Pharmaceutical Technology

JULY 29, 2022

In 2013, the US Food and Drug Administration (FDA) approved Ravicti (glycerol phenylbutyrate), manufactured by Hyperion Therapeutics, which has since then been acquired by Horizon Pharma. Ultragenyx uses adeno-associated virus 8 (AAV8) gene therapy to induce stable OTC gene expression.

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XTalks

DECEMBER 20, 2023

Given its ability to precisely edit genes, there is tremendous promise for the revolutionary technology to offer cures for genetic disorders and to treat various diseases. This year, the very first CRISPR-based therapy received FDA approval for sickle cell disease.

Life Science 119

Life Science Gene Editing Development Clinical Trials 119

Camargo

DECEMBER 10, 2021

Biomarin’s stock shot up after the drug was first approved in the EU this past August, and on the day the news broke of the FDA approval, the stock improved 10.3%. Subsequent revisions of the document reflect the experience gained as gene therapy products of this type have been approved.

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Development Genetics Production Clinical Trials 176

XTalks

FEBRUARY 27, 2024

The US Food and Drug Administration (FDA) has approved a number of new rare disease drugs in recent years, including gene therapies. In 2020, 31 out of 53 novel drug approvals were for rare or orphan diseases​​. Both therapies were awarded their approvals on the same day in December 2023.

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Research Drugs Gene Therapy Clinical Trials 59

XTalks

APRIL 24, 2023

Awareness of rare diseases is growing, and with a better understanding of the pathophysiology of many rare diseases, innovative treatment options are emerging, like gene therapies that can treat the root cause of rare genetic diseases and potentially provide long-term symptom relief, or even a definitive cure.

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Gene Therapy Gene Trials Clinical Trials 95

Pharmaceutical Technology

FEBRUARY 13, 2023

Although recently approved drugs can treat seizures more safely, they do not treat the comorbidities that patients experience. Several biotech companies and researchers are now exploring medical devices and gene therapies to address not just common forms of epilepsy, but also rare conditions such as Dravet Syndrome.

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XTalks

NOVEMBER 30, 2023

New FDA-Approved Treatments For HIV HIV treatment involves the administration of combined antiretroviral therapy (ART) to effectively suppress the viral load, maintain or enhance immune function and reduce the risk of opportunistic infections and cancers commonly associated with HIV. aiming to end the HIV epidemic by 2030.

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XTalks

MARCH 25, 2021

Biomedical Research and Technologies : Presenting the latest biomedical research from both industry and academia, including preclinical studies as well as technological innovations in genomics, single cell analysis, proteomics, metabolomics and much more.

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Life Science HR Clinical Trials Clinical Trials 105

XTalks

SEPTEMBER 12, 2023

Biomedical Research and Technologies: Get access to the most recent biomedical research news from both industry and academia, including preclinical studies and technological breakthroughs in genomics, single cell analysis, proteomics, metabolomics and much more.

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Life Science Branding Clinical Trials Clinical Trials 105

XTalks

DECEMBER 4, 2023

In a statement outlining its investigation, the FDA said it received reports of the T-cell malignancies from clinical trials and post-marketing safety monitoring. The vectors can potentially cause cancer by inserting genetic material into a patient’s genome, particularly near cancer-related DNA sequences or suppressors​​​​.

Gene Therapy 69

Gene Therapy Gene Clinical Trials Clinical Trials 69

Delveinsight

DECEMBER 15, 2020

The initial data from the trial demonstrated no statistically significant reduction in the number of hospitalized COVID-19 patients receiving ruxolitinib plus SoC therapy who experienced severe complications including death, mechanical ventilation or ICU care by Day 29, compared to SoC treatment alone.

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Vaccination Vaccine Gene Therapy Gene 54

The Pharma Data

AUGUST 11, 2020

In the field of immune cell therapy, this includes registrational CAR T cell agents for numerous diseases, and a growing early-stage pipeline that expands cell and gene therapy targets, and technologies. 1%) as determined by an FDA-approved test, with no EGFR or ALK genomic tumor aberrations.

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Trials Hormones Clinical Trials Clinical Trials 52

The Pharma Data

AUGUST 11, 2020

In the field of immune cell therapy, this includes registrational CAR T cell agents for numerous diseases, and a growing early-stage pipeline that expands cell and gene therapy targets, and technologies. 1%) as determined by an FDA-approved test, with no EGFR or ALK genomic tumor aberrations.

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Trials Hormones Clinical Trials Clinical Trials 52

The Pharma Data

MAY 5, 2021

FDA approves 100th monoclonal antibody product ( Nature Reviews Drug Discovery ). Affinia Therapeutics raises $110 million for gene therapy treatments ( STAT ). New variant-hardy AbCellera-Lilly mAb shows utility of viral genomics surveillance ( BioCentury ). Medtech.

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Vaccination Vaccine Gene Therapy Contract Packaging 52

XTalks

DECEMBER 24, 2020

From isolating SARS-CoV-2 in early January to sequencing its genome shortly thereafter and having a prototype vaccine against it within days, scientific process and progress have held steadfast throughout the pandemic. Infectious disease expert, epidemiologist and director of the Yale Institute for Global Health at Yale University, Saad B.

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Life Science Vaccination Vaccine Gene 111

XTalks

JANUARY 6, 2025

For example, antisense oligonucleotide (ASO)-based therapies have gained traction, with 100 Phase I clinical trials initiated and around 25 percent of these advancing to Phase II or Phase III trials in recent years. As of March 18, 2024, the FDA has approved 36 gene therapies, with an additional 500 therapies in the pipeline.

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