Bio Pharma Dive

AUGUST 16, 2023

The struggling biotech also plans to offload other cell therapy assets, including a multiple myeloma therapy, and focus exclusively on gene editing medicines.

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Pharmaceutical Technology

FEBRUARY 15, 2023

In the last three years alone, there have been over 633,000 patents filed and granted in the pharmaceutical industry, according to GlobalData’s report on Immuno-oncology in Pharmaceuticals: Gene therapy delivery using viral vectors. Sanofi is one of the leading patent filers working with viral vectors.

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Pharmaceutical Technology

MARCH 10, 2023

On 10 March, the National Health Service Blood and Transplant (NHSBT) opened a new Clinical Biotechnology Centre (CBC) with the aim of improving the UK’s ability to develop and manufacture cell and gene therapies. Personalised medicines will also be developed at the centre.

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Pharmaceutical Technology

DECEMBER 23, 2022

Eli Lilly and Company has expanded a licencing and partnership agreement with ProQR Therapeutics to discover, develop and market new genetic medicines. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. The companies entered the initial agreement in September last year. By Cytiva Thematic.

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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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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

Bio Pharma Dive

FEBRUARY 28, 2023

An alliance with the startup brings Vertex an RNA-based medicine for the disease, adding to a portfolio that already includes a gene editing treatment.

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Pharmaceutical Technology

JANUARY 5, 2023

Capsida Biotherapeutics and Eli Lilly and Company ’s wholly owned subsidiary Prevail Therapeutics have announced a partnership for the development of non-invasive gene therapies for central nervous system (CNS) diseases. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. By Cytiva Thematic.

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STAT News

OCTOBER 3, 2022

A Swedish scientist won the 2022 Nobel Prize in medicine or physiology on Monday for his groundbreaking research into the evolutionary history of humankind. Pääbo unlocked scientists’ understanding of how genes from these extinct relatives have been passed down to present-day humans. Read the rest…

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Bio Pharma Dive

JULY 14, 2021

Formed around research from the Broad Institute's David Liu and Andrew Anzalone, Prime has attracted significant funding from a wide range of high-profile biotech backers.

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Drug Discovery World

AUGUST 7, 2023

Evox Therapeutics has agreed a research collaboration and option agreement with the Icahn School of Medicine at Mount Sinai (Icahn Mount Sinai) in New York, NY, US. The collaboration will work on developing exosome-encapsulated AAV (exoAAV) vectors as a novel gene delivery technology aimed at improving treatments for heart disease.

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Worldwide Clinical Trials

NOVEMBER 27, 2023

Casgevy, the commercial product formerly known as exa-cel, is administered by taking stem cells out of a patient’s bone marrow and editing a gene in the cells in a laboratory, with the modified cells then infused back into the patient after conditioning treatment to prepare the bone marrow. In June 2023, the U.S.

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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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Drug Discovery World

MAY 8, 2024

Recombinant adeno-associated viruses (rAAVs) are considered a promising delivery system for gene therapy medicines. This webinar will detail how Oxford Nanopore evaluated long-read nanopore sequencing as a comprehensive and sensitive QC method for rAAVs which is supporting internal research activities at AstraZeneca.

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Gene Therapy Gene DNA Genome 69

Medical Xpress

MARCH 13, 2023

Researchers have identified two previously unknown genes linked to schizophrenia and newly implicated a third gene as carrying risk for both schizophrenia and autism.

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Pharmaceutical Technology

MARCH 24, 2023

Moderna has entered a strategic partnership with Generation Bio for the development of non-viral genetic medicines. Moderna’s biological and technical expertise will be combined with core technologies of the non-viral genetic medicine platform from Generation Bio.

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Genetics Medicine Development In-Vivo 130

Scienmag

JANUARY 19, 2022

Research from the University of Virginia School of Medicine suggests how a newly developed gene therapy can treat Dravet syndrome, a severe form of epilepsy, and potentially prolong survival for people with the condition.

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Gene Therapy Gene Research Medicine 78

Pharmaceutical Technology

APRIL 21, 2023

Moderna has signed an agreement with IBM to explore quantum computing and generative artificial intelligence (AI) for advancing and expediting its mRNA research. The company stated that bringing together advanced formulation discovery and generative AI will help in designing new mRNA medicines with optimal safety and performance.

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Pharmaceutical Technology

NOVEMBER 21, 2022

RVAC Medicines has signed a master research partnership agreement with the Agency for Science, Technology and Research (A*STAR) for analysing and developing solutions to build messenger ribonucleic acid (mRNA) manufacturing and analytics expertise in Singapore.

