01 / 05
Life-Changing Cystic Fibrosis Treatment Wins $3-Million Prize

Nature | Health & Medical Care

Life-Changing Cystic Fibrosis Treatment Wins $3-Million Prize

“The triple-drug combination Trikafta has given a new lease of life to 90% of people with cystic fibrosis, an inherited disorder that affects the lungs and other organs. Now, the trio of researchers who spearheaded its development has won one of this year’s US$3-million Breakthrough prizes — the most lucrative awards in science.”

From Nature.

World Health Organization | Vaccination

Historic Step Towards Broader Vaccination against Malaria

“Shipments of the world’s first WHO-recommended malaria vaccine, RTS,S, have begun with 331 200 doses landing last night in Yaoundé, Cameroon…

A further 1.7 million doses of the RTS,S vaccine are expected to arrive in Burkina Faso, Liberia, Niger and Sierra Leone in the coming weeks, with additional African countries set to receive doses in the months ahead.”

From World Health Organization.

The Atlantic | Science & Technology

The CRISPR Era Is Here

“Four years ago, [Victoria Gray] joined a groundbreaking clinical trial that would change her life. She became the first sickle-cell patient to be treated with the gene-editing technology CRISPR—and one of the first humans to be treated with CRISPR, period. CRISPR at that point had been hugely hyped, but had largely been used only to tinker with cells in a lab. When Gray got her experimental infusion, scientists did not know whether it would cure her disease or go terribly awry inside her. The therapy worked—better than anyone dared to hope. With her gene-edited cells, Gray now lives virtually symptom-free. Twenty-nine of 30 eligible patients in the trial went from multiple pain crises every year to zero in 12 months following treatment.”

From The Atlantic.

The Economist | Health & Medical Care

The World’s First Pathway for Individually Designed Drugs

“The Medicines and Health-care products Regulatory Agency is working with Genomics England, Oxford University and Mila’s Miracle Foundation, a charity, to develop a regulatory pathway to allow one-off drugs to be designed and approved for use in individual patients in less than a year.”

From The Economist.

Wall Street Journal | Health & Medical Care

Gene Editing and Health Investing May Change Medicine

“Gene editing is part of a broader therapeutic revolution that encompasses genetic and cellular medicine. The pills and injections we are all familiar with generally target proteins or pathways in the body to treat disease. With gene and cell therapy, we can now target the root cause of disease, sometimes curing patients.”
From Wall Street Journal.