pharmaphorum

SEPTEMBER 28, 2022

Vertex Pharma and partner CRISPR Therapeutics will start a rolling marketing application in the US for their gene-editing drug for sickle cell disease (SCD) and beta thalassaemia later this year. The post Vertex, CRISPR prep filing for gene-editing blood disorder therapy appeared first on.

Gene Editing 59

Gene Editing Gene In-Vivo Clinical Trials 59

XTalks

DECEMBER 13, 2023

The US Food and Drug Administration (FDA) has approved the first gene therapies for the treatment of sickle cell disease, approving two on the same day. Both gene therapies are approved for individuals 12 years of age and older with sickle cell disease. million after discounts.

Gene Therapy 98

Gene Therapy FDA Approval Gene In-Vivo 98

XTalks

DECEMBER 29, 2022

For example, on the pharma and biotech side, there has been growing demand for new drugs and biologics, from new classes of dual-action diabetes and weight loss drugs like Ozempic and Mounjaro to life-saving gene therapies. As of December 16, 2022, there are seven US Food and Drug Administration (FDA) approved gene therapies.

Life Science 52

Life Science Gene Therapy Gene Manufacturing 52

pharmaphorum

JANUARY 10, 2022

The first mRNA-based vaccines were brought onto the market in 2020, with Pfizer/BioNtech and Moderna receiving emergency use authorisation from the US Food and Drug Administration (FDA) for the prevention of COVID-19.

RNA 98

RNA Vaccination Vaccine Pharma Companies 98

XTalks

DECEMBER 22, 2021

At the beginning of the year, Genentech, a member of the Roche Group, struck a deal with Ribometrix to leverage its discovery platform to discover, develop and commercialize small molecule drug candidates against RNA targets. RNA therapeutics hold promise as powerful treatments for diseases, including those that are genetic-based.

Life Science 98

Life Science RNA Vaccination Vaccine 98

Drug Discovery World

FEBRUARY 22, 2024

Lu Rahman looks at neuroscience drug discovery – advances and challenges – and how breakthroughs in this field are helping address areas of unmet need. Recent advances in biomarkers, research into the central nervous system and breakthroughs in human iPSC cell models, have bolstered neuroscience drug discovery research.

Drugs 59

Drugs Gene Clinical Trials Clinical Trials 59