Drug Discovery World

APRIL 30, 2024

Messenger RNA (mRNA) has come into focus within the drug discovery and development as an exciting tool to deliver genetic information. Whilst research into mRNA has been ongoing for decade, interest has sparked in recent years and research has begun to expand into areas of vaccinology, infectious diseases and other indications.

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Therapies RNA Vaccine Small Molecule 130

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.

Immune Response 52

Immune Response Vaccine Virus RNA 52

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.

Treatment 81

Treatment Small Molecule Clinical Trials Therapies 81

The Pharma Data

JANUARY 10, 2021

Forge Therapeutics received FDA clearance to initiate a Phase I/II trial of its FBX-101 for patients with Krabbe disease. Krabbe disease is a rare and fatal pediatric leukodystrophy caused by mutations in the galactosylceramidase (GALC) gene. ENPP1 deficiency is a rare, chronic, systemic, and progressive disease.

Clinical Trials 52

Clinical Trials Trials FDA Protein Expression 52

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. CRISPR libraries: Why do we need them?

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DNA RNA Protein Expression Drugs 130

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.

Drug Development 52

Drug Development Treatment FDA Approval Therapies 52

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.

Therapies 52

Therapies RNA Engineering Vaccine 52

The Pharma Data

SEPTEMBER 22, 2020

Novartis Gene Therapies remains confident in the overall benefit-risk profile for patients on treatment. All patients deserve a gene therapy designed to address the genetic root cause of their disease with a single dose. Zolgensma is approved in the US, Japan and, most recently, Brazil, for patients with SMA under the age of two.

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Clinical Development Therapies Clinical Trials Treatment 52