XTalks

MARCH 21, 2025

Following Neffys FDA approval , ARS Pharma reported $2.3 The company recently reported topline results from its Phase I/II trial of AURN001, an allogeneic cell therapy for corneal edema. 10x Genomics 10x Genomics reported $707 million in 2024 revenue. million in fourth-quarter (Q4) 2024 net revenue.

Genome 59

Genome Genomics FDA Approval Trials 59

Pharmaceutical Technology

JUNE 29, 2022

The US Food and Drug Administration (FDA) put a high-profile bluebird bio trial for sickle cell disease on partial clinical hold, and advisory panels deliberated over decisions involving gene therapies for amyotrophic lateral sclerosis (ALS), cerebral adrenoleukodystrophy (CALD), and beta-thalassemia.

Drugs 246

Drugs FDA Approval Development Marketing 246

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.

RNA 245

RNA Gene Expression Gene Gene Therapy 245

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.

FDA Approval 111

FDA Approval Gene Therapy Clinical Trials Clinical Trials 111

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

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.

Development 176

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.

Research 59

Research Drugs Gene Therapy Clinical Trials 59

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.

Life Science 105

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.

Life Science 105

Life Science Branding Clinical Trials Clinical Trials 105

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.

Vaccine 54

Vaccine Vaccination 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.

Trials 52

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.

Trials 52

Trials Hormones Clinical Trials Clinical Trials 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.

Life Science 111

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.

Gene Therapy 111

Gene Therapy RNA Gene Editing Gene 111

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.

Vaccination 52

Vaccination Vaccine Gene Therapy Contract Packaging 52