Rare & Genetic Diseases
| 5 min read
Automation, digital manufacturing, and regulatory reform are reshaping how the UK competes in cell and gene therapy as the field moves toward routine delivery.
| 9 min read
Industry leaders predict this year will be shaped by AI-driven discovery, human-relevant safety testing, and tighter capital discipline.
| 5 min read
While faster reviews could bring life-saving treatments to patients sooner, compressed timelines could also limit regulatory scrutiny and strain FDA resources.
| 4 min read
By reengineering small-molecule chemistry for automation, Excelsior aims to remove a long-standing barrier to AI-driven drug discovery.
| 4 min read
The FDA’s National Priority Voucher program aims to cut standard review times from potentially years to months, raising questions about safety, access, and public health impact.
| 5 min read
Emerging optogenetic, gene, and cell therapies are moving beyond single-gene treatments, offering hope for a broader range of patients with retinitis pigmentosa.
| 2 min read
The milestone marks meaningful progress toward developing new therapeutic options for Alport syndrome, with first-patient dosing expected in early 2026.
| 3 min read
The partnership leverages real-world patient data and advanced analytics to support faster, more patient-centered drug discovery.
| 4 min read
Cross-industry collaborations, coupled with technology innovations, could help bring CRISPR therapies to millions of patients with genetic diseases.
| 6 min read
New AI tools promise to speed up and simplify CRISPR experiments, but their real-world impact is still being tested.
| 5 min read
A new study finds that non-core and non-essential procedures may add significant burden to participants and research sites.
| 4 min read
Greater focus on non-animal testing methods in the UK, a new CRISPR treatment to lower cholesterol, and positive results for a BTK inhibitor in MS led the news this week.
