Business Wire23d
Arcturus Therapeutics Announces Initiation of Dosing in Phase 2 Multiple Ascending Dose Studies for Cystic Fibrosis (CF) and Ornithine Transcarbamylase (OTC) Deficiency
“ARCT-810 is the only mRNA therapy ... Fibrosis Cystic fibrosis is a life-shortening disease with a worldwide prevalence. Mutations in the cystic fibrosis transmembrane conductance regulator ...
labiotech6d
mRNA, RNAi, circRNA, ASOs: A comparative guide to RNA therapeutics
In this review of RNA therapies, we paint an overview of what’s going on in a field that is addressing a number of diseases.
River Journal19d
New York Medical College Clinical Trial Becomes First to Administer Novel Inhaled mRNA Therapy to a Patient with Cystic Fibrosis in the United States
My body took time to adjust over the holidays, but I am hopeful that 2025 will bring success in advancements in gene therapy for cystic fibrosis. I can’t wait ... inhaled therapy for CF based on mRNA.
Cystic Fibrosis Foundation7d
Restore CFTR: Exploring Treatments for Nonsense and Rare Mutations
The Cystic Fibrosis ... of CFTR mRNA to cells, allowing them to produce healthy CFTR protein. This type of therapy would not affect a person’s own DNA, and the effects would not be permanent, meaning ...
Independent.ie3d
‘Time is precious’ – children with rare cystic fibrosis gene denied access to potentially life-extending therapies
Children whose cystic fibrosis is caused by a rare gene variant are excluded from a new treatment which could potentially slow the decline in their lung function, it has emerged. A group of ...
Independent.ie3d
Wicklow mother calls for access to life-saving therapy for son with rare form of Cystic Fibrosis
His rare gene means he is currently excluded from modulator therapy Kaftrio ... are being supported by Cystic Fibrosis Ireland, and believes the only solution for people with CF like Aaron ...
Medscape25d
Cystic Fibrosis Gene Therapy: Successes, Failures and Hopes for the Future
Uta Griesenbach, Eric W.F.W. Alton, Department of Gene Therapy, Faculty of Medicine ... proof-of-principle for gene transfer into the cystic fibrosis (CF) lung. It is currently unknown whether ...
FierceBiotech27d
Pfizer cuts losses on near-approval hemophilia gene therapy, adding to troubled Sangamo's woes
Pfizer gave Sangamo Therapeutics coal for Christmas, terminating a hemophilia A gene therapy pact to deprive the cash-strapped biotech of up to $220 million in milestones. Pfizer’s decision sent ...
The New England Journal of Medicine8d
Platelet-Targeted Gene Therapy for Hemophilia A with Inhibitor History
Factor VIII gene was introduced into hematopoietic stem cells in men with hemophilia. The annualized bleeding rate went from 10 to 0. No worrisome gene integrations were seen. The content of this ...
Medscape14d
Gene Therapy in Hemophilia: High Hopes and Costs, Low Uptake
Gene therapy has been available since 2022 for hemophilia B and since 2023 for hemophilia A, yet some major medical institutions have barely treated any patients so far. What’s the holdup?
Yahoo Finance28d
Why Moderna Inc. (MRNA) Is Skyrocketing
Shares of Moderna (NASDAQ:MRNA) closed the year on a strong note, rallying by 5.59 percent at $41.58 apiece as investors resorted to bargain-hunting after analysts regarded the company’s ...