Table 2.
Disorder | Treatment | Biotech/Pharmaceutical Company | Stage of Development |
---|---|---|---|
PKU | Microbe therapy (SYNB1618) | Synlogic (https://www.synlogictx.com/) | Phase II clinical trail |
mRNA therapy (mRNA-3283) | Moderna (https://www.modernatx.com/) | Preclinical development | |
Small molecule therapy | Agios Pharmaceuticals (https://www.agios.com/), Camp4 (https://www.camp4tx.com/) | Preclinical development | |
Red blood cell therapy (RTX-134) | Rubius Therapeutics (https://www.rubiustx.com/) | Discontinued in Phase 1b trial |