- In April 2023, the U.S. FDA granted accelerated approval to QALSODY (tofersen), an antisense oligonucleotide (ASO), for the treatment of amyotrophic lateral sclerosis (ALS) in adults with a superoxide dismutase 1 (SOD1) gene mutation. This approval highlights a significant advancement in treating a specific genetic form of ALS, although tofersen is administered intrathecally, it underscores the broader progress in oligonucleotide therapeutics
- In August 2023, the FDA approved IZERVAY (avacincaptad pegol) for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration. While this is an intravitreally injected pegylated RNA aptamer, its approval signifies the versatility and expanding therapeutic applications of nucleic acid-based drugs in various disease areas, including ophthalmology
- In January 2023, Agilent Technologies, Inc. announced a significant investment of USD 725 million to double its manufacturing capacity for therapeutic nucleic acids. This substantial investment in their Frederick, Colorado facility reflects the strong demand for high-quality active pharmaceutical ingredients (APIs) and scalable production capabilities essential for the growing pipeline of nucleic acid drugs, including siRNAs, antisense, and CRISPR guide RNA molecules
