A baby boy with a rare genetic condition was treated with a custom-made CRISPR therapy, raising hopes for others struggling with similar diseases. The treatment, created by a collaboration of companies working together at no cost, is a rare success story in the field of rare diseases.
According to Mark Veich, CEO of Advancium Health Network, one-third of children with rare diseases will not survive to five years old. This is a public health emergency that requires immediate attention.
Veich notes that pharmaceutical companies tend to avoid developing treatments for rare diseases due to low financial incentives and limited patient pools. As a result, most rare diseases have no FDA-approved treatments available.
However, advancements in CRISPR technology and gene editing could lead to more successful therapies. The FDA may also take notice of this success story and prioritize reviews for rare diseases, potentially easing the regulatory process for smaller patient groups.
Veich emphasizes that rare diseases are often overlooked, but communities and families affected by these conditions will continue to rally around those who need help. By highlighting successes like KJ’s treatment, experts aim to raise awareness and push for more investment in research and development for these underserved populations.
Source: https://www.biospace.com/drug-development/crispr-success-in-single-baby-highlights-rare-disease-crisis