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Pharmaceutical Technology

FEBRUARY 23, 2023

Moderna has entered a strategic research and development partnership with ElevateBio’s Life Edit Therapeutics to discover and develop new in-vivo mRNA gene editing therapies. Under the deal, both companies will partner on the research and preclinical studies, which will be funded by Moderna.

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Medical Xpress

JANUARY 5, 2023

Indiana University School of Medicine researchers are studying how the reduction of a gene variant found in the brain's immune cells could diminish the risk of late-onset Alzheimer's disease.

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Drug Discovery World

MARCH 20, 2023

Researchers have identified two previously unknown genes linked to schizophrenia and newly implicated a third gene as carrying risk for both schizophrenia and autism. The investigators found the two risk genes, SRRM2 and AKAP11, by comparing the gene sequences of people with schizophrenia to those of healthy controls.

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Medical Xpress

APRIL 17, 2023

Genes are full of clues about a person's health. A recent UC Davis Health study published in BMC Medicine suggests that this may be possible, thanks to time-sensitive gene analysis that allows for faster stroke diagnosis and treatment. They might also show the way for stroke recovery.

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Drug Discovery World podcast

APRIL 4, 2022

This is the latest episode of the free DDW podcast, “What does gene-based medicine have to offer?”. The articles are called “Lipidomics in biomedical research: chance and challenges” and “Exploring gene-based medicine’s true potential”. Listen here:

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Medical Xpress

MAY 1, 2023

Gene-editing therapy aimed at two targets—HIV-1, the virus that causes AIDS, and CCR5, the co-receptor that helps the virus get into cells—can effectively eliminate HIV infection, new research from the Lewis Katz School of Medicine at Temple University and the University of Nebraska Medical Center (UNMC) shows.

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Gene Editing Gene Medicine Drugs 124

Medical Xpress

MAY 9, 2023

Researchers at Baylor College of Medicine have linked specific variants or mutations of the gene myocardin-related transcription factor B (MRTFB) with a novel neurodevelopmental disorder.

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Gene Medicine Research Genetics 97

Medical Xpress

MARCH 21, 2023

An international study led by the Molecular Physiology Laboratory at the UPF Department of Medicine and Life Sciences (MELIS) identifies new genes that modulate the toxicity of the protein β-amyloid, responsible for causing Alzheimer's disease.

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Protein Gene Life Science Research 98

Pharmaceutical Technology

MAY 24, 2023

In the last three years alone, there have been over 633,000 patents filed and granted in the pharmaceutical industry, according to GlobalData’s report on Innovation in Pharmaceuticals: Recombinant viral vector-based medicinal preparations. However, not all innovations are equal and nor do they follow a constant upward trend.

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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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Scienmag

MAY 6, 2021

Jude Children’s Research Hospital scientists have developed a highly efficient method to address a major challenge in biology–identifying the genetic ‘switches’ that regulate gene expression. Jude Children’s Research Hospital St. Credit: St.

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STAT News

OCTOBER 5, 2022

Scientists rely on gene synthesis technologies as a research tool for everything from basic research to vaccine development and drug target identification. Ever since the inception of gene synthesis, there have been concerns about possible misuse of synthetic genes. Read the rest…

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Gene DNA Engineer Scientist 98

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).

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Medical Xpress

MAY 10, 2023

How colorectal cancer develops is not well understood, but a team led by researchers at Baylor College of Medicine reports in the Journal of Experimental & Clinical Cancer Research that silencing the gene p16, even though the DNA itself does not change, can drive colorectal cancer progression in animal models.

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Pharmaceutical Technology

AUGUST 22, 2022

Porton Advanced Solutions has raised $80m in a Series B funding round to expand its end-to-end gene and cell therapy (GCT) contract development and manufacturing organisation (CDMO) platforms. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Roots Analysis

AUGUST 29, 2023

Over the years, cell and gene therapies (CGT) and other advanced therapy medicinal products (ATMPs) have managed to capture the interest of both drug developers and healthcare investors. It is worth highlighting that some therapeutic interventions can be considered both cell and gene therapies.

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Gene Therapy Gene Research Manufacturing 40

Bio Pharma Dive

NOVEMBER 17, 2021

Chroma Medicine's launch is the latest step in a decadeslong quest by drugmakers to capitalize on research into epigenetics, a way of controlling gene expression without altering DNA.

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XTalks

MAY 17, 2023

The Foundation for the National Institutes of Health (FNIH) announced this week that the Accelerating Medicines Partnership Bespoke Gene Therapy Consortium (AMP BGTC) has selected eight rare diseases for its clinical trial portfolio.

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