pharmaphorum

JANUARY 18, 2023

In 2016, scientists behind a study called the Resilience Project analysed genetic data from 589,000+ people and found 13 adults who carried genetic variants that should have resulted in serious – even deadly – childhood disease, but who were apparently healthy. Giving participants something in return.

Genetic Disease 88

Genetic Disease Genetics Research Genome 88

Drug Discovery World

JULY 3, 2023

The OXEGENE dataset contains anonymised genotype data including disease stage and infertility status, from 1,000 surgically confirmed patients. There are currently no approved diagnostic biomarkers or cures for the disease.

Genetics 52

Genetics Genotype Medicine Licensing 52

The Pharma Data

MARCH 7, 2022

The IGA results in the equivalent genotype (genetic make-up) and short-hair coat trait seen in some conventionally bred cattle, known as a “slick” coat. This is the FDA’s first low-risk determination for enforcement discretion for an IGA in an animal for food use. Solomon, D.V.M., director of the FDA’s Center for Veterinary Medicine.

Genome 52

Genome Genomics Production Marketing 52

The Pharma Data

NOVEMBER 2, 2020

ONWARD is investigating Adial’s lead drug candidate, AD04, as a therapeutic agent for the treatment of Alcohol Use Disorder (AUD) in persons with certain target genotypes related to the serotonin transporter and receptor genes. This communication contains certain “forward-looking statements” within the meaning of the U.S.

Trials 52

Trials Genotype Marketing Production 52

XTalks

MARCH 3, 2021

The US Food and Drug Administration (FDA) has granted approval to Nulibry (fosdenopterin) for reducing the risk of death associated with molybdenum cofactor deficiency type A (MoCD Type A), making it the first treatment for the ultra-rare, genetic metabolic disorder. The approval was granted to BridgeBio Pharma, Inc.

FDA Approval 64

FDA Approval Genotype Clinical Trials Clinical Trials 64

XTalks

SEPTEMBER 15, 2020

A vaccine developed by Swedish company Diamyd Medical has demonstrated significant treatment efficacy in a predefined genetic subgroup of individuals with type 1 diabetes in a Phase IIb clinical trial. Specifically, the vaccine contains human recombinant glutamate decarboxylase 65 (GAD65) protein conjugated to aluminum hydroxide (GAD-alum).

Vaccination 103

Vaccination Vaccine Insulin Trials 103

XTalks

APRIL 24, 2023

Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. One substantial obstacle is from the additional regulatory requirements of gene therapies containing genetically modified organisms (GMOs). Rare diseases can often be progressive, chronic and fatal.

Gene Therapy 95

Gene Therapy Gene Trials Clinical Trials 95