Home / Rare & Genetic Diseases
Rare & Genetic Diseases
| 2 min read
A disease once synonymous with a shortened lifespan is now a model for how drug discovery can directly alter the natural history of genetic disorders.
| 6 min read
Avidity remains on track to submit Biologics License Application at the end of 2025 for accelerated approval
Available on Demand
Large-scale clinicogenomic data sheds light on the biology of rare kidney disorders and opens doors to new treatment possibilities.
| 4 min read
Long-term treatment with givinostat can significantly delay loss of key mobility functions in patients with DMD
| 2 min read
Industry experts and consulting firms warn that proposed import tariffs could drive up drug prices, disrupt global supply chains, and divert investment away from critical R&D pipelines.
| 2 min read
The state-of-the-art facility will deliver CGMP plasmid DNA development and manufacturing services by late 2025.
| 5 min read
Methylation profiling of cell-free DNA is opening new possibilities for detecting cancer at its earliest, pre-symptomatic stages, offering a potential breakthrough in how and when disease is found.
| 4 min read
A well-chosen CDx development partner can streamline drug development, reduce delays, and pave the way for successful regulatory approval and market entry.
| 3 min read
This recognition signals momentum for next-gen RNA therapeutics designed to enhance delivery and efficacy in treating rare genetic diseases.
| 4 min read
For rare and inherited kidney diseases, genetic testing is solving the clinical trial recruitment crisis and reshaping how precision therapies get to patients.
| 5 min read
A wave of FDA rejections reveals how CMC issues are becoming a leading barrier to approval in cell and gene therapy development.
| 4 min read
Another death linked to Sarepta’s gene therapy, a promising glioblastoma trial, a rejection for Replimune, and more led the news this week.
