Promise and Challenges of Gene-Editing and Other Genomic Medicines — 2024 NEXT Report
April 11, 2024
When scientists completed the Human Genome Project in 2003, it provided, among other things, a reference genome against which the sequencing of an individual could be compared to help uncover variations that might underlie a person’s disease. The so-called reference genome was not a single person’s DNA but constructed from the DNA of multiple genomes. One of the challenges in translating genome research to practice is the lack of ethnic diversity in human genomic studies. But there is promise — researchers have been working on a pangenome that captures the genetic diversity of human population.
This is one of the several topics covered in the 2024 NEXT Report.
The NEXT report provides an overview of developments across the rare disease landscape and highlights trends in research, diagnosis, development, and treatment, as well as the changing regulatory and financial environment. Other topics covered include:
- The need to think differently about the challenges for rare, ultra-rare, and hyper-rare conditions
- How changing sequencing technologies are improving diagnostic success
- How non-profit and for-profit entities are crafting business models to enable sustainable development of N-of-1 therapies
- The need for regulators to exercise their flexibility to address ultra-rare therapies
- How drug developers or working to rethink payment models for one-time, curative therapies
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