Genetic Disease Life Science Regulation Research 
XTalks
DECEMBER 29, 2022
The life sciences and healthcare are among the biggest industries globally, and their significance was particularly highlighted during the past couple of years by the COVID-19 pandemic. Given the hyperfocus on the life sciences thanks to COVID, consumers appear to be more autonomous and vocal about their medical demands and choices.
Drug Discovery World
JUNE 19, 2023
Anoat Therapeutics’ goal is to bring a transformative and mutation-agnostic treatment option to CF patients for whom existing therapies are not effective – for example, patients not eligible or responsive to CFTR (Cystic Fibrosis Transmembrane Conductance Regulator) modulator therapies.
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XTalks
OCTOBER 7, 2020
A tiny child with a devastating genetic disease who wasn’t supposed to blow out the candles on his first birthday cake. Not only did this baby survive to do all these things, but he became a poster child for gene therapy with the regulators at the U.S. One chapter discusses how to form early partnerships with regulators.
Delveinsight
FEBRUARY 11, 2021
Verily is partnering with Johnson & Johnson’s Janssen division to witness the body’s earliest immune responses to a coronavirus infection, with people participating in the research from within their homes. . Moreover, those that did become ill showed relatively mild disease, from which they recovered quickly.
XTalks
APRIL 24, 2023
Rare diseases can often be progressive, chronic and fatal. Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. Sadly, one-third of children with rare diseases die before their first birthday.
XTalks
NOVEMBER 10, 2023
cTTP is a very rare, inherited and life-threatening blood clotting disorder caused by a disease-causing mutation in the ADAMTS13 (A disintegrin and metalloproteinase with thrombospondin motifs 13) gene, which encodes the ADAMTS13 enzyme that regulates blood clotting by cleaving the von Willebrand factor (VWF) protease.
XTalks
AUGUST 6, 2020
TSC is a rare genetic disease that affects approximately 1 in 6,000 people. Defects in hamartin or tuberin lead to a loss of regulation of mTOR, resulting in aberrant differentiation and development, which causes the generation of enlarged cells, as seen in TSC brain lesions.
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