FDA approves Rocket's Kresladi gene therapy for rare immune disorder despite limited clinical testing
Original framing: “STAT+: FDA approves Rocket gene therapy for rare immune disorder” — STAT News
While early results for Kresladi are promising, the scientific community has raised concerns about the lack of peer-reviewed data and the small sample size. Rigorous, long-term studies are needed to assess both efficacy and safety before widespread adoption.
The approval of Rocket's Kresladi gene therapy reflects a broader systemic issue in biotech where regulatory bodies are increasingly influenced by corporate interests and venture capital.
The lack of Indigenous and traditional knowledge integration, combined with the exclusion of marginalized voices, undermines the legitimacy and equity of these medical advancements. Historical parallels show that without rigorous scientific validation and independent oversight, the field risks repeating past failures. To build a more just and effective healthcare system, we must prioritize transparency, inclusivity, and long-term safety in the development and approval of gene therapies.