The Pharma Data

AUGUST 16, 2021

The CureVac-GSK COVID-19 collaboration announced in February 2021 extends the existing strategic mRNA technology partnership both companies started in July 2020, which focuses on the development of new products based on CureVac’s second-generation RNA-technology for different targets in the field of infectious diseases.

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Conversations in Drug Development Trends

MAY 30, 2024

Since marketing authorization for the first breakthrough treatment in 1994, the steady increase in clinical trials reflects the community’s commitment to finding effective ALS treatments despite the numerous hurdles associated with clinical trial design, from proof-of-concept to pivotal trials. The MAA file in the E.U.

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The Pharma Data

JANUARY 10, 2021

INZ-701 is a soluble, recombinant protein containing the extracellular domain of native human ENPP1 fused to the Fc domain of the immunoglobulin IgG1. Ilya Pharma completed recruitment in its Phase I trial of ILP100-Topical for wound treatment. The drug is an RNA interference (RNAi) therapeutic. Most Read Today. Source link.

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Drug Discovery World

FEBRUARY 28, 2023

With only 10% of these conditions indicated for an approved therapy, there is a sizable opportunity for scientists and companies to develop treatments for patients with no other options to turn to. The post Unlocking the potential of mRNA for the future treatment of rare diseases appeared first on Drug Discovery World (DDW).

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Drug Discovery World

MAY 2, 2023

Today, scientists leverage this defence mechanism to learn more about the genomic alterations in diseased states as well as the impact of drug treatments on the human genome. Bacterial RNA is replaced by a synthetic guide RNA (gRNA) that can recognise a specific target DNA sequence.

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Alta Sciences

APRIL 17, 2024

Five Promising Treatment Areas in Early-Phase Drug Development in 2024 aasimakopoulos Wed, 04/17/2024 - 15:52 Early-phase drug development is an ever-changing landscape, as emerging science leads to new promising areas of research for the treatment of human health issues.

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Broad Institute

JUNE 27, 2024

By Greta Friar, Whitehead Institute June 27, 2024 Images of a mouse brain show the effect of a technology called CHARM in turning off the expression of a gene in the brain. Vallabh soon found out that she inherited the same disease-causing mutation in the prion protein gene. Credit: Neumann EN, Bertozzi TM, et al.

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Codon

APRIL 9, 2023

Of those, 17 children responded to the treatment, and the 3-year overall survival rate was 60 percent. Small snippets of double-stranded RNA were sprayed onto hot pepper plants to control a pest, called Frankliniella occidentalis. The RNA strands shut down a specific gene in the insects, and killed half after 7 days.

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The Pharma Data

SEPTEMBER 22, 2020

Novartis Gene Therapies remains confident in the overall benefit-risk profile for patients on treatment. The company’s commitment to SMA extends beyond gene therapy to branaplam (LMI070), an oral, once-weekly RNA splicing modulator also currently under development, to expand the treatment options for SMA patients. .

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