Luxturna was the first FDA-approved gene therapy for a disease caused by mutations in a specific gene. It helped establish ...
Two Philadelphia doctors who led the groundbreaking gene-editing treatment of baby boy from Delaware County are included ...
Molecular biologist Jean Bennett and ophthalmologist Albert Maguire will share the $3 million (£2.2m) "Breakthrough prize" ...
The global gene therapy market size is projected to expand significantly, with an estimated value of USD 19.3 billion by 2034 ...
Cancer cells excel at evading detection, but subtle chemical differences set them apart from healthy cells. Now, a team of ...
Pfizer announced in February 2025 that it was taking Beqvez (fidanacogene elaparvovec), its gene therapy for hemophilia B, ...
Investigating a common gene in three very different species—salamanders, mice and zebrafish—scientists have discovered the ...
The treatment for primary immunodeficient diseases is varied. As mentioned before, the most common type of primary immunodeficient disease are antibody deficiencies. Either patients don't make enough ...
From billionaire-backed biotech to promising animal studies, this early trial will test whether partial reprogramming can ...
Pfizer and BioMarin have pulled their hemophilia gene therapies, citing low demand. To prepare for his presentation at the ...
Dr. Swee Lay Thein and Dr. Stuart Orkin won the $3 million Breakthrough Prize in Life Sciences for their work toward a ...
Ultragenyx (NASDAQ:RARE) is one of the 10 Overlooked Growth Stocks to Buy Now.
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