Medical Xpress

MAY 16, 2023

They study gene variation and mutations that cause rare genetic diseases. A mutation is a permanent change in the genetic material. That was the case when Thilini Gamage was to carry out one of the studies in her doctoral work with Professor Eirik Frengen at the Institute of Clinical Medicine, University of Oslo.

In-Vitro 98

In-Vitro In-Vivo Genetic Disease Genetics 98

Pharmaceutical Technology

MAY 4, 2023

The approval allows KALYDECO to be used in infants who have at least one mutation in their cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to the therapy, on the basis of clinical and/or in vitro assay results. This cohort showed a safety profile identical to that found in older children and adults.

In-Vitro 246

In-Vitro Genetic Disease Medicine Genetics 246

Drug Discovery World

JUNE 19, 2023

Anoat will use the funds to conduct additional in vivo and in vitro pharmacology assessments and select a lead candidate. Cystic fibrosis is a progressive genetic disease that affects the lungs, pancreas and other organs.

Development 52

Development Life Science In-Vivo In-Vitro 52

Delveinsight

JANUARY 12, 2021

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. Bluebird bio recently announced its plans to split its genetic disease and oncology businesses. and Europe.

Licensing 52

Licensing Licensing Antibody Gene Therapy 52

Pharma Mirror

APRIL 21, 2024

This represents an increasingly promising field of precision medicine, holding significant prospects for preventing and treating numerous challenging or genetic diseases. In vitro transcribed (IVT) mRNA has been successfully The post General Design Methods for mRNA Drugs appeared first on Pharma Mirror Magazine.

In-Vitro 130

In-Vitro Drugs Genetic Disease RNA 130

The Pharma Data

DECEMBER 20, 2020

On May 14, the company presented data from the vibegron EMPOWUR 52-week extension study and data by age groups from the 12-week placebo-controlled EMPOUR study at the 2020 American Urological Association Annual Meeting. The drug is a once-daily, beta-3 adrenergic agonist. David Staskin, principal investigator of EMPOWUR and a urologist with St.

In-Vitro 52

In-Vitro FDA Approval Manufacturing Drugs 52

Drug Discovery World

FEBRUARY 28, 2023

As we turn our focus to new potential applications and disease areas for the platform, scientists and companies must consider the potential life-saving impact on rare, inherited diseases, says Archana Gupta , PhD, staff scientist in genetic sciences at Thermo Fisher Scientific.

Genetics 52

Genetics Genetic Analysis Vaccination Vaccine 52