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

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

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

XTalks

DECEMBER 13, 2023

Bolstered by world-class R&D capabilities, a highly productive scientific platform and an established management team, Alpine is dedicated to crafting first- or best-in-class multifunctional immunotherapies through distinctive protein engineering technologies, all aimed at enhancing the quality of life for patients.

Genetics 111

Genetics Vaccination Vaccine DNA 111

XTalks

MAY 4, 2021

Related: 2020 Year in Review: COVID-19, CRISPR and Immunotherapies Define the Year for the Life Sciences. To develop CRISPRoff, the researchers generated a single “dead” Cas9 fusion protein capable of mediating DNA methylation and repressive histone modifications, but devoid of any DNA cleavage function. pyogenes dCas9.

DNA 98

DNA Gene Gene Silencing Genome 98

XTalks

MAY 4, 2021

Related: 2020 Year in Review: COVID-19, CRISPR and Immunotherapies Define the Year for the Life Sciences. To develop CRISPRoff, the researchers generated a single “dead” Cas9 fusion protein capable of mediating DNA methylation and repressive histone modifications, but devoid of any DNA cleavage function. pyogenes dCas9.

DNA 52

DNA Gene Gene Silencing Genome 52