovery project has now become the breakthrough strategy used by countless researchers working to help improve the human condition,” said Doudna. “I

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Gene Editing Bacteria DNA Gene 101

Pharmaceutical Technology

FEBRUARY 15, 2023

Editas Medicine is one of the most important players concerning innovation surrounding CRISPR nucleases. The company is largely involved in genome editing using CRISPR/Cas9 and CRISPR/Cas12a systems. Biocad and Sangamo Therapeutics are in the second and third positions, respectively.

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In-Vivo Genome Genomics Gene Editing 162