Pharmaceutical Technology

AUGUST 17, 2022

These programmes will include therapies and vaccines in infectious disease and oncology areas. oRNA molecules have been demonstrated to possess increased stability in vivo compared to linear mRNA and can potentially create more quantities of therapeutic proteins within the body. .

RNA 147

RNA Vaccination Vaccine In-Vivo 147

pharmaphorum

JANUARY 10, 2022

The German group is paying $40 million upfront to kick off the alliance, which is focusing on the development of up to five CRISPR drugs that can be delivered in vivo for liver-targeted diseases. – are around one-third the size of the more commonly used Cas9.

In-Vivo 52

In-Vivo Gene Editing Gene Therapy Gene 52

Delveinsight

JANUARY 12, 2021

Birinapant is a clinical-stage SMAC mimetic that degrades Inhibitors of Apoptosis Proteins (IAPs) by binding to them, ultimately leading to cell death in tumor cells. The companies believe that their candidate in its original IgG format has shown potent neutralization activity in in vitro assays and in an in vivo animal model.

Licensing 52

Licensing Licensing Antibody Gene Therapy 52

XTalks

DECEMBER 13, 2023

Both gene therapies are approved for individuals 12 years of age and older with sickle cell disease. Sickle cell disease is a group of inherited red blood cell disorders that affect approximately 100,000 people in the US, most of whom are Black. Casgevy is also the first ever CRISPR/Cas9-based therapy approved in the US.

Gene Therapy 98

Gene Therapy FDA Approval Gene In-Vivo 98

The Pharma Data

AUGUST 3, 2021

While disease progression is slower in patients with later-onset SMA, there are significant unmet needs. “We Food and Drug Administration (FDA) has determined that OAV-101 intrathecal (IT) clinical trials for spinal muscular atrophy (SMA) patients may proceed, thereby lifting the partial clinical trial hold initiated in October 2019.

Clinical Trials 52

Clinical Trials Clinical Trials Gene Therapy Trials 52

Drug Discovery World

FEBRUARY 22, 2024

Dementia is the only major cause of death without a treatment to prevent, slow or stop disease progression. “In With the breadth and range of central nervous system disorders – for example Parkinson’s disease and multiple sclerosis – facing the global population there is an unmet need within this area of drug discovery.

Drugs 59

Drugs Gene Clinical Trials Clinical Trials 59