Researchers recommend a new long-read DNA test as the first choice for rare genetic disorders, showing a 3% higher diagnosis rate. Separately, a Chinese AI diagnostic system called DeepRare achieved 57.18% accuracy using only patient symptoms, with accuracy exceeding 70% when genetic data is included.
The mRNA synthesis and manufacturing market is projected to grow from USD 2.23 billion in 2024 to USD 2.96 billion by 2029 at a 5.8% CAGR. Consumables led in 2023, while Asia Pacific is expected to be the fastest-growing region.
Whole genome sequencing, comprehensive genomic profiling, and spatial multiomics are transforming precision medicine from research tools to clinical applications in oncology, rare diseases, and neonatal care, enabling faster diagnoses and personalized treatments.
The global CRISPR and prime editing market is forecast to grow at 24.1% CAGR through 2031, driven by therapeutic advances and regulatory progress. Scribe Therapeutics achieved a second milestone with Eli Lilly, while new RNA production and gene editing technologies accelerate research.
The U.S. orphan drug market is projected to exceed $200 billion by 2031, driven by innovation in oncology and genetic disorders. More than 1,400 FDA-designated orphan drugs are in development or marketed, with over 800 currently in clinical trials.
The mRNA therapy market is forecast to grow from USD 13.6 billion in 2025 to USD 70.0 billion by 2035 at a 17.8% CAGR, driven by expanding applications beyond vaccines into oncology, rare diseases, and infectious disease treatments including genital herpes.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT07348926 |
The Relationship Between Functional Independence and Family Well-being in Children With Rare Genetic Disorders |
RECRUITING | |
| NCT07039084 |
Using a Speech-Generating Device to Support Communication in Rare Genetic Conditions |
RECRUITING | NA |
| NCT02365376 |
Collection and Storage of Human Biospecimens for Research Into Rare Diseases and Medical Conditions |
UNKNOWN |
