pharmaphorum

SEPTEMBER 6, 2021

BioMarin Pharma has had another setback in its gene therapy development programme, announcing this morning that the FDA has placed a phase 1/2 trial of its candidate for phenylketonuria (PKU) on hold while it investigates a safety signal. Five of the mice had adenomas and one had a hepatocellular carcinoma (HCC).

Gene Therapy 80

Gene Therapy Gene Genetic Disease DNA 80

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. The PASTE gene editing technique was recently published in Nature Biotechnology.

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Gene Editing DNA Gene Gene Sequencing 98

Pharmaceutical Technology

MARCH 28, 2023

Vertex Pharmaceuticals has signed a new non-exclusive licensing agreement with CRISPR Therapeutics to expedite the development of its hypoimmune cell therapies to treat type 1 diabetes (T1D). The gene-editing technology allows for precise, directed changes to genomic DNA.

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Gene Editing DNA Gene Gene Therapy 235

Drug Discovery World

SEPTEMBER 20, 2022

Breakthroughs in gene therapy are only possible with an exact understanding of the genetic underpinnings of disease. To develop safe and effective gene therapies, researchers need confidence that genomic data is both complete and accurate.

Gene Therapy 52

Gene Therapy Gene Genome Genomics 52

Drug Discovery World

SEPTEMBER 6, 2022

DDW Editor Reece Armstrong looks at the cell and gene therapy landscape, examining the challenges facing developers and the trends we can expect to see throughout the year. . There’s no doubt that cell and gene therapies present some of the most exciting opportunities for emerging drugs.

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Gene Therapy Gene Manufacturing Medicine 52

pharmaphorum

APRIL 27, 2021

Dosing can start once again in uniQure’s phase 3 trial of its haemophilia B gene therapy, after the FDA concluded that a case of liver cancer seen in the study was unlikely to be caused by the treatment. . The post FDA lifts hold on uniQure gene therapy after cancer case review appeared first on.

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Gene Therapy Gene Genome Genomics 52

pharmaphorum

MARCH 10, 2021

bluebird bio is to ask regulators to restart clinical studies of its LentiGlobin for sickle cell disease, after an investigation concluded that a case of acute myeloid leukaemia (AML) was “very unlikely” to be caused by the gene therapy. bluebird bio’s Philip Gregory.

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Gene Therapy Gene Trials Gene Expression 53

Roots Analysis

FEBRUARY 27, 2024

Over the last few decades, various viral vector manufacturing have been developed, optimized and standardized for introduction of therapeutic DNA / gene of interest into a patient’s body / cells. On the other hand, among non-viral gene delivery tools, plasmid DNA has emerged as the preferred option.

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Manufacturing Gene Therapy Gene DNA 52

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

Pharmaceutical Technology

JUNE 4, 2023

Within the emerging innovation stage, cell therapy for ocular disorders, coronavirus vaccine components, and DNA polymerase compositions are disruptive technologies that are in the early stages of application and should be tracked closely. The company has developed various assets based on lentivirus vectors.

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Gene Therapy DNA Gene Genetics 246

Advarra

MARCH 31, 2023

The use of engineered genetic materials in clinical trials is rapidly expanding, with potential applications for genetic vaccines, gene-modified cellular therapies, and gene therapies. A key part of the IBC’s evaluation is assessing the risks posed by the engineered genetic materials.

Engineer 52

Engineer Genetics Clinical Research Research 52

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. Credit: St. Jude Children’s Research Hospital St.

Gene Expression 97

Gene Expression Regulation Gene DNA 97

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

The Pharma Data

FEBRUARY 22, 2022

State-of-the-art facility will headquarter research efforts in Boston and New York to accelerate the development of RNA and DNA-based medicines. Adams, vice president of genetic medicine at Lilly and co-director of the Institute. ” The Institute will be headquartered in 334,000 sq.

Genetics 52

Genetics Medicine RNA Scientist 52

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. Moderna will also assume further development, manufacturing, and commercialisation responsibilities on exercising a target option.

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Gene Editing Gene In-Vivo Gene Therapy 147

The Pharma Data

DECEMBER 5, 2020

5, 2020 — A pair of new gene therapies promise a potentially lasting cure for sickle cell disease by subtly altering the genetic information in patients’ bone marrow cells, researchers report. Both of the new gene therapy studies were published online Dec. SATURDAY, Dec.

Gene Therapy 52

Gene Therapy Gene Gene Editing Genetics 52

Delveinsight

FEBRUARY 18, 2021

Bluebird suspends gene therapy trials after two sickle cell patients develop cancer. Bluebird bio has stopped two clinical trials of its sickle cell disease gene therapy after participants developed acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS).

Gene Therapy 52

Gene Therapy Gene Trials Clinical Trials 52

Drug Discovery World

AUGUST 14, 2023

Lung-I Cheng, Vice President and Head of Cell & Gene Therapy Service Line at AmerisourceBergen, and Cori Gorman, Senior Director of CMC and Regulatory Affairs at Biopharma Excellence, offer advice on navigating the different and sometimes contradictory regulatory requirements in the US and EU. billion in 2020 to $15.5 globally.

Gene Therapy 52

Gene Therapy Marketing Gene DNA 52

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

Roots Analysis

FEBRUARY 23, 2022

NGS has emerged as an evolutionary technology in modern biotechnology and healthcare research, enabling researchers to develop a better understanding of the cause and consequences of disease. Currently, close to 60 industry players are actively offering NGS kits for preparing DNA / RNA libraries. Players Engaged in the Domain.

