Drug Discovery World

JANUARY 11, 2024

The team, led by University College London (UCL), King’s College London and Moderna scientists, found that mRNA could be used to correct a rare genetic liver disease known as argininosuccinic aciduria in a mouse model. mRNA contains instructions that direct the cells to make proteins.

Research 59

Research Gene Therapy RNA Gene 59

XTalks

AUGUST 6, 2020

Epidiolex is the only FDA-approved formulation that contains CBD derived from the cannabis plant. CBD is the most abundant non-psychoactive cannabinoid compound found in the cannabis plant, making up about 40 percent of the plant’s extract, which contains over 100 different cannabinoid compounds.

FDA Approval 64

FDA Approval Protein Containment Genetic Disease 64

XTalks

FEBRUARY 27, 2023

The lysosomal storage disorder is caused by mutations in the MAN2B1 gene, which codes for lysosomal alpha-mannosidase, an enzyme that degrades glycoproteins (proteins attached to sugar residues). A lack of this enzyme results in a toxic build-up of mannose-containing sugars in the cells of the body, including the central nervous system (CNS).

FDA Approval 52

FDA Approval Life Science Containment Genetics 52

XTalks

NOVEMBER 10, 2023

The US Food and Drug Administration (FDA) has approved Takeda Pharmaceuticals’ Adzynma, the first recombinant protein product for prophylactic (preventive) or on‑demand enzyme replacement therapy (ERT) in adult and pediatric patients with congenital thrombotic thrombocytopenic purpura (cTTP), an ultra-rare blood clotting disorder.

FDA Approval 59

FDA Approval Gene Therapy Gene Clinical Trials 59

The Pharma Data

OCTOBER 18, 2020

Telomeres degrade and shorten with age and can become excessively damaged in certain genetic diseases, as well as from lifestyle factors such as smoking, poor diet, and chronic stress. Shortening of telomeres is associated with the symptoms of aging, heart disease, DNA damage and uncontrolled cell replication, which can lead to cancer.

DNA 52

DNA Scientist Genetic Disease Research 52

The Pharma Data

AUGUST 3, 2021

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. Novartis today announced that the U.S. SVP, Chief Medical Officer, Novartis Gene Therapies. “We

Clinical Trials 52

Clinical Trials Clinical Trials Gene Therapy Trials 52

XTalks

DECEMBER 13, 2023

The company’s sustained investment in R&D has led to a rapid expansion of its pipeline in 2023, with notable milestones such as four infectious disease vaccines in Phase III trials, including the recently submitted respiratory syncytial virus (RSV) vaccine for regulatory approval. million and $38.8 million and $33.9

Genetics 111

Genetics Vaccination Vaccine DNA 111