Accelerating Therapies
for Ultra-rare Diseases
Leveraging an International Coalition to Improve Access to Gene Therapies
Most rare diseases are too small to justify investment in the development of treatments.
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 life-saving gene therapies by those who need them most.
More than 10,000 rare diseases have been identified; most have prevalence of < 1:100,000
72% of all rare diseases have a genetic origin
Around 70% of the patient population are children
350 million individuals globally are affected by rare diseases
Only 5% of rare diseases have available treatments
The economic burden of rare diseases in the US in 2019 was ~$1 trillion
Building on Dr. Jim Wilson's 40-year career pioneering the development and delivery of gene therapies for rare diseases
RareTx is helping overcome the challenges faced by ultra-rare patient populations through:
novel platform-based development paradigms
next-generation technologies for gene delivery and manufacturing
innovative commercial models for worldwide distribution
global engagement with regional centers of excellence