Genetics 52

Genetics DNA RNA Reagent 52

pharmaphorum

OCTOBER 12, 2022

Now, he leads Whitelab Genomics as its artificial intelligence (AI) platform powers the development of genomic therapies – an emerging field in which genetic sequences are injected into cells to target and repair damaged genes. We have been very impressed by their vision and the relevance of their platform.”.

Genome 99

Genome Genomics Medicine Development 99

Scienmag

FEBRUARY 11, 2021

Study in The CRISPR Journal reports the app advances CRISPR gene therapy R&D; 18-year-old software savant helped lead the app’s development Wilmington, DE, Feb. The app reveals potentially risky DNA […].

Gene 73

Gene Gene Therapy DNA Scientist 73

XTalks

DECEMBER 4, 2023

The FDA said it is investigating the identified risk of developing the T-cell malignancies as secondary cancers with serious outcomes, including hospitalization and death, and also looking at the need for regulatory action. These studies are intended to assess the long-term risk of secondary malignancies following treatment.

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Gene Therapy Gene Clinical Trials Clinical Trials 69

Delveinsight

DECEMBER 3, 2020

The company anticipates the test can aid in tracking the success of vaccines and recognize people who have recovered from infection and could serve as convalescent plasma donors or help in the development of new therapies. Janssen buys a gene therapy asset. Zimmer Biomet buys chest surgery toolmaker A&E Medical.

Gene Therapy 52

Gene Therapy Gene Antibody Research 52

XTalks

NOVEMBER 6, 2020

Canadian clinical-stage biotech company Symvivo Corporation has developed an oral COVID-19 vaccine that entered clinical trials this week. The gene therapy-based vaccine targets mucosal tissues in the gastrointestinal tract, allowing for greater selectivity and targeted delivery compared to traditional vaccines.

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Clinical Trials Clinical Trials Vaccination Vaccine 75

XTalks

JANUARY 26, 2024

Technological advancements are revolutionizing cancer research and therapies. Key breakthroughs at the forefront include novel approaches to cancer treatment and diagnosis, the integration of artificial intelligence (AI) in oncology and the development of advanced preventive measures and care.

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Research Gene Editing Gene Genetics 118

XTalks

DECEMBER 20, 2023

As we step into 2024, the life sciences continue to evolve at an unprecedented pace, driven by technological innovation, a deeper understanding of human biology and the application of new technologies in areas like drug development and health wearables. Regulatory bodies are also taking note of the applications of AI in drug development.

Life Science 119

Life Science Gene Editing Development Clinical Trials 119

pharmaphorum

OCTOBER 3, 2022

DMD is a severe, progressive muscle-wasting genetic condition caused by the lack of a protein called dystrophin. A gene therapy, it permits the body to read over the DNA mutation and still continue to produce dystrophin. Eventually, they will need assistance with breathing.

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Gene Therapy Gene FDA Approval DNA 91

XTalks

MARCH 13, 2024

Biomarker testing is being pursued to identify biological signs of the disease and provide an approach to develop personalized treatment plans for each patient. As this technology continues to evolve, it is expected to help identify glaucoma in its nascent stages of development.

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Gene Therapy Gene Life Science DNA 111

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. billion in funding.

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Gene Editing Gene Gene Therapy In-Vivo 144

XTalks

NOVEMBER 30, 2023

Global entities such as the WHO, the Global Fund and the Joint United Nations Programme on HIV/AIDS (UNAIDS) have developed comprehensive strategies aligned with Sustainable Development Goal (SDG) target 3.3, These findings signify a significant step forward in the development of AGT103-T as a potential gene therapy for HIV.

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FDA Approval Gene Therapy Clinical Trials Clinical Trials 111

XTalks

MAY 4, 2021

Researchers at the University of California San Francisco (UCSF) and the Whitehead Institute have developed a novel CRISPR-based tool called “CRISPRoff” that can switch off genes in human cells through epigenetic editing without altering the genetic sequence itself. It’s a great tool for controlling gene expression.”.

DNA 98

DNA Gene Gene Silencing Genome 98

Delveinsight

DECEMBER 17, 2020

Following that hypothesis, scientists designed oral inhibitors, which could target mitochondrial DNA (mtDNA). The data promote further development of the drugs as potential first-in-class mitochondria inhibitors for the cancer treatment, the researchers stated in the paper. million in financing.

Gene Therapy 96

Gene Therapy Gene DNA Genetics 96

Drug Discovery World

APRIL 17, 2024

As for gene-modulating approaches, I want to point out that our therapeutic candidates do not in any way modulate the gene itself. Increasing gene expression via protein replacement or gene therapy has been shown to have limitations and drawbacks. Can you give some examples? What’s in the pipeline?

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Gene Expression Gene Genetic Disease RNA 59

XTalks

MARCH 8, 2022

From leading COVID-19 vaccine developments to being at the helm of the latest gene therapies and inventing ground-breaking DNA technologies like CRISPR, women have been at the forefront of some of the most leading-edge scientific discoveries in recent years, both as innovators and leaders.

Life Science 105

Life Science Gene Therapy DNA Vaccination 105

NY Times

FEBRUARY 22, 2021

Two patients in a gene therapy study developed cancer years after treatment. It is not clear whether the therapy was responsible.

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Gene Therapy Trials Research Gene 54

Roots Analysis

FEBRUARY 21, 2022

Since the introduction of a gene sequencing method by Frederick Sanger in 1977, the field of genomic data collection and analysis has evolved significantly. Advancement in DNA sequencing technologies have resulted in noteworthy developments in various healthcare-related research fields, such as diagnostics and personalized medicine.

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Marketing Gene Sequencing DNA Genome 52