Rare Therapeutics

RareTx is a clinical-stage therapeutics company, and its portfolio includes the lysosomal storage diseases GM1 gangliosidosis (GM1), Krabbe disease, and metachromatic leukodystrophy (MLD). An affiliate of GEMMABio, RareTx will enhance the quality and affordability of gene therapy products, leading to greater access and a sustainable pipeline of treatments. Treatments will be delivered through an international coalition of public-private partnerships, helping to ensure affordable access to lif...