STEMCELL Technologies, Canada’s largest biotechnology company, is pleased to announce the acquisition of Cellular Highways ...

Machine learning models have seeped into the fabric of our lives, from curating playlists to explaining hard concepts in a ...

In the first part of this series, we explored how early genetic screening and gene therapy transform the lives of newborns ...

UniQure said it has aligned with the FDA on components of a statistical analysis plan for a registrational trial of AMT-130.

Scientists at the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), Milan, have found that gene editing using ...

In comments posted in response to the Trump administration’s pharma tariff investigation, companies and industry groups ...

While vepdegestrant outperformed a standard treatment option in a subtype of patients, the benefit on progression-free ...

As newborn screening and rapid DNA sequencing become routine, we are poised to catch and treat inherited diseases at their ...

Neuroscientists can now make precise genetic tweaks to the neurons that are most affected by brain diseases such as Parkinson ...

AMT-130 has been granted the FDA’s Regenerative Medicine Advance Therapy (RMAT) designation and Breakthrough Therapy designation, the first therapy for Huntington’s disease to receive an RMAT ...

Greater numbers of circulating stem cells increase the efficacy of in vivo gene therapy techniques, potentially reducing the price tag of lifesaving treatments.

This comes at a challenging time for gene therapies, as multiple serious AEs and fatalities have been recently reported.