Regeneron Pharmaceuticals’ gene therapy for otoferlin-related hearing loss, a genetic cause of deafness, has restored hearing to normal levels in one deaf child within just 24 weeks.
Neurogene, a clinical-stage company developing genetic medicines, is expanding its ongoing phase 1/2 clinical trial investigating NGN-401 as a treatment for female pediatric patients with Rett Syndrome.
A new genetic sequencing technology from Element Biosciences has helped researchers from the Translational Genomics Research Institute (TGen), part of the City of Hope, identify the likely genetic causes of disorders in six of nine children from Sonora,...
Camena Bioscience, a synthetic biology company providing genes to the pharmaceutical and biotechnology industries, has closed a $10 million Series A financing round, led by Mercia.
The US medical research agency plans to offer funding over five years through a newly launched consortium, expected to include 30 sites across the country.
While last month saw many pharma executives leaving their positions to head towards smaller biotech companies, this month saw members of the industry climbing the ladder from various different starting points.
Spark Therapeutics has received US approval for Luxturna (voretigene neparvovec-rzyl), its one-time gene therapy treatment for an inherited form of vision loss.
Plasticell has teamed up with encapsulation and screening tech firm Sphere Fluidics to develop genetic modification technology for cell therapy production.
Most Brits would want to know if they carry genes linked to life-threatening diseases according to UK researchers who say this could have implications for drug development.
Gene-editing technology CRISPR-Cas9 must not be used to alter human DNA until the risks have been researched and the ethical issues considered, say scientists.
Gentra Systems has introduced its new versaGene DNA Cell
Purification Kit, which responds to the growing need for
high-quality DNA for sensitive downstream applications. The kit
uses chemistry and column design to purify DNA with...
