Gene therapy has helped a 9-year-old boy regain enough muscle strength to run. If successful in others, the treatment could change the lives of thousands of children with Duchenne muscular dystrophy.
The first gene therapy for hemophilia could be approved by the FDA within six months, according to the drugmaker, raising hopes among families. But the drug's price could be $3 million per patient.
The new coronavirus has spurred biotech labs in universities and companies to try to find new approaches to stopping the virus — from blocking its key enzymes to interfering with its genetics.
Recent advances in biotech make scientists optimistic that they might have a vaccine that has passed basic tests of human safety and efficacy ready to go to clinics as soon as this fall.
Labs are churning out more and more synthetic DNA for scientists who want to use it to reprogram cells. Some say the technology has outpaced government safety guidelines put in place a decade ago.
Some tech firms and researchers say plans to impose export restrictions on "emerging and foundational technology" would make it hard to thwart cyber threats and to cooperate globally in science.
Sometimes discoveries derived from patients' medical data become the foundation of new profit-making companies. A fledgling industry wants to help patients get a cut of the cash.
A committee of experts examined about a dozen different synthetic biology technologies that could be potentially misused. For each, they considered how likely it was to be usable as a weapon.
The Republican tax plan could reduce or eliminate federal tax credits that reward companies for developing drugs to treat rare diseases. People who benefit from the medicines have objected.