RNA therapeutics
| 6 min read
With the first wave of FDA-approved siRNA drugs under their belt, companies are racing to expand into new tissues and indications.
| 6 min read
Avidity remains on track to submit Biologics License Application at the end of 2025 for accelerated approval
| 3 min read
By combining synthetic biology, computational design, and novel RNA formats, these therapies adapt in real time to disease signals, enabling previously impossible treatments.
| 3 min read
This recognition signals momentum for next-gen RNA therapeutics designed to enhance delivery and efficacy in treating rare genetic diseases.
Available on Demand
Cell-free gene synthesis removes key bottlenecks in mRNA synthesis, accelerating the development of lifesaving therapies.
| 5 min read
Michelle Werner and her team at Alltrna are developing novel tRNA therapeutics to overwrite mutations shared by hundreds of rare genetic diseases.
| 3 min read
New dual AAV vector delivery overcomes challenges in delivering large genes.
| 5 min read
The durability and capacity to boost protein expression of circular RNA makes it a promising candidate for next-generation vaccines and gene therapies.
| 8 min read
Alnylam Pharmaceuticals develops therapeutics based on the RNA interference (RNAi) pathway discovered in 1998.
| 5 min read
Paolo Martini leads the biotech company’s rare disease research program with the hope of developing the very first treatments for genetic metabolic disorders